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Stem Cell Study in ALS Patients Gets FDA Green Light

Mesenchymal stromal cells secrete neurotropic factors

The FDA has OK’d the start of a phase II clinical trial of NurOwn (BrainStorm Therapeutics) in patients with amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease.

The trial will be launched initially at the Massachusetts General Hospital in Boston; at the University of Massachusetts Memorial Hospital in Worcester; and at the Mayo Clinic in Rochester, Minnesota.

MSC-NTF cell therapy (NurOwn) is based on the transplantation of autologous bone marrow-derived mesenchymal stromal cells (MSCs), which are enriched from the patient’s own bone marrow, propagated ex vivo, and induced to secrete neurotropic factors (NTFs). The autologous MSC-NTF cells are back-transplanted into the ALS patient into the sites of damage, the spinal cord, and the muscles.

NTFs are potent survival factors for embryonic, neonatal, and adult neurons and are considered to be potential therapeutic candidates for ALS. The delivery of appropriate NTFs to the immediate environment of affected neurons in ALS patients is expected to improve the neurons’ survival, slow disease progression, and alleviate symptoms.

The new phase II trial is a randomized, double-blind, placebo-controlled study designed to evaluate the safety and efficacy of the transplantation of MSC-NTF cells compared with that of placebo (excipient) in 48 patients with ALS. The MSC-NTF cells will be administered via combined intramuscular and intrathecal injection. Patients will be followed monthly for approximately 3 months before transplantation and for 6 months after transplantation. The study’s primary endpoint will be the treatment’s safety and tolerability, as defined by a measure of the number of patients with adverse events. Secondary endpoints will measure the change in the ALS Functional Rating Scale (ALS-FRS) and the change in Slow Vital Capacity (SVC) slopes.

The estimated study completion date is December 2015.

Earlier clinical trials showed that treatment with MSC-NTF cells was safe and well-tolerated. In a phase I study, MSC-NTF therapy successfully met the primary and secondary endpoints. Moreover, there were “encouraging signals of efficacy” in individuals receiving MSC-NTF cells.

Sources: BrainStorm Cell Therapeutics; April 28, 2014; and ClinicalTrials.gov; February 17, 2014.

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