Pharma

Siddhartha Mukherjee on cancer drug development: We need to focus on failing earlier

You know you’re dealing with a compelling topic when Ken Burns is interested in making a documentary about it and cancer certainly is that. Dr. Siddhartha Mukherjee’s Pulitzer Prize-winning book, “The Emperor of All Maladies: a biography of cancer,” is the subject of that documentary series and he gave an overview at Pennsylvania Bio’s Life […]

You know you’re dealing with a compelling topic when Ken Burns is interested in making a documentary about it and cancer certainly is that. Dr. Siddhartha Mukherjee’s Pulitzer Prize-winning book, “The Emperor of All Maladies: a biography of cancer,” is the subject of that documentary series and he gave an overview at Pennsylvania Bio’s Life Sciences Future conference in Philadelphia today. The Columbia Medical School professor and physician also highlighted some life science innovation trends shaping the development of cancer treatments.

Mukherjee emphasized that there needs to be a shift in attention from the late stage of drug development to the early stages where it is less costly to fail. If testing is more rigorous at the earlier stages, then companies needn’t go through the expense of failing later when there is more money at stake.  “We want trials to fail early so they can clear the way for real science and real medicine to advance.”

Historically, the problem has been that few pharma companies view the entrepreneur culture of failing as a way to get to the right answer as something to be encouraged, generally speaking. At least one member of the audience from a big pharma company said he wished more companies embraced that idea. Failure can end careers, he noted.

Mukherjee said he’s encouraged that cancer trials are becoming smaller, allowing for more personalized treatments to improve outcomes. He praised the Whitehead family’s brave decision to permit their daughter, Emily, to receive an experimental T cell immunotherapy. The treatment for her acute lymphoblastic leukemia was developed by researchers at Penn and Children’s Hospital of Philadelphia. She has since become the symbol of its success after the treatment killed the cancer. Novartis licensed the therapy and is advancing it in clinical trials.

Mukherjee likened the size of the cancer fight we currently face to an inverted pyramid with hundreds of thousands of genetic mutations and relatively few targeted therapies. He emphasized a need to reverse the trend.