Giusti: The $40 million multiple myeloma dataset will help predict risk and clinical response

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Paul Giusti

Paul Giusti

President and CEO of MMRF

Our hope is that this will inspire other nonprofits and research foundations to look at the GDC as a common data aggregation site where we can all place data and be able to provide it to researchers to access.

This article is part of The Cancer Letter's To The Moon series.

The data on the second most common blood cancer—with genomic information from about 1,400 patients—was compiled by the Multiple Myeloma Research Foundation through a genome mapping initiative and a $40 million network of clinical trials.

“There are 38 other cancers on the GDC site, and multiple myeloma is now the largest by far, given this data contribution,” said Paul Giusti, president and CEO of MMRF. “One of the things we’re very proud of is that we are the first research foundation to contribute data to the GDC.

“Our hope is that this will inspire other nonprofits and research foundations to look at the GDC as a common data aggregation site where we can all place data and be able to provide it to researchers to access.”

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

Matthew Ong: What was the impetus for MMRF to contribute data to the GDC? How did those conversations come about?

Paul Giusti: It’s very much within the mission of the MMRF to place data into the public domain. We feel that by having more researchers, more individuals having access to the data, that the research will be faster, you’ll have more people looking at it, it will advance the science more quickly, and we’ll get to a cure faster.

So, we have always been advocates of getting data into the public domain as quickly as we can, and we saw this opportunity with the GDC and working with Lou Staudt [director of the NCI Center for Cancer Genomics] as a way that we can demonstrate leadership in that regard.

In our conversation with Lou, he was very interested in having a nonprofit foundation that has such well-curated data get that into the GDC. We were very excited about working with him to make that happen.

Did this involve the Harvard Business School Kraft Precision Medicine Accelerator that Kathy [Giusti] is working on?

PG: The data transfer was directly between the MMRF and the GDC. However, the MMRF participated in a meeting as part of the Kraft Precision Medicine Accelerator in June where one of the big topics that we talked about was data sharing. The Kraft Accelerator’s goal is to accelerate precision medicine so it certainly helped to move this along. Kathy has a long history of working with Lou Staudt, as does Daniel Auclair, who is our senior vice president. The HBS/Kraft effort also did a terrific job in outlining the data landscape.

Since we’re talking about data, we’re talking about the aggregation, we’re talking about analytics, and this acted as a catalyst to move the effort forward.

Kathy is part of the White House Precision Medicine Initiative, and Daniel Auclair monitors this very closely. We certainly have kept abreast with all of this, and we are well attuned to what was going on in the efforts on the GDC.

How many patient cases will the CoMMpass genomic study contribute to GDC, say, by 2020, or when the study has been completed?

PG: The CoMMpass data increases the amount of data stored in the GDC by 50 percent. We are contributing data from our CoMMpass study as well as our Multiple Myeloma Genomics Initiative (MMGI) that we had instituted a while ago.

There are 38 other cancers on the GDC site, and multiple myeloma is now the largest by far, given this data contribution. One of the things we’re very proud of is that we are the first research foundation to contribute data to the GDC. Our hope is that this will inspire other nonprofits and research foundations to look at the GDC as a common data aggregation site where we can all place data and be able to provide it to researchers to access.

It’s not only that we are the first, but also that we are the largest, that helped move the needle in regard to the amount of data on the GDC.

What is unique about the data that MMRF will be contributing?

PG: The reason the CoMMpass data is so valuable is that we took almost 1,200 newly-diagnosed multiple myeloma patients and, before they received treatment, we did full genomic sequencing of those patients. So, we have that initial genomic profile at the time of their diagnosis and before they received any treatment.

Then, these patients are all being followed in a longitudinal study. In other words, we’re keeping track of what treatments they are getting, when they are getting those treatments, how they respond to those treatments, and we’re doing that for a period of eight years. In the event that a patient progresses and their disease advances, there will be another bone marrow biopsy and another genomic profile done to see what’s changed and why that has changed.

First of all, it’s hard to do this and it’s expensive. This is a more than $40 million effort, so not everybody is doing this and they just can’t afford to conduct a study like CoMMpass put the data in the public domain.

What’s unique is that it is one of the best-curated databases, it’s the largest genomic database in all cancers and it follows these patients all longitudinally for all these years so that you can see trends and progress. Our hope is, someday, that you’ll be able to look at their genomic information, their profile, at the point of diagnosis and be predictive about whether they were high risk, whether they’re going to be great responders to treatment, and what treatments are they responding best to.

All of this is in the real world, it isn’t just a clinical trial with a couple of arms, and this is why it’s so exciting. The database is very well curated and very well organized, and it’s the gold standard of myeloma data. It helps drive the precision medicine model forward, because we’re very proud to have one of the only—or the only—end-to-end precision medicine models in oncology in that we have a data bank, a learning network, and the clinic.

That’s where we’re able to take data—and CoMMpass is a big part of generating that data—and then look at it in this learning network and do these correlative studies so that we can see whether this looks predictive, whether it works, and then based on that, put together clinical trials that are able to drive new treatments for our patients.

You mentioned that this is a $40 million effort—how did MMRF raise the funds?

PG: It was a combination: roughly half of it was from our pharmaceutical partners, and half of it was from philanthropists—we went out as the MMRF and raised that money and then our pharma partners contributed half of it in a precompetitive consortium. What that allowed them to do is to get a first look at the data, and before it all went out into the public domain. It was a 50-50 partnership.

What else is MMRF currently working on?

PG: We’re looking at three major initiatives. First, we will continue to advance the precision medicine model by aggregating more data—building on our CoMMpass database. Second, we will use data analytics to better understand that data. In particular, with 10 new drugs approved by the FDA to treat multiple myeloma in the past 10 years, we need to better understand which drugs in which combinations are most efficacious for each patient. Third, we need to innovate in clinical trials to speed the process. This includes work in using minimal residual disease (MRD) as a surrogate endpoint for clinical trial design.

While we have experienced remarkable progress, there is so much more to do. We always keep our patients at the center of everything—answering the questions they’re asking, helping them make decisions in their treatment and ultimately finding a cure.

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