Five UC Comprehensive Cancer Centers form consortium to pool patient data for translational research

Five UC Comprehensive Cancer Centers form consortium to pool patient data for translational research

By Matthew Bin Han Ong

Five academic cancer centers within the University of California system are putting together a single consortium to integrate their electronic health records, forming a clinical trials monolith that could be used by pharmaceutical companies doing research in the Golden State.

The UC Cancer Consortium, announced Sept. 11, consists of the following NCI-designated comprehensive cancer centers:

  • University of California, Davis Comprehensive Cancer Center,

  • The Chao Family Comprehensive Cancer Center at University of California, Irvine,

  • The Jonsson Comprehensive Cancer Center at University of California, Los Angeles,

  • University of California, San Diego Moores Cancer Center, and

  • University of California, San Francisco Helen Diller Family Comprehensive Cancer Center.

The UC cancer centers treat 16 percent of all cancer patients in California. The UC centers administered more than 1,600 clinical trials in the past year, accruing more than 29,000 patients. Collectively, these centers are the nation’s top recipient of research grants from NIH and of cancer research grants from NCI. They are also the largest university holder of U.S. biomedical patents.

“Our research mission and high level of skill mean that our centers often care for patients with rare cancers who cannot be treated in other hospitals that may lack the expertise or access to clinical trials using the latest experimental drugs,” John Stobo, executive vice president of UC Health, said in a statement.

Now, for the first time, these centers will face the challenge of developing the infrastructure for streamlining collaborative research protocols and raising funds together—comprehensively, on a statewide level.

The consortium will use a central IRB, create a uniform patient enrollment protocol, and develop IP agreements. These systems will save academic researchers and their industry collaborators the effort of reinventing the wheel each time they want to conduct a clinical trial across institutions, said Alan Ashworth, inaugural chair of the consortium, director and president of the UCSF Helen Diller Family Comprehensive Cancer Center, senior vice president for cancer services at UCSF Health, professor of medicine in the Division of Hematology/Oncology, and E. Dixon Heise Distinguished Professor in Oncology.

“We want to capitalize on the scale we have, to do more,” Ashworth said to The Cancer Letter. “I’m not going to minimize the amount of effort that is. But what we want to say is that this will be available for partnership with pharma, we bring the power of the scale and the power of diversity to problems.

“So, this is not a finished consortium, this is something that’s going to evolve rapidly over time, hopefully. We’re going to build on all these components.”

A conversation with Ashworth appears here.

The UC cancer centers are among the best funded academic institutions in the U.S., but the consortium will need additional resources, Ashworth said.

“We don’t have a budget as such at this point. What we have is each cancer center pulling resources in to build the consortium,” Ashworth said. “We decided to build it and show value first.”

That value will become apparent once the consortium makes patient health records interoperable across the UC cancer centers, Ashworth said.

“What we will be able to do is to take snapshots of the data and then convert it into a form where it could be aggregated,” he said. “I think that’s where real value will come from, and then one overlays precision medicine on clinical data.

“We were also stimulated by the challenges thrown down by former VP Joe Biden and the Cancer Moonshot to collaborate and share data. So, there are multiple reasons why this is the right thing to do and why now is the right time.”

The UC Cancer Consortium’s five goals are: advancing precision medicine, increasing access to clinical trials, improving population health sciences, harnessing the power of Big Data, and engaging in politics and patient advocacy. In 2013, cancer centers at institutions that belong to the Big Ten athletic conference formed the Big Ten Cancer Research Consortium that had similar goals and principles (The Cancer Letter, May 31, 2013).

The Big Ten consortium, which includes public and private NCI-designated cancer centers located at several states, is a success, said Steven Rosen, provost and chief scientific officer for City of Hope, director of the Comprehensive Cancer Center, and director of Beckmann Research Institute and the Irell & Manella Graduate School of Biological Sciences.

“I have not been engaged now for four years, but it took off,” said Rosen, who was watching a Big Ten game in 2011 when he came up with the idea for creating the Big Ten consortium. He was, at the time, director of Northwestern University’s Robert H. Lurie Comprehensive Cancer Center.

“I think that what they’re doing with the University of California cancer centers is fabulous. It’s the way it should be,” Rosen said to The Cancer Letter. “Obviously, if they approached us, we would be very open to working with them. We always reflect on that and make a decision that is appropriate for our population and our investigators. But I think it’s great.”

Indeed, the Big Ten consortium comes to mind, UCSF’s Ashworth agreed. “There is the Big Ten consortium, which is perhaps the most geographically diverse,” he said. “The idea is to build this as a consistent thing across the University of California system. So, this is almost a safe haven where we can just go and share things and learn and develop, really, at quite a considerable scale.

“We can implement clinical trials for rare cancers in which it would be difficult for one of our centers to meet accrual goals. Also, we’re eager to see how we can implement what we know in terms of precision public health. The state’s diversity will teach us a great deal about tailoring interventions and treatments by ethnicity and culture.”

The UC cancer centers have the reach and leverage to do great things, Rosen said.

“I wish them great success,” Rosen said. “We currently have independent collaborative activities with many of the institutions, but I think that, as a consortium, they have the power to do some important things, and that they hopefully will do much to benefit humanity.”

 

UC directors: What we bring to the consortium

In phone calls and emails, directors of the consortium’s five UC cancer center described to The Cancer Letter their institution’s expertise, and what projects they plan to catalyze through the new consortium:

Primo_Lara_Jr

As the only NCI-designated cancer center serving inland Northern California, including the Central Valley, UC Davis Comprehensive Cancer Center provides access and critical knowledge to the area’s many underserved populations. Our expertise in early-phase clinical trials development, comparative oncology and cancer health disparities research that are so essential to our region can be leveraged across the UC Cancer Consortium.

Primo Lara, Jr.
Acting director, associate director for translational research, and co-leader of the Cancer Therapeutics Program at UC Davis Comprehensive Cancer Center

 

Richard_VanEtten

Part of the power of the UC Cancer Consortium is the access to the 14 million patients under UC Care for clinical trials. Dr. David Fruman of UCI’s Chao Family Comprehensive Cancer Center is developing a clinical trial testing the ability of statin cholesterol-lowering medications to synergize with cell-death-inducing drugs in lymphoid cancer, and we plan to launch this investigator-initiated trial through the Consortium.

Richard Van Etten
Director of the Chao Family Comprehensive Cancer Center and professor in the Division of Hematology/Oncology at UC Irvine

 

michael_teitell

The Jonsson Cancer Center is a renowned leader in pioneering new cancer treatments, particularly immunotherapy using checkpoint inhibitors, such as anti-PD-L1 and anti-CTLA-4, and dendritic cell vaccines for brain and other cancers. But in order to develop truly transformative new treatments and bring them to the clinic, it’s important for our scientists to have access to the whole spectrum of research activities. The consortium offers an unprecedented opportunity for UCLA researchers to communicate cross-institution, and obtain the level of understanding of immune response and resistance that is essential to future advances.

Personalized healthcare is another area that is of critical importance to us. To truly fulfill its promise, it requires both developing considerable informatics and pipeline infrastructures to collect and analyze enormous amounts of data from large populations. In January, we launched the UCLA Institute for Precision Health. Headed by JCCC researcher Dr. Daniel Geschwind, the goal is to connect us to the other UC campuses and create a ‘virtual lab’ that can perform large numbers of extended sequencing cases to screen for hereditary cancers, pharmacogenomic profiles, cardiac risk, or different diseases associated with genetic mutations. Researchers from all UC cancer centers will now be able to access large databases to search for genetic abnormalities across highly diverse patient populations and ethnic groups. I’m incredibly excited for what we can achieve when we work together as a system.”

Michael Teitell
Director of the UCLA Jonsson Comprehensive Cancer Center and president of the Jonsson Cancer Center Foundation

 

Scott Lippman

The consortium is a force multiplier, and the concept is already bearing fruit. Here’s an example, which happened just this week:

“Pancreatic cancer is relatively rare, but ranks among the deadliest of malignancies, accounting for just 3 percent of all cancer cases in the United States, but 7 percent of all cancer deaths. At Moores Cancer Center, we have one of the finest groups of pancreatic cancer specialists in the world, led by Andrew Lowy and Tannishtha Reya. In fact, they are part of Stand Up To Cancer’s international dream team focused on pancreatic cancer research and treatment.

“But because pancreatic cancer is complicated, deadly and comparatively rare, it’s especially challenging when it comes to developing new approaches and clinical trials leading to new, better therapies. Through the Consortium, we began discussions this week with our colleagues at UCSF to combine our strengths, resources and patient pools to more aggressively and effectively treat pancreatic cancers.

“It’s a first step, of course, but it’s a big step and one that a lot of places or institutions cannot take.”

Scott Lippman
Director of Moores Cancer Center at UC San Diego Health, professor of Medicine at UC San Diego School of Medicine, senior associate dean and associate vice chancellor for cancer research and care, and Chugai Pharmaceutical Chair in Cancer

 

Part of the power of the UC Cancer Consortium is the access to the 14 million patients under UC Care for clinical trials. Dr. David Fruman of UCI’s Chao Family Comprehensive Cancer Center is developing a clinical trial testing the ability of statin cholesterol-lowering medications to synergize with cell-death-inducing drugs in lymphoid cancer, and we plan to launch this investigator-initiated trial through the Consortium.

Alan Ashworth
Inaugural chair of the UC Cancer Consortium, and director of the UCSF Helen Diller Family Comprehensive Cancer Center

Ashworth’s challenge: Build one very big data sharing system across the University of California cancer centers

Conversation with The Cancer Letter

Ashworth’s challenge: Build one very big data sharing system across the University of California cancer centers

Alan Ashworth

Inaugural chair of the UC Cancer Consortium,
Director of the UCSF Helen Diller Family Comprehensive Cancer Center

 

As the inaugural chair of the University of California Cancer Consortium, Alan Ashworth has to do a little cheerleading and a lot of pushing for integration of the electronic health records across the UC cancer centers.

All five cancer centers use Epic, but that doesn’t mean much. “We’re all on Epic—but they’re all different instances,” Ashworth said to The Cancer Letter. “So, we need another solution to put all these things together.

“It’s not practical to think that we can all move on to exactly the same instance of Epic. The cost and logistics would be enormous. What we will be able to do is to take snapshots of the data and then convert it into a form where it could be aggregated.”

With an interoperable system across the UC centers, researchers would be able to discern patient characteristics, patterns of care and the cost of treatment.

“For example, questions we can ask are: what is the differential use of specific therapeutics across the five campuses?” Ashworth said. “We take an example like ipilimumab—who uses more, who uses less? It’s not an issue of who’s right, who’s wrong. Rather, we need to get at what the underlying reasons are for that selection, and then share and discuss it. Can we come up with a policy for the use of high-end expensive drugs? Can we do this as a group?

“Another example is: what is the proportion of ER-positive breast cancers at each site? How is that designated? This is something that is generally well standardized, but once we start to look across the system, then maybe we would find some differences that might reflect differences in populations.

“That, I think, will lead to discussions about how to improve quality. Also, there may be opportunities to reduce cost. That’s not the primary objective of this consortium, but we do think it could be an important aspect of what we do.”

Ashworth spoke with Matthew Ong, a reporter with The Cancer Letter.

 

Matthew Ong:

What was the inspiration for this effort, and whose idea was it?

Alan Ashworth:

This is all about the power of scale to combat cancer. There were multiple drivers to establish the consortium. First, the science is telling us there is considerable heterogeneity even within common cancers. If we are to study these subsets or rare cancers in detail, we need numbers.

Another issue is expertise. Cancer research is increasingly a team game—even though we are all leading centers, none of us can do everything. Proper analysis of patient level data is facilitated by bigger numbers.

Finally, we were also stimulated by the challenges thrown down by former VP Joe Biden and the Cancer Moonshot to collaborate and share data. So, there are multiple reasons why this is the right thing to do and why now is the right time. Collaboration and co-operation is an imperative, not just a good to-do.

The idea came about from discussion amongst the UC cancer center directors and the head of UC Health, Jack Stobo.

 

MO:

What were the relationships between the UC cancer centers like before the consortium?

AA:

The five UC cancer center researchers have already been collaborating on a number of projects, such as a hematologic malignancies group and the Athena breast health project. However, these were one-offs, and we decided to formalize the collaboration to have a much broader vision and to facilitate future interactions.

 

MO:

And how is California going to be different, with five world-class cancer centers now formally working together?

AA:

California is the most populous—39 million people—and ethnically/racially diverse state in the U.S. In some counties here, cancer is already the leading killer; in others, it is just behind cardiovascular disease, but it will catch up.

Of course, decreasing incidence and mortality are ultimate goals, but we know that takes time. In the meantime, there are a number of ways in which the consortium can improve things for patients. We can sync our electronic medical records for easier sharing—to ease the way for patients who need to visit more than one of our centers; and for sharing data for larger projects.

Given that, combined, the five centers see 16 percent of California’s cancer patients; we can implement clinical trials for rare cancers in which it would be difficult for one of our centers to meet accrual goals. Also, we’re eager to see how we can implement what we know in terms of precision public health. The state’s diversity will teach us a great deal about tailoring interventions and treatments by ethnicity and culture.

Finally, together, the five UCs can have a compelling voice in advocating for patients on cancer policy and ensuring that our elected local and state officials are fully briefed on the latest research and issues. These things are all incredibly difficult, but we believe the path may be made a little easier because we are all part of the same system.

 

MO:

What do you think would be the first and most tangible objective that only a consortium like this can achieve?

AA:

Sharing and analyzing patient records and acting upon what we see would be an extremely powerful demonstration of the power of working together.

 

MO:

What is the business model of the consortium? Will there be a central CRO?

AA:

We are already in the same entity—UC—and standards for data sharing and IP are already harmonized. A lot of contracting is done centrally. This made thinking about the consortium considerably easier than it would have been if we were completely separate entities.

The business model will be to do things together where it makes sense for each participant and their patients to add value to the system. Over time, we will look to measuring the impact and return on investment of what we do to refine our projects.

 

MO:

Is the consortium aiming to become a one-stop translational pipeline for pharmaceutical companies looking to do business in the Golden State?

AA:

There are already examples of this, and we want to capitalize on the scale we have to do more. There are a couple of UC-wide things. We have the Hematological Malignancies Consortium that’s been active for a couple of years. There’s also the Head and Neck Consortium, a UC consortium that has worked with AstraZeneca, for which the results were just announced at ESMO.

I’d have to say these are one-offs, and each time pretty much we have to reinvent the wheel. The idea then is to have it structured in a way that it becomes much easier and facile to do it. It’s not that anybody’s putting impediments in the way, but as you know, UC is a huge organization, and that means that sometimes, it doesn’t move as fast as it could.

 

MO:

And the consortium would streamline all the protocols and SOPs and IP agreements.

AA:

Yes, and I’m not going to minimize the amount of effort that is, but there’s also already a common IRB that can be used. But this is to really give it focus and say, “Well, this is what we want to be seeing happening.”

There will still be examples, of course, many examples where cancer centers will do things on their own; or maybe two or three will work together. But what we want to say is that this will be available for partnership with pharma. We bring the power of the scale and the power of diversity of the California population to problems.

So, this is not a finished consortium. This is something that’s going to evolve rapidly over time, hopefully.

 

MO:

Who is funding the consortium, and what does the budget look like? 

AA:

Collectively, the UC cancer centers are among the best funded in the country, but we recognize we will need additional resources to make this work optimally. We think there could be a financial return in the medium term. We are in discussions with various bodies about start-up funds, but for now the costs are being met by the partner cancer centers. The launch has generated a lot of interest.

We don’t have a budget as such at this point. What we have is each cancer center pulling resources in to build the consortium.

Of course, there’s always a balance in setting these kinds of things up. Do you get a lot of resources in advance, in which case, people don’t know what it is? Or do you launch it and then try and build resources? We decided to build it and show value first.

 

MO:

How does the consortium leverage and build, for instance, specific projects and initiatives at UCSF Helen Diller? 

AA:

This initiative is about leveraging the strength of each cancer center so that we can all grow and improve via the partnership. One of the projects at UCSF that we aiming to build on is our SF CAN program which is a citywide, community-driven plan to reduce the burden of cancer in San Francisco. This could be adapted for other cities or counties across the state. More generally, cancer prevention and a reduction in disparities of incidence and outcome is a major goal of the consortium.

 

MO:

Will the consortium also invest in interoperable infrastructure? In its focus on precision medicine, can UC oncologists and faculty look forward to a system-wide database that would integrate health records, streamline clinical trial matching, and improve guidance for clinical decision-making?

AA:

Already there are efforts underway to integrate the medical records of the UC health system. We will piggyback on these to develop cancer-specific clinical data to drive improvements in quality, safety and to reduce costs. Precision medicine and sharing genetic and genomic testing will also be a priority.

Obviously, it’s a very large patient population involved here, the difficulties in integrating medical records have been well discussed, even when one is on the same type of medical record. We’re all on Epic—but they’re all different instances. So, we need another solution to put all these things together.

Atul Butte is leading these efforts for the University of California. He’s a faculty member at UCSF, and he’s leading our Institute for Computational Health Sciences. This effort will be a key part of what the cancer groups will do. He’s looking at the whole health system and very closely at all sort of issues related to health care provision.

So, for example, questions we can ask are: what is the differential use of specific therapeutics across the five campuses? We take an example like ipilimumab—who uses more, who uses less? It’s not an issue of who’s right, who’s wrong. Rather, we need to get at what the underlying reasons are for that selection, and then share and discuss it. Can we come up with a policy for the use of high-end expensive drugs? Can we do this as a group?

Another example is: what is the proportion of ER-positive breast cancers at each site? How is that designated? This is something that is generally well standardized, but once we start to look across the system, then maybe we would find some differences that might reflect differences in populations.

That, I think, will lead to discussions about how to improve quality. Also, there may be opportunities to reduce cost. That’s not the primary objective of this consortium, but we do think it could be an important aspect of what we do.

 

MO:

Will the UC cancer centers use an interface program to connect the electronic data warehouses?

AA:

It’s not practical to think that we can all move on to exactly the same instance of Epic. The cost and logistics would be enormous. What we will be able to do is to take snapshots of the data and then convert it into a form where it could be aggregated.

I think that’s where real value will come from, and then one overlays precision medicine on clinical data. We all have different efforts in testing germline genetics and tumor genetics. How can we share that information?

While there’s been a lot of focus on sharing genomic data, there’s less focus on how to action it in a common way and how to learn from each person’s experience and join forces. For example, molecular tumor boards. We’ll have some version of a molecular tumor board, but trying to work out what are the best aspects of each one and put that together and learn from these experiences.

And of course, it’s always possible to visit someone else’s shop and learn these things. The idea is to build this as a consistent thing across the University of California system. So, this is almost a safe haven where we can just go and share things and learn and develop, really, at quite a considerable scale.

 

MO:

If you had to compare, what other cancer consortium in the U.S. would be most similar in design and operation as the UC’s? What’s the same, and what’s different?

AA:

There are a few that come to mind. There is the Big Ten consortium, which is perhaps the most geographically diverse, and there are a number of smaller groups, mostly built around a major site with affiliates.

I suppose the difference is that we are five NCI-designated comprehensive cancer centers—of 49 overall—located in California and all part of the same state UC system. With this model, we believe we will be best placed to serve the people of California. In different parts of the country, other ways of getting together could be equally valid approaches.

 

MO:

Do you foresee the consortium accruing member institutions—other academic cancer centers or oncology establishments in California—over time?

AA:

We started with the UC family of cancer centers for the reasons discussed above, but in the future, if this works, there’s no reason we could not work with other organizations.

 

MO:

You are the inaugural chair of the consortium: what is your immediate focus in getting the consortium up and running?

AA:

Maintaining momentum and making sure everyone sees value in working together. It’s important to do this when everyone is so busy with their own center.

 

MO:

What do you envision we’ll be hearing from the consortium, say, five years from now? Are there plans for working with other large networks and consortiums nationwide; perhaps even a federated model in the future?

AA:

The UC Cancer Consortium members are fully committed to collaborating in any way that will benefit patients. It will be interesting to see who might want to collaborate with us, and we’re all open to it as long as we can stay nimble.

While our immediate focus is on California, we’re all dedicated to trying to change things for as many people as possible. Many multi-institutional and national initiatives didn’t exist five years ago, so it’s intriguing to think about what’s ahead.

We will see where we go with the structure of the consortium in the future; for now we are concentrating on the huge immediate potential.

 

MO:

What do you envision we’ll be hearing from the consortium, say, five years from now? Are there plans for working with other large networks and consortiums nationwide; perhaps even a federated model in the future?

AA:

I hope so. Obviously, far be it from me—as a non-U.S. citizen—to comment on politics, but one of the things that is clear is that people need to look at acting locally and the state level as well as the national.

What I want to do is to be thinking very much about California: how California deals with its $14 billion cancer problem and if we can show the way to others, then all the better!

We will aim to help others if we have successes. Some will be applicable to other states, some won’t. But our priority at this point is California. That’s a big enough problem in and of itself.

 

MO:

What about sustainable increases for the NIH budget?

AA:

Absolutely, we will advocate for that. We’ll be one voice, perhaps a powerful voice in scale. That’s a national issue; this is very much a state-driven initiative at this point. We’ll see what happens in the future.

I’m being careful that we don’t try to do everything at once, that we don’t try and claim we’re going to change the entire world. We’ll start locally and do what we can.

 

MO:

Right. Overpromising, God forbid!

AA:

We’re not going to say we’re going to cure cancer in five years, or by any date whatsoever. The consortium hopefully will be able to move things a little faster and a little bit more smoothly, but it’s still a very, very big problem.

At the moment, this is what it is. It’s a big step forward, but we’re not going to try and over-claim anything huge until we can prove that we can do something.

Gottlieb: Oncology center shows how FDA can improve regulation, lower development costs

Gottlieb: Oncology center shows how FDA can improve regulation, lower development costs

FDA has a legitimate role to play in slowing down the cost of developing drugs, and it can do so by relying on good regulatory science, the agency’s commissioner Scott Gottlieb said.

Speaking at a Washington event sponsored by Friends of Cancer Research and focused on precision medicine, Gottlieb said the agency’s Oncology Center of Excellence demonstrates what the agency can do to streamline the drug development process.

“There are a lot of scientific opportunities to modernize our approach to drug regulation and also make it more rigorous. We’ve seen this, in particular, in oncology,” Gottlieb said.

The center has “thought about things like seamless trials, master protocols, site-agnostic clinical trials, and other ways to try to develop drugs through methodologies that not only could be more efficient and potentially lower-cost, but will yield a lot more information about how those drugs can work in clinical practice,” he said.

A partial transcript of Gottlieb’s remarks at the FOCR meeting Sept. 13 follows:

We’ve talked a lot about my concern about drug prices as a public health issue, because we do concern ourselves at FDA with issues of access, and to the extent that patients don’t have access to the medications they need because of the way they are priced that is something that should concern us all.

To the extent that there are things within the context of FDA’s regulatory portfolio that we think, in fact, of competition and marketplace in a way that could help lower prices and increase opportunities to patients, that is something that we will and are concerning ourselves with.

A lot of what we’ve done today has focused on generic drugs and things that we can do to try and accelerate competition in the space of generic drugs, and we focused on a couple of areas in particular.

First is complex drugs, which in the generic industry refers to high-value drugs. These are often drugs that have difficult formulations and aren’t easy to copy within the confines of the existing generic drug regulatory architecture.

We just don’t have good policy or good science to be able to genericize these drugs, and so you see a lot of these branded drugs remain branded drugs in perpetuity, long after patents have expired, long after Congress intended there to be vigorous competition. We are going to have much more to say about steps we think we can take to help bring more competition into this category.

The second bucket of issues that we’ve taken on is what I see as regulatory arbitrage that exists in the market right now, where someone can come in, pick off a low-volume generic drug, jack up the price a lot, and know that it takes up to 42 months on average—or historically it’s taken up to 42 months on average—for a second generic competitor coming to the market, because of the way the process works and the fact that applications undergo multiple cycles of review.

We want to end that arbitrage.

We want to prioritize generic applications in categories where there isn’t competition and bring down the amount of time it takes for a generic drug to be approved.

We’ve seen the review times come down substantially on the new drug side in large measure because we don’t have as many multiple-cycle reviews anymore. But on the generic drug side, we still see a lot of cycling of applications, and we think we can reduce that dramatically and make it a much more fluid process to the market.

So if someone comes into the market and buys a low-volume generic drug and thinks they are going to raise the price, they might have a monopoly for six months or eight months, but that’s about it.

And that’s going to reduce the incentive to do that, and I think it’s just good government that we have good process and we are prioritizing places of unmet need.

The third category of issues we’ve talked about is what I see as gaming of the system, places where I think drug companies are taking advantage of our rules, rules are intended for one purpose and using it as a way to prolong monopolies way beyond the point that Congress intended under Hatch-Waxman.

I talked a lot about the REMS Program where you see some of this activity. Congress is talking about it too, and I think there’s a lot of things we can do.

I have more to say about this, I think you’ll see us putting out a lot more policies on all these fronts.

I wanted to touch on briefly here is the new drug side of the house.

There are a lot of things we are doing and going to be doing to try to bring more innovation to the market and create more competition when it comes to new drugs by reforming the process, making it more modern and efficient.

There are a lot of places where we can really have a win-win and incorporate new science into how we evaluate new drugs and how we ask sponsors to evaluate drugs and allow them to come to market perhaps more quickly through a development process that might be lower-cost while still increasing our assurance of safety and fairness.

This isn’t a zero-sum game. If you somehow reform the process to make it more efficient and maybe low-cost, it doesn’t mean you are necessarily sacrificing on safety, and I think too many people treat this as a binary equation where you take something off one side you are taking something off the other. It’s really not the way we should be thinking about this.

There are a lot of scientific opportunities to modernize our approach to drug regulation and also make it more rigorous. We’ve seen this, in particular in oncology.

I think we’ve seen it in oncology in part owing to the leadership of Rick Pazdur, who has been very forward-leading and thinking about the scientific methodologies. He has some of the best people in the agency working for him.

His team have thought about things like seamless trials, master protocols, site-agnostic clinical trials, and other ways to try to develop drugs through methodologies that not only could be more efficient and potentially lower-cost, but will yield a lot more information about how those drugs can work in clinical practice.

We’re doing these things because we should do them for patients, because we want to see these products come to the market efficiently. And in some cases, certain products might not even come to the market at all if we go back to how we regulate them.

If you look at the immunological treatments now they are used for oncology, if the oncology division didn’t think about using master protocols that allow this to be tested in different applications simultaneously, we might not have the uses for them that we have right now and it might have taken much longer to get certain information.

But we also need to think about these things because of the cost of drugs development.

There was a debate going on Twitter on Monday. I made a comment that a lot of the drug development programs cost $1 billion in direct out-of-pocket cost, not even factoring in cost of capital and other things that the Tufts people include when they talk about the cost of development.

There was a study that came out and said, “No it’s $650 million looking at just 10 drugs.”

It’s surprising what happened at the debate that ensued, because either way, the number is too large. Either way, that’s unsustainable whether it’s at three quarters of a billion dollars or a billion dollars or $2.5 billion, we are on an unsustainable course and if the costs continue to go up at the percentages that they are, we’re going to see a lot few drugs get developed.

Or we are not going to see the second and third drug come to the market and that’s going to decrease competition and ultimately increase prices to patients.

People think about R&D and the investments in terms of the direct costs that it takes to develop a drug program. So, if it’s a billion dollars to develop drugs, someone might say, “Well, you know there should be a 4x return on that.” You hear that talk in Washington.

Unfortunately, that’s not how investment decisions get made, and some of you have been on the other side of the table. I’ve been on the other side of the table.

People think about making investment decisions based on what the cost of the capital is, and the cost of the capital to develop a drug isn’t just the direct cost. It’s the time cost of capital. It’s also the risk-adjusted cost of capital. So, you always need to adjust for the risk, and so you know in the market place where someone is weighing whether or not to invest in the next biotech innovation or invest in the next Twitter, it would be a real shame if more and more the capital went towards the next Twitter and away from the next biotech innovation.

I think we need to think about these things, think about them in terms of how we modernize the process scientifically to make it more efficient, hopefully to make it low-cost and to also bring more applications. Because right now if the costs continue to go up at the rate that they are for developing products, we’re just going to see fewer innovations as a consequence. And I think that that would be ultimately, obviously, bad for patients. Some things may not get done at all, and you may see less competition in product categories.

We had CVS come in to the agency to talk to us about their experience in the marketplace recently. We’re talking to a lot of payers right now to help us think through drug pricing issues, and also think through our response to the opioid crisis. They made a point that when they see the second or third drug in a category come to the market, they see tremendous price competition, price reductions, even in excess of what they experienced in the European market where there are price controls.

So, we want to see that innovation come to the market, because it provides useful differentiation to patients but it also provides all kinds of savings that ultimately leads to greater access. So, we’re thinking about all these things.

You may hear me more and more talking about stuff we are doing on the new drug along with the stuff we are doing on generic drugs. I want to put it in the right context, so folks understand, this is ultimately about patient access and new innovations pertaining to patients, but it also is about trying to make sure we continue staying on a sustainable course and continue to see treatments that are going to ultimately benefit patients.

The Next Step: Neil Hayes picks up stakes at UNC to build an NCI-designated cancer program in Memphis

Conversation with The Cancer Letter

The Next Step: Neil Hayes picks up stakes at UNC to build an NCI-designated cancer program in Memphis

Neil Hayes

Scientific director, University of Tennessee West Institute for Cancer Research.

 

The Next Step is an occasional series of conversations in which The Cancer Letter will focus on cancer researchers in the midst of transition from one position to another.

Here we sit down with Neil Hayes, who after 15 years at the UNC Lineberger Comprehensive Cancer Center, where he was most recently a co leader of the Clinical Research Program, is leaving for Memphis to become the scientific director of the University of Tennessee West Institute for Cancer Research.

He will be the Van Vleet Endowed Professor in Medical Oncology at the University of Tennessee Health Science Center, with appointments at the Department of Medicine and the Department of Genetics, Genomics, and Informatics and the Department of Preventive Medicine in the College of Medicine of the University of Tennessee Health Science Center.

His recruitment was a part of a collaboration between UT and St. Jude Children’s Research Hospital.

“As I zeroed in on my decision, I ultimately had an epiphany,” Hayes said in a conversation with The Cancer Letter. “The mentors at UNC who created such an incredible institution for me at Chapel Hill—Joe Pagano, Shelley Earp, Gene Orringer, and others—had actually made the same decision I was in the process of making. They came to UNC less than a generation after the founding of the medical school. At that time, Chapel Hill was a provincial backwater. There was no cancer center or research facilities. Yet, over the course of a career, they founded one of the great research institutions of our time. That is the challenge I wanted and now see before me.”

At UNC, Hayes was a professor in the Division of Hematology/Oncology and the Department of Otolaryngology/Head and Neck Cancer Surgery, and director of Clinical Bioinformatics at UNC. He is in his tenth year as a co-principal investigator of The Cancer Genome Atlas project focused on characterizing head and neck cancers.

In Tennessee, Hayes will assume direction of a $25 million fund raised through the combined efforts of The West Cancer Center, University of Tennessee Health Science Center and Methodist Le Bonheur Health Care. He will use the money to recruit additional scientists to the UT/West Institute for Cancer Research to create a program of precision cancer medicine.

West Cancer Center’s ultimate goal is to become an NCI designated cancer center, which is anticipated to take seven to ten years with an additional investment of $100 million.

Hayes’s wife, Liza Makowski, a former professor at the UNC Department of Nutrition whose research focuses on the mechanisms underlying metabolic reprogramming of immune cells, became a professor of medicine  at UTHSC.

Hayes spoke with Paul Goldberg, editor and publisher of The Cancer Letter.

 

Paul Goldberg:

It’s always interesting to see an academic oncologist move to a place that would ordinarily have been described as community medicine. Why did you decide to take this job?

Neil Hayes:

In my case, the motivation for change was a sense that I had gone about as far as I could in the situation I was in. I was not unhappy by any means, but I felt that I had more to offer than I was able to contribute without moving towards a leadership position.

This is to say, I was at a great institution with colleagues I respect, and a place that was well-resourced, and where I had been productive… however, there were still goals I was struggling to achieve. Most importantly, I wanted to deliver on the promise of personalized and precision medicine I felt was only possible from the top down rather than the bottom up.

In fact, in deciding to take this position, I looked at a number of leadership opportunities and I reached a conclusion. I concluded that at the more established programs, the bounds of what is possible clinically and scientifically are often restrained by the very same circumstances that created their prior successes. By their nature, most prominent programs are characterized by powerfully siloed researchers and entrenched programs. A leadership position in that setting consists of building bridges while new initiative programs seemed inherently a secondary priority.

To me, the possibility to build a program from the ground up, despite the challenges, fit best with my academic goals.

As I zeroed in on my decision, I ultimately had an epiphany. The mentors at UNC who created such an incredible institution for me at Chapel Hill—Joe Pagano, Shelley Earp, Gene Orringer and others—had actually made the same decision I was in the process of making. They came to UNC less than a generation after the founding of the medical school. At that time, Chapel Hill was a provincial backwater. There was no cancer center or research facilities. Yet, over the course of a career, they founded one of the great research institutions of our time. That is the challenge I wanted and now see before me.

Having said all of that, I wouldn’t characterize the University of Tennessee Health Science Center as a community practice in that they do have all the relevant structures of academic administration and their institutional support of research.

UTHSC is not just a medical school, but a collection of 6 colleges, including a graduate school, dentistry, and pharmacy, with a full complement of labs and core facilities integrated into the medical campus of three hospitals. It is definitely fair to say that the partnerships with community providers are much stronger than for most academic centers. In fact, it is just those community relationships and outreach opportunities that most medical schools are struggling to foster at this moment in our health care ecosystem.

A blank slate alone would not have been enough to tempt me to leave my academic home of nearly 15 years. There were additional features of the UTHSC program that were specifically attractive. Firstly, the West Cancer Center with whom UTHSC is affiliated has strong leadership team including but not limited to Lee Schwartzberg. The West leadership had established a broad base of community support and a uniquely well-run clinical trials office that are ultimately vital to this
research program.

Complementing the clinical practice leadership was equal support from the Methodist hospital (Methodist Le Bonheur Health Care) as well as the Dean’s and Chancellor’s office at the UTHSC medical school. A final key component, without which I don’t think the move would have been possible, was the support of St. Jude Children’s Hospital.

I have spent the better part of the last 20 years in cancer genomics, and it would have been impossible to consider a move that did not include a community capable of supporting that part of my career. Again, I thank the leadership of the UTHSC medical school, Dean David Stern, as well as Dr. James Downing, president and CEO of St. Jude and Dr. Jinghui Zhang in the St. Jude’s Department of Computational Biology for having the creativity to craft a role for me within this world-class institution.

I should also mention that I am part of a two-career family and my wife Liza Makowski had a say in things as well. She is a molecular biologist who was a tenured associate professor at UNC as well so as you can imagine, that took some thought. In an unlikely turn of events, one of her PhD committee members, a longtime friend and mentor, Guy Reed, just so happened to be the chair of medicine at UTHSC making a recruitment for Liza enticing as well.

So, at the end of the day, it was really a serendipitous combination of circumstances that lead to the move.

 

PG:

Memphis is an interesting place. There is St. Jude, which is a place like no other in pediatric oncology. But there has not been a great academic center in adult oncology. Why was that? And is this changing? 

NH:

I was unquestionably moved and inspired by the St. Jude story. Here, in the middle of the country, far away from the traditional academic centers and without even a graduate school, this institution has quietly and consistently had a major role in the cure of pediatric cancer. It is really an amazing story, and I think about it often. While it is an oversimplification, I think it is true to say that St. Jude exists because of an idea hatched by one man or at least a small number of folks, and executed by skillful physicians, researchers, and administrators. Clearly, the community must be part of the reason that this happened here, as opposed to somewhere else. Memphis is known for its generosity. If they can have this impact in childhood cancer, we asked “why not for adults?”

At the risk of sounding naïve, the answer may be because no one has yet had the vision to try. I think being in the middle of the US is actually a plus in many ways. While there is great value in the culture that arises in close academic quarters, it comes with some baggage as well. I wonder if some of our committees, routines, silos, and inherent hierarchy might occasionally create bounds on the range of what is considered possible. The distance in miles from the academic routine may in some cases create daylight for new ideas and the peace and quiet to execute them.

So, I have asked myself, if the folks at St. Jude can do it, literally on the banks of the Mississippi with the support of the community and dedicated cancer leadership, could it be possible that we too could create a revolution in personalized medicine for adult cancer? Again, this is a naïve and simplistic question. However, it is not that different from the one asked by the founders of St. Jude, so I’m going to stick with it for the time being.

 

PG:

You hear—let me paraphrase—I hear people complain endlessly about all the hardships associated with getting and keeping an NCI designation. Yet, an NCI designation remains quite a desirable commodity. And here you are, wanting one of those things. What’s the rationale?

NH:

As I understand it, the purpose of the NCI Cancer Center mechanism was originally to bring together researchers on campuses across the country to harmonize their collective research and resources rather than having faculty compete internally.

This concept of building a single cancer community within an institution, irrespective of whether or not it is associated with NCI designation, is valuable. Teams of researchers are usually stronger than individuals.

Resources are scarce and coordination to build a community of cancer researchers when done well is invaluable. This type of team-building has been a huge part of my scientific experience over the last decade or more, both as part of the NCI-designated Lineberger Comprehensive Cancer Center and as part of international team science opportunities such as The Cancer Genome Atlas. In short, I consider the process of working towards NCI designation as bringing value to the institution independent of the award itself. It goes without saying, however, that like any milestone, NCI designation brings peer-review and recognition to an institution in a way that few other grants can do.

Having said that, there are clearly points of diminishing returns for both institutions and individuals involved solely in the process of getting designation. My vision is that working towards the model of team science and shared resources should be the near-term goal, and if this is successful, NCI designation along with other successes will follow.

 

PG:

How do you plan to do it? What’s the war chest like? 

NH:

I don’t think it is helpful at this point to open up the RFA for the P30 program and to start making a to-do list.

Firstly, I am continuing with my own palate of team science grants in cancer genomics, which I think brings a new skill set to the institution. Importantly, I plan to expand my own activities from retrospective cancer genome characterization, such as we did in The Cancer Genome Atlas, to a series of prospective clinical interventional trials run through the West Clinic and collaborators.

I have no doubt whatsoever that with modest investments and recruitments, the existing strong clinical trials operation already on the ground will perform at a level on par with any with which I am familiar. On the mechanistic side, cancer genomics has allowed me to participate in research teams, especially in aerodigestive tumors, developing animal models of cancer and therapeutic targets. I plan to continue this work as well.

In terms of the NCI model of programs and core facilities, the institution has strengths worthy of strategic investment and recruiting. I think my role is to bring the faculty together individually, in small groups, and at the institutional level to solidify programs and the cores that serve them.

For an institution as early in the process as we are, there is a lot of latitude for bringing in strategic partners, recruiting leadership positions, growing strong internal candidates, and making targeted resource investments. In fact, the pace of growth is already stunning particularly in terms of clinical and translational leadership. I am not the only new recruit convinced of the potential at UTHSC. The last 1-2 years have seen national external hires in many key cancer positions, including Dave Shibata (chief of surgery) formerly of Moffitt, Mahul Amin (pathology chair) formerly of Cedars-Sinai Medical Center, Boyd Gillespie (ENT Chair) formerly of MUSC, and Matt Ballo (radiation oncology) formerly of MD Anderson, just to name a few.

These recruitments are one piece of evidence at the tens of millions recently invested in cancer at UTHSC and the West Cancer Institute. I have been empowered to recruit and equip at a similar pace and breadth on the science and translational side with an initial commitment of $25 million.

More importantly than this amount of money, which is considerable, is the clear-eyed recognition that a cancer research program will require an ongoing and significant level of support from the hospital, clinical enterprise, and the community. In return, the social contract we are writing with our patients and supporters is revolutionary progress in adult cancer care based on personalized and precision medicine.

Teitell named director of the UCLA Jonsson Comprehensive Cancer Center

In Brief

Teitell named director of the UCLA Jonsson Comprehensive Cancer Center

Michael Teitell, a molecular immunologist and biochemist, was named director of the UCLA Jonsson Comprehensive Cancer Center and president of the Jonsson Cancer Center Foundation.

Teitell, who was chosen after a national search, has been a member of the faculty at the David Geffen School of Medicine at UCLA since 1999. He is the Latta Endowed Professor of Pathology and holds joint appointments in the departments of pediatrics and bioengineering. Also, he serves as chief of the Division of Pediatric and Neonatal Pathology.

Teitell will succeed Dr. Kenneth Dorshkind, who has served as interim director since 2014.

The Jonsson Comprehensive Cancer Center has more than 450 researchers and clinicians engaged in cancer research, prevention, detection, control, treatment and education. Its physicians and scientists handle more than 20,000 patient visits per year.

At UCLA, research in Teitell’s laboratory began with studies of fundamental mechanisms of cancer formation and progression, particularly for B lymphocyte malignancies. This led to projects in stem cell and cancer cell metabolism and the collaborative development of new approaches in cell engineering and response detection. The research lab has received continuous funding since 1999.

Teitell serves as co-director of the UCLA Tumor Immunology Training Program, co-director of the Broad Stem Cell Research Center Bioengineering Core, an associate director of the UCLA-Caltech Medical Scientist Training Program, and the NCAA faculty athletics representative for intercollegiate athletics at UCLA. Prior to being named director, he served as the cancer center’s director of basic and translational research.

 

AACR calls for sound policy, sustained funding increases

In its seventh annual progress report on cancer, the American Association for Cancer Research made the following policy recommendations:

  • Continue to support robust, sustained, and predictable growth of the NIH budget by providing an increase of $2 billion for NIH in FY 2018, for a total funding level of $36.2 billion.

  • Increase the FDA budget in FY 2018 to $2.8 billion, an $80 million increase above its FY 2017 level, to ensure support for regulatory science and to accelerate the pace of development of medical products that are safe and effective.

  • Ensure that funding designated through the 21st Century Cures Act for initiatives and programs, such as the Beau Biden Cancer Moonshot and the FDA Oncology Center of Excellence, is fully appropriated in FY 2018.

  • Negotiate a bipartisan budget deal to raise the discretionary budget caps for FY 2018 and beyond, which would allow our nation’s policy makers to continue to invest in priority areas, such as the biomedical research funded by the NIH.

The 122-page document, titled the “AACR Cancer Progress Report 2017; Harnessing Research Discoveries to Save Lives,” is posted here.

The report was released on Sept. 13, as 400 organizations, AACR among them, met in Washington for a Rally for Medical Research Hill Day to call for robust, sustained, and predictable growth in funding for NIH. NIH Director Francis Collins, Sens. Roy Blunt (R-MO), Christopher Van Hollen (D-MD) and Jerry Moran (R-KS), as well as Rep. Cole (R-OK) spoke at rally events.

 

Allison, Schreiber win 2017 Balzan Prize

James Allison and Robert Schreiber won the Balzan Prize for their contributions to immunological approaches in cancer therapy.

According to the prize committee, “Professors Schreiber and Allison have played transformative roles in the field of tumor immunology, Schreiber by demonstrating that the immune system is crucially involved in fighting tumours and by introducing the concept of immune editing and immune escape, and Allison by unravelling the molecular bases of this escape and succeeding to block it by monoclonal antibody therapy increasing for the first time the survival of patients with metastatic melanoma. Both have collaborated recently in the identification of tumor specific neoantigens, together with other scientists, an approach that might lead to the development of effective personalised cancer-specific vaccines.”

Allison is chairman of the Department of Immunology in the Division of Basic Science Research, executive director of the Immunotherapy Platform, associate director of the Center for Cancer Immunological Research and Deputy Director of the David H. Koch Center for Applied Research of Genitourinary Cancers at MD Anderson Cancer Center.

Schreiber is the Andrew M. and Jane M. Bursky Distinguished Professor at Washington University School of Medicine.

The Prizes will be presented during the award ceremony to be held in Berne on November 17. The amount of each prize is 750,000 Swiss Francs ($790,000).

Every year, the four Balzan Prizes are awarded to scholars and scientists who are distinguished in their fields on an international level. The aim of the Balzan prizes is to foster culture, the sciences and the most outstanding humanitarian initiatives of peace and brotherhood among peoples, regardless of nationality, race or creed.

The four subject areas for the awards change every year. As stipulated in the Articles of the Balzan Foundation, they are selected from among “literature, the moral sciences and the arts” and “the physical, mathematical and natural sciences and medicine.”

Other prize winners are:

  • Aleida and Jan Assmann (Germany), Universities of Constance and Heidelberg, for Collective Memory,

  • Bina Agarwal (India), University of Manchester (UK), for Gender Studies,

  • Michaël Gillon (Belgium), University of Liège, for The Sun’s Planetary System and Exoplanets.

The 2016 Balzan Prizewinner for International Relations, History and Theory, not awarded last year, is Robert Keohane, (USA), Woodrow Wilson School – Princeton University.

 

Mannel Appointed as an NRG Oncology Group Chairman

NRG Oncology, one of the five funded network groups within NCI’s National Clinical Trials Network said Robert Mannel will serve as one of its three group chairmen. He joins Walter Curran and Norman Wolmark as the NRG Oncology group chairmen and principal investigators of the group’s federal awards.

Mannell, director of the Stephenson Cancer Center, the Rainbolt Family Chair in Cancer, and a professor of obstetrics and gynecology at the University of Oklahoma, follows Philip DiSaia, in the role upon Disaia’s retirement after 46 years of service collectively to NRG Oncology and the Gynecologic Oncology Group.

Prior to being appointed as an NRG Oncology group chairman, Mannell served as chair of the NRG Oncology Gynecologic Committee, a member of the NCI Gynecologic Cancers Steering Committee, and corresponding PI for the University of Oklahoma LAPS site for the NCTN. Mannel will now serve as the Chair of the NRG Oncology Foundation Board of Directors.

 

Marcus named associate director for basic research, shared resources at Winship

Adam Marcus, director of Emory University’s Integrated Cellular Imaging Shared Resource, has been appointed as Winship Cancer Institute’s new associate director for basic research and shared resources.

He replaces Paul Doetsch, who served in the position since 1999. Doetsch has been designated to become the deputy scientific director in the Division of Intramural Research of the National Institute of Environmental Health Sciences.

Marcus, an associate professor in the Department of Hematology and Medical Oncology and a member of Winship’s Cancer Cell Biology Program, is a funded investigator with several NCI grants supporting a diverse portfolio of research. His research is focused on metastasis.

“Since joining Winship in 2006, Adam has established himself as a leader in several research realms, including how a common mutation of the LKB1 protein in lung cancer is associated with a high metastatic potential and how this knowledge can be utilized to develop new therapeutic strategies.” Walter Curran, Winship’s executive director, said. “Adam has been an outstanding leader of the Integrated Cellular Imaging Shared Resource, and we all know he will be an outstanding associate director for Winship.”

Marcus is leading the effort to stimulate critical thinking and enthusiasm for sciences in children, kindergarten through 12th grade, in the state of Georgia. Along with Theresa Gillespie, he received a $1.2 million R25 Science Education Partnership Award from the National Institutes of Health in 2016. The grant led to the creation of the Center for Advancing Health and Diversity through Citizen Science where he serves as co-director.

Marcus has served as a Georgia Cancer Coalition Distinguished Scholar and is an American Cancer Society Research Scholar award recipient.

 

Nominations open for AACR-Waun Ki Hong Award for translational and clinical cancer research

The American Association for Cancer Research is accepting nominations for the 2018 AACR-Waun Ki Hong Award for Outstanding Achievement in Translational and Clinical Cancer Research.
The AACR-Waun Ki Hong Award for Outstanding Achievement in Translational and Clinical Cancer Research will recognize a worthy cancer researcher who has conducted highly meritorious translational and clinical cancer research who has not yet reached 51 years of age at the time of the award presentation.

The AACR established the award in recognition of the extraordinary contributions of Past President Waun Ki Hong. His work during his long, brilliant career as a physician-scientist led to advances in cancer research, care, and prevention.

The recipient of the award will present a 30-35-minute lecture at the AACR Annual Meeting 2018, to be held April 14-18, in Chicago, and receive an honorarium of $10,000. The deadline for nominations is Sept. 20, 2017

 

Vanderbilt’s Penson named to JNCI editorial post

David Penson, chair of the Department of Urologic Surgery at Vanderbilt University Medical Center, was named associate editor of The Journal of the National Cancer Institute.

Penson, the Paul V. Hamilton and Virginia E. Howd Professor in Urologic Oncology, also serves as director of the Center for Surgical Quality and Outcomes Research at Vanderbilt University Medical Center.

He joined the Vanderbilt faculty in 2009 as a professor of Urologic Surgery and Medicine and also serves as a faculty member at the VA Tennessee Valley Geriatric Research, Education, and Clinical Center.

 

Roswell Park joins the Oncology Information Exchange Network

Roswell Park Cancer Institute joined joined the Oncology Information Exchange Network (ORIEN), a network of centers collaborating to share information and translate pooled resources into personalized treatments for cancer patients.

With Roswell Park, the number of ORIEN members has expanded to 16.

Formed in 2014, ORIEN seeks to give oncologists ready access to a searchable repository of medical evidence to enhance treatment decision-making and, at the same time, make any new clinical insights or precision treatment approaches informed by this data available to as many eligible patients as possible.

ORIEN allows clinicians and researchers at member centers to leverage multiple data sources and match patients to targeted treatments. Partner centers use a common protocol, known as Total Cancer Care, to track the molecular, clinical and epidemiological data of consenting patients throughout their lifetimes.

The resulting databank represents one of the world’s largest clinically annotated cancer tissue repositories, comprising data from more than 200,000 participating patients.

Researchers at ORIEN member institutions access this shared data warehouse to ask new research questions, develop more effective grant submissions and participate in cross-institutional collaborations and biomarker-discovery projects with academic partners and pharmaceutical companies. This rapid-learning environment allows scientists to analyze and share findings so that that research can be retested and insights re-applied.

M2Gen, a health-informatics company formed to support ORIEN and its members, analyzes data from all participating ORIEN centers to more quickly connect patients with clinical trials based on their molecular profile. ORIEN provides the information and tools needed to study the long-term effects of disease, while assisting cancer center member institutions in their development of precision medicine.

 

Kimmel Cancer Center to open welcome center

A new welcome center at Sidney Kimmel Cancer Center at Thomas Jefferson University will be officially open Oct. 2.

The addition is a part of the cancer center’s patient support system. There, patients and families will be introduced to supportive care services and cancer-related information. The resources are aimed at educating those affected, fostering a sense of community and offering family members a better understanding of cancer diagnosis and management.

Jefferson Oncology Medical Associates are recognized by the NCQA for patient-centered specialty practice and Oncology Medical Home. Both are three-year terms for the achievement status. The patient-care award is the first-ever recognition of its kind by the organization.

SKCC won high marks in the following areas :

  • Track and Coordinate Referrals. The practice coordinates with primary care and referring clinicians to ensure timely exchange of information.

  • Provide Access and Communication. The practice provides timely access to culturally and linguistically appropriate clinical advice and care.

  • Identify and Coordinate Patient Populations. The practice systematically records patient information for patient populations and uses it to coordinate patient care.

  • Plan and Manage Care. The practice collaborates with referring clinicians and the patient/family/ caregiver to plan and manage care and to enable patient self-care.

  • Track and Coordinate Care. The practice systematically tracks tests and referrals to coordinate care with subspecialists and facilities.

  • Measure and Improve Performance. The practice systematically monitors its performance and carries out activities to improve clinical outcomes, efficiency and patient experience.

  • Quality Improvement. The practice monitors at least six standardized oncology quality measures.

  • Oncology Practice Responsibilities. The practice orients patients and their families/caregivers, explains patient and practice responsibilities, discusses available services and care coordination and instructs patients/families/caregivers how to obtain clinical advice 24/7.

  • Comprehensive Health Assessment. The practice has a process for comprehensive health assessment in relevant areas.

  • Evidence-based Pathways. The practice adopts diagnostic and therapeutic, evidence-based pathways and pathways for symptom management.

  • Coordinating Patient-Centered Support During Treatment. The practice works with specialists, consultants, community partners and others in the patient care team to ensure appropriate support.

Bayer’s Aliqopa gets FDA accelerated approval for relapsed follicular lymphoma

Drugs and Targets

Bayer’s Aliqopa gets FDA accelerated approval for relapsed follicular lymphoma

FDA granted accelerated approval to Aliqopa (copanlisib) for the treatment of adults with relapsed follicular lymphoma who have received at least two prior systemic therapies.

Aliqopa, a kinase inhibitor that works by blocking several enzymes that promote cell growth, is sponsored by Bayer Healthcare Pharmaceuticals Inc.

Further clinical trials are required to confirm Aliqopa’s clinical benefit and the sponsor is currently conducting these studies.

The approval of Aliqopa was based on data from a single-arm trial that included 104 patients with follicular B-cell non-Hodgkin lymphoma who had relapsed disease following at least two prior treatments. The trial measured how many patients experienced complete or partial shrinkage of their tumors after treatment (overall response rate). In the trial, 59 percent of patients had a complete or partial response for a median 12.2 months.

“For patients with relapsed follicular lymphoma, the cancer often comes back even after multiple treatments,” said Richard Pazdur, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the Center for Drug Evaluation and Research. “Options are limited for these patients and today’s approval provides an additional choice for treatment, filling an unmet need for them.”

Common side effects of Aliqopa include high blood sugar levels (hyperglycemia), diarrhea, decreased general strength and energy, high blood pressure (hypertension), low levels of certain white blood cells (leukopenia, neutropenia), nausea, lower respiratory tract infections, and low levels of blood platelets (thrombocytopenia).

Serious side effects include infections, high blood sugar levels (hyperglycemia), high blood pressure (hypertension), inflammation of the lung tissue (non-infectious pneumonitis), low levels of certain white blood cells (neutropenia), and severe skin reactions. Women who are pregnant or breastfeeding should not take Aliqopa because it may cause harm to a developing fetus or newborn baby.

Aliqopa was was previously granted the Priority Review and Orphan Drug designations.

 

FDA approves Amgen’s Mvasi, a bevacizumab biosimilar

FDA approved Mvasi (bevacizumab-awwb) as a biosimilar to Avastin (bevacizumab) for the treatment of multiple types of cancer. Mvasi is the first biosimilar approved in the U.S. for the treatment of cancer.

Mvasi is sponsored by Amgen Inc. Avastin, approved in February 2004, is manufactured by Genentech Inc.

“Bringing new biosimilars to patients, especially for diseases where the cost of existing treatments can be high, is an important way to help spur competition that can lower healthcare costs and increase access to important therapies,” FDA Commissioner Scott Gottlieb said in a statement. “We’ll continue to work hard to ensure that biosimilar medications are brought to the market quickly, through a process that makes certain that these new medicines meet the FDA’s rigorous gold standard for safety and effectiveness.”

Mvasi is approved for the treatment of adult patients with certain colorectal, lung, brain, kidney and cervical cancers. Specifically, the approved indications include:

  • Metastatic colorectal cancer, in combination with intravenous 5-fluorouracil-based chemotherapy for first- or second-line treatment. Mvasi is not indicated for the adjuvant treatment of surgically resected colorectal cancer.

  • Metastatic colorectal cancer, in combination with fluoropyrimidine-irinotecan- or fluoropyrmidine-oxaliplatin-based chemotherapy for the second-line treatment of patients who have progressed on a first-line bevacizumab product-containing regimen. Mvasi is not indicated for the adjuvant treatment of surgically resected colorectal cancer.

  • Non-squamous non-small cell lung cancer, in combination with carboplatin and paclitaxel for first line treatment of unresectable, locally advanced, recurrent or metastatic disease.

  • Glioblastoma with progressive disease following prior therapy, based on improvement in objective response rate. No data is available demonstrating improvement in disease-related symptoms or survival with bevacizumab products.

  • Metastatic renal cell carcinoma, in combination with interferon alfa.

  • Cervical cancer that is persistent, recurrent, or metastatic, in combination with paclitaxel and cisplatin or paclitaxel and topotecan.

The approval is based on review of evidence that included extensive structural and functional characterization, animal study data, human pharmacokinetic and pharmacodynamics data, clinical immunogenicity data and other clinical safety and effectiveness data that demonstrates Mvasi is biosimilar to Avastin. It has been approved as a biosimilar, not as an interchangeable product.

 

Cemiplimab receives FDA breakthrough designation for advanced cutaneous squamous cell carcinoma

Regeneron Pharmaceuticals Inc. and Sanofi said FDA has granted Breakthrough Therapy designation status to cemiplimab, an investigational human, monoclonal antibody targeting PD-1– (REGN2810) for the treatment of adults with metastatic cutaneous squamous cell carcinoma and adults with locally advanced and unresectable CSCC.

Regeneron and Sanofi previously reported positive preliminary results for cemiplimab from two expansion cohorts involving 26 advanced CSCC patients in a phase I study of nearly 400 patients, at the American Society of Clinical Oncology Annual Meeting in June 2017.

EMPOWER-CSCC 1, a phase II, potentially pivotal, single-arm, open-label clinical trial of cemiplimab is currently enrolling patients with metastatic CSCC and locally advanced and unresectable CSCC.

Cemiplimab was discovered using Regeneron’s proprietary VelocImmune technology that yields optimized fully-human antibodies, and is being jointly developed by Regeneron and Sanofi under a global collaboration agreement.

Pending results, the companies anticipate submitting a biologics license application for cemiplimab with the FDA in the first quarter of 2018.

Breakthrough Therapy designation serves to expedite the development and review of drugs that target serious or life- threatening conditions. Drugs qualifying for this designation must show credible evidence of a substantial improvement on a clinically significant endpoint over available therapies, or over placebo if there is no available therapy.

The designation includes all of the Fast Track program features, as well as more intensive FDA guidance and discussion.

DOD Kidney Cancer Research Program publishes funding opportunities for FY17

Funding Opportunities

DOD Kidney Cancer Research Program publishes funding opportunities for FY17

The FY17 Defense Appropriations Act provides $10 million to the Department of Defense Kidney Cancer Research Program to support United States Army Medical Research Acquisition Activity.

As directed by the Office of the Assistant Secretary of Defense for Health Affairs, the Defense Health Agency J9, Research and Development Directorate manages the Defense Health Program Research, Development, Test, and Evaluation appropriation. The managing agent for the anticipated Program Announcements/Funding Opportunities is the Congressionally Directed Medical Research Programs.

The KCRP is providing the information in this pre-announcement to allow investigators time to plan and develop applications. FY17 KCRP Program Announcements and General Application Instructions for the following award mechanisms are anticipated to be posted on the Grants.gov website in October 2017. Pre-application (Letter of Intent) and application deadlines will be available when the program announcements are released. This pre-announcement should not be construed as an obligation by the government.

Consortium Development Award

  • Investigators at or above Assistant Professor (or equivalent)

  • Supports infrastructure development to establish the necessary collaborations among a Coordinating Center and Clinical Sites

  • Multi-institution collaboration required

  • Supports clinical trials of novel interventions with the potential to have a significant impact on patient care in kidney cancer

  • Proposed trials may be Phase 0, Phase 1, or Phase 2

  • Minimum of three separate institutes: one Coordinating Center and at least two Clinical Sites (other than the Coordinating Center)

  • Maximum funding of $1.6 million total costs

  • Maximum period of performance is 2 years

  • Awardee will be eligible to apply for FY19 Consortium Award, if funds are available

Idea Development Award

Established Investigators: Independent investigators at or above the level of Assistant Professor (or equivalent) and 10 years or more from a terminal degree; or

Early Career Investigators: Investigators at the level of Assistant Professor, Instructor, or Assistant Research Professor (or equivalent) and less than 10 years from a terminal degree (excluding time spent in medical residency or family medical leave) at the time of application submission deadline are eligible.

  • Supports new ideas that represent innovative, high-risk/high-gain approaches to kidney cancer research, and have the potential to make an important contribution to kidney cancer.

  • Preliminary data is required; need not be in kidney cancer.

  • Innovation and Impact are the most important review criteria.

  • Clinical Trials are not allowed

Areas of Interest include:

  • Microenvironment, Metabolism, Chromatin and Gene Regulation, Rare Cancers, Screening, Early Detection, Novel Imaging Technologies, Liquid Biopsy, Biomarker Development, Prognosis, Targeted Therapies, Immunotherapies, Resistance, Novel Interventions, Surgical, Ablation, Radiation, Prognosis, Managing Toxicity, Survivorship and Patient Experience, Surveillance, Genetic Risk Factors

  • Maximum funding of $400,000 in direct costs (plus indirect costs)

  • Period of performance not to exceed 3 years

Concept Award

  • Investigators at all academic levels

  • Supports highly innovative, untested, potentially groundbreaking concepts in kidney cancer

  • Emphasis on innovation

  • Clinical trials not allowed

  • Preliminary data not allowed

  • Blinded review

  • Maximum funding of $75,000 for direct costs (plus indirect costs)

  • Maximum period of performance is 1 year

 

Translational Research Partnership

  • Investigators at or above the level of Assistant Professor (or equivalent)

  • Supports partnerships between clinicians and laboratory scientists that accelerate ideas in kidney cancer into clinical applications

  • Supports translational correlative studies

  • Preliminary data required

  • Funding for clinical trials not allowed

  • Maximum funding of $600,000 for direct costs (plus indirect costs)

  • Maximum period of performance is 3 years

A pre-application (letter of intent) is required and must be submitted through the electronic Biomedical Research Application Portal (eBRAP) at prior to the pre-application (letter of intent) deadline. All applications must conform to the final Program Announcements and General Application Instructions that will be available for electronic downloading from the Grants.gov website. The application package containing the required forms for each award mechanism will also be found on Grants.gov. A listing of all CDMRP funding opportunities can be obtained on the Grants.gov website by performing a basic search using CFDA Number 12.420.

Applications must be submitted through the Federal Government’s single-entry portal, Grants.gov. Submission deadlines are not available until the Program Announcements are released. For email notification when Program Announcements are released, subscribe to program-specific news and updates under “Email Subscriptions” on the eBRAP homepage. For more information about the KCRP or other CDMRP-administered programs, please visit the CDMRP website.

EC approves Merck’s Keytruda for locally advanced, metastatic urothelial carcinoma

Drugs and Targets

EC approves Merck’s Keytruda for locally advanced, metastatic urothelial carcinoma

The European Commission approved Merck’s Keytruda (pembrolizumab) for the treatment of certain patients with locally advanced or metastatic urothelial carcinoma.

Keytruda is approved for use as monotherapy for the treatment of locally advanced or metastatic urothelial carcinoma in adults who have received prior platinum-containing chemotherapy, as well as adults who are not eligible for cisplatin-containing chemotherapy.

The approval in patients previously treated with platinum-containing chemotherapy was based on superior overall survival for Keytruda versus investigator-choice chemotherapy (paclitaxel, docetaxel, vinflunine) (HR, 0.73 [95% CI: 0.59, 0.91], p=0.002), as demonstrated in the randomized, phase 3 KEYNOTE-045 trial.

The approval in patients ineligible for cisplatin-containing chemotherapy was based on phase II data from the KEYNOTE-052 trial, which demonstrated an overall response rate of 29 percent (95% CI, 25-34). The approval allows for the marketing of Keytruda in these two new indications in all 28 EU member states plus Iceland, Lichtenstein and Norway at a dose of 200 mg every three weeks until disease progression or unacceptable toxicity.

“Despite advances, there remain limited treatment options available to patients with locally advanced or metastatic urothelial carcinoma who are either not eligible to receive cisplatin-containing chemotherapy – which is platinum-based and currently the standard of care – or for those patients whose cancer returns after receiving prior platinum-containing chemotherapy,” said professor Ronald de Wit, group leader for experimental systemic therapy of urogenital cancers, Erasmus MC Cancer Institute. “It is exciting that with this approval of Keytruda, we now also have a new treatment option for patients previously treated with platinum-containing chemotherapy that has shown a clinically meaningful and improved overall survival benefit versus chemotherapy in this difficult-to-treat population.”

The approval in patients previously treated with platinum-containing chemotherapy is based on data from a multicenter, randomized, controlled trial, KEYNOTE-045, investigating Keytruda (pembrolizumab) in patients with locally advanced or metastatic urothelial carcinoma with disease progression on or after platinum-containing chemotherapy.

Patients must have received a first-line platinum-containing regimen for locally advanced/metastatic disease or as neoadjuvant/adjuvant treatment, with recurrence/progression ≤12 months following completion of therapy. Patients were randomized (1:1) to receive either Keytruda 200 mg every three weeks (n=270) or investigator’s choice of any of the following chemotherapy regimens, all given intravenously every three weeks (n=272): paclitaxel 175 mg/m2, docetaxel 75 mg/m2, or vinflunine 320 mg/m2.

Patients were treated with Keytruda until unacceptable toxicity or disease progression, or for up to 24 months in patients without disease progression. The study excluded patients with autoimmune disease, a medical condition that required immunosuppression and patients with more than two prior lines of systemic chemotherapy for metastatic urothelial cancer. The primary efficacy outcomes were OS and progression-free survival (as assessed by BICR using RECIST v1.1); secondary outcome measures were ORR (as assessed by BICR using RECIST v1.1) and duration of response.

In the study, Keytruda demonstrated a statistically significant improvement in OS compared to chemotherapy. Findings demonstrated that KEYTRUDA resulted in a 27 percent reduction in the risk of death compared to chemotherapy – with 155 events (57%) observed in the Keytruda arm, compared to 179 events (66%) in the chemotherapy arm (HR, 0.73 [95% CI: 0.59, 0.91], p=0.002); the median OS was 10.3 months (95% CI: 8.0, 11.8) in the Keytruda (pembrolizumab) arm, compared to 7.4 months (95% CI: 6.1, 8.3) in the chemotherapy arm.

There was no statistically significant difference between Keytruda and chemotherapy with respect to PFS. There were 218 events (81%) observed in the Keytruda arm, compared to 219 events (81%) in the chemotherapy arm (HR, 0.98 [95% CI: 0.81, 1.19], p=0.416). The median PFS was 2.1 months (95% CI: 2.0, 2.2) in the Keytruda arm, compared to 3.3 months (95% CI: 2.3, 3.5) in the chemotherapy arm.

The ORR was 21 percent (95% CI: 16, 27) for patients receiving Keytruda, with a complete response rate of 7 percent and a partial response rate of 14 percent. In the chemotherapy arm, the ORR was 11 percent (95% CI: 8, 16), with a complete response rate of 3 percent and a partial response rate of 8 percent (p=0.001). The median duration of response for patients treated with Keytruda had not yet been reached (range: 1.6+ to 15.6+ months), compared to 4.3 months (range: 1.4+ to 15.4+ months) in the chemotherapy arm.

The approval in patients ineligible for cisplatin-containing chemotherapy is based on data from a multicenter, open-label study, KEYNOTE-052, investigating Keytruda in 370 patients with locally advanced or metastatic urothelial carcinoma who were not eligible for cisplatin-containing chemotherapy. Patients received Keytruda at a dose of 200 mg every three weeks until unacceptable toxicity or disease progression, or for up to 24 months in patients without disease progression.

The study excluded patients with autoimmune disease or a medical condition that required immunosuppression. The primary efficacy outcome measure was ORR as assessed by BICR using RECIST 1.1; secondary efficacy outcome measures were duration of response, PFS, and OS.

The efficacy analysis, with a median follow-up time of 9.5 months, showed an ORR of 29 percent (95% CI: 25, 34), a complete response rate of 7 percent, and a partial response rate of 22 percent. The median duration of response had not been reached (range: 1.4+ to 19.6+ months).

The safety analysis supporting the European approval of Keytruda was based on 3,830 patients with advanced melanoma, non-small cell lung cancer, classical Hodgkin lymphoma, or urothelial carcinoma across four doses (2 mg/kg every three weeks, 200 mg every three weeks, or 10 mg/kg every two or three weeks) in clinical studies.

In this patient population, the most common adverse reactions (>10%) with Keytruda (pembrolizumab) were fatigue (21%), pruritus (16%), rash (13%), diarrhea (12%) and nausea (10%). The majority of adverse reactions reported were of grade I or II severity. The most serious adverse reactions were immune-related adverse reactions and severe infusion-related reactions.

Keytruda is an anti-PD-1 therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. Keytruda is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Studies of Keytruda – from the largest immuno-oncology program in the industry with more than 550 trials – include a wide variety of cancers and treatment settings. The Keytruda clinical program seeks to understand factors that predict a patient’s likelihood of benefitting from treatment with Keytruda, including the exploration of several different biomarkers across a broad range of tumors.

Keytruda is administered as an intravenous infusion over 30 minutes every three weeks for the approved indications. Keytruda for injection is supplied in a 100 mg single-dose vial in the U.S.

FDA grants orphan drug status to Cellect’s ApoGraft for acute and chronic GvHD

Cellect Biotechnology Ltd. said the FDA has granted orphan drug designation for Cellect’s ApoGraft for the prevention of acute and chronic graft versus host disease in transplant patients.

GvHD is a transplant associated disease representing an outcome of two immune systems crashing into each other. In many transplantations from donors, and especially in Bone Marrow Transplantations, the transplanted immune mature cells, as opposed to stem cells, attack the host, patient receiving the transplant, and create severe morbidity and in many cases even death.

This disease happens as a result of current practices being unable to separate the GvHD causing cells from the much needed stem cells. Cellect’s ApoGraft was designed to eliminate immune responses in any transplantation of foreign cells and tissues.