Promise of revolutionary orally-administered drugs to combat Alzheimer's began journey 8 years ago
"Finding a needle in a haystack - 4 molecules out of a million"
NEW YORK, Aug. 6 /PRNewswire-USNewswire/ -- New therapeutics funded by the Alzheimer's Drug Discovery Foundation (ADDF), have achieved a significant milestone in their development as they advance toward the clinic, the ADDF announced today. PharmatrophiX, a biotechnology company founded in 2005 with funding from ADDF, has entered into an exclusive collaboration with Elan Corporation to research, develop, and commercialize a portfolio of small-molecule compounds that show promise in treating Alzheimer's disease and other neurodegenerative conditions.
"The road to this discovery was a search for a needle in a haystack," says Howard Fillit, M.D., Executive Director of ADDF, the only public charity whose sole mission is to fund drug discovery and development to prevent, treat, and cure Alzheimer's disease and cognitive aging.
The journey began when Frank Longo, M.D., Ph.D., began receiving funding from ADDF eight years ago while at UCSF (and then later at University of North Carolina) to find small molecules with the features of large biological agents that could both effectively combat neurodegenerative damage and be administered orally rather than intravenously - a challenge that has eluded researchers for years.
Dr. Longo, with the help of his UCSF colleague Stephen Massa, M.D., Ph.D., set out to find small molecules that could activate a receptor on brain cells (p75), protecting the cell from damage caused by the toxic amyloid molecules associated with Alzheimer's disease, but not induce the side effects triggered by the much larger natural biologic molecules. Out of the more than one million small molecules he studied, he ultimately found 4 that protected the cells and that had profiles suitable for further drug development. Once these molecules were identified, Dr. Longo formed PharmatrophiX as a biotech company in order to partner with industry.
PharmatrophiX is funded through a special hybrid investment vehicle begun by ADDF eighteen months ago - the Fund for Alzheimer's Drug Discovery. This novel philanthropic funding program is modeled after a traditional, "for profit" venture capital fund. It allows individual donors to participate more directly in accelerating drug discovery for Alzheimer's disease with greater accountability of their donations and potential for a return on investment that can further more research. Ninety-eight percent of the dollars in this fund go directly to drug discovery.
Using this "venture philanthropy" model, the foundation has supported more than 250 drug discovery programs, with 40% of these having advanced forward at least two key stages in the drug development process.
"This work we are supporting at PharmatrophiX is truly revolutionary and shows great promise in preventing and treating this terrible disease," says Dr. Fillit. "And this all started with an idea from Frank Longo that many thought was a little improbable. But ADDF is able to take chances on high-risk ideas - we know that this is often where the best solutions come from."
"And PharmatrophiX's exclusive collaboration with a major biotechnology company like Elan validates our funding to PharmatrophiX, and it underlines the significant impact of Dr. Longo's discovery in the field of Alzheimer's drug development," says Dr. Fillit.
"At ADDF, we are about results - bridging the gap between basic research and later stage drug development in humans. Alzheimer's is the fifth leading cause of death in the elderly and the third most costly disease overall. We're seeking to reduce the growing consequences this disease has on individuals and families."
About the Alzheimer's Drug Discovery Foundation
ADDF (www.alzdiscovery.org) is the only public charity whose sole mission is to accelerate the discovery and development of drugs to prevent, treat and cure Alzheimer's disease, related dementias and cognitive aging. We award grants to leading scientists conducting breakthrough drug discovery research. ADDF uses a venture philanthropy model to bridge the worldwide funding gap between basic research and later-stage development, using any return on investment to support new research. Since 1998, we have received over 1,600 requests to fund new ideas for Alzheimer's drugs. For all, we provided expert reviews and recommendations to advance their programs. We granted more than $35M to fund over 240 Alzheimer's drug discovery programs in academic centers and biotechnology companies in 12 countries.
SOURCE Alzheimer's Drug Discovery Foundation