While some people with systemic mastocytosis (SM) only have milder symptoms, others have symptoms so severe that their ability to go about their daily lives is shut down. It is only human to feel overwhelmed by a chronic disease, especially when you experience symptoms that can’t be controlled by the medications currently available. In these circumstances, can experimental therapies be of help?
What are experimental therapies?
First, it’s important to understand what an experimental therapy is — and what it is not. An experimental therapy is a therapy that is currently being tested in clinical trials, but has not yet been approved. In the U.S., the Food and Drug Administration (FDA) is responsible for approving new medications. A new drug is only approved by the FDA after extensive testing, usually over the course of many years.
New drugs are tested in clinical trials so there is proof they are both effective and safe. Some new drugs create significant side effects: many experimental therapies don’t make it past testing because they led to unexpected adverse reactions in trial participants. Hence, it is for your own safety that these drugs are not available on the market before testing is complete.
Read more about SM testing and diagnosis
Accessing experimental therapies
Despite the potential risks, many patients want to pursue these therapies. There is U.S. legislation called “The Right to Try Act” which allows individuals facing a life-threatening illness to try experimental therapies as a last hope. The criteria around this is strict, and it is best to talk to your doctor if you have questions about it.
Alternatively, you can try to get into a clinical trial that is testing one of these experimental therapies; however, you might receive a placebo instead of the experimental therapy. Once again, the best person to speak to is your doctor.
If you choose to pursue an experimental treatment, be prepared that there may be many bureaucratic hurdles to navigate before you can access the drug.
Current experimental therapies
There are a few experimental therapies for SM currently being researched.
One is called bezuclastinib, and is a tyrosine kinase inhibitor that is currently being tested for advanced SM. Early studies indicate that this therapy shows promise in alleviating mast cell infiltration and the symptoms typically associated with advanced SM. This therapy is being developed by Cogent Biosciences.
Another promising experimental therapy of note is elenestinib, which works by blocking mast cell proliferation and is being investigated for use in indolent SM. Early results show that it reduces symptoms and is associated with few side effects. Elenestinib is being developed by Blueprint Medicines.
The bottom line is that there are a number of experimental therapies currently being tested, and they may soon become available for use — but as of now, they aren’t as well understood as drugs that have been approved for SM. Talk to your doctor if you would like to learn more about these drugs and the timeline of their development.
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