Research at Health Department's Wadsworth Center Holds Promise for Alzheimer's, Parkinson's, Huntington's Diseases
ALBANY, NY, August 22, 2005 – Scientists at the New York State Health Department's Wadsworth Center have shown that a novel therapy initially developed to fight cancer and HIV/AIDS also holds promise for treating neurodegenerative diseases, such as Alzheimer's, Parkinson's and Huntington's Diseases.
The findings prove that engineered antibody fragments directed at brain cells can lessen or slow degeneration caused by altered proteins that clog neurons and disrupt motor and cognitive functions.
"While science has not yet unlocked the secrets to these debilitating diseases, this finding by Wadsworth scientists takes us another step forward toward understanding and, perhaps, someday preventing and treating these diseases," said State Health Commissioner Antonia C. Novello, M.D., M.P.H., Dr.P.H.
The study led by Drs. Anne Messer and William Wolfgang was reported online in the August 1, 2005, early edition of the Proceedings of the National Academy of Sciences. It appeared in the journal's August 9, 2005, print edition.
The gene that causes Huntington's disease (HD) produces altered proteins that amass within the brain cells. Using a fruit fly model with a human gene for HD, the scientists demonstrated that flies treated with an engineered antibody against the protein all matured to adulthood, increased their life span by 30 percent, and accumulated fewer altered proteins than untreated flies. There is no known effective treatment or cure for the estimated 200,000 Americans at risk for inheriting Huntington's.
"The promising research of our Wadsworth Center scientists puts New York State on the cutting-edge of scientific medical research. As our population ages, and the incidence of brain disorders such as Alzheimer's, Parkinson's and Huntington's diseases grows, any steps that move us closer to potential new treatments are encouraging," Dr. Novello said.
This is the first successful use of an engineered antibody (intrabody) therapy against a neurodegenerative disease in a living organism. Engineered antibody fragments are directed against proteins within a cell. Like all antibodies, they bind readily and specifically to their target molecule and can interfere with protein folding or protein to protein interactions.
Dr. Messer earlier showed in cell and tissue culture that engineered antibodies successfully interfered with the formation of altered brain cells that cause neurodegenerative diseases. The long-term goal of Dr. Messer's investigation is the development of direct gene therapies against neurodegenerative diseases.
Support for Dr. Messer's research comes from the Huntington's Disease Society of America; the Hereditary Disease Foundation; the Cure Huntington's Disease Initiative; and the National Institutes of Health. Dr. Messer also holds a faculty appointment in the School of Public Health, University at Albany. Her co-authors include Todd W. Miller and Jack M. Webster, formerly her graduate student and postdoctoral fellow, respectively, James S. Huston of IntraImmune Therapies; the EMD Lexigen Research Center; and Leslie M. Thompson and J. Lawrence Marsh of University of California Irvine.
For more information on the Wadsworth Center, please visit its web site (http://www.wadsworth.org).