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The Neuro Collaborative: An Engine for ALS Therapeutic Discovery

In 2013, the Golden West Chapter of The ALS Association, led by Advisory Trustees Jim Barber and Linda Della, developed the concept of a novel scientific initiative called the Neuro Collaborative. The idea was to build a synergetic model that brought together complementary expertise within the ALS research community to accelerate scientific discovery and de-risk investment in drug development.

In 2014, this research initiative, spearheaded by the Golden West Chapter, was one of the first to receive ALS Association funding from the global outpouring of support generated by ALS Ice Bucket Challenge.

About the Scientists

The Neuro Collaborative established a formal partnership between the Golden West Chapter, The ALS Association and three leading laboratories in California – including Don Cleveland, PhD at University of California San Diego; Steven Finkbeiner, MD, PhD. at Gladstone Institutes, affiliated with University of California San Francisco; and Clive Svendsen, PhD at Cedars-Sinai Regenerative Medicine Institute in Los Angeles. From sharing information to attracting industry investment, the Neuro Collaborative is a forward looking initiative designed to drive potential therapeutics for ALS quickly toward FDA approval.

Since 2009, these principal investigators have also served on the California ALS Research Network, and help to organize the annual California ALS Research Summit, presented by the Golden West Chapter. The Summit is an annual gathering of researchers, investigators, clinicians, biotech companies, government representatives, partner organizations, and advocates in ALS and related fields. The three scientists have also participated in many of the Golden West Chapter's educational events presented for people with ALS and their families.

Dr. Don Cleveland, professor at the University of California, San Diego’s Ludwig Institute for Cancer Research has been working with The ALS Association since 2004 to develop DNA-based "designer drugs" to silence ALS genes, called antisense gene silencing or antisense technology (ASO). In 1999, The ALS Association and the American Academy of Neurology awarded Dr. Cleveland with the prestigious Sheila Essey Award for ALS Research for his significant contributions. He also received the 2014 Commitment to a Cure Essey Award from the Golden West Chapter, along with Richard Smith, MD, Center for Neurologic Study and C. Frank Bennett, PhD, of Ionis Pharmaceuticals, Inc -- for their ASO research which may lead to a treatment for people with ALS and related diseases. Dr. Cleveland was awarded the $3 million prestigious 2018 Breakthrough Prize in Life Sciences for his numerous accomplishments in neurodegenerative disease research, including establishing ASO in animal models of ALS and demonstrating the promise of ASO drugs for neurodegenerative diseases including ALS. Dr. Cleveland’s efforts have led to ASO drug trials directly targeting genes responsible for the major inherited forms of ALS, as well as one gene implicated in sporadic ALS.

Dr. Steven Finkbeiner is the Director of the Center for Systems and Therapeutics and the Taube/Koret Center for Neurodegenerative Disease Research and Investigator at the Roddenberry Center for Stem Cell Biology and Medicine at Gladstone Institutes. He is also a Professor in Neurology and Physiology at University of California, San Francisco. Dr. Finkbeiner is well known for inventing robotic microscopy, a new form of imaging that has helped illuminate relationships in amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease), Huntington’s, Alzheimer’s and other neurodegenerative diseases. Dr. Finkbeiner received the 2017 Commitment to a Cure Essey Award from the Golden West Chapter for his pioneering work with neurodegenerative diseases and urgent focus on bringing effective treatments to the ALS community.

Dr. Clive Svendsen is the Executive Director of the Cedars-Sinai Regenerative Medicine Institute and serves as a Professor In-Residence, at UCLA Medicine. Dr. Svendsen has won numerous scientific awards, including the 2010 Sheila Essey Award for ALS Research from the American Academy of Neurology. He is the principal investigator on multiple research projects studying stem cells and ALS that have been awarded more than $36 million dollars in grant funding from the California Institute of Regenerative Medicine. He is currently working to develop novel ways of using stem cells modified to release powerful growth factors to treat patients with neurological diseases such as ALS, Huntington’s, Alzheimer’s and Parkinson’s. Dr. Svendsen serves as the Co-Director of the Answer ALS program. He is also the Chair for the California ALS Research Network, an initiative that was launched by the Golden West Chapter.

How the Neuro Collaborative accelerates the search for treatments and cures for ALS

The Neuro Collaborative is an initiative designed to drive potential therapeutics for ALS quickly toward FDA approval. The goal of the Collaborative is to efficiently advance ALS drug development together with industry partners. TThis synergetic model leverages open dialogue and the scientific expertise of leading researchers to achieve therapeutic milestones as quickly as possible. It also reduces risk in the drug discovery process and attracts pharmaceutical companies to invest in drug development and clinical trials for ALS. With success, potential therapies for ALS will move more quickly than ever toward FDA approval and the open market.

Some examples of goals of the Neuro Collaborative include:

  • Identify small molecules designed to eliminate SOD1 and C9orf72 – the two most common genetic causes of ALS (i.e. antisense therapy).

  • Discovery of novel, potential ALS drugs.

  • Move drug discoveries into clinical trials through partnerships with pharmaceutical companies in order to leverage various expertise in drug development (chemistry, toxicology, regulatory), which is lacking in academia.

  • Reinforce the clinical trial process to move drugs toward FDA approval.

  • Provide tools to the broader community and industry partners such as available induced pluripotent stem cell (iPSC) lines and screening capabilities through the Brain Bot and other emerging technologies.

Each laboratory affiliated with the Neuro Collaborative focuses on different areas of ALS research to maximize shared knowledge and understanding of the complexities of the disease while increasing industry interest and investment in ALS drug development. The Neuro Collaborative partners with prestigious scientific advisors including: Dr. John Dunlop, Ph.D., Vice President of Neuroscience Innovative Medicines at AstraZeneca, Fred Gage, Ph.D., Professor in Laboratory Genetics at the Salk Institute and Harry Orr, Ph.D., Professor of Laboratory Medicine and Pathology at University of Minnesota. In support of their efforts, the Neuro Collaborative was awarded an additional $3 million dollars in support by The ALS Association in 2017.

Laboratories that represent the Neuro Collaborative

The Cleveland lab at the University of California San Diego:

“With The ALS Association’s ongoing support, my work in [the] development of designer DNA drugs, which is frequently called antisense technology, for therapy of neurodegenerative diseases – once widely considered by almost everyone to be doomed to failure – has led to clinical trials that could help people with ALS and other neurodegenerative diseases.” - Dr. Cleveland

The Finkbeiner lab at the Gladstone Institutes, which is affiliated with UCSF:

The support for the Neuro Collaborative from The ALS Association has allowed us to approach therapeutics development for ALS with an urgency and at a scale that we could never do before. It has enabled us to greatly accelerate the work, make unexpected and promising new discoveries. In less than a year, we already formed our first partnership with a drug company entirely dedicated to ALS." -Dr. Finkbeiner

The Svendsen lab at Cedars-Sinai in Los Angeles:

“The funding from The ALS Association has allowed us to generate a set of valuable ALS stem cell lines and develop robust protocols for making motor neurons from them. As these are the cells that die in ALS, they should help us understand the underlying mechanisms of this disease.” -Dr. Svendsen