Consider that with more than 10,000 known diseases affecting our world today, there are treatments available for only 500 of them. Philanthropy accounts for just about 3 percent of all medical research investment made annually in the US, but it can have an outsized impact if deployed using smart, and precise, giving strategies to invest in high-risk biomedical research with the potential to make significant impact on medical innovation.

The Growing Field of Precision Medicine

Medical advances such as the mapping of the human genome have revolutionized research, and the field of “precision medicine” allows doctors and patients to make informed decisions about treatments targeted specifically to address a patient’s individual molecular profile. In other words, precision medicine means getting the right medicine to the right patient at the right time.

The field of precision medicine gained attention earlier this year when President Barack Obama announced the Precision Medicine Initiative, which aims to revolutionize medicine and generate the scientific evidence needed to move the concept of precision medicine into everyday clinical practice. In addition to cancer, medical advances from precision medicine have shown promise for neuroscience, including Alzheimer’s disease, one of the greatest health challenges of our time.

The Challenge of Alzheimer’s

Alzheimer’s disease is a neurodegenerative disease that severely impairs memory, cognition, and a person’s ability to conduct common daily activities. It is the sixth leading cause of death in the United States, claiming the lives of more than 500,000 people in the US each year. Currently, more than five million Americans are living with Alzheimer’s disease. The economic impact of the disease is significant, costing the United States $214 billion in 2014 and on pace to escalate to more than $1 trillion over the next four decades. In fact, it was estimated in 2010 that Alzheimer’s disease and dementia generally cost our global economy over $600 billion, which was about 1% of our global GDP.

There is no cure for Alzheimer’s disease, and currently the three therapies approved by the US Food and Drug Administration treat the symptoms but do not modify the disease to cure or slow it down. According to a study in Scientific American, Alzheimer’s drugs have a 99.6% failure rate, compared with 81% for cancer. New and effective treatment options are desperately needed.

The Promise of Precision Treatments

Philanthropy plays a key role in developing treatments for Alzheimer’s disease. As part of its Philanthropy Advisory Service, a program that offers families and foundations resources and insights to help maximize the return on their philanthropy, we at the Milken Institute undertook a project to understand the state of Alzheimer’s science — unmet needs, research roadblocks, and promising opportunities for philanthropy — to accelerate the efforts to find a cure.  With input from over a dozen leading Alzheimer’s experts, we created “Alzheimer’s Disease: A Giving Smarter Guide to Accelerate Development of New Therapies.”

One exciting opportunity related to precision medicine is the development of a new preclinical drug screening model, termed “Alzheimer’s in a Dish,” developed by Dr. Rudolph Tanzi of Massachusetts General Hospital. This technology could help researchers better understand how an experimental therapy will work before it’s ever tried in humans, thereby avoiding costly and time-consuming clinical trials for non-efficacious treatments.

Using a small skin biopsy from an Alzheimer’s patient to form stem cells, researchers can program these cells to evolve into neurons – the brain cells most impacted by Alzheimer’s disease. These neurons are then grown in a special 3D matrix gel that maintains the key pathological features of the disease. This is important because many of the cellular and animal models historically used to study Alzheimer’s do not adequately simulate human Alzheimer’s pathology.

The 3D matrix of neurons can then be used to screen a pharmacopeia of approved drugs to identify agents that may be effective in the original patient as well as in other Alzheimer’s patients with similar disease signatures. If successful, the approach can be automated and scaled to investigate a wider array of patients with other Alzheimer’s disease signatures. The approach represents a much-needed departure from traditional drug development strategies, and capitalizes on emerging genetic, pharmacologic, and imaging technology.

The PAS team has identified other important and exciting opportunities where philanthropy can play an outsized role in finding a cure for Alzheimer’s disease, including:

  • identifying new druggable molecular targets,
  • developing reliable biomarkers that signal presence and progression of disease,
  • promoting collaboration among researchers,
  • funding young investigators,
  • increasing public awareness about the opportunities for research, and
  • advocating for public policy that invests in cures.

Precision medicine has opened the door to new ways to conduct research. By investing in promising therapies in a precise manner, philanthropy can play an important role in seizing the opportunities that this growing field of medical research has provided. The targeted approach of precision medicine, combined with targeted funding, will result in the greatest return on philanthropy for Alzheimer’s disease and other conditions that urgently need effective treatments.

Melissa Stevens is executive director of the Milken Institute Philanthropy Advisory Service, which provides resources and insights to foundations and philanthropists to build high-impact philanthropy portfolios across medical research, education, and public health. LaTese Briggs, PhD, is the director of the Philanthropy Advisory Service at the Milken Institute and oversees the medical research portfolio. Briggs previously served as a pharmaceutical market analyst for Decision Resources, a Boston-based research and consulting firm serving the biopharmaceutical industry.