Patient dosing has finished in a Phase 2 clinical trial testing NurOwn, BrainStorm Cell Therapeutics’ investigational cell-based therapy for people with progressive multiple sclerosis (MS), the company announced.
The trial’s top-line results are expected by April.
“Now that all dosing of the NurOwn Phase 2 progressive MS clinical trial is complete, we will shift our focus to completing all remaining patient clinical trial assessments and biomarker analyses,” Ralph Kern, MD, BrainStorm’s president and chief medical officer, said in a press release.
“We look forward to generating clinical trial top line results by the end of the first quarter 2021 and to advancing this promising treatment option based on a thorough analysis of the clinical trial outcomes,” Kern added.
Stacy Lindborg, PhD, BrainStorm’s executive vice president and head of global clinical research, noted that the full study data will be shared “with the MS scientific community at upcoming conferences and in subsequent publications” to “help advance understanding of progressive MS and the potential benefits of NurOwn as a treatment.”
NurOwn involves collecting mesenchymal stem cells (MSCs) from a patient’s own bone marrow, and expanding and maturating them into cells that produce high levels of neurotrophic factors (NTF) — molecules that promote nerve cell growth and survival. MSCs are stem cells that can generate a variety of other cell types.
The mature cells — called MSC-NTF cells — are then injected into the patient’s spinal canal to promote and support nerve cell repair. Using a patient’s own cells minimizes the risk of an immune reaction, as might occur with cells from a donor.
In MS, NurOwn is believed to promote the repair of myelin — the protective sheath around nerve fibers that is lost in MS — lessen the likelihood of further damage, and potentially slow disease progression.
A previous preclinical study suggested that NurOwn may also be able to suppress the damaging immune responses that contribute to MS progression, by promoting a cellular and molecular shift toward an anti-inflammatory state.
The open-label Phase 2 trial (NCT03799718) is evaluating NurOwn’s safety and effectiveness in 20 adults, ages 18 to 65, with either primary progressive MS or secondary progressive MS. Patient enrollment started in March 2019, and the trial is taking place at five U.S. sites.
Participants received a total of three injections of NurOwn, directly into the spinal canal, over a 16-week period (about four months), and will now be followed for 12 weeks (about three months) for safety and effectiveness measurements.
The study’s main goal is to assess the safety of the procedure. Effectiveness goals include changes in patients’ walking abilities or finger dexterity, as well as in the levels of neurotrophic and immunomodulatory biomarkers in their cerebrospinal fluid (the liquid that bathes the brain and the spinal cord).
“Completed dosing of the Phase 2 progressive MS clinical trial is a major milestone for BrainStorm as we advance our goal to develop the NurOwn platform technology in neurodegenerative diseases,” said Chaim Lebovits, BrainStorm’s CEO.
“We are very thankful for the dedicated efforts of the investigators and their teams and to the patients who participated in this important clinical trial. Through their combined efforts, we hope to bring a promising therapeutic option to those affected by progressive MS,” Lebovits added.
BrainStorm also is evaluating NurOwn as a potential therapy for other neurodegenerative diseases, such as amyotrophic lateral sclerosis, Parkinson’s disease, and Huntington’s disease, as well as for autism spectrum disorder.
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