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Immunotherapy is a treatment that boosts or manipulates the immune system to fight diseases, including cancer. For instance, by stimulating an immune response through vaccinations against viruses that cause cancers, like hepatitis B virus and human papillomavirus, these diseases can be prevented. Nonetheless, some cancer cells can avoid the immune system due to their rapid mutation and division. The immune response to many cancers involves three phases: elimination, equilibrium, and escape.
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Redefining multiple sclerosis with CAR-T cell therapy.

Yan-Ruide Li1, Yuning Chen1, Lili Yang2

  • 1Department of Microbiology, Immunology & Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA; Department of Bioengineering, University of California, Los Angeles, Los Angeles, CA 90095, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|December 12, 2025
PubMed
Summary
This summary is machine-generated.

Chimeric antigen receptor (CAR)-T cell therapy shows promise for treating multiple sclerosis (MS) by targeting autoimmune responses. Further research is needed to optimize safety and efficacy for long-term disease control.

Keywords:
CAR-T cell therapyCD19-targeting CAR-TCRSMSautoimmune diseasesautoreactive B cellscytokine release syndromemultiple sclerosisneurotoxicity

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Area of Science:

  • Neuroimmunology
  • Cellular Therapy
  • Autoimmune Disorders

Background:

  • Multiple sclerosis (MS) is a chronic central nervous system autoimmune disease causing progressive neurological impairment.
  • Current therapies for MS offer limited remission and do not reverse existing damage.
  • Chimeric antigen receptor (CAR)-T cell therapy, successful in oncology, is emerging as a potential treatment for autoimmune diseases like MS.

Purpose of the Study:

  • To review recent advancements in applying CAR-T cell therapy to multiple sclerosis.
  • To discuss challenges and future directions for CAR-T cell therapy in neuroimmunology.
  • To explore the potential of CAR-T cells for achieving long-lasting disease control in MS.

Main Methods:

  • Review of preclinical studies and early-phase clinical trials of CAR-T cell therapy in MS.
  • Analysis of lessons learned from CAR-T cell applications in oncology and other autoimmune diseases.
  • Discussion of strategies for optimizing antigen targets and minimizing toxicities.

Main Results:

  • CAR-T cell therapy targeting CD19+ B cells has demonstrated encouraging efficacy in preclinical and early clinical studies for autoimmune conditions, including MS.
  • The approach holds potential for selective elimination of autoreactive immune cells, offering a pathway to immune system reset.
  • Significant challenges persist, including toxicity management, sustained therapeutic benefit, and scalable clinical implementation.

Conclusions:

  • CAR-T cell therapy represents a promising frontier for managing multiple sclerosis, potentially offering durable remission and reversal of pathology.
  • Overcoming challenges in antigen selection, safety, and manufacturing is crucial for translating CAR-T cell therapy into standard clinical practice for MS.
  • Further research and clinical trials are essential to establish CAR-T cells as a transformative treatment in neuroimmunology.