Samples From ALS Patients in Phase 3 NurOwn Trial Open to Researchers
BrainStorm donates NurOwn placebo group's samples to NEALS repository
Biological samples from amyotrophic lateral sclerosis (ALS) patients who took part in the Phase 3 clinical trial of NurOwn, a cell-based therapy, are being donated by BrainStorm Cell Therapeutics to a repository for use by the research community.
The samples include blood and spinal fluid (cerebrospinal fluid or CSF) collected from participants on a placebo throughout the trial. They will be given to the Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) biorepository.
NEALS is a nonprofit group of 147 research sites around the world working to translate clinical research into potential new treatments for ALS. The consortium’s biorepository includes blood, CSF, DNA, and urine samples from people who took part in various ALS clinical trials.
Blood and CSF samples taken in NurOwn trial among those donated
“As a clinical research organization, NEALS is committed to using the NEALS Biorepository to help ALS researchers around the globe identify biomarkers and novel therapies as rapidly as possible,” James Berry, MD, director of the Neurological Clinical Research Institute at Massachusetts General Hospital, said in a company press release.
“We are deeply appreciative of this contribution of samples, which adds to the biorepository and helps facilitate that goal,” Berry added.
With NurOwn, mesenchymal stem cells are harvested from a patient’s bone marrow and matured in the lab to produce large amounts of signaling molecules that promote nerve cell survival and growth. The modified cells then are returned to the patient via a spinal canal injection to potentially slow disease progression.
The therapy was assessed in a Phase 3 trial (NCT03280056) that enrolled 189 adults with rapidly progressing ALS. Patients were randomized to either three injections of NurOwn or a placebo every other month, and then were followed for up to about seven months (28 weeks).
The trial failed to meet its primary goal of slowing progression across treated patients, but later analyses supported benefit in those with less severe ALS at the start of the therapy’s use.
Samples were collected from patients up to seven times over 20 weeks of the trial, the company reported. They were used to evaluate biomarker changes in each patient over time and to compare biomarker levels in the blood and CSF.
“Our Phase 3 trial included one of the largest longitudinal specimen collection protocols in trial participants with ALS, which has allowed us to perform extensive and unique biomarker analyses,” said Stacy Lindborg, PhD, co-CEO of BrainStorm.
“These analyses have greatly increased our understanding of both the mechanisms driving ALS progression, as well as the biological changes in participants treated with NurOwn,” Lindborg added. “We are pleased to be providing the research community with access to our clinical samples, which will drive new research and can facilitate the discovery of new breakthroughs for patients with ALS.”
The donation is connected to a $500,000 biomarker research grant awarded to the company in 2020 by The ALS Association and I AM ALS.
The combined grant aimed to gain insights from clinical measures and tissue samples to identify biomarkers associated with NurOwn response. As part of the award, the company agreed to share samples with the ALS community to further advance biomarker research.
Ultimately, access to biomarker data provides information that could aid in disease understanding and developing targeted therapies.
“We would like to thank The ALS Association and I AM ALS for providing important funding for our biomarker analyses, as well as NEALS for housing our clinical samples for future use by the research community,” said Chaim Lebovits, BrainStorm’s co-CEO.
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