CAR-T therapy shows promise for stiff person syndrome

Summarised from 3 outlets · Updated 9 Nov, 16:20 · Archive

CAR-T cell therapy has led to dramatic improvements in patients with stiff person syndrome, the rare neurological disorder affecting singer Celine Dion.

CAR-T cell therapy, a form of immunotherapy, has produced dramatic improvements in patients with stiff person syndrome (SPS), the rare neurological disorder that affects singer Celine Dion. The treatment teaches the body's immune system to recognise and attack the cells causing disease. CAR-T is already approved on the NHS for certain conditions, including some cancers, and research is ongoing.

Stiff person syndrome is a progressive condition causing stiffness in the body, spine, arms and legs. Symptoms can include muscle spasms, severe leg cramps and difficulty walking. As the disease progresses, sudden noises can trigger muscle rigidity and falls. It was previously understood that there was no cure for SPS, with treatment focusing on managing symptoms.

In the recent US clinical trial, two patients saw particularly striking results. Jonathan Walden had reached a point where he needed family help with walking and basic daily tasks. After receiving the one-off CAR-T treatment, he returned to work and resumed hunting and fishing. "I can go to work every single day. I can do everything I did before," he said. "I have not had one episode since receiving treatment." He added that when first offered the trial, he almost declined. "But realising the outcome of either direction, I was better off going ahead and taking the risk for the trial, which has so far been the best thing I've ever done."

Shari St Germain, diagnosed in 2021, had suffered severe leg cramps and difficulty walking. As her condition worsened, standard treatments no longer controlled her symptoms. In the months after receiving CAR-T, she began noticing improvements, such as being able to walk on a treadmill again.

Dr Anastasia Zekeridou, a neurologist at the Mayo Clinic in Rochester who led the trial, commented on the scale of improvement observed. "What was most striking was the degree of improvement we saw in some patients. With existing treatments, we may be able to improve symptoms, but we very rarely see patients return to their previous level of function," she said. Celine Dion announced her diagnosis of stiff person syndrome in December 2022.

How it is being reported

• Celine Dion stiff person syndrome breakthrough - scientists may have found a cureCeline Dion brought stiff person syndrome to global attention and now scientists are trialling a gene therapy that appears to halt terrifying 'statue' attacksThe Mirror · 9 Nov, 16:10
• Breakthrough treatment for Celine Dion's condition sees patients walk againCanadian singer Celine Dion has suffered since 2022 from stiff person syndrome, a rare and incurable neurological conditionThe Independent · 9 Nov, 16:00
• One-off drug offers hope to people with same disorder as Celine DionDion announced she had been diagnosed with stiff person syndrome in December 2022.Evening Standard · 9 Nov, 16:00

In this story: Celine Dion · Jonathan Walden · Shari St Germain · Dr Anastasia Zekeridou · Mayo Clinic · NHS · Rochester

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