Crusading for Science
Regina Kelder

Crusading for Science

With the pharmaceutical sector more reticent about drug discovery, disease foundations are increasingly driving the science, and in the quest for novel treatments, cures and vaccines they are turning more to CROs

In 1991, a handful of breast cancer survivors launched the grass-roots National Breast Cancer Coalition to raise money and awareness. The group successfully lobbied US legislators to expand research and worked strategically to get Genentech’s novel targeted therapy Herceptin—one of the first monoclonal antibodies—into the clinic (see our post on Eureka.)

Clearly, this disease advocacy model works, but today’s not-for-profit disease foundations also want a seat on the bench. What’s interesting to us (for obvious reasons) is that these foundations are not only working with a diverse array of research players—including contract research organizations (CROs)— to advance their scientific goals both, they are also  helping to accelerate the often gelatinous pace of translational research.

Foundations have established research consortiums to search for groundbreaking brain imaging tools, and partnered with laboratories—including CROs—to develop novel drugs for lethal and largely untreatable genetic disorders.  Disease foundations are dominant players in the search for a vaccine that could help end AIDS, and in some cases they remain the only viable funder of research for neglected diseases, particularly those ravaging the developing world.  They consistently seek out CROs—whose clients historically came from the biopharmaceutical sector—to help bridge the gap in discovery and preclinical science.

How important are CROs in helping charitable organizations advance their science? The Alzheimer’s Drug Discovery Foundation recently created a platform called ACCESS to help foundations work with CROs. The program gives academic and small biotech communities online access to a marketplace of CROs and the industry standard drug discovery and development services they provide.

While the faces behind these foundations range from the famous to the obscure, but they share one thing in common: A passionate, almost obsessive desire to find a cure, vaccine or better therapies for diseases that have touched them personally. Television and film star Michael J. Fox started his foundation in 2000 after being diagnosed with early-onset Parkinson’s disease at the age of 31. Warren Buffet’s reinsurance chief Ajit Jain launched a foundation in 2005 to find a cure for a type of muscular dystrophy caused by dysferlin protein deficiency that afflicts his son.  And Ted Stanley, who amassed a fortune selling collectibles, drew headlines in July when he donated US$650 million to the Broad Institute in Cambridge, MA to study schizophrenia (see our post on Eureka.) Stanley created The Stanley Medical Research Institute in 1989 (a non-profit) to fund psychiatric research after seeing the toll bipolar disorder took on his college-age son.  On a more global scale, Microsoft founder and billionaire Bill Gates and his wife Melinda launched their foundation in 2000—now the largest grant-making institution in the world in terms of total assets ($37 billion) and giving ($3.1 billion)—to eradicate poverty, reduce child deaths and fight disease.  And the ALS Association leapt to stardom with Ice Bucket Challenge that went viral over social media this summer and raise over US$100 million.

The expanding influence of disease foundations is disproportionate to the retrenchment on the part of biopharmaceutical companies, where R&D budgets are shrinking. Driven by a flurry of mergers and acquisitions along with economic pressures stemming from patent expirations, the economic downturn and increased competition from smaller biotechs, drug developers have shed more than 156,000 jobs in the US alone since 2009, see data cited in a recent Wall Street Journal article. As the article points out, that is the size of your average suburban community. Government-funded research labs aren’t faring much better. The US National Institutes of Health (NIH), the largest global funder of biomedical research, has been contending with the painful aftermath of sequestration that has left more grants unfunded, particularly work by young and early career investigators, and is destroying morale.

The pharmaceutical and government sectors still account for the bulk of R&D spending, of course. But a 2013 report from the Milken Institute’s Faster Cures found that patient advocacy and disease foundations initiated 10% of the 250 research consortia created to accelerate biomedical research. Nearly half are on single-minded missions to discover disease-specific drugs, devices or vaccines, but some of the consortia have much broader missions—to create broadly-used tools applicable across many diseases, or to unlock the research gates and make certain biomarker research data publicly available.

CROs are benefiting from this trend toward more disease foundation-sponsored research, but disease foundations are also benefiting from the work that contract labs provide.  Here are a few examples:

  • The Michael J. Fox Foundation created a research consortia that, two years ago, started hunting for radiotracers that could identify and track clumps of the protein alpha-synuclein that accumulate in the brains of people with Parkinson’s disease. Identifying these radiotracers could be useful as both a biomarker of disease and disease progression, as well as a tool for drug development. The consortia group includes BioFocus—a UK division of Charles River—along with Massachusetts General Hospital, Washington University in St. Louis and the University of Pittsburgh.
  • The Institute for OneWorld Health (iOWH), now part of the Seattle-based non-profit PATH, is about to start a Phase II trial in Bangladesh for a drug designed to alleviate severe rehydration related to diarrheal diseases, which kill two million children under the age 5 every year in developing countries. BioFocus—one of a number of collaborators on this project—performed medicinal chemistry, screening and ADME studies in a partnership that would identify the eventual candidate.
  • The CHDI Foundation, a non-profit working on the degenerative neurological disorder Huntington’s disease, functions like a virtual biotech and works directly with a large number of academic and commercial research groups, including Charles River and other CROs. Unlike more traditional nonprofits that award grants to scientists, CHDI contracts directly with research groups and plays a much more active role in directing the science.
  • The Myelin Repair Foundation focuses on developing new treatments for multiple sclerosis. It describes itself as the only research organization addressing every phase in the drug discovery process—from research all the way to licensing by commercial drug developers—and partners with CROs for critical validation and drug discovery studies.

Will any of these foundations put their stamp on a cure or vaccine? That’s certainly the goal. At the very least, they are pushing the pace of research in interesting and innovative directions. That alone ought to earn them a seat on the bench.