Paul Allen, philanthropist and co-founder of Microsoft, has devoted many millions of dollars to unlocking the mysteries of the brain, and today he’s taking on a new challenge: Alzheimer’s disease. The Paul G. Allen Family Foundation, launched by the tech mogul and his sister, Jody Lynn, in 1988, has awarded $7 million in grants to five teams of researchers who are working on new ways of combating the degenerative brain disease.

The new grants were largely driven by Allen’s desire to overcome the hurdles plaguing the field of Alzheimer’s drug development, says Judy Lytle, manager of medical research for the organization’s life sciences portfolio. Clinical trials of experimental therapies to treat the disease have suffered a 99.6% failure rate. There have been a number of high-profile failures in recent years, including bapineuzumab from Pfizer, Johnson & Johnson, and Elan Pharmaceuticals in 2012.

Allen, who has often spoken about his mother’s battle against Alzheimer’s, has a policy of thinking outside the box when it comes to funding medical research, Lytle says. The most recent tranche of money, for example—which was awarded under an ongoing program called the Allen Distinguished Investigator (ADI) grants—went to teams that include both neurology experts and scientists who have other areas of expertise, such as immunology.

By supporting such inter-disciplinary teams, Allen hopes to further research that will unravel clues to how Alzheimer’s disease develops, Lytle says. “We convened a board of experts from all over the country to discuss where they thought the holes in this field of Alzheimer’s disease research were,” she says. “They determined that there needs to be more work done to understand what’s going on at the cellular level—to really start to appreciate what the disease does at the early stages.”

Although the ADI program has been in place since 2010, today’s grants are the first to be devoted to Alzheimer’s, Lytle says. The five winning teams were chosen from a pool of about 50 applicants.

Three of the five grant winners are pursuing projects aimed at understanding the characteristic plaques and tangles that develop in the brains of Alzheimer’s patients. A University of Cambridge team is examining whether damage to the white matter in the brain contributes to the formation of plaques and tangles, while a group at the University of California at San Francisco will use genetic sequencing technologies to study how certain drug combinations interact with genes to affect those abnormalities. A second UCSF team will look at the question of why neurons in the brain are unable to clean up “garbage” proteins that cause the accumulation of plaques and tangles.

The two other grant recipients are focused on understanding the role of aging in Alzheimer’s. Researchers at Oregon Health and Science University are using imaging technology to determine how age affects the “glymphatic system,” a newly discovered process by which the body clears plaques and other unwanted substances from the brain.

Finally, Fred “Rusty” Gage—the scientist at the Salk Institute who is best known for demonstrating that the brain can continue to generate new neurons in adulthood—won an ADI grant to further his research in aging and neuron function. He plans to use cell culturing and RNA sequencing to compare gene expression in aging with specific changes that drive Alzheimer’s.

These projects could provide a deeper understanding of what’s going on in the brain before Alzheimer’s symptoms develop, Lytle says—and that could be essential to developing better treatments for patients. Although a handful of Alzheimer’s drugs have made it onto market, they merely lessen the symptoms and delay the progression of the disease. “If we understand that progression, maybe we can actually intervene at the appropriate time. But the first step is to understand what’s happening there.”

The Alzheimer’s program is the latest milestone in Allen’s plan to improve the world’s understanding of the brain. In May, the Allen Institute for Brain Science, which is backed by $500 million of his money, launched a cell types database containing information on 240 neurons.

Funding from philanthropists like Allen can be an important bridge for medical researchers who hope to ultimately win support from deep-pocketed investors like Big Pharma companies looking for fresh research to fill their pipelines. Federal funding for Alzheimer’s research currently stands at about $586 million a year, according to the Alzheimer’s Association, which recently celebrated a Senate Appropriations Committee proposal to boost that amount by 60%.

That’s good news, to be sure, but cross-disciplinary teams such as the ones Allen is supporting might have trouble accessing that money, Lytle says. Allen hopes to close that funding gap. “Paul is very interested in Alzheimer’s disease research—it hits close to home for him,” she says. “We’re in the process of putting together a 10-year plan for the topics we want to go after, and given that interest, I can see a role for neurodegenerative disease and Alzheimer’s specifically.”

Source: Forbes