A UCLA research team has secured $7.49 million in state funding to bring a groundbreaking cell-based therapy for multiple sclerosis closer to human trials, offering hope for a treatment that could provide lasting disease control rather than just managing symptoms. The funding, awarded by the California Institute for Regenerative Medicine (CIRM), will support development of a CAR-NKT cell therapy that works differently from existing MS medications by targeting two major drivers of the disease simultaneously.

What Makes This MS Therapy Different From Current Treatments?

Most approved MS medications focus on suppressing the acute immune attacks that trigger relapses, but they do little to stop the chronic inflammation that gradually damages nerve cells and worsens disability over time. The new therapy takes a different approach by using specialized immune cells called natural killer T-cells, or NKT cells, that are engineered in the laboratory to attack both B-cells and inflammatory myeloid cells.

The key innovation lies in the dual-action strategy. Unlike conventional CAR T-cell therapies that only target B-cells, the CAR-NKT cells leverage their natural ability to recognize and destroy myeloid cells, which accumulate in the brain and spinal cord and fuel the chronic inflammation driving ongoing nerve damage.

"Regular CAR-T cells will wipe out the bad B cells, and we can do the same, but we do more than that. The CAR-NKT cells can also use their natural receptor to attack the inflammatory myeloid cells that are causing so much of the central nervous system damage. They work on two fronts. That's very important for dealing with this disease," explained Lili Yang, PhD, a professor and leader of the Yang Engineering Immunity Lab at UCLA.

Lili Yang, PhD, Professor and Researcher, Yang Engineering Immunity Lab at UCLA

How Could This Therapy Provide Long-Lasting Disease Control?

One of the most promising aspects of this approach is its potential for durability. Because CAR-NKT cells can persist in the body for months or even years, and it takes time for newly formed B-cells to become capable of triggering autoimmune attacks, researchers believe a single treatment course could be enough to reset the immune system and keep serious attacks at bay for an extended period.

The therapy also differs fundamentally from existing CAR T-cell treatments in terms of manufacturing and cost. While conventional CAR T-cell therapies are custom-made for each individual patient, making them expensive and time-consuming to produce, CAR-NKT cells can be manufactured in advance from umbilical cord blood, frozen, and stored until needed.

"We estimate the cost to produce one dose could be as low as $5,000. And those cells are universal, we don't need to customize them for each individual patient. They're made. They're ready," said Yang.

Lili Yang, PhD, Professor and Researcher, Yang Engineering Immunity Lab at UCLA

According to researchers, a single cord blood donation could yield enough cells to produce thousands of treatment doses.

What Evidence Supports Moving Forward With Human Trials?

The research team has already demonstrated promising results in established mouse models of MS. In these studies, CAR-NKT cell treatment prevented mice from developing paralysis, showing real, measurable benefits rather than just laboratory improvements. The team also met with the U.S. Food and Drug Administration in a pre-investigational new drug meeting to discuss the pathway toward human testing.

Over the next 2.5 years, the scientists plan to complete several critical milestones:

• Manufacturing: Develop a clinical-grade version of the therapy that meets regulatory standards for human use
• Production Scale: Produce hundreds of treatment doses to support initial clinical trials and establish manufacturing feasibility
• Regulatory Approval: Seek investigational new drug clearance from the FDA, which would allow clinical trials to begin in human patients

If successful in MS, the platform could potentially be adapted to treat other autoimmune diseases, including lupus and rheumatoid arthritis, expanding its impact beyond multiple sclerosis.

What Other MS Research Is Advancing Treatment Options?

While the UCLA team pursues immune cell engineering, other researchers are exploring complementary approaches. A separate clinical trial at the University of California, San Francisco is investigating clemastine fumarate, a drug that may promote myelin repair in MS patients. Myelin is the protective coating around nerve fibers that becomes damaged in MS, and clemastine was identified as a potential remyelinating agent using advanced screening techniques.

In a different area, a systematic review of clinical trials found that nanocurcumin, a supplement containing a turmeric compound, may significantly improve disability outcomes in people with relapsing-remitting MS. Across three trials involving 150 people with RRMS, participants who took 80 milligrams of nanocurcumin daily for six months had significantly better disability scores than those given a placebo. However, researchers emphasized that larger, well-designed clinical trials are needed to confirm these preliminary findings.

Why Does This Funding Matter for MS Patients?

Multiple sclerosis affects the central nervous system when the immune system mistakenly attacks healthy tissue in the brain and spinal cord, disrupting communication between nerve cells and causing fatigue, movement problems, and cognitive decline. While more than 20 disease-modifying therapies are now approved for MS, most focus on immune suppression rather than addressing the underlying damage or providing a potential cure.

The $7.49 million investment represents a significant commitment to exploring fundamentally different treatment strategies.

"With this approach, we hope to achieve a much more significant delay, or even a cure. If we can truly reset the immune system, we may be able to keep another serious attack at bay indefinitely," stated Yang.

Lili Yang, PhD, Professor and Researcher, Yang Engineering Immunity Lab at UCLA

This optimism reflects the potential of cell-based therapies to move beyond symptom management toward disease modification and possible long-term remission.