First Clinical Results Announced for In-Body CAR-T Therapy for MS and Autoimmune Diseases

A historic achievement in the fields of genetic engineering and cellular therapy has been recorded in the medical world. A new study published in the prestigious medical journal *The New England Journal of Medicine* announced to the public the first clinical results of in vivo CAR-T cell therapy developed against MS (Multiple Sclerosis) and similar severe autoimmune diseases.

While traditional methods require taking blood cells from the patient, genetically editing them in the laboratory, and reinfusing them into the body ("ex vivo")—a process that takes weeks—this new method, which enables the body to produce therapeutic cells internally through a single injection, has significantly accelerated the process.

IMMUNE SYSTEM IS RESET WITH A SINGLE INJECTION

16 patients suffering from MS and autoimmune disorders causing muscle breakdown participated in the study conducted jointly by Huazhong University of Science and Technology and medical centers in China. Researchers instructed the patients' T cells to produce chimeric antigen receptors (CAR) via a genetically modified, harmless lentivirus.

Multiplying rapidly within the body, these protective cells destroyed the faulty B cells and autoantibodies that were attacking healthy tissues. Following the treatment, it was observed that the new B cells produced by the body no longer attacked healthy tissues, meaning the immune system essentially returned to its "factory settings."

SIGNIFICANT IMPROVEMENT IN MOTOR SKILLS AND MUSCLE STRENGTH

While significant improvements were recorded in the motor and mental (cognitive) functions of MS patients receiving the treatment, their complaints of chronic fatigue were seen to decrease. In other patients experiencing muscle inflammation, it was documented that muscle strength scores increased and inflammation regressed.

Experts report that the inflammatory reactions observed in most of the patients were mild and that side effects could be controlled within two weeks.

LONG-TERM FOLLOW-UP REQUIRED AGAINST CANCER RISK

Stating that cellular therapy has created great excitement in the medical world, German and Chinese scientists warn that caution is necessary for the method to become clinically widespread. It is stated that the genetic material transferred via the virus carries the risk of mutating over the long term and causing cells to become cancerous.

For this reason, it was reported that patients participating in the study will be closely monitored for at least 10 years against possible cancer cases, infections, and the possibility of disease relapse. Following Phase II and III trials to be conducted with large control groups, the aim is for the method to become a routine treatment protocol worldwide.