December 11, 2006
Research Update — from ALSA’s National Office
Human Motor Neurons from Stem Cells to Serve as
Drug Discovery Tools with Funds from The ALS Association
Roberta Friedman, Ph.D., ALSA Research Department Information Coordinator
[Quick Summary: In a focused drug discovery effort funded by TREAT ALS, proprietary stem cells and gene manipulation technologies will be combined in a search for targets for candidate therapies for the disease].
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, also known as Lou Gehrig's disease). The $3 million funding from The ALS Association’s translational program, Translational Research Advancing Therapy for ALS (TREAT ALS), is the largest to date focused on drug development.
TREAT ALS seeks new avenues to therapeutics and puts into place a clinical trials process that will be ready when any new candidates are identified. Several pilot clinical trials of existing candidates are already underway with TREAT ALS funding.
Partnering with The ALS Association are the companies, Galapagos NV (Euronext & LSE: GLPG), and Stem Cell Innovations, Inc. (SCI, SCLL.OB). Funding will be made in steps that depend on the companies’ success at specific points in the project, that is, the project is milestone driven.
“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 Lucie Bruijn, Science Director and Vice President at 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 has a proprietary stem cell technology based on cells that are exempt from the President’s ban. The 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.”
Galapagos NV has a technology that brings in or removes, in turn, large numbers of genes to lab-grown cells.
The technology can be carried out with the cells growing and responding in vast numbers of tiny chambers scanned automatically by robotic imaging equipment, termed high throughput functional array analysis.
If manipulating these genes rescues the cells when they are challenged by stimulation with toxic factors such as excessive levels of glutamate or by withdrawal of support molecules, then the genes should represent targets at which to aim drugs.
“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 expertise and on our strong franchise in working with non-profit health organizations to identify disease-modifying drug targets for unmet medical needs.”
Please see The ALS Association’s web site under the research tab for further information about laboratory models http://www.alsa.org/research/article.cfm?id=812 and TREAT ALS http://www.alsa.org/news/article.cfm?id=643 and http://www.alsa.org/research/article.cfm?id=1048.
