A US clinical trial has shown significant success for CAR-T cell therapy, an immunotherapy used in treating some cancers, in treating people with stiff person syndrome (SPS), the rare neurological condition that affects singer Celine Dion. Two participants in the study, published in JAMA Neurology, recovered essential everyday capabilities previously lost to SPS after a single dose of the treatment.
The trial, led by the Mayo Clinic in Rochester, treated patients with SPS, a progressive condition thought to be autoimmune that causes muscle stiffness and affects an estimated 8,000 people worldwide. Celine Dion announced her diagnosis in December 2022.
Patient Jonathan Walden, who had become reliant on family for help with walking and daily tasks, said: “I can go to work every single day. I can do everything I did before. I go hunting; I can go fishing.” He added that he had almost talked himself out of the trial but said it had so far been the best thing he had ever done.
Shari St Germain, diagnosed with SPS in 2021, had experienced severe leg cramps and difficulty walking. By 2024, standard treatments were no longer controlling her symptoms. After receiving CAR-T therapy, she began noticing improvements. "After the treatment, I got up one day and I'm like 'I'm feeling really good', and I went to walk on the treadmill," she said. "So every day, it just gets better and better."
Dr Anastasia Zekeridou, a neurologist and researcher at the Mayo Clinic, which led the trial, said: “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 noted that the key question now is whether the improvements will be sustained over time.
Dr Sean Pittock, chairman of the department of neurology at the Mayo Clinic, said the results offer hope for people with SPS and potentially for patients with other immune-mediated neurologic conditions. “CAR-T therapy has already transformed the treatment of some cancers, and we are now beginning to see its potential in autoimmune neurologic diseases,” he said.
Yasmin Sheikh, head of policy and public affairs at Anthony Nolan, said the research provides more evidence that CAR-T therapies could have applications beyond blood cancer. She stressed the need for specialist workforce and infrastructure to deliver such treatments in practice.
The study could pave the way for larger trials in people with SPS.
Analysis
Why This Matters
- The success of CAR-T therapy for SPS opens the door to treating other autoimmune neurological disorders with similar approaches.
- Patients like Walden and St Germain have regained their independence, demonstrating the potential for life-changing improvements.
- The trial represents a significant step beyond cancer for CAR-T technology, expanding its therapeutic reach.
Background
CAR-T cell therapy involves modifying a patient's own immune cells to target and destroy specific cells causing disease. It is currently approved by the NHS for certain cancers. Stiff person syndrome (SPS) is a rare autoimmune neurological condition that causes progressive muscle stiffness and affects around 8,000 people worldwide. The condition gained international attention when singer Celine Dion revealed her diagnosis in 2022.
What to Watch
- Whether larger clinical trials confirm these early findings.
- The durability of the improvements in patients, as follow-up studies continue.
- Potential application of CAR-T therapy to other immune-mediated neurological conditions.
Checked
12 claims in this story checked against 1 source: 12 supported.
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- SupportedThe immunotherapy trial helped patients with stiff person syndrome walk again.The Independent UK: “Breakthrough treatment for Celine Dion's condition sees patients walk again.”
- SupportedAn early study showed promising results for CAR-T cell therapy.The Independent UK: “A form of immunotherapy has shown significant success in treating patients with stiff person syndrome (SPS)”
- SupportedTwo trial participants regained everyday capabilities they had lost to SPS after a single dose.The Independent UK: “Receiving the single-dose treatment allowed two trial subjects to recover essential everyday capabilities previously lost to SPS.”
- SupportedThe US trial was led by the Mayo Clinic in Rochester.The Independent UK: “Dr Anastasia Zekeridou, a neurologist and researcher at Mayo Clinic in Rochester in the US, which led the trial.”
- SupportedThe study was published in JAMA Neurology.The Independent UK: “The new study, published in Jama Neurology, could now lead to larger trials in people with SPS.”
- SupportedJonathan Walden said, “I can go to work every single day. I can do everything I did before. I go hunting; I can go fishing.”The Independent UK: ““I can go to work every single day. I can do everything I did before. I go hunting; I can go fishing,” he said.”
- SupportedWalden said he had almost talked himself out of joining the trial, but that it had been the best thing he had ever done.The Independent UK: “He added that when offered the trial, he almost talked himself out of it. “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.””
- SupportedShari St Germain said, “After the treatment, I got up one day and I'm like 'I'm feeling really good', and I went to walk on the treadmill,” and, “So every day, it just gets better and better.”The Independent UK: “She said: “After the treatment, I got up one day and I’m like ‘I’m feeling really good’, and I went to walk on the treadmill. “My husband comes running in because I’m crying and he’s like ‘Are you OK?’ And I’m like ‘I just walked for seven minutes on the treadmill’. “..So every day, it just gets better and better.””
- SupportedDr Anastasia Zekeridou said existing treatments rarely return patients to their previous level of function and that the key question is whether improvements will last.The Independent UK: ““With existing treatments, we may be able to improve symptoms, but we very rarely see patients return to their previous level of function. “These early results are very encouraging, and the key question now is whether those improvements will be sustained over time.””
- SupportedDr Sean Pittock said CAR-T therapy is beginning to show potential in autoimmune neurological diseases.The Independent UK: ““CAR-T therapy has already transformed the treatment of some cancers, and we are now beginning to see its potential in autoimmune neurologic diseases.””
- SupportedSPS affects an estimated 8,000 people worldwide and is thought to be an autoimmune condition.The Independent UK: “SPS affects an estimated 8,000 people worldwide. ... SPS is also thought to be an autoimmune condition”
- SupportedYasmin Sheikh said CAR-T could have applications beyond blood cancer and that specialist workforce and infrastructure are needed to deliver such treatments.The Independent UK: “This research is an exciting development, providing more evidence that CAR-T cell therapies could have applications beyond blood cancer and offer new hope to people living with other conditions, including SPS. ... This means ensuring the right specialist workforce, infrastructure and resources are in place”
