Using a novel TCR therapy to treat children with a rare brain tumor.
STAMFORD, Conn., April 6, 2023 — Thanks to a $500,000 grant from Alliance for Cancer Gene Therapy (ACGT), scientists at the University of California San Francisco (UCSF) are planning to launch an innovative clinical trial to study engineered T cells to treat children with a rare brain cancer.
The grant was awarded to Hideho Okada, MD, PhD, a Professor of Neurological Surgery at the UCSF School of Medicine and the Director of UCSF’s Brain Tumor Immunotherapy Center. Dr. Okada conducted one of the first immune gene therapy trials in patients with malignant glioma. Now, with ACGT’s support, he is developing a T-cell receptor therapy – also called a TCR therapy – to target a specific protein mutation expressed in diffuse midline glioma, which is a type of rare brain tumor that mostly affects children. The therapy will be used in a phase 1 clinical trial for children with diffuse midline glioma, or DMG, at the UCSF Helen Diller Family Comprehensive Cancer Center.
“I’m optimistic about cell and gene therapy for treating patients with these tumors,” says Dr. Okada. “I’m very grateful for the support of ACGT for this clinical trial because it’s so hard to implement a phase 1 clinical trial for cell and gene therapy as an academic researcher.
“This project has the potential to significantly impact the treatment approach for a disease for which we have not achieved any improvement for the last several decades and is the first of its kind for this devastating disease.”
Children who are newly diagnosed with diffuse midline glioma that expresses the H3.3K27M mutation may be eligible for this clinical trial. Patients will undergo collection of their own T cells, and these T cells will then be engineered in the laboratory to recognize the specific mutation. These modified T cells will then be given back to the participants once they completed standard of care radiation therapy and a short chemotherapy course. The hope is that these modified T cells will now be able to kill the cancer cells carrying the specific H3.3K27M mutation. The phase 1 clinical trial is being planned to assess if such a therapeutic approach is feasible and safe for patients.
“We have been able to show in the laboratory that we can make T cells that specifically recognize DMG cells with the H3.3K27M genetic mutation and that these specific T cells can kill tumor cells,” says Dr. Okada. “Based on these exciting data, we propose to test this new therapy approach in the clinic.”
“Dr. Hideho Okada’s proposal is an exciting opportunity to develop a cell and gene therapy for a patient population much in need – children with brain cancer,” says Kevin Honeycutt, ACGT chief executive officer and president. “Success in developing a T-cell receptor therapy for children with this brain cancer would have a significant impact – not just for this cancer, but many others as well.”
“ACGT’s Scientific Advisory Council is excited by the potential of this clinical trial to advance cell and gene therapies for one of the most difficult to treat cancers.”
For more information, visit acgtfoundation.org or join the Alliance for Cancer Gene Therapy community on Facebook, Twitter, LinkedIn, Instagram and YouTube.
# # # # #
Alliance for Cancer Gene Therapy (ACGT)
For more than 20 years, Alliance for Cancer Gene Therapy has funded research that is bringing innovative treatment options to people living with deadly cancers – treatments that save lives and offer new hope to all cancer patients. Alliance for Cancer Gene Therapy funds researchers who are pioneering the potential of cancer cell and gene therapy – talented visionaries whose scientific advancements are driving the development of groundbreaking treatments for ovarian, prostate, sarcoma, glioblastoma, melanoma and pancreatic cancers.
100% of all public funds raised by Alliance for Cancer Gene Therapy directly support research and programs. For more information, visit acgtfoundation.org, call (203) 358-5055, or join the Alliance for Cancer Gene Therapy community on Facebook, Twitter, LinkedIn, Instagram and YouTube.
About UCSF and the Helen Diller Family Comprehensive Cancer Center
The Helen Diller Family Comprehensive Cancer Center, which is affiliated with UCSF and the UCSF School of Medicine, is a National Cancer Institute-designated comprehensive cancer center. The “comprehensive” designation is NCI’s highest ranking and affirms UCSF’s commitment to pursuing scientific excellence in the treatment of cancer patients.
The Helen Diller Family Comprehensive Cancer Center offers patient care at five medical centers in San Francisco: UCSF Medical Center at Mount Zion, UCSF Medical Center at Mission Bay, UCSF Medical Center at Parnassus, Zuckerberg San Francisco General Hospital and Trauma Center, and the San Francisco Veterans Affairs Medical Center.
For more information about UCSF, visit the Helen Diller Family Comprehensive Cancer Center website.
