A stem cell treatment is safe and well tolerated in patients with ischemic stroke and may improve outcomes, research indicates.
The data was presented at the 2016 International Stroke Conference.
MultiStem, a stem cell product featuring multipotent adult progenitor cells and created by biotech company Athersys, was the focus of a phase 2, double-blind, placebo-controlled trial conducted at 33 sites in the US and UK. The product’s safety and efficacy has previously been shown in other clinical indications and pre-clinical models of stroke.
In the current study, 126 patients with ischemic stroke were randomized 1:1 to receive either 1.2 billion MultiStem cells (n=65) or placebo (n=61) within 24-48 hours of symptoms. Primary efficacy endpoints included Global Recovery (mRS ≤2, NIHSS Δ ≥75% and BI ≥95) and Excellent Outcome between groups (mRS ≤1, NIHSS ≤1, BI ≥95) at 3 months. Safety endpoints included neurologic worsening, secondary infections, adverse events, and mortality.
Overall, a greater percentage of patients treated with MultiStem had an Excellent Outcome (P=0.10); however it did not have a significant benefit compared to placebo. The stem cell treatment was associated with lower rates of infection and pulmonary events, and a reduction in hospitalization, life threatening adverse events, and death. Patients who received MultiStem in 36 hours or less from symptoms had better Global Recovery (41.9% vs 17.3%, P<0.01) and Excellent Outcome (18.5% vs 3.8%, P=0.03) compared to placebo, and had a significantly better recovery by mRS shift analysis (P=0.03) and reduced hospitalization (6.7d vs 10.3 days, P<0.01).
“These results are very promising and this therapy would be an important adjunct to acute stroke care to reduce the amount of brain injury from which patients need to recover,” said Cathy Sila, MD, a principal investigator at UH Case Medical Center. “But the results are still preliminary and need to be reproduced.”
Notably, the Multistem system appears to hold benefits for patients up to 36 hours after stroke, expanding the current limited treatment window.
“There is only one drug, tPA, that is FDA approved for acute ischemic stroke, and tPA needs to be administered within hours of onset of a stroke,” Dr Sila said in a statement. “Despite public education and the development of stroke centers, only about 7% of all ischemic stroke patients nationwide receive tPA therapy and less than 2% undergo catheter-based clot removal therapy. New treatments are needed to reverse the effects of a stroke and promote recovery from stroke, and they need to be effective in a wider time window to help more patients.”
Other studies are ongoing and more are planned.
For more coverage of ISC 2016, go here.
Hess DC, Auchus AP, Uchino K, et al. Abstract 71. Final Results of the B01-02 Phase 2 Trial Testing the Safety and Efficacy of MultiStem® in Treatment of Ischemic Stroke. Presented at: International Stroke Conference; Feb. 16-19, 2016; Los Angeles.