CU Anschutz Researcher Receives Alzheimer's Association Grant

The Alzheimer’s Association, in partnership with a fundraising initiative led by philanthropist Michaela “Mikey” Hoag, announces a new $7 million investment in clinical trials that target brain inflammation as an innovative avenue for Alzheimer’s disease therapy. Among the four clinical trials included in the newly-funded research is one led by noted Alzheimer’s researcher Huntington Potter, Ph.D., of the University of Colorado Anschutz Medical Campus.

The Part the Cloud Challenge on Neuroinflammation targets a critical gap in understanding and treating Alzheimer’s, and absorbs some of the financial risk associated with advancing these studies across a space in drug development where many promising ideas stall due to lack of funding. This innovative funding program is the vision of Mikey Hoag, of Atherton, California, whose family is affected by Alzheimer’s disease.

“When my father passed away with Alzheimer’s, I decided to use my personal story to rally others in support of Alzheimer’s research. When my mother started to show signs of the disease, I knew I had to kick these efforts into high gear,” said Hoag. “We hope the competition we’re creating for additional funding will speed the rate of discovery and deliver a new and effective treatment or prevention strategy to doctors’ offices and people’s medicine cabinets more quickly.”

Each study will receive $1 million to advance current research to the next stage of clinical trials. A unique, goal-driven competition offers an additional $3 million to the clinical trial that demonstrates the most promise for treating this devastating disease.

“The importance of the Part the Cloud Challenge from the Alzheimer’s Association cannot be overstated,” said Dr. Potter, who is director of the Rocky Mountain Alzheimer’s Disease Center and professor and director of Alzheimer’s disease research, Department of Neurology, Linda Crnic Institute for Down Syndrome, University of Colorado School of Medicine. “The million dollars, with a potential for another $3 million after two years, will certainly propel novel research forward towards a therapy for Alzheimer’s. For my research, the hope is that Leukine, an FDA-approved drug, may slow or even prevent the progression of Alzheimer’s disease.  That would be a home run for everyone.”

“There has not been a genuinely new Alzheimer’s drug in more than a decade, and there is currently no drug that stops or slows the progression of this devastating disease,” said Linda Mitchell, president and CEO of the Alzheimer’s Association of Colorado. “The Association’s Part the Cloud Challenge is a much-needed and inventive approach to complement mainstream drug development that we hope can change the current situation.”

Increasing evidence suggests neuroinflammation plays an important role in the brain changes that occur in Alzheimer’s and other neurodegenerative diseases. By further understanding the role and the timing of neuroinflammation and immune responses, there is an opportunity to further accelerate novel candidate Alzheimer’s therapies.

Inflammation is a natural immune system response to infection and injury where defense cells are directed to fight infection or repair damaged tissue. However, persistent or misdirected inflammation can damage otherwise healthy tissue, such as the destruction of joint cartilage that occurs in arthritis or nerve damage in multiple sclerosis. Similarly, inflammation in the brain may help protect it from harm, such as the formation of the hallmark amyloid plaques of Alzheimer’s, but too much inflammation may damage the brain’s delicate nerve cells and intricate connections.

The four selected projects will receive $1 million over two years for either a Phase I or Phase II trial. Projects will be evaluated for their ability to advance in human testing, such as being safe for use in people and the ability to influence the underlying biological process they are meant to target. The project that demonstrates the most viable translation to advanced clinical trials will be eligible to receive an additional award of up to $3 million to further therapy development. Three of the four studies are testing potential therapies developed for other conditions that are being repurposed for Alzheimer’s.

“This funding from the Alzheimer’s Association is a testament to the incredible potential that Dr. Potter’s work holds for the future,” said CU Anschutz Medical Campus Chancellor Donald M. Elliman, Jr. “As the director of the Rocky Mountain Alzheimer’s Disease Center, he is ideally positioned to bring together teams of the brightest minds in medicine across the University of Colorado system to unravel the mysteries of Alzheimer’s disease. This generous investment will accelerate Dr. Potter’s work, bringing us closer to novel therapies that could impact countless lives in Colorado and around the world.”

“I like to think we have the brightest, most hard working scientists who also have huge hearts for our families,” said Michelle Sie Whitten, president and CEO of the Global Down Syndrome Foundation – an affiliate of the Linda Crnic Institute for Down Syndrome. “We are so pleased Dr. Potter has received $1 million from the Alzheimer’s Association to advance his research.  His work is shining a light on the link between Down syndrome and Alzheimer’s disease, and his team is focused on several innovative paths towards better treating and preventing Alzheimer’s.”

The funded projects are:

• A Phase II clinical trial of the FDA-approved drug Leukine, to determine whether it is safe and can help slow or prevent the progression of Alzheimer’s, led by Huntington Potter, Ph.D., Professor and Director of Alzheimer’s disease research, Department of Neurology, Linda Crnic Institute for Down syndrome, University of Colorado Anschutz Medical Campus. Leukine is approved for reducing and preventing infection in people who have received chemotherapy.
• A Phase II clinical trial to determine if the drug Sativex, a cannabis-based liquid medication that was previously tested for the alleviation of cancer-related pain, reduces brain inflammation and helps slow the progression to Alzheimer’s disease in people with mild cognitive impairment, led by Isidro Ferrer, M.D., Ph.D., Coordinator of the group Neuropathology at CIBERNED (Network Center for Biomedical Research in Neurodegenerative Diseases), Institute of Health Carlos III, Barcelona, Spain.
• A study to test if treatment with the drug Senicapoc can reduce brain inflammation, alter the rate of brain amyloid accumulation, and improve memory in people with early Alzheimer’s disease or mild cognitive impairment. In previous research, a drug similar to Senicapoc helped to reduce brain inflammation, prevent nerve cell damage, and improve memory in mice with an Alzheimer's-like condition. The project includes a Phase II clinical trial led by John Olichney, M.D., Professor and Neurologist at the University of California, Davis. Senicapoc has been shown to be safe in clinical trials of sickle cell anemia and asthma, but has yet to be tested in people with Alzheimer's.
• A Phase I clinical trial to examine the safety and efficacy to reduce brain inflammation of a novel therapy manufactured by Longeveron LLC using stem cells derived from healthy adult donors and that are delivered into the bloodstream of people with mild Alzheimer’s disease. Anthony Oliva, Ph.D., senior scientist at Longeveron, will serve as principal investigator, and Bernard Baumel, M.D., will serve as the clinical investigator of the trial at the University of Miami Miller School of Medicine. Longeveron is a life sciences company located in Miami, Florida. In past research, this type of stem cell has demonstrated the ability to target and reduce inflammation, promote tissue repair, and improve brain function in mouse models of Alzheimer’s disease.

Individuals interested in applying to participate in the research should register for the Alzheimer’s Association’s TrialMatch program or call the Association’s 24/7 Helpline at 800-272-3900.