NEW YORK—Target ALS Foundation, a collaborative research consortium focused on discovering treatments and a cure for amyotrophic lateral sclerosis (ALS), has received a $5-million donation from Biogen that will fund critical ALS research across a network of academic laboratories. The Biogen donation helps establish the Industry Fund for ALS Research and launches a challenge to raise $20 million for Target ALS from other industry donors to support precompetitive research of value to the whole ALS community. Biogen has further committed to matching 50 percent of contributions by biotech and pharmaceutical companies to the fund through 2018. Companies contributing more than $1 million will be given the title of Founding Member.
ALS, also known as Lou Gehrig’s disease, is a debilitating and fatal disorder that strikes patients and their families in the prime of their lives. In the 150 years since it was first described, no effective treatment has been discovered, but recent progress in biological, genetic and clinical research has raised hope that we may be making progress toward slowing or stopping the devastating paralysis that characterizes the disease.
“Our goal is to increase the number, robustness and intensity of active ALS drug development programs in the biopharmaceutical industry by supporting the most promising preclinical research and fostering vital interactions between academia and industry,” says Dr. Manish Raisinghani, president of Target ALS Foundation. “We are grateful for the generous support of Biogen and other organizations that fund our important mission and look forward to welcoming new industry collaborators to our urgent search for a treatment for people living with ALS.”
Donations to Target ALS fund the exploration of potent ideas that might not have advanced otherwise and have enabled the creation of new models for scientific collaboration. Philanthropic support has sparked more collaborative relationships between academic and industrial partners, yet few therapeutic targets—essential for drug development and later clinical trials—have been validated. Target ALS, working together with other ALS foundations, aims to fill this gap.
“Biogen is deeply committed to ALS research and will continue to contribute both intellectually and financially to the search for a treatment for this devastating disease,” says Dr. Spyros Artavanis-Tsakonas, chief scientific officer of Biogen. “We challenge our industry peers to join us in precompetitive support of Target ALS and their groundbreaking mission to drive research collaboration across the public and private sectors.”
According to a recent statement by the ALS Association, the disease shares certain attributes with other neurodegenerative afflictions such as Parkinson’s disease. The association notes that “While each disease is unique in its symptoms and ultimate causes, researchers increasingly believe that all neurodegenerative diseases, including Alzheimer’s and Parkinson’s, share some important features. The presence of these common features means that an increased understanding of one disease is likely to have major benefits in understanding, treating and ultimately curing each of the others.”
The features of ALS that most, and perhaps all, neurodegenerative diseases share include:
- Neurons highly sensitive to stress, which could include exposure to environmental toxins.
- Misfolded proteins and reduced protein recycling. Proteins must fold into their proper three-dimensional form to function correctly. Misfolded proteins are normally recycled. In neurodegenerative diseases, protein misfolding increases and recycling decreases, leading to accumulation of potentially toxic proteins.
- Toxic proteins spread from one neuron to another. Researchers increasingly see evidence that once neurodegeneration begins in a small group of cells, it can spread from cell to cell, spreading the problem of misfolded proteins described above.
- Neuroinflammation. An inflammatory response triggered by the immune system is believed to contribute to the worsening of neurodegenerative diseases.
As if on cue, Dr. Stevin Zorn, executive vice president of research at Lundbeck, a 70-year old foundation-owned Danish company with a focus on neuroscience, commented on how the more they learn about Alzheimer’s disease, the more they realize they are likely not searching for a single trigger, but rather a host of factors (genetic and biological) that manifest differently for every patient. Like cancer, Alzheimer’s disease is not one disease but multiple diseases with underlying genetic abnormalities that cause damage from different angles.
Lundbeck takes a multifaceted approach to public-private collaborations by partnering across the spectrum in the Alzheimer’s space, including academic partnerships for early-stage research, such as the Gladstone Institute, and participation in the Global Alzheimer’s Platform, which is designed to remove logistical hurdles that interfere with the speedy enrollment of clinical trials. Lundbeck is currently in Phase 3 testing of idalopirdine, a drug designed to improve symptoms and neurocognitive functions. Lundbeck is also in Phase 3 trials to see if Rexulti (brexpiprazole), the recently approved schizophrenia drug and add-on treatment to an antidepressant medication to treat adults with manic depressive disorder, can help Alzheimer’s patients suffering from agitation.