These researchers have dedicated their careers to finding new treatments and cures for people with cancer.
What is a clinical trial?
A clinical trial is when one or more drugs that are not specifically approved by the Food and Drug Administration for a particular disease are being tested to see how they work for people and what the side effects may be. All trials are different in terms of how “experimental” they are – some trials may be testing a new drug for the very first time in people, some are testing combinations of drugs that have already been approved to see if they work better together, or some trials may use approved drugs or drug combinations but for a different type of cancer than the drugs have already been approved.
How are patients selected for clinical trials?
The clinical trial must be designed with very specific criteria for which patients can participate. Some factors include the type of cancer and whether there are curative treatments already existing for that individual, previous treatments that have been tried, as well as general health and safety factors to make sure patients are not at unreasonable risk by participating in the study. Every trial has different specific criteria.
People who've discovered your breakthrough story with Brittany want to know what treatment helped her - can you share?
Brittany was treated on a combination clinical trial of two drugs, axitinib and pembrolizumab. Both drugs are approved for other types of cancer, but the combination had never been tested for patients with sarcomas, bone and muscle tumors.
Does that mean the same treatment would work for other patients with the same type of cancer?
What we learned in the trial was that patients with her specific type of sarcoma, alveolar soft part sarcoma (ASPS), did quite well with this treatment. More than half of the patients with this type of sarcoma treated on the trial had more than a 30 percent decrease in the size of their tumors. For some patients with ASPS, the tumors grew on treatment, or the drugs stopped working after a period of time. There are other trials using immune-based drugs like pembrolizumab that are also looking effective for ASPS that are ongoing, so I think in general for patients with ASPS where other drugs have not helped, looking to immune therapy is a very good idea.
Will this treatment be available to all sarcoma patients one day?
We learned patients with other types of sarcoma did not do as well as the ASPS patients. There were still a few patients with other sarcomas who had tumor shrinkage or stabilization, some for nearly one year. However, other patients seemed to have their tumors grow even more aggressively after treatment. We are currently studying blood and tumor biopsies from patients treated on this study to understand which patients benefitted from the treatment and why, in hopes that a blood or tumor test might one day be able to pick out those patients in advance who will do well with immune-based treatments and those who likely will not. While this treatment is probably not going to be available for all patients as an approved therapy, there are newer and, hopefully, better clinical trials that will help more patients in the works, so it’s important to talk with your doctor about what trials you might want to consider.