The University of California, San Diego, The Salk Institute for Biological Studies and Life Technologies Corporation (NASDAQ: LIFE) today announced the initiation of studies which may lead to the future development of cell transplant therapy for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease. Currently, there is only one FDA-approved pharmaceutical addressing this lethal disease, which affects approximately 30,000 people in the United States.
“Our scientists will bring together diverse technologies across multiple businesses at Life Technologies to provide a clinically compliant process intended to enhance the safety profile of this innovative therapeutic approach”
ALS is a progressive disease that attacks the motor neurons, long and complex nerve cells controlling voluntary movement that reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. Degeneration of the motor neurons in ALS leads to progressive loss of muscle control, paralysis and untimely death. Most people are diagnosed with ALS between the ages of 45 and 65. Typically, ALS patients live only one to five years after initial diagnosis.
As part of the UC-San Diego- and Salk-led effort - made possible by $11.5 million in funding from the California Institute for Regenerative Medicine (CIRM) - investigators aim to conduct novel, cutting-edge stem cell research, based on a special type of cell called an astrocyte progenitor.
Astrocytes are cells that help support the proper functioning and insulation of neurons. In ALS disease, the insulation critical to neuron function decays over time, and it is thought that administration of an astrocyte-based cell therapy might help regenerate neuron insulation and reverse the debilitating effects of the disease or halt its progression. UCSD and Salk scientists will study two methods of administering astrocyte progenitors in animal models including cervical and lumbar spinal cord injections. By carefully and rigorously testing the safety and ability of these cells to prevent or slow down the deterioration of motor neurons, this team of world-class experts is hoping this research provides proof-of-principle, paving the path for bringing this approach to clinical trials within five years.
Through Invitrogen's Primary and Stem Cell Systems business, Life Technologies is providing support through its expertise in stem cell biology, cell separation, next-generation sequencing and bio-production to help the UCSD and Salk teams test their pre-clinical research concepts, with the hope that the results may pave the way for future clinical applications for individuals diagnosed with ALS. Life Technologies' involvement in these ground-breaking efforts demonstrates the company's commitment to creating workflow solutions with integrated tools and reagents that will allow researchers to accelerate the development of cell based therapeutics for patients with unmet medical needs.
"Developments resulting from this study could be game-changing for thousands of people affected by neurodegenerative diseases like ALS," said Larry Goldstein, PhD, professor of Cellular and Molecular Medicine and Director of the UC San Diego Stem Cell Program.
"Any novel treatment option would not only be clinically competitive, but could have a major impact for thousands of patients currently battling this disease," added Don Cleveland, PhD, professor of Medicine, Neurosciences and chair of the UCSD Department of Cellular and Molecular Medicine. Cleveland is also head of the Laboratory of Cell Biology at the Ludwig Institute for Cancer Research based at UC San Diego. "This approach has the potential to lead to the development of new therapies that could significantly extend the lifespan of individuals living with this disease, and improve their quality of life."
"Our scientists will bring together diverse technologies across multiple businesses at Life Technologies to provide a clinically compliant process intended to enhance the safety profile of this innovative therapeutic approach," said Paul Pickering, General Manager of the Cell Therapy Systems segment of Primary and Stem Cell Systems business at Life Technologies. "Our hope is that our involvement in the UCSD and Salk study will lead to a reproducible approach that may be applied to other diseases that have huge unmet medical need. We are pleased to be involved in this innovative investigation and hope that our efforts help accelerate the process of bringing novel therapeutics to the general public."
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