Benefunder Joins UC Davis Symposium on Rare Disease Research & Precision
Medicine
First-Time Conference Highlights Alternative Funding Models & Cutting-Edge
Technologies That Can Better Treat Conditions Affecting Millions of People
Feb 25, 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.