Spotlight on SM Q&A with one of the world’s leading SM doctors
Srdan Verstovsek, M.D., Ph.D., an oncologist at MD Anderson Cancer Center and one of the world’s leading clinical researchers in systemic mastocytosis (SM), speaks about the need – and growing hope – for better SM treatments.
The first patient with SM came to me by chance. It was about 2002, a couple years after completing my fellowship. A man in his late 60s came in with mast cell leukemia, the most aggressive form of SM. The only thing we had to offer was chemotherapy. Very little was known about SM at the time. There were only about 20 published cases of mast cell leukemia in any medical journal at that time, and there was nothing going on in terms of developing new drugs.
The patient died of complications within two months, and spent most of that time in the hospital in pain with a very poor quality of life. It was terrible for him and his family, and for me as a physician. That made a mark on me.
Better treatment. Given the nature of my practice, I see SM patients with advanced forms of the disease or with a heavy symptom burden. In advanced SM, mast cells accumulate in organs, compromising organ function and shortening life expectancy. Chemotherapy is still our most effective approach for these patients, and average survival has not changed.
In indolent SM, life span isn’t affected, but the abnormal proliferation of mast cells causes a host of symptoms, including hives, shortness of breath, headache and pain, that often erode quality of life. A cure is always the ultimate goal, but shy of that, we need therapies that slow or stop the disease and that quiet symptoms without causing harmful side effects. Improving survival for advanced SM patients is critical, but so is improving quality of life for all SM patients
Much more hope. The biology of SM is being dissected, and that will lead to new medicines. We’ve uncovered abnormalities, including a genetic mutation known as KIT D816V that is the primary disease driver in most SM patients. The path from biological discovery to a new medicine is long, but these discoveries are bearing fruit. I’ve been involved in a number of clinical trials of new investigational therapies over the past seven years for patients with SM. In the 1990s, there wasn’t a single study.
Targeted medicine. As we learn more about SM, we are moving away from chemotherapies, which are toxic to both healthy and diseased cells, and toward targeted approaches that attack the abnormal accumulation of mast cells in SM with little to no effect on healthy cells.
Become informed, participate in clinical trials, and help raise awareness about the disease. Patients are at the heart of clinical research. They are crucial to helping researchers gain a better understanding of the disease and study the potential of novel therapies in development. The goal of every interaction I have with patients is to help and encourage them to become as educated as possible, because at the end of the day, they are the ones who are going to take the investigational drug, and only with their participation, will we be able to develop the medicines patients need.
The goal of every interaction I have with patients is to help and encourage them to become as educated as possible, because at the end of the day, they are the ones who are going to take the investigational drug, and only with their participation, will we be able to develop the medicines patients need.
Srdan Verstosek, M.D., PhD.MD Anderson Cancer Center