Stiff person syndrome (SPS) is a rare, progressive disorder that causes painful muscle spasms. It occurs when the immune system attacks a key protein in the nervous system, leading to stiffening and spasms. While there is no cure for SPS, researchers are exploring new treatment options.
A recent case report published in the journal *Neurology* highlights a potential new treatment for SPS using CAR T-cell therapy, a treatment typically used for cancer. This therapy involves modifying a patient’s T cells, which are immune cells, to target specific cells. In this case, the CAR T cells were designed to target antibody-producing B cells, which are thought to play a role in the development of SPS.
The patient in the case report had been diagnosed with SPS in 2014 and had experienced progressive worsening of symptoms. Despite previous treatments, her condition deteriorated in 2023. The researchers decided to try CAR T-cell therapy, targeting the B cells responsible for producing antibodies against the protein GAD.
The treatment had a rapid effect. Within six months of the one-time treatment, the patient’s walking speed doubled, and she was able to walk further distances. She also experienced a reduction in other symptoms, including fatigue and stiffness. Importantly, she was able to discontinue other immunotherapies and reduce her use of benzodiazepines, which help manage GABA function.
While this case report is promising, it is important to note that this treatment remains experimental. Further research, including clinical trials with a larger group of patients, is needed to confirm its effectiveness and safety. However, this case study provides hope for a new treatment option for individuals with SPS, a disease that currently has limited treatment options.
