The skills of basic scientists, clinician-scientists, and medicinal chemists are almost completely synergistic, says Stephen Hauser of UCSF, but silos have developed that can limit their interactions. A new alliance seeks to change that.
SAN FRANCISCO—The University of California, San Francisco (UCSF) and the University of California, Berkeley (UC Berkeley) recently joined a long-term research partnership with Genentech and its parent company Roche for the development of new therapeutics for brain diseases and disorders of the central nervous system (CNS).
The partnership will target diseases like Alzheimer’s, Parkinson’s, Huntington’s, amyotrophic lateral sclerosis (ALS), and autism. UCSF and UC Berkeley will receive up to $53 million in funding from Genentech over the course of a 10-year collaboration.
“At Genentech and Roche, we are strongly committed to discovering and developing breakthrough medicines to help improve the lives of people living with neurological disorders, which are some of the most complex conditions to understand and treat,” commented James Sabry, global head of pharma partnering at Roche. “We are excited to join forces with Weill Neurohub researchers and clinicians to accelerate scientific and clinical advancements for neurological disorders so that we can slow—or even one day reverse—the progression of these conditions.”
The partnership, called the Alliance for Therapies in Neuroscience (ATN), is a major milestone for Weill Neurohub—a joint research collaboration between UCSF, UC Berkeley, and the University of Washington. The Innovative Genomics Institute will also be a key collaborator. Weill Neurohub was launched in 2019 with a focus on disorders of the brain and nervous system. The Genentech/Roche partnership will put the collaborative project into action, with a focus on developing much needed therapies for the targeted disorders.
“This represents a perfect opportunity to align the scientific expertise of our great public institutions with innovation that thrives at Genentech and Roche. [The partners] have longstanding commitments to the neurosciences, and the strongest possible motivation to find better answers for the billion people affected each year by a brain disorder,” said Stephen Hauser, the Robert A. Fishman Distinguished Professor of Neurology at UCSF and director of the Weill Institute for Neurosciences. “We also have a long history of working together, so this new partnership accelerates and strengthens our longstanding relationships and collaborations.”
“The skills of basic scientists, clinician-scientists, and medicinal chemists are almost completely synergistic—they should be interlocking—yet for numerous reasons, silos have developed which can limit the interactions with each other and ability to form close partnerships,” continued Hauser, who will co-chair the joint steering committee for the ATN. “Finding solutions to this problem is [the core focus] behind the ATN initiative. We believe that by combining our strengths and working closely together to advance translational research, we can speed discovery of underlying disease processes and develop novel therapeutics that would otherwise not be possible.”
“The initial projects funded under the ATN will focus on developing biomarkers to track disease progression and the effectiveness of future therapies; understanding molecular pathways of protein breakdown that drive disease; parsing genetic and protein profiles linked to CNS disorders; and developing therapeutics for brain diseases based on CRISPR technologies,” noted Ehud Isacoff, the Evan Rauch Chair in Neuroscience at UC Berkeley, and head of the Helen Wills Neuroscience Institute.
The collaboration will focus on topics including neurodegeneration, CRISPR-based therapeutics, protein degradation, functional genomics in human brain cells, and sleep mechanisms and circuits.
“In many neurodegenerative diseases, sleep impairment occurs long before diagnosis, and can impact the progression of the disease. Disrupted sleep is also a central feature of various neurodevelopmental disorders. Understanding sleep mechanisms and circuits in healthy and disease states could offer insights for addressing an unmet medical need,” Hauser points out. “Restoration of normal sleep would greatly benefit the lives of patients and families, and may have the potential to modify disease progression.”
Joint project teams will use the expertise and resources between academia and industry to better understand disease pathologies, discover novel targets, generate new preclinical models, and develop novel translatable biomarkers. The partnership hopes this research will accelerate the development of potential therapeutics for patients with CNS disorders.
“What we’ve announced is just the starting point of a long-lasting relationship that has the potential to go in new directions. In fact, we hope that by collaborating, we may discover new therapeutic areas over the next decade that we haven’t even considered yet,” added Hauser. “This partnership is not about picking off the low-hanging fruit—it’s about transforming the treatment of neurological and psychiatric disease, which will require long-term investment and the flexibility to go where the science guides us.”
“Weill Neurohub is especially well placed to lead in the areas of neuroimaging, neuroengineering, neurogenomic and neuromolecular therapeutics, and neurocomputation/data analytics. These pillars are strongly positioned to deliver high-impact results, including the development of next-generation devices and therapies positioned for rapid translation to the clinic. It is really hard to imagine that significant progress will not be made in each of these areas, because the scientific expertise and passion across the teams are so strong,” Isacoff concluded. “What I am hoping for is that some of these things lead to a breakthrough where eventually we are looking back a decade or two from now and saying, ‘Holy moly! That is when we turned the corner on neurological disease.’ That’s the dream.”
University of California, San Francisco https://www.ucsf.edu/
University of California, Berkeley https://www.berkeley.edu/
Weill Neurohub https://www.weillneurohub.org/
University of Washington https://www.washington.edu/
The Innovative Genomics Institute https://innovativegenomics.org/