Last week, the New York Times published an article featuring a new approach to drug testing for people with unusual cancers that show particular genetic mutations. Instead of the old protocol, in which people with the same kind of tumor are given a new remedy to try, and comparisons are made with the outcomes of standard treatments, this new approach looks for people whose cellular mutations are the same, regardless of the organ in which the cancer first appeared. Thus, people with cancers that are typically considered unrelated may qualify for these new tests as long as their tumors show these mutations.
Gina Kolata, a long-time science writer for the Times, writes with exceptional enthusiasm about the promise of this approach, but notes possible problems as well. I'll offer some teaser quotes here, but in the interest of fair use I'd prefer you to go read the original. It's not long. We'll wait. ;)
The studies of this new method, called basket studies because they lump together different kinds of cancer, are revolutionary, much smaller than the usual studies, and without control groups of patients who for comparison’s sake receive standard treatment.
Basket studies became possible only recently, when gene sequencing became so good and its price so low that doctors could routinely look for 50, 60 or more known cancer-causing mutations in tumors. At the same time, more and more drugs were being developed to attack those mutations. So even if, as often happens, only a small percentage of patients with a particular tumor type have a particular mutation, it was possible to find a few dozen patients or more for a clinical trial by grouping everyone with that mutation together.
The new studies pose new problems. With no control groups, the effect has to be enormous and unmistakable to show it is not occurring by chance. When everyone gets a drug, it can be hard to know if a side effect is from the drug, a cancer or another disease. And gene mutations can be so rare that patients for a basket study are difficult to find.
If I had advanced cancer for which no other treatment was successful, I also might jump at the chance to participate in such a trial. What's impressive for these treatments is that the responses can be surprisingly good in a high percentage of those who try it. Unfortunately, as with all cancer treatments, nothing is 100% effective, and even this last-ditch hope doesn't pan out for everyone.
But what do you think? Would you try something like this, if you had no other options? Have you heard about anything else with this kind of novelty in traditional clinical trials? Curious minds want to know.
Thanks for sharing tonight; as always, this is an Open Thread. No topic is irrelevant if it concerns your health and recovery.
Monday Night Cancer Club is a Daily Kos group focused on dealing with cancer, primarily for cancer survivors and caregivers, though clinicians, researchers, and others with a special interest are also welcome. Volunteer diarists post Monday evenings between 7:30-8:30 PM ET on topics related to living with cancer, which is very broadly defined to include physical, spiritual, emotional and cognitive aspects. Mindful of the controversies endemic to cancer prevention and treatment, we ask that both diarists and commenters keep an open mind regarding strategies for surviving cancer, whether based in traditional, Eastern, Western, allopathic or other medical practices. This is a club no one wants to join, in truth, and compassion will help us make it through the challenge together.