Efforts to Advance Cell and Gene Therapies for Children With Cancer

September is Childhood Cancer Awareness Month and an opportunity to share the progress in advancing cell and gene therapy research for children with cancer—and how these therapies have already helped some children defeat cancer.

Carl June, MD, of the University of Pennsylvania

According to the American Childhood Cancer Organization, each year in the United States there are an estimated 15,800 children between the ages of birth and 19 years old who are diagnosed with cancer. Cancer is also the second leading cause of death in US children under the age of 15. While standard treatment options can help some children, there is a dire need for new options—and Alliance for Cancer Gene Therapy (ACGT) has long filled this void.

It was 20 years ago when ACGT funded the research of Carl June, MD, of the University of Pennsylvania. Dr June used funding from ACGT to research and develop the first successful CAR T-cell therapy for cancer. This CAR T-cell therapy was featured in a landmark clinical trial for people diagnosed with leukemia. The clinical study included numerous patients who have been cancer-free for years and are considered cured.

Hideho Okada, MD, PhD, of the University of California, San Francisco

The trial resulted inthe first FDA-approved CAR T-cell therapy for cancer: Kymriah’s approval in 2017 for patients up to 25 years old with acute lymphoblastic leukemia. Since then, there have been five additional CAR T-cell therapies approved by the FDA for various blood cancers in both children and adults.

The successes in cell and gene therapy for blood cancers has inspired ACGT to aim for the same breakthroughs with solid tumors.

ACGT has funded several research projects focused on cell and gene therapies for pediatric cancers. The foundation recently awarded a grant to Hideho Okada, MD, PhD, of the University of California, San Francisco. Dr Okada is running a clinical trial for children with a rare type of brain cancer called diffuse midline glioma.

Visit the ACGT website for the full article about the foundation’s efforts to beat childhood cancer through advancing cell and gene therapy research.