The ALS Association

ALS Ice Bucket Challenge Progress

Neuralstem's Phase 2 Trial of NSI-566 Therapy Deemed "Well Tolerated" for Study's Participants

March 12, 2015

This week, pharmaceutical firm Neuralstem released findings from its Phase 2 clinical trial involving its NSI – 566 therapy for ALS.

The trial, which involved inserting spinal cord-derived neural stem cells into 15 people with the disease, met primary endpoints for safety for use of the therapy. Ninety-three percent, or 14 participants, involved in the study “well tolerated” the maximum dose of 16 million cells, which were transplanted directly inserted into specific areas of the spinal cord where the cells join with host motor neurons. Motor neurons are the nerve cells that make up the central nervous system (CNS), which convey impulses initiating muscle contraction in an individual. The trial’s participants received up to 16 million cells through 40 injections, an amount that the study’s researchers believe is the maximum dosage that participants can endure.

“Although this study is small, it provides interesting findings on the potential use of these stem cells for people with ALS,” said Lucie Bruijn, Ph.D. M.B.A. Chief Scientist for The ALS Association. “The Association is pleased to have been an integral part of funding the early preclinical work and funding support for Dr. Eva Feldman to expand the number of sites providing this treatment approach. I look forward to seeing further results from additional research of this therapy.”

The researchers will include the trial’s complete results in a document to be published at a later date. Another trial, which should involve more participants, should begin later this year and will be available through multiple locations around the U.S. The United States Food and Drug Administration (FDA) has designated NSI-566 as an orphan drug for ALS.

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