Our research capabilities are growing constantly, breakthroughs are happening more frequently, and we are able to not only save more and more children, but also to help them thrive as cancer survivors. We believe hope may be on the horizon for medulloblastoma, a common pediatric brain tumor.
There is more hope for young cancer patients now than at any point in the past.
What is the most common brain tumor in children?
Medulloblastoma, the most common childhood brain tumor, starts in the lower back part of the brain (cerebellum) and spreads quickly. The cerebellum is responsible for muscle coordination, balance and movement, so kids with this type of brain tumor often have trouble with all three.
Are there any clinical trials for medulloblastoma?
There is a clinical trial underway for medulloblastoma. The trial uses a medication previously only used for heart conditions to treat the tumor. The drug in question is called digoxin, made from the foxglove plant, which was itself well known as a heart treatment hundreds of years ago.
The foxglove is known for its tall, showy flowers that grow on a stalk up to five feet. While it is native to Europe, western Asia and parts of Africa, it’s often grown ornamentally in many parts of the world and is highly toxic if consumed.
The Drug Enforcement Administration (DEA) approved digoxin for heart treatment long ago, and until now that’s all it’s been used for.
Digoxin, from the foxglove plant, has a special chemical makeup that fights against a genetic target.
Ching Lau, MD, the Martin J. Gavin Chair of Hematology and Oncology and Director of Research for the Center for Cancer & Blood Disorders, discovered digoxin’s potential in treating brain tumors in children through an ambitious screening program.
Dr. Lau identified the genetic targets in medulloblastoma tumors and, using a partnership with IBM’s Smash Childhood Cancer network of thousands of linked computers, he began screening several million existing drugs to see if any of them had the chemical makeup that might make them effective against that target. Digoxin was one of those.
Then, after this encouraging discovery, Dr. Lau got to work.
- He tested the drug against tumor cells in a lab, where it worked well.
- That was followed by testing in mice that were carrying actual human tumor tissue. Once again, it appeared to be quite effective.
- With those results, the way was clear for human trials, and those trials are now being developed here at Connecticut Children’s, along with several other hospitals around the country.
About Connecticut Children’s Center for Cancer & Blood Disorders
In addition to the cutting-edge research being conducted, the Center for Cancer & Blood Disorders is providing comprehensive, compassionate care for approximately 200 children with cancer. And those children have access to more than 100 clinical trials, providing real hope in the face of a terrifying disease.