Pilot trial sees tumors shrink or disappear in 78% of patients

The study brings much hope to patients with brain and spinal cord cancers.
Loukia Papadopoulos
An illustration of brain cancer tumors.jpg
An illustration of brain cancer tumors.

Mohammed Haneefa Nizamudeen/iStock 

A small pilot trial involving patients with lymphoma of the brain and/or spinal cord has shown that CAR-T-cell therapy known as axicabtagene ciloleucel (axi-cel) can be a viable treatment option for patients who often have little hope, according to a press release by Dana-Farber Cancer Institute investigators published on Sunday. “For many patients with lymphoma of the central nervous system, there aren’t great treatment options,” said Dana-Farber’s Caron Jacobson, MD, MMSc, who led the trial.

“Our early results suggest that expanding the applicability of CAR-T cells to this indication could improve patient outcomes.”

Lymphomas typically feature aggressive tumors that can spread to the brain and spinal cord after they originate in other parts of the body. These cancers are often difficult to treat and patients do not survive more than two years.

Pilot trial sees tumors shrink or disappear in 78% of patients
An illustration of brain cancer.

CAR-T cells are genetically re-wired to bind to and destroy tumors and have proven effective in other forms of lymphoma. Now, Jacobson and her colleagues have embarked on a pilot clinical trial to assess the safety of axi-cel treatment for brain and spinal cord cancers.

Nine patients

The trial consisted of nine patients, 78 percent of which saw their tumors shrink or disappear due to the treatment, and 67 percent of which showed a complete response, in which their tumors disappeared.

The experiment isolated and analyzed both CAR- and non-CAR-T-cells using advanced single-cell techniques, including single-cell RNA sequencing and single-cell sequencing of T-cell receptors.

"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,” said Gerdemann.

“Moreover, we can compare identical cell populations in both blood and CSF, gaining a high-resolution picture of T-cell dynamics and a deeper understanding of what drives therapeutic responses.”

Further studies needed

The researchers analyzed over 125,000 individual cells, representing a technical and clinical tour de force. The massive breadth and depth of the researchers’ inquiry enabled them to reveal a molecular signature, unique to CAR-T-cells, that could be crucial to driving anti-tumor responses in brain and spinal cord cancer.

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“Further studies of these cells promise to shed even more light on their unique biology and their activities in the body,” said Kaminski. “This has been a really tremendous effort and we are extremely grateful to the patients, clinicians, and researchers involved who made it happen.”

Although further clinical studies are still required, the data suggest that axi-cel yields responses are potentially more durable than current treatments for brain and spinal cord lymphoma, offering much-needed hope to patients with few options. The Dana-Farber Cancer Institute is described as one of the world’s leading centers of cancer research and treatment.

The firm is dedicated to a unique and equal balance between cancer research and care, translating the results of discovery into new treatments for patients locally and around the world, offering more than 1,100 clinical trials.