St. Jude Children’s Research Hospital Scientist Receives ACGT Grant to Accelerate Gene Therapy Treatment for Pediatric Sarcoma

Feb 03, 2020

Approach will use Genetically Engineered Immune Cells to Attack Pediatric Sarcoma

STAMFORD, Conn. — An innovative approach to tackling the challenges of pediatric sarcomas is being pursued by researchers at St. Jude Children’s Research Hospital (Memphis, Tennessee) with a new grant from Alliance for Cancer Gene Therapy (ACGT).

The $500,000 ACGT grant supports Stephen Gottschalk, MD, chair of Bone Marrow Transplantation and Cellular Therapy at St. Jude’s, in exploring the use of genetically engineered immune cells to attack pediatric sarcoma. Dr. Gottschalk and his team target not only the cancer, but the blood vessels supporting tumor growth. Dr. Gottschalk has conducted more than 25 previous clinical trials and most recently worked on gene therapies for treating infants with the “bubble boy” disease.

“There is an urgent need to develop meaningful treatments for children with solid tumors,” says Kevin Honeycutt, CEO and president of ACGT. “Dr. Gottschalk’s research combines state-of-the-art technologies and methodologies to attack two targeted gene proteins found in pediatric sarcomas. The members of the ACGT Scientific Advisory Council are enthusiastic about Dr. Gottschalk’s vision and very promising preliminary data.”

“Pediatric sarcoma, which comes back after initial therapy or is present at multiple sites, is difficult to treat even with the most aggressive, currently available therapies,” noted Dr. Gottschalk. “Because Sarcomas are a relatively uncommon group of cancers, accounting for about 15 percent of childhood cancers, improving outcomes has been challenging. This ACGT grant enables me to further investigate how we can leverage different proteins to attack not only abnormal sarcoma cells but also the blood vessels that provide nutrients to tumors. We’ve seen this approach work in mice. Given the resources from ACGT, we can move ahead much faster to complete all the preclinical studies that are needed to evaluate our approach in the clinic.”

Dr. Savio Woo, chairman emeritus of the ACGT Scientific Advisory Council and recently retired from the Icahn School of Medicine at Mount Sinai (New York, New York), dedicated his career to the pursuit of fundamental science and technology development in gene and cell therapy, and translating those laboratory advances into direct patient benefit. Dr. Gottschalk, who did a post-doctoral session with Dr. Woo from 1992 to 1995, acknowledges that it was Dr. Woo’s commitment to ACGT that drove his quest to secure a grant from the organization.

“Being connected to the organization that Dr. Savio Woo helped direct on the scientific side, is a great honor,” said Dr. Gottschalk.