Doctors encouraged by early-stage trial of MS stem cell therapy

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Doctors are cautiously optimistic about a new multiple sclerosis treatment after learning that injecting stem cells into patients’ brains is safe and can prevent further damage from the disease.

The small, early-stage trial was only to assess whether injecting the cells directly into the brain was well tolerated by patients, but in post-treatment tests, researchers found that the cells could have long-lasting beneficial effects. .

“We don’t know yet whether this is the start of a remarkable journey or not, but the results are very strong and very consistent,” said Stefano Pluchino, a professor at the University of Cambridge.

More than 2 million people worldwide suffer from multiple sclerosis. While most existing drugs initially target Repeated vomiting The stage of the disease, two-thirds of patients are still 30 years after the diagnosis to the second stage, gradually and increasingly weak stage.

The disease occurs when the body’s immune system attacks the protective myelin sheaths around nerve fibers, causing damage that disrupts how electrical signals are transmitted around the nervous system.

For the first in-human treatment trial, researchers injected 5m to 24m neural stem cells directly into the brains of 15 patients with advanced MS. Instead of rebuilding damaged tissue, stem cells are thought to reduce the inflammation that triggers the disease.

Some patients in the trial experienced side effects, one from tremors and another from steroid-induced psychosis, but all recovered with treatment.

In the following year, tests on patients did not report any worsening of symptoms or an increase in disability, although most of them were using a wheelchair before the study and may not have been impaired anyway. Brain scans showed that patients who received higher doses of stem cells had less brain shrinkage, possibly because the stem cells reduced inflammation.

What really surprised scientists was the experiments on the cerebrospinal fluid that bathes the brain and spinal cord. They found that patients who received more stem cells had higher levels of carnitine It is intended to protect nerve cells from harm. “What does it mean? I have no idea. But it’s incredibly exciting,” Pluchino said. Details published in Cell stem cell.

The researchers now want to do a larger trial to see if the injection changes the course of the disease. One factor that needs to be avoided is any effect of immunosuppressive drugs that patients take to prevent stem cell rejection.

Professor Paolo Muraro, a neuroimmunology expert at Imperial College London, who was not involved in the research, said the trial set a benchmark for the production and quality control of the cell-based drug. But more challenges remain. One is scaling up the treatment and making it affordable for larger trials to test how effective the treatment is. “It’s a long journey for Hope, but it’s definitely worth it,” Muraro said.

Kathleen Astbury, from the MS Society, said it was a “really exciting study” that built on previous research funded by the charity. “These results show that specialized stem cells injected into the brain are safe and well tolerated in people with secondary progressive MS,” she said. “Also, this treatment modality can slow the progression of disability. We’ve known for some time that this modality has the potential to help protect the brain from MS progression.”

“This was a very small preliminary study and we need more clinical trials to see if this treatment has a beneficial effect on this condition. But this is an encouraging step towards a new way of treating some people with MS.”



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