CAR-T cell therapy has offered good results against some blood tumors, but its effectiveness is much less in the case of solid tumors. But according to a study published in the magazine ‘Nature‘, this treatment may be a solution for patients with a disease that has a poor prognosis, diffuse midline glioma, a tumor of the nervous system considered incurable.
The researchers of Stanford Medicine (USA) shows that this immune cell therapy managed to reduce brain tumors in children, restore neurological functions, and in one case, eliminate all detectable signs of a brain cancer with a very poor prognosis.
This clinical trial marks a milestone in the fight against solid tumors in the brain, using immune cells Modified CAR-Toffering hope for children with lethal tumors such as diffuse intrinsic pontine glioma (DIPG).
For Manel Juan, head of the Immunology Service at the Hospital Clínic of Barcelona«This is a very important study in a disease that has a terrible prognosis. With only 11 patients in a phase 1 trial to demonstrate safety, it provides enough response data (9 of 11 patients) to be able to say that we are in a previous but crucial year for the treatment of tumors of the nervous system with therapy. CAR-T be approved for systematic use, because unlike other solid tumors where the results are usually clearly insufficient for the CAR-T studied, in brain tumors since February 2024, several very clear and more than promising proposals have been published. ».
Although it is early to talk about a cure, one of the participants, Drew, is healthy four years after his diagnosis. Drew was diagnosed with DIPG in 2020 and his tumor caused loss of mobility and balance. He is currently in his third year of college and it is hoped that his case will inspire improvements in therapy. Drew continues to receive treatment every few months as the Stanford team investigates how to optimize this therapy.
“This disease is usually lethal, but we have found a therapy that achieves tumor regression and clinical improvement,” he says. Michelle Monjestudy leader.
In statements to Science Media CenterMarta María Alonso Roldán, of the TOP and the Clínica Universidad de Navarra, says that “work like this, which is different from continuing to test small molecules or conventional chemotherapy, is very relevant.”
And he highlights that it is a different approach based on cell therapy and CAR-T, “which have given very good results in hematological tumors but whose effectiveness in solid tumors has yet to be demonstrated.”
DIPG and other diffuse gliomas affect hundreds of children each year, with a median survival of one year and less than 1% surviving five years. Because the Tumors mix with healthy cells in the brain, are not operable and chemotherapy is ineffective. Given this scenario, the research team decided to test CAR-T cells. These patient’s own T cells are modified to attack a specific marker on tumor cells, generating an immune response against cancer.
Although it is early to talk about a cure, one of the participants, Drew, is healthy four years after his diagnosis
“The study indicates that brain tumors, especially DIPG in children, could be successfully treated with CAR-T cells, providing hope for a disease without long-term survival options. Furthermore, recent research in glioblastoma and other solid tumors suggests that CAR-T may be effective beyond hematological cancers, where they have already shown successful results in thousands of patients, Manel Juan tells SMC.
The participants, with an average age of 15 years and recently diagnosed, received CAR-T cells after chemotherapy to avoid immune attacks. After the first intravenous infusion, participants were monitored for side effects and subsequently received doses into the cerebrospinal fluid, with fewer adverse effects.
The response was positive: In 9 participants the tumor shrank or their neurological symptoms improved. Four experienced a significant reduction in tumor size and recovery of lost skills, such as walking and controlling neuropathic pain.
For Manuel Juan, the results suggest that administering CAR-T cells directly into the central nervous system is more convenient, less toxic and potentially more effective.
Ignacio Melero, CIMA researcher and co-director of the department of Immunology and Immunotherapy of the Clínica Universidad de Navarraassures that the study suggests that the “intracavitary administration of CAR-T cells maximizes their effectiveness in solid tumors, reduces systemic side effects and, although it can cause inflammation and risk of intracranial hypertension, it is a promising avenue.”
This therapy opens the possibility of making an incurable disease chronic.
Furthermore, Manel Juan points out, this therapy opens the possibility of “chronizing an incurable disease, since, unlike chemo or radiotherapy, the immunotherapy treatment can be maintained in the body as a living drug.”
The trial is ongoing, and researchers are refining the treatment to increase its effectiveness and reduce side effects.
As trials progress, CAR-T therapy is expected to represent new hope for children with brain tumors.
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