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Human Motor Neurons from Stem Cells to Serve as Drug Discovery Tools for ALS

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The ALS Association has partnered with two biotech companies to harness new stem cell and gene manipulation technologies to the search for effective treatment for the disorder, amyotrophic lateral sclerosis (ALS).

The $3 million funding from The ALS Association’s program, Translational Research Advancing Therapy for ALS (TREAT ALS), is the largest from this program to date focused on new drug candidates. Several pilot clinical trials of existing candidates are already underway with TREAT ALS funding.

Partnering with The ALS Association are the companies Galapagos NV and Stem Cell Innovations, Inc. (“SCI”). Funding is contingent on the companies’ success at specific points in the project.

“The unique ability provided by SCI to screen human motor neurons at large scale, and Galapagos’ target discovery engine, will open new approaches to developing medicines that may stop ALS,” said Dr. Lucie Bruijn, science director and vice president of The ALS Association.

“We see this alliance as an important initiative within The ALS Association’s mission to find a cure for and improve living with ALS.”

Stem Cell Innovations claims that it has a proprietary human stem cell technology based on cells that are exempt from the presidential ban. These pluripotent stem cells are able to produce motor neurons that can grow robustly in the lab.

“The human motor neuron cultures derived from our PluriCells will form the basis of this exciting alliance,” said Stem Cell Innovations CEO Dr. James Kelly.

“We are very pleased to be collaborating with The ALS Association and Galapagos on this discovery project.”

According to Galapagos NV it has a technology that brings in or removes, in turn, large numbers of genes to lab-grown cells that reflect aspects of a disease of interest. This identifies which genes might serve as drug targets in the disease. The Galapagos technology will be applied to the SCI motor neuron cells.

“We are proud to work with the ALS Association in the fight against ALS,” said Onno van de Stolpe, CEO of Galapagos.

“This alliance builds on both our CNS (central nervous system) expertise and on our strong franchise in working with non-profit health organizations to identify disease-modifying drug targets for unmet medical needs.”