ADDF Grantees Headline NIH Alzheimer’s Summit

Earlier this month, Dr. Howard Fillit and I attended the 2018 NIH Alzheimer's Disease Research Summit as representatives of the Alzheimer’s Drug Discovery Foundation. ADDF-supported research was at the forefront of the Summit and the National Plan to Address Alzheimer's.

The NIH Summits, held every three years, are planning meetings to advance the National Plan and its goal to treat and prevent Alzheimer's disease by 2025. The purpose of this year's meeting was to enable the development of effective treatments for Alzheimer’s with a focus on precision medicine approaches.

The Summit took a holistic approach to Alzheimer's treatment. Presenters began by discussing what we know about the complex biology of Alzheimer’s before moving on to how biomarkers and big data are advancing drug development. Presenters emphasized the importance of expanding existing and building new resources to drive drug discovery and clinical development. The Summit also featured two sessions on emerging drugs now in trials. (Note: day two presentations were postponed due to inclement weather.)

The first session of the meeting—on the diverse biological pathways or systems that can lead to Alzheimer's—included ADDF grantees Carol Colton (Duke University, ADDF funding in 2014) and Elizabeth Bradshaw (Columbia University, funded since 2016). Dr. Colton is investigating immune suppression and the role of arginine in Alzheimer’s. Dr. Bradshaw is also focused on immune function, specifically the role of the brain's immune cells (i.e., microglia) in protecting neurons from damage. Both of these researchers are translating their findings into drugs thanks to ADDF support.

ADDF grantee Allan Levey (Emory University, funded since 2011) chaired the session on research tools and NIH-sponsored initiatives that aid in drug development. It featured presenter Jacob Hooker (Massachusetts General Hospital, funded since 2016), who develops PET ligands to illuminate brain processes. With ADDF support, Dr. Hooker developed a PET tracer that allows us, for the first time, to see epigenetic changes in the brain. His exciting work is accelerating the development of epigenetic therapies for Alzheimer's.

Many presenters in the sessions on emerging therapeutics were ADDF grantees. The first, Frank Longo (Stanford University, funded since 2000), is conducting a phase 2 trial of his drug LM11A-31. It targets a receptor that may protect neurons for multiple sources of damage and slow the progression of Alzheimer's. Roberta Diaz Brinton (University of Arizona, funded since 2004) shared her development of the neuro-steroid allopregnanolone as a treatment for Alzheimer's. Allopregnanolone has shown potential to regenerate neurons and interim phase 2 results have been positive. Mark Gurney (Tetra Therapeutics, funded since 2016) is preparing for a phase 2 trial of BPN14770, which is designed to protect synapses and preserve memory. Mark Tuszynski (University of California, San Diego, funding began in 2000) is developing therapies that target nerve growth factors. Linda Van Eldik (University of Kentucky, funded in 2014) and Martin Watterson (Neurokine Therapeutics, funded since 2001) are developing MW189 and MW150, respectively, which target aspects of inflammation in the brain. These drugs, on which Dr. Van Eldik and Dr. Watterson collaborated during early discovery, are both expected to enter phase 1 trials this year. Finally, Michela Gallagher (Agenebio, funded since 2010) is preparing for phase 3 trials of her drug AGB101, which tames hippocampal overactivity. Initial results suggest that AGB101 can slow the progression of mild cognitive impairment to Alzheimer’s and may act as a preventative therapy.

I and my colleagues at the ADDF are proud to contribute to the National Plan to Address Alzheimer's. The Summit made clear that our early investments in novel ideas to treat this disease are now a critical component of that plan. We will continue to support these and other researchers who are forging new paths toward a cure.