A new study by researchers at Tisch MS Research Center, New York, shows that intrathecal administration of autologous neural progenitors can be safe and well tolerated in patients with progressive multiple sclerosis. On top of that, the majority of patients demonstrated improvement in their Expanded Disability Status Scale (EDSS) score. The results of a first-of-its-kind study were presented at 2016 annual meeting of the American Academy of Neurology in Vancouver, British Columbia.
The author of the study Dr Sadiq organized it because there is no conventional treatment to reverse disability in disabled patients with progressive MS. Encouraged with the positive clinical results of intrathecal administration of neural progenitors in animal models, the doctor and his colleagues aimed to establish the safety and tolerability of their approach in humans.
The phase 1 trial enrolled 20 patients with MS who had established disability (range, 3.5-8.5) but relatively stable disease as demonstrated by less than 1-point change in EDSS score in the last year, and stable magnetic resonance imaging disease burden with no enhancing lesions in the previous 6 months.
For the study, Dr Sadiq and colleagues administered to the patients neural progenitors, derived from patients own (autologous) bone marrow stem cells. Neural progenitors were injected three times (once in three months) in a dose of up to 10 million cells per injection.
Lack of adverse effects was the primary concern of the scientists, and the trial showed that the treatment was safe and overall well-tolerated. Some minor adverse events were reported, such as transient headache and/or fever.
Although the trial was not designed to determine efficacy of the therapy, the scientists measured a number of disability indicators, such as EDSS score, timed 25-foot walk, peg test, paced auditory serial addition test, evoked potentials, and urodynamics testing. After treatment, some patients demonstrated neurologic improvement, including better EDSS scores and peg tests, as well as better bladder function.
The researchers plan to conduct a multicenter phase 2 double-blind trial to determine the efficacy of their method for reversing disability in patients with multiple sclerosis.