CAR-T cell therapy offers hope for rare syndrome

In the multi-center study, in which 26 people with Stiff Person Syndrome participated, a single dose of CAR-T cell therapy was applied to the patients in order to reorganize the immune system and alleviate the symptoms of the disease.

Significant improvement was noted in the approximately 7.6-meter walk test performed 16 weeks after CAR-T treatment.

Jonathan Walden, one of the patients participating in the study, had reached a point where he needed his family's help to walk and meet his basic daily needs. However, after CAR-T cell therapy, it started working again.

Neurologist and researcher Anastasia Zekeridou from Mayo Clinic in the USA, one of the centers where the study was conducted, stated that existing treatments can alleviate the symptoms of the disease, but it is rarely possible for patients to return to their previous levels of functionality.

Zekeridou stated that the improvement they observed in some patients was remarkable and emphasized that it should be investigated whether the gains achieved with the treatment can be maintained in the long term.

Sean Pittock, Head of the Department of Neurology at Mayo Clinic, stated that the results regarding Stiff Person Syndrome are promising not only for those living with this disease, but also for other immune system-related neurological diseases for which effective treatment options are limited.

The results of the research were published in the journal "JAMA Neurology".

Rigid Person Syndrome

Stiff Person Syndrome causes muscle stiffness and painful muscle spasms that can be triggered by factors such as loud noises or light touch.

Although the cause is unknown, this syndrome is thought to be one of "autoimmune diseases" in which the immune system mistakenly attacks the body's normal tissues.

Famous singer Celine Dion also announced that she suffered from Rigid Person Syndrome in 2022 and took a break from her concerts due to her illness.