Finding New Treatments for Pediatric Cancer

Finding New Treatments for Pediatric Cancer

At Vanderbilt University Medical Center, we're aggresively pursuing new treatments for children who have cancer. We're striving to create new drugs that target the genetic factors behind Ewing sarcoma, a rare type of cancer that forms in bone.

Why is Vanderbilt a leader in drug discovery?
Vanderbilt has a unique culture of collaboration between experts from many fields. The Vanderbilt Institute of Chemical Biology (VICB) includes team members with experience in the pharmaceutical industry and offers the most advanced technology available to support drug discovery. Also, nonprofit academic centers like Vanderbilt can focus on rare diseases that drug companies may not pursue.

What is Vanderbilt doing to advance treatment for Ewing sarcoma?
Patrick Grohar, M.D., Ph.D., recently joined the team at the Monroe Carell Jr. Children’s Hospital at Vanderbilt. He came to Vanderbilt from the National Cancer Institute (NCI) to work with our drug discovery team on his research to develop drugs to target a specific genetic change (mutation) that is involved in Ewing sarcoma.

Is this treatment available for patients yet?
Only in an early-stage clinical trial. If the trial proves successful, it may be offered to patients at Vanderbilt and other centers across the country.

Does this research apply to other types of cancer?
Yes. The gene Dr. Grohar studies is a type of gene that turns on other genes that contribute to cancer. Many other types of cancers have mutuations in similar genes. So far, no one has figured out how to target this type of gene; they're considered “undruggable.” If Dr. Grohar and his colleagues can block the action of this gene in Ewing sarcoma, it could open the door to a new way to treat other cancers.

Sarcoma Care at Vanderbilt
Our team includes some of the best orthopaedic oncology surgeons in the country. Learn more here.


Make an appointment with a pediatric cancer specialist: call (615) 936-1762.


Learn more about the Childhood Cancer Program here.