There is currently just one disease-modifying therapy for Alzheimer’s disease (AD). Increasingly, AD R&D is being performed by academic groups and early-stage biotech companies. Many of these programs stall in the “valley of death”, due to insufficient funding, expertise, and tools required to develop and commercialize drugs. To bridge this gap, the venture philanthropy model has emerged as a complimentary driver of translational research amongst public and private funders. Venture philanthropy combines deep disease-focused expertise and networks, with funding in high-risk/high-reward drug programs. Funding is structured to enable returns on investment, which are reinvested into further drug development projects. Venture philanthropies have contributed to advancing more preclinical AD candidates into clinical trials and helping academic and early-stage biotech programs reach critical scientific and business milestones. With recent examples of successful returns on investment, more capital is available to feed the AD therapeutic pipeline, expand clinical trials, and develop biomarkers to support these programs.

Alzheimer’s disease (AD) drug development is a complex process that proceeds from identification of a biological target; to testing of candidate therapies in in vitro assays; assessment of efficacy in animal models and assessment of safety in several animal species; clinical testing in humans in Phase1, Phase 2, and Phase 3 clinical trials; regulatory review by agencies in all countries in which the drug might be marketed; and eventual commercialization. This process requires more than a decade to accomplish. The process involves substantial infrastructure resources; multiple stakeholders; and funding from a variety sources along the developmental pathway. This is the complex ecosystem that supports AD drug development.

While Alzheimer’s disease (AD) remains one of the very few common chronic diseases of aging and old age without any effective treatments to slow or prevent the illness, an historical perspective can provide the context for why this is true. The same historical perspective demonstrates that this lag is primarily a frame shift in time, rather than due to excess difficulties in developing drugs for the disease. Indeed, the historical perspective suggests that today, we are “right on time” for an explosion in new drugs for the disease after 40 years of rapid progress in basic and clinical research. The first drugs (cholinergic agents) for AD were approved after only about 15-20 years of research in the field, including the time needed for basic discovery, clinical development and regulatory approval.  Today, after 40 years of AD research, many disease-modifying drugs are in clinical development. Considering that cancer, diabetes and hypertension research began more than 80-100 years ago, the rapid progress in AD research can actually be viewed as impressive progress.