CAR-T cell therapy brings hope for Stiff Person Syndrome patients

In a multicenter study conducted on 26 patients battling Stiff Person Syndrome, a rare condition targeting the immune system, it was observed that a single dose of CAR-T cell therapy provided significant improvement in patients' walking test and daily functionality levels.

In the study involving 26 patients with Stiff Person Syndrome, a single dose of CAR-T cell therapy was administered to reorganize the immune system and alleviate symptoms. Approximately 16 weeks after the application, it was determined that significant progress was recorded in walking tests of about 7.6 meters. It was reported that some patients, such as Jonathan Walden, who could not meet their basic needs without family assistance prior to treatment, started working again after the treatment.

Anastasia Zekeridou, a neurologist and researcher from the US-based Mayo Clinic, one of the centers where the study was conducted, stated that current treatments can alleviate symptoms, but it is rarely possible for patients to return to their former level of functionality. Zekeridou emphasized that the improvement they observed was remarkable, but it needs to be investigated whether these gains can be maintained in the long term. Sean Pittock, Head of the Mayo Clinic Department of Neurology, noted that the results obtained are promising not only for those living with this syndrome, but also for other immune-system-mediated neurological diseases with limited effective treatment options. The study results were published in the journal JAMA Neurology.

The exact cause of Stiff Person Syndrome, which manifests with muscle stiffness and painful spasms that can be triggered by factors such as loud noises or gentle touches, is unknown. However, the syndrome is thought to be an autoimmune disease in which the immune system mistakenly attacks the body's normal tissues. Famous singer Celine Dion had also announced in 2022 that she was suffering from this syndrome and had to take a break from her concerts.