New hope is on the horizon for pediatric patients with brain tumors. A novel treatment based on engineered immune cells, known as CAR-T therapy, has shown promise in shrinking tumors and restoring neurological function in children with aggressive forms of cancer. The results of this clinical trial, conducted at Stanford Medicine in California, were published in the journal Nature.
The trial involved eleven young patients, of whom nine showed significant improvements. Of these, four saw a reduction in tumor volume of more than half. One exceptional case saw a patient achieve a complete response: the tumor disappeared from brain scans. Although it is premature to declare the patient definitively cured, his health remains stable four years after diagnosis.
The study’s lead author, Michelle Monje, said: “This cancer is a universally fatal disease, and we have found a therapy that can cause significant tumor regression and clinical improvement.” These results represent one of the first successes in the fight against solid tumors using CAR-T therapy. This innovative form of treatment recently received Advanced Therapy Designation from the U.S. Food and Drug Administration (FDA), allowing researchers to accelerate the approval process.
Diffuse midline gliomas, which can develop in the brain or spinal cord, have a median survival of about a year without effective therapies. CAR-T therapy involves a process in which a patient’s own T cells are harvested and genetically modified to attack a specific molecular target: the tumor antigen GD2. These engineered cells are then reinfused into the patient, triggering a targeted immune response against the tumor cells. Although CAR-T cells have been approved for the treatment of blood cancers since 2017, their application to solid tumors has shown more challenges.
With the encouraging results to date, CAR-T therapy could represent a significant change in the treatment of pediatric brain tumors. Although there are still many unknowns to address, the advancement of immunological technologies offers new hope and opportunities to improve the life expectancy of young patients affected by these devastating diseases.
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