A University of California, Los Angeles (UCLA) research team has secured a $7.49 million grant from the California Institute for Regenerative Medicine (CIRM) to advance a novel off-the-shelf, cell-based immunotherapy for multiple sclerosis (MS). Led by Lili Yang, PhD, the project aims to utilize engineered natural killer T-cells (NKT cells) to halt both immune system attacks and chronic inflammation, targeting a transition from symptom management to long-term disease control.
How do CAR-NKT cells differ from standard therapies?
Current MS treatments primarily target acute immune attacks, often failing to address the chronic inflammation that leads to long-term nerve damage. Standard CAR T-cell therapies involve harvesting a patient’s own cells, engineering them, and re-infusing them, a process that is both time-consuming and expensive.
According to Yang, the UCLA team’s platform uses NKT cells derived from umbilical cord blood. Unlike traditional T-cells, these engineered CAR-NKT cells work on two fronts: they destroy disease-causing B-cells while simultaneously using their natural receptors to attack inflammatory myeloid cells. These myeloid cells accumulate in the central nervous system and are known drivers of chronic nerve damage.
Because CAR-NKT cells are universal—meaning they don’t require customization for each patient—a single cord blood donation could potentially produce thousands of treatment doses.
What is the projected cost of this treatment?
The off-the-shelf nature of this therapy significantly lowers production barriers. Yang estimates that the cost to produce a single dose could be as low as $5,000. This is a sharp contrast to conventional CAR T-cell therapies, which are custom-manufactured for individual patients, resulting in significantly higher expenses.
The cells can be manufactured in advance, frozen, and stored until needed. This logistical advantage allows for immediate availability, eliminating the wait times associated with personalized cell manufacturing.
What are the next steps for clinical trials?
The research team has already completed a pre-investigational new drug (IND) meeting with the U.S. Food and Drug Administration (FDA) to outline the requirements for human testing. Over the next 2.5 years, the UCLA team plans to manufacture a clinical-grade version of the therapy and produce hundreds of treatment doses.
The therapy has already shown efficacy in mouse models of MS, preventing the development of paralysis. Researchers believe that because these cells can persist in the body for months or years, a single treatment course could potentially reset the immune system, providing a durable defense against the disease.
Potential for other autoimmune conditions
If the clinical trials prove successful, the platform may have broader applications. Yang suggests the CAR-NKT technology could be adapted to treat other autoimmune disorders, including lupus and rheumatoid arthritis, by utilizing the same dual-targeting mechanism against B-cells and inflammatory myeloid cells.
Frequently Asked Questions
What is the primary goal of the UCLA MS therapy?
The goal is to reset the immune system to halt both acute immune attacks and chronic inflammation, potentially providing long-lasting disease control or a cure.
Why are NKT cells used instead of T-cells?
NKT cells possess a natural ability to target inflammatory myeloid cells, a feature that standard T-cells lack. This allows the therapy to address two distinct drivers of MS-related nerve damage.
Is this therapy currently available for patients?
No. The therapy is currently in the development stage. The team is working toward obtaining FDA clearance for clinical trials over the next several years.
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