Stem cell treatment improved neurological symptoms of 75% of people with multiple sclerosis in a Phase 1 clinical trial, say researchers in the USA.
Results were so encouraging that they prompted the team, at Tisch MS Research Center of New York, to start a Phase 2 trial. This is to allow them to assess further the safety and effectiveness of the therapy.
I must say that it is important to remember that this stem cell treatment is NOT HSCT. Importantly, the treatment being trialed does not involve suppressing and rebuilding the autoimmune system. Instead, this trial involves injecting mesenchymal stem cell-derived nerve cell progenitors directly into the spinal canal.
The study, “Phase I Trial of Intrathecal Mesenchymal Stem Cell-Derived Neural Progenitors in Progressive Multiple Sclerosis,” was published in the journal EBioMedicine.
In the open-label trial (NCT01933802):
- 80% of patients had secondary progressive MS (SPMS);
- Half were unable to walk at the beginning of the study. They had Expanded Disability Status Scale (EDSS) scores of 7 or higher;
- Eight required assistance to walk. They had EDSS scores of 6 to 6.5;
- Two were able to walk without assistance. They EDSS scores between 3.5 and 5.5.
Stem cell treatment led to improvements in neurological symptoms of 75% of the patients. The condition of 10% worsened, 15% remained the same.
Interestingly, 70% of the patients had better muscle strength after the treatment, mainly in the lower limbs.
Of those who were able to walk when the trial began, 40% improved their scores on a measure of exercise capacity — the timed 25-foot walk test — by at least 20%. Additionally, those who couldn’t walk at the start were later able to complete the walk test with assistive devices.
Disability scores improved
Researchers say that those who responded to the treatment improved their disability scores by 0.5 to 2.0 EDSS points.
Other key points of the trial were:
- The stem cell treatment improved the bladder function of half the patients with a problem. Some were able to stop taking their bladder medications.
- There were no serious adverse effects associated with the therapy. About 85% of the patients experienced headaches and fever in the first 24 hours after the treatment. Doctors managed these with standard medications.
- The treatment consisted of three injections of stem cells derived from the patients, administered three months apart. Patients received antibiotics to reduce the risk of infection.
Researchers used fresh cells, rather than cells preserved by refrigeration, and say this may have contributed to the good results.
Senior research scientist at the Tisch center, Dr Violaine Harris said: “The protocol we created allowed for delivery of these stem cells within 30 minutes of harvesting” them.
Tisch center’s director and senior author of the study, Dr Saud A Sadiq added that the Phase 1 results “justify the initiation of a planned FDA-approved Phase 2 trial in a larger group of patients.
“We anticipate that these novel studies may form the therapeutic basis of reversing disability in patients with MS, and if successful, these studies could have positive implications for treating other neurological diseases,” he said.
Researchers are now seeking to recruit about 50 patients with primary progressive or secondary progressive MS for the Phase 2 trial (NCT03355365). Patients must have an EDSS between 3 and 6.5. For additional information, click here.
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50shadesofsun.com is the personal website of Ian Franks, a freelance medical writer and editor for various health information sites. He enjoyed a successful career as a journalist, from reporter to editor in the print media. He gained a Journalist of the Year award in his native UK. Ian received a diagnosis of MS in 2002 and now lives in the south of Spain. He uses a wheelchair and advocates on mobility and accessibility issues.
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