Stephen Skapek, M.D. Answers Questions On: Rhabdomyosarcoma

We are learning that childhood cancers are very different from adult cancers, biologically speaking. Adult cancers usually arise from environmental factors that cause DNA damage, such as cigarette smoking. In childhood cancer, it seems to be a genetic influence that affects cells as they’re developing.

Rhabdomyosarcoma cells look and behave a lot like skeletal muscle cells when they’re still forming in the womb. They proliferate rapidly and they migrate easily to different places in the body, just like developing muscle cells do. In a sense, these cancer cells have co-opted these normal developmental processes to become a cancer.

One main focus in my research lab is to understand the relationship between the rhabdomyosarcoma cells and the normal skeletal muscle cells. Instead of using lots of powerful chemotherapy to try to kill all the cancer cells – which frankly we have a hard time doing – we could someday reprogram these cells to behave like normal muscle cells.

Effective treatment of rhabdomyosarcoma starts with choosing a center that has a lot of experience and expertise in treating this type of cancer – even if just to make sure you have the right diagnosis. Rhabdomyosarcoma fits within the larger group of childhood cancers that can all look the same under the microscope. It takes a trained oncologist to make an accurate diagnosis.

From there, cancers like rhabdomyosarcoma generally involve a multidisciplinary approach that includes surgical removal of the parts of the tumor that can be removed, radiation treatment for the tumor that you can see but can’t remove, and then intensive chemotherapy to attack the cancer cells in the body that can’t be seen. That type of multifaceted, multidisciplinary treatment can last up to three to nine months. But for a lot of that time the child is out of the hospital, recovering at home and just going in for frequent check-ups to make sure everything is going in the right direction.

Today there are subsets of rhabdomyosarcoma in which 80 to 90 percent of children can expect to be cured. That’s a big improvement from 30 years ago.

But for the kids who have the most advanced disease, the survival really hasn’t improved very much. And even the kids who are cured are being cured at great cost, because they get really intensive chemotherapy, and many of them suffer longstanding side effects from the treatment.

I really think we’re on the cusp of having major breakthroughs that lead quickly to better treatments. We’re in the midst of an explosion of biological, technological, and computational advances, which is going to allow us to merge computing power with the molecular and genetic understanding of cancer to develop new therapeutic agents. So I think, in the not-too-distant future, we’ll be able to help the kids with this disease, that’s currently hard to cure, and also make the treatment easier for all children who develop rhabdomyosarcoma.