Taube to fund $3m Huntington’s disease research in US

Program will use gene editing and stem cell therapies to find treatments, cure for devastating disease

Shoshanna Solomon was The Times of Israel's Startups and Business reporter

Philanthropist Tad Taube speaks at the groundbreaking of the Taube Family Campus on June 29, 2016 at the Jerusalem-based HUC-JIR rabbinical school. (Avi Hayun)
Philanthropist Tad Taube speaks at the groundbreaking of the Taube Family Campus on June 29, 2016 at the Jerusalem-based HUC-JIR rabbinical school. (Avi Hayun)

Taube Philanthropies, founded by US-based Jewish businessman Tad Taube, said it would donate $3 million to support a collaboration between Stanford University School of Medicine and the Gladstone Institutes in California focused on research related to Huntington’s disease.

The donation will support a new program with three medical research groups and, for the first time, introduce gene editing and stem cell therapies for treatments and, eventually, a potential cure for Huntington’s disease, the philanthropic group said in a statement.

Researchers from Stanford School of Medicine and the Gladstone Institutes’ Taube-Koret Center for Neurodegenerative Disease Research, will collaborate with clinical trials at the Memory and Aging Center of the University of California, San Francisco (UCSF).

“Huntington’s disease is a cruel genetic disorder that robs its victims of physical and mental control,” said Taube, the chairman of Taube Philanthropies. “The time and research that Stanford, UCSF and Gladstone Institutes have committed to this cause is remarkable.”

Breakdown of nerve cells in the brain

Huntington’s disease is a genetic neurodegenerative disease that causes the progressive breakdown of nerve cells in the brain. It causes physical and mental abilities to deteriorate, eventually leading to premature death. There is no cure or approved drugs to slow the progression of the disorder. Huntington’s disease belongs to a family of neurodegenerative diseases that includes Alzheimer’s, Parkinson’s and ALS. Today, there are approximately 30,000 symptomatic Americans and more than 200,000 at risk of inheriting the disease.

“We have made considerable progress in advancing drug therapy developments for patients with neurodegenerative diseases since Taube Philanthropies started supporting my research, nearly a decade ago,” said Dr. Steve Finkbeiner, director of the Gladstone Institutes’ Taube-Koret Center. “With this renewed commitment and concerted approach focusing on Huntington’s disease, I am optimistic that we will continue advancing toward our research goals.”

The $3 million donation is a continuation of Taube Philanthropies’ aim to finding treatments and cures for neurodegenerative diseases. Over the last 12 years, the organization has donated over $5 million to Gladstone Institutes and $1 million to the Stanford University School of Medicine for related research. Taube Philanthropies started funding Huntington’s disease research under the direction of Nobel laureate Dr. Stanley Prusiner at UCSF’s department of neurology.

“We have been working to develop similar therapies for neurodegenerative diseases for many years with some promising results,” said Dr. Matthew Porteus, associate professor of pediatrics at Stanford. “Employing the latest technology, including CRISPR/Cas9 — a powerful new tool that enables scientists to make precise genome edits — I believe we have the opportunity to make great strides toward developing a therapy specifically for Huntington’s disease.”

The funds will be distributed to the researchers in annual payments through 2021, to account for the long-term process of translating scientific discoveries into clinical trials and applications. The research team will take on a multi-year program to develop genetic therapy for the disease. Stanford is already doing stem cell research and hopes to begin conducting clinical trials with Huntington’s patients soon, the statement said.

“Having studied Alzheimer’s for many years, I am all too familiar with the devastating effects of neurodegenerative diseases,” said Dr. Frank Longo, professor of neurology at Stanford. “As researchers with different area specialties convene for this unique joint project, I am optimistic about our capacity to come even closer to finding treatments and cures for these degenerative conditions.”

Research into Huntington’s disease has until recently suffered from a lack of significant funding. Due to the progress made in the field by researchers, new US federal funding has become available and major pharmaceutical companies have started funding research to develop possible medications. However, collaborative efforts still rely on contributions from individuals and private foundations, the statement said.

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