Ira Pastan, distinguished investigator at NCI, has received the Paul A. Volcker Career Achievement Medal from the Partnership for Public Service for his discovery of moxetumomab pasudotox-tdfk (Lumoxiti), which is indicated for the treatment of relapsed or refractory hairy cell leukemia.
Pastan’s discovery led to the finding of a new class of drugs, recombinant Immunotoxins.
“Dr. Pastan is now building on the success of this new class of drugs he developed called recombinant immunotoxins that could also be effective against solid tumors such as pancreatic and lung cancer, and mesothelioma, in addition to leukemia,” Thomas Misteli, director of cancer research at NIH, said in a statement.
In 2018, FDA approved Lumoxiti to treat relapsed or refractory hairy cell leukemia. Lumoxiti, is the result of decades of research by Pastan, whose discovery has led to a class of drugs that can kill cancer cells while leaving healthy cells intact and save patients’ lives.
When Pastan first came up with his idea of using bacterial toxins for treating cancer, “it was not popular and most immunologists said it would never work, but he has taken this idea and this dream and turned it into reality,” Michael Gottesman, deputy director for Intramural Research at NIH, said in a statement.
“Lumoxiti fills an unmet need for patients with hairy cell leukemia whose disease has progressed after trying other FDA-approved therapies,” Richard Pazdur, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, said in a statement at the time the drug was approved.
Reflecting on the FDA approval, Pastan, who continues to work at age 88, said, “I am very excited about that. It is how things usually begin. Once a drug is approved for one kind of cancer, you try to make it useful for treating other types of cancer.”
Pastan’s research focuses on bacterial protein toxins that are toxic to human and other animal cells.
He worked to direct the biotoxin to target cancer cells. The agents are termed “recombinant immunotoxins,” and they kill cells by interfering with the cell’s ability to build proteins and grow, a mechanism not employed by other anti-cancer agents.
Once Pastan and his lab partners had a drug in hand, they started clinical trials and awaited the results. Pastan said he would always remember the moment he got word of the trial’s effect on patients.
“I was on vacation, and I got a call from my clinical colleague. ‘Ira! The leukemia counts have fallen by 50% and it’s only day one.’ The cancer went away entirely for many patients,” Pastan said. “Eight or 10 years later, some of those patients have survived without any detectable cancer. So, the drug can cure many people.”
Pastan is also known for mentoring other scientists, including Nobel Prize winners Harold Varmus and Robert Lefkowitz as well as Doug Lowy, deputy director of NCI.
