CAR-T therapy successfully treats previously incurable brain tumors in children

The first blow of science against cancer, which is considered incurable. A clinical trial of a CAR-T cell-based treatment showed efficacy against diffuse intrinsic pontine glioma (DIPG), a primary tumor of the central nervous system; that is, it originates in the brain or spinal cord and is more common in children than adults.

Experts say the study, developed largely at Stanford University in California and led by neuro-oncologist Michelle Monnier, is overtly promising about the utility of CAR-T therapy for treating nervous system and brain tumors.

Results published in the journal Natureindicate that treatment improved functional status in 9 of 11 patients in the study who had previously received first-line radiation and chemotherapy. One in four people who showed an excellent response remains healthy four years later.

The patient survives a type of glioma that has a life expectancy of 10 months from diagnosis.

Diffuse midline gliomas are extremely aggressive, invariably fatal, and have few treatment options. The average life expectancy is 10 months from diagnosis, and the 5-year survival rate is less than 1%. Palliative radiotherapy is the only recognized treatment option.

Stanford’s work, praised in the scientific world for its quality, rigor of design, and reliability of its results, has led to improved survival data. “This demonstrates that this type of adaptive cell immunotherapy will become the reference treatment for brain tumors in the short term, and paves the way for the future evolution of this type of therapy to improve their effectiveness,” Luis Alvarez said in a statement to SMC Spain. – Wallina, Head of the Department of Immunotherapy at the 12 October Hospital and the National Cancer Research Center.

In his opinion, the results obtained (a case of complete remission of an incurable pathology) represent a “real push” for CAR-T therapy in terms of safety and effectiveness.

CAR-T (a therapy that involves extracting a patient’s blood and genetically modifying immune T cells to attack cancer cells) has shown very good results in hematological tumors: 40,000 patients have been treated worldwide, but its effectiveness against tumors has not yet been confirmed . be demonstrated.

“These immunotherapies will become the reference treatment for brain tumors in the short term.”

Luis Alvarez-VallinaNational Cancer Research Center

The study “reinforces the idea that brain tumors can be treated with CAR-T,” Manel Juan, head of the immunology service at the Barcelona Clinic, told SMC. “We are in an early but decisive year for the treatment of nervous system tumors with CART-T therapy, which must be approved for systemic use, because unlike other solid tumors, where results are usually clearly insufficient for the CAR-T studied, Regarding brain tumors, several very clear and more than promising proposals have been published since February 2024,” he states.

The authors describe the study as “promising early experience” that “lays the foundation for further optimization of this approach to central nervous system cancers.” However, the results are superior to those achieved with any other treatment to date. According to Dr. Huang, “there is clear hope for effective treatment for those who do not have it.”

“This opens up the possibility of turning a disease that is currently incurable into a chronic disease (an important concept in immunotherapy, which in a sense distinguishes it from radiation therapy and chemotherapy, since immunotherapy treatment can last and be integrated into the body itself as it is ). A live drugwhereas radiation therapy and chemotherapy only work as long as they are used,” says Manel Juan.

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Joseph Corbella