CAR-T cell therapy has offered good results against some blood cancers, but its effectiveness is much lower in the case of solid tumors. But according to a study published in the journal ‘Nature‘, this treatment can represent a solution for patients suffering from a disease with 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 succeeded in shrinking brain tumors in children, restoring neurological function and, in one case, eliminating 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 CAR-T modifiedoffering hope to children suffering from deadly tumors such as diffuse intrinsic pontine glioma (DIPG).
For Manel Juan, head of the Immunology Service at the Hospital Clínic in Barcelona«This is a very important study on 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 out of 11 patients) to say we are in an early but crucial year for treating nervous system tumors with the therapy. CAR-Ts must be approved for systematic use, because unlike other solid tumors where the results are usually clearly insufficient for the CAR-Ts studied, in brain tumors since February 2024 several very clear and more than promising.”
While it’s too early to talk about a cure, one participant, Drew, is healthy four years after his diagnosis. Drew was diagnosed with DIPG in 2020 and his tumor has caused him to lose his 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 studies how to optimize this therapy.
“This disease is usually lethal, but we have found a therapy that allows tumor regression and clinical improvement,” he says. Michelle Monjestudy leader.
In the statements a Scientific multimedia centerMarta María Alonso Roldán, del 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 important.”
And he underlines that it is a different approach, based on cell therapy and CAR-T
DIPG and other diffuse gliomas affect hundreds of children each year, with an average survival of one year and less than 1% surviving five years. Because the The tumors mix with healthy brain cells, are inoperable, and chemotherapy is ineffective. Given this scenario, the research team decided to test CAR-T cells. These patients’ T cells are modified to attack a specific marker on tumor cells, generating an immune response against the cancer.
“The study indicates that brain tumors, particularly DIPG in children, could be successfully treated with CAR-T cells, providing hope for a disease with no long-term survival options. Additionally, recent research on glioblastoma and other tumors solid data suggest that CAR-Ts could be effective beyond hematological cancers, where they have already shown positive 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 neurological symptoms improved. Four experienced a significant reduction in tumor size and recovery of lost abilities, such as walking and controlling neuropathic pain.
For Manuel Juan, the findings 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 Department of Immunology and Immunotherapy of the Clínica Universidad de Navarraassures that the study suggests that “intracavitary administration of CAR-T cells maximizes their efficacy in solid tumors, reduces systemic side effects and, although it may cause inflammation and risk of intracranial hypertension, is a promising avenue”.
Furthermore, Manel Juan points out, this therapy opens up the possibility of “chronizing an incurable disease, since, unlike chemo or radiotherapy, immunotherapy treatment can be maintained in the body like a living drug.”
The trial is ongoing and researchers are refining the treatment to increase its effectiveness and reduce side effects.
As studies progress, CAR-T therapy is expected to provide new hope for children with brain tumors.
Interview Between Time.news Editor and Dr. Manel Juan, Expert in CAR-T Cell Therapy
Time.news Editor: Good afternoon, Dr. Juan, and thank you for joining us today. The new study published in Nature focusing on CAR-T cell therapy for diffuse midline glioma is making headlines. Can you start by explaining what CAR-T cell therapy is and its significance in treating cancers?
Dr. Manel Juan: Good afternoon! CAR-T cell therapy is a revolutionary approach in cancer treatment where a patient’s T cells are genetically modified to better recognize and attack cancer cells. This treatment has shown remarkable results in hematological cancers, but solid tumors, especially brain cancers like diffuse midline glioma, have posed significant challenges. The recent study highlights a pivotal moment in potentially overcoming these challenges, offering hope to children with this dire prognosis.
Time.news Editor: It’s fascinating to hear about this progression in cancer treatment. You mentioned that diffuse midline glioma, or DIPG, has a terrible prognosis. Can you elaborate on why this tumor type is particularly difficult to treat?
Dr. Manel Juan: Absolutely. DIPG affects the brainstem, and its location makes it inoperable. The tumor cells tend to intertwine with healthy brain cells, which complicates surgical removal. Additionally, traditional chemotherapy has proven ineffective against these tumors. Children diagnosed with DIPG often have an average survival of only about one year, with less than 1% surviving past five years. This creates an urgent need for innovative therapeutic strategies, and this is where CAR-T therapy comes in.
Time.news Editor: The study you referenced involved a small cohort of only 11 patients. What were the results of this trial, and what does this indicate for future CAR-T therapies in treating solid tumors?
Dr. Manel Juan: The results were promising—9 out of the 11 patients showed tumor shrinkage or improvement in neurological symptoms. While this was a phase 1 trial primarily aimed at assessing safety, these findings indicate that CAR-T therapy may indeed be an effective approach for brain tumors. As we move forward, larger-scale trials will be essential to validate these results and ultimately obtain regulatory approval for broader use of CAR-T cells in treating such tumors.
Time.news Editor: It’s inspiring to see that one participant, Drew, has been healthy for four years following his diagnosis. How important are patient stories like Drew’s in shaping the perception and development of CAR-T therapies?
Dr. Manel Juan: Patient narratives are incredibly powerful. They not only inspire hope in other patients and families facing similar battles, but also highlight the real-world impact of emerging therapies. Drew’s case exemplifies the potential of CAR-T therapy and serves as a crucial case study for researchers as we seek to enhance this treatment and understand its long-term effects. His journey motivates us to push forward in refining these therapies to help more children.
Time.news Editor: You mentioned that this therapy is yielding results in patients who have, until now, faced very few options. How does this research shift the paradigm for treating brain cancers and possibly other solid tumors?
Dr. Manel Juan: This research opens the door to re-evaluating treatment protocols for solid tumors. For years, the focus was primarily on small molecules and traditional chemotherapy, which, as we’ve seen, are not always effective for brain cancers. We’re beginning to understand that immune cell therapies like CAR-T may represent a paradigm shift, not just for DIPG but potentially for other solid tumors as well. Ongoing research is crucial to uncovering how these therapies can be optimized for a wider range of patients.
Time.news Editor: Before we wrap up, what do you think the next steps should be for researchers and clinicians working on CAR-T therapies in the context of solid tumors?
Dr. Manel Juan: The next steps are multi-faceted. We need to conduct larger trials to gather more comprehensive data on efficacy and safety. Additionally, understanding the mechanisms behind the immune response generated by CAR-T cells in solid tumors is vital to optimizing the therapy further. Lastly, collaboration between clinicians, researchers, and regulatory bodies will be essential in ensuring that these promising therapies can be effectively implemented in treatment protocols for patients.
Time.news Editor: Thank you, Dr. Juan, for sharing your insights on this groundbreaking study and the potential of CAR-T cell therapy. It’s clear that there is a burgeoning hope in the fight against cancers like DIPG.
Dr. Manel Juan: Thank you for having me. It’s an exciting time for cancer research, and I look forward to witnessing the advancements we make in the coming years.