New Options for Rare Cancer Mutations: Basket and 'Just-in-Time' Trials

Maurie Markman, MD


May 08, 2020

This transcript has been edited for clarity.

Hello. I'm Dr Maurie Markman from Cancer Treatment Centers of American in Philadelphia.

I wanted to discuss briefly the important and perhaps controversial topic of basket trials in oncology. It is increasingly clear that the era of precision cancer medicine is upon us. As a result, we are seeing testing being done on patients with advanced cancers which demonstrate the presence of potentially actionable abnormalities, mutations, and rearrangements of genes that appear to be functional. The loss of function or increase in function may lead to an abnormality that, if interfered with, could result in major benefit for the patients. The problem is that many of these abnormalities occur in 2% or less of patients with any given type of cancer. A basket trial includes multiple tumor types, and if a particular genetic abnormality is found, the drug that might interfere with that abnormality is tested across the board in terms of tumor types.

In our own practice, we have seen a large number of patients (in my opinion) who have potentially benefited from this approach—individuals found to have molecular abnormalities and who may go on a variety of clinical trials.

There is also a new concept of trials now, so called "just-in-time" trials. The pharmaceutical industry and the US Food and Drug Administration have put together trials where the trial would not open until there is a particular patient with a particular abnormality. The reason for this is that in any given year, an individual practice might see one or no patients with these abnormalities. Opening a trial quickly that includes these abnormalities could lead to the possibility of a patient benefiting as well as information related to that drug for that particular tumor type. This concept is gaining favor. A paper recently in Nature described an entire country's effort in looking at the presence of abnormalities in patients with advanced cancers in order to determine the value of a particular off-label drug or class of drugs against an abnormality across tumor types in a trial setting.

This is a very important concept and one that I hope we will see much more of in the near future. The goal, of course, is to benefit our patients but also to make certain that if we are going to use a drug against a particular molecular abnormality, we have evidence for the benefit of that strategy. I would encourage those of you who are interested in this concept to read more about basket trials. Certainly in your own practices, if there is the opportunity for you to participate in such trials, I would encourage it.

Maurie Markman, MD, is president of medicine and science at Cancer Treatment Centers of America in Philadelphia. He has more than 20 years of experience in cancer treatment and gynecologic oncology research.

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