Toni Dimaano Science 12.12.2022 06:Dec AM EST

CAR-T Cell Therapy Pilot Trial Demonstrates Successful Treatment Among Patients With Brain, Spinal Cord Lymphoma

A small pilot trial for patients with brain or spinal cord lymphoma has shown that CAR-T cell therapy, known as axicabtagene ciloleucel (axi-cel), can be a viable treatment option.

According to Interesting Engineer ing, the Dana-Farber Cancer Institute investigators announced on Sunday that early results suggest the applicability of CAR-T cells to improve patient outcomes.

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The research features an in-depth molecular study of individual CAR-T cells separated from the blood and cerebrospinal fluid (CSF), which surround the brain and the spinal cord.

The analysis was conducted in collaboration with the Cellular Therapeutics and Systems Immunology Lab (CTSI), directed by Dr. Leslie Kean.

The study reveals two surprising differences in the two CAR-T populations in terms of the cells in the CSF displaying a molecular signature that show activation of the interferon pathway.

According to Dana-Farber's Dr. Carson Jacobson, who led the trial, there are limited treatment options for patients with lymphoma in the nervous system, and this study provides a great breakthrough.

Lymphomas are aggressive tumors that can spread from one place, such as the brain and the spinal cord, to other parts of the body, making it difficult to fight and treat.

However, with CAR-T, cells are generally rewired to bind to and destroy tumors, which have been proven to be effective in other forms of lymphoma.

This is the reason why Jacobson and her colleagues have decided to pilot a clinical trial to assess the safety of such treatment on brain and spinal cord cancers.

Medical Express writes that CAR-T cells are made from the patients' own disease-fighting T-cells, which were used in this trial to pause and assess treatment-limiting toxicities that emerged.

To get to these results, the researchers took blood and CSF samples during the period when CAR-T cells spread the fastest, in an approach more frequent yet less painful than the traditional spinal tap.

The Trial Shows Promising Results

Interesting Engineering reports that the trial consisted of nine patients show that 78% of the participants had their tumor shrink or disappear with the treatment.

Additionally, 67% of them showed a complete response to the CAR-T therapy during the duration of the trial.

"This approach gives us an unprecedented opportunity to explore the immune environment of the central nervous system and determine how that compares to the peripheral blood," Dr. Ulrike Gerdemann says.

He adds that through this research, they can compare identical cell populations in the blood and the CSF to gain a high-resolution picture of the T-cell dynamics to understand therapeutic responses.

The researchers analyzed as many as 125,000 individual cells during the trial, enabling them to reveal a molecular signature and unique CAR-T that is crucial to driving anti-tumor responses.

However, while the research seems to have proven initial success, scientists at Dana-Farber say that further trials are still necessary to yield more responses for the current treatment for lymphoma.