More than five million people across the United States and some 30 million worldwide suffer from Alzheimer’s, a devastating disease that attacks the brain and leads to memory loss, the inability to perform daily tasks and functions, and eventually death.
Neil Buckholtz, the director of the Division of Neuroscience at the National Institute on Aging, has been the driving force behind an ambitious public-private partnership focused on finding the biological markers that show how Alzheimer’s progresses in the brain so that researchers can more quickly target the testing of new drugs to slow or stop the disease.
“Alzheimer’s disease is a major public health problem which will only get much worse if new ways to treat and prevent it are not found,” said Buckholtz. “It is critical for the federal government to facilitate the discovery, development and testing of new therapeutics to help people and their families who are desperately in need.”
This multi-million dollar project, started in 2004 and now in its second phase, has led to clinical trials at 57 sites in the United States and Canada, and involved participation of the National Institutes of Health, the Food and Drug Administration, universities, nonprofits and numerous pharmaceutical, diagnostic and biotechnology companies.
The initiative is unique not just because of its ambitious goals and the broad coalition of participants, but because of the requirement conceived by Buckholtz and colleagues that all of the data and findings be made available on a public database for use by scientists around the world. The database contains thousands of brain scan images, clinical and neuropsychological data, genetic information, and blood and cerebrospinal fluid analyses.
Unlike most clinical trials, no one owns this data or can submit patent applications, but companies could eventually benefit from any drugs or imaging tests that are developed from use of the information. There are only a handful of medications approved for treatment of Alzheimer’s in the United States, but they do not stop the disease and have only marginal effects on the symptoms.
The project, formally known as the Alzheimer’s Disease Neuroimaging Initiative, has led to more than 170 scientific papers, helped lead to new insights into people at high risk of Alzheimer’s progression, and accelerated the testing of new medications. This collaboration also has been a model for biomarker studies in other diseases such as Parkinson’s and Down syndrome.
“This collaborative effort has deepened our understanding of this complex disorder. Numerous scientific laboratories are processing data, writing abstracts and papers, and reporting the results at meetings,” said Buckholtz. The results have been extremely positive and concrete and are being used extensively in academic and industry clinical trials.”
Dr. Richard Hodes, the director of the National Institute on Aging, said Buckholtz is an “outstanding scientist, administrator and public servant” who had a vision, worked with a group of diverse partners and has achieved remarkable success so far with additional progress on the horizon.
“Neil commands a great deal of respect,” said Hodes. “People have confidence and faith in his abilities and his desire to achieve a common goal, and they know he is even-handed and fair.
This project that he led is renowned for what it has accomplished.”
Buckholtz joined the National Institute of Mental Health in 1983 and later served as chief of the National Institute of Aging Dementias of Aging Branch from 1993-2012, where he managed the Alzheimer’s project. He briefly retired in 2012 before growing restless and coming back as director of the Division of Neuroscience, where he now oversees the project along broader responsibilities to reviewing grants and other research involving Alzheimer’s and dementia.
“I actually attempted to retire, but found that I am still drawn to the field and hope to continue to make contributions for as long as I can,” said Buckholtz. “I am motivated by what is going on in science and the chance we have to test and develop new drugs. It will be very gratifying if I can help in some way.”
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