Press Release

First-Time Conference Highlights Alternative Funding Models & Cutting-Edge Technologies That Can Better Treat Conditions Affecting Millions of People

Feb 26, 2015– It is estimated that over 25 million Americans are affected by a rare disease. Developing a treatment for even a common disease takes an average of 14 years at a cost of

$2 billion, with a 95 percent failure rate. Patients, researchers, and clinicians fighting rare diseases face added problems of restricted resources, small patient populations, and limited specialists and researchers that understand the rare disease. In addition, 50 percent of rare diseases affect children, and 30 percent of patients die before their fifth birthday. These challenges demand urgent and new, innovative approaches for rare-disease researchers.

Benefunder, an organization that works with wealth management firms to connect donors with top researchers across the nation, will join other industry and academic leaders to discuss solutions to these challenges on Thursday, March 5 during the  UC Davis Department of Chemistry’s 15th annual R. Bryan Miller Symposium. The symposium, which will be held at the UC Davis Conference Center, is the first of its kind to take a pioneering look at rare disease research, the obstacles, and new approaches that are shaping the future of research, and how to treat difficult diseases. Speakers and panels from academia, industry and patient advocacy groups will share perspectives on driving innovation towards finding cures for people living with rare diseases. The event is held in conjunction with Rare Disease Day, on Feb. 28.

Symposium topics will include emerging technologies, such as stem cell therapy, gene therapy and RNAi therapy, as well as new research, fundraising models and partnerships, the importance of diagnostics, and integrative biologic strategies.

As a presenter and moderator during the conference, Gert Lanckriet, Academic Ambassador and Cofounder of Benefunder, will discuss alternative funding models to support basic and fundamental research and for pushing applied research into the market.

“As federal dollars are increasingly harder to attract for research, top scholars and scientists are looking for novel funding methods to further fundamental understanding of our world and life, and to help advance knowledge, cures, therapies, and new technologies, and to bring them to the masses,” Lanckriet said. “Benefunder, which connects top researchers with philanthropists, provides an important alternative funding channel that helps advance research and innovation.”

The Benefunder panel discussion will include:

• Dr. Peter Stacpoole, a University of Florida physician who has developed a novel treatment for combating mitochondrial diseases
• Elva Diaz, a UC Davis researcher who is taking a groundbreaking approach to uncovering cures for brain diseases
• Petr Janata, a UC Davis psychology professor whose novel research links music to brain function

Dr. Stacpoole -- who has devoted his life to giving children with a rare disease a longer lease on life -- said rare disease research, especially that involving clinical trials, is extremely challenging.

“Rare disease research is traditionally grossly underfunded and must compete for federal dollars more commonly allocated to common diseases, such as diabetes and cancer,” he said. “The number of physician-scientists with the knowledge to develop and conduct scientifically and ethically sound clinical trials in a rare disease is very small. Thus, it is not surprising that a major unmet challenge facing rare disease patients and researchers alike is overcoming the so-called ‘Valley of Death,’ in which lack of funding prevents new laboratory discoveries from being translated into promising treatments through clinical trials.”

Dr. Stacpoole has developed effective treatment options for a rare and previously fatal mitochondrial disease, called pyruvate dehydrogenase complex deficiency (PDCD), with an investigational drug called dichloroacetate (DCA). Further studies of DCA by Stacpoole and other scientists show promise in combating other diseases, including brain and other cancers. PDCD is a devastating neurological and muscular degenerative disease. However, some of Dr. Stacpoole’s patients with mitochondrial diseases have been treated with DCA since infancy for over 20 years, with encouraging results. These results have led him and his team to work with the FDA and an international PDCD family advocacy group to develop a clinical trial of DCA in children with PDCD who would be recruited from throughout the U.S.— the first study of its kind. If the trial results are positive, it could lead to DCA becoming the first FDA-approved treatment for PDCD.

 

About Benefunder

Benefunder is a unique hybrid organization -- part foundation and part tech startup -- that has created a new funding channel for research and innovation while providing philanthropists with a smarter and more meaningful way to give. Through its Charitable Innovation Fund, Benefunder connects donors and the nation’s top researchers across all fields of study, including healthcare and life sciences, advanced technologies, the environment, and the arts and humanities. More information at www.benefunder.org.

 

About the R. Bryan Miller Symposium

The annual Miller Symposium honors late chemistry professor R. Bryan Miller, whose research laid the groundwork for current thriving programs in pharmaceutical chemistry and chemical biology at UC Davis. This year the symposium has added an extra day to focus on rare-disease research. The symposium is sponsored by the UC Davis Department of Chemistry in partnership with CheckOrphan and RARE Science. More information at http://chemistry.ucdavis.edu/seminar/miller_symposium/index.html.