St. Jude researcher receives grant to develop gene therapies for pediatric sarcoma
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Alliance for Cancer Gene Therapy awarded a $500,000 grant to Stephen Gottschalk, MD, to develop gene therapies for pediatric sarcoma.
Gottschalk, chair of the department of bone marrow transplantation and cellular therapy at St. Jude Children’s Research Hospital, has conducted more than 25 clinical trials. He most recently worked on gene therapies for patients with X-linked severe combined immunodeficiency.
The grant will support Gottschalk’s lab in its effort to develop a cell therapy approach to treating pediatric sarcoma using genetically engineered immune cells that attack the disease.
“We are investigating cell therapies with T cells that are genetically modified to recognize tumor cells and the blood vessels that support their growth,” Gottschalk told Healio. “We are planning to evaluate our approach in preclinical models that closely mimic human disease.”
Meaningful treatments for children with solid tumors are urgently needed, according to Kevin Honeycutt, CEO and president of Alliance for Cancer Gene Therapy (ACGT).
“Dr. Gottschalk’s research combines state-of-the-art technologies and methodologies to attack two targeted gene proteins found in pediatric sarcomas,” Honeycutt said in a press release. “The members of the ACGT Scientific Advisory Council are enthusiastic about Dr. Gottschalk’s vision and very promising preliminary data.”
Although chimeric antigen receptor and other T-cell therapies have demonstrated success in hematologic malignancies, they have been less effective in clinical studies for treating solid tumors. This lack of clinical success extends to sarcoma.
“Limited efficacy is most likely multifactorial and includes a limited array of targetable solid tumor-specific antigens and the solid tumor microenvironment that actively inhibits T-cell function. Several approaches are currently being developed to overcome these limitations, including ours in which we aim to target multiple antigens and destroy the tumor vasculature,” Gottschalk told Healio.
Developing cell-based therapies requires a team approach in which clinicians and researchers work closely in “iterative cycles of ‘bench-to-bedside-and-back’ translational research,” he said. This typically means initial clinical testing of disease-specific therapies that do not result in high cure rates.
“In the long run, we hope that our cell therapy approach benefits patients with recurrent or refractory disease who fail conventional therapies,” Gottschalk said. “Cell therapy approaches like ours have the potential to reduce treatment complications for all patients because they have greater tumor specificity than conventional therapies.” – by Drew Amorosi
For more information:
Stephen Gottschalk, MD, can be reached at St. Jude Children’s Research Hospital, MS 321, Room I4112, 262 Danny Thomas Place, Memphis, TN 38105-3678; email: stephen.gottschalk@stjude.org.
Disclosure: Gottschalk reports patents and patent applications in the field of cell and gene therapy for cancer; a research collaboration with TESSA Therapeutics; and a data safety monitoring board role with Immatics US.