Foundations Invest to Accelerate Cures for Rare Disease
Patients with rare disease and their advocates are impatient with the time it takes to bring forth new therapies.
The Pharmaceutical Research and Manufacturers of America estimates it takes, on average, 10 years and $2.6 billion to bring a new drug to market. Thus, disease foundations are carving out niches and using creative models to support drug development.
Learning how they are doing this was the focus of the first of the two-day Invest in Cures & Rare Disease Forum, hosted at the North Carolina Biotechnology Center in late September. The Invest in Cures component, presented by LaunchBio, featured disease foundation representatives to share how North Carolina life science companies can access these unique opportunities. In addition, attendees heard from a senior associate from the Milken Institute, which advises high-net worth individuals and foundations in using strategic philanthropy to spur drug discovery.
The commitment to cancer research
Derrick Rowe, J.D., Ph.D., a registered patent attorney in the Washington, D.C., office of Wilson Sonsini Goodrich & Rosati, moderated the first panel of representatives from foundations, which focused on rare cancers. First, each panelist described what resources they have to offer drug developers, and, second, how companies can access them.
The panelists were: John Hallberg, CEO of the Children’s Cancer Research Fund, Annette Baker, Ph.D., president and CSO of the Children’s Tumor Foundation, and David Heenan, director of operations of the American Cancer Society’s (ACS) BrightEdge investing arm.
“We are interested in for-profit companies positioned to put cures in patients’ hands today or imminently … and then accelerate that market adoption” said BrightEdge’s Heenan. “You could have a great solution, but if providers aren’t prescribing it, and the industry isn’t buying it, then it’s not getting into patients’ hands.”
Additionally, he said through their Boost program, they make $250,000 grants to support small companies preparing to apply for a National Institutes of Health Small Business Innovation Research grant.
On the other hand, Baker explained that her comparatively smaller foundation analyzed the R&D ecosystem and “decided to be a convener rather than an investor.” CTF, which focuses exclusively on neurofibromatosis (NF), a rare children’s cancer-risk tumor condition, developed the NF Accelerator. This is an enabling platform to accelerate treatment development through a patient registry to bolster clinical trial recruitment and a clinician network to collaboratively develop endpoints and work with the FDA.
“We will not invest in your company, but we will help you save time and money,” she said.
Finally, Hallberg explained that, while the CCRF has donated more than $130 million to cancer research since its founding in 1981 through academia, it is “just now getting into the venture philanthropy investment space.”
"Thoughtful philanthropy" drives drug development
Kristie Keller, Ph.D., senior associate of the Milken Institute’s Center for Strategic Philanthropy, presented findings from its recent report, “Nonprofits: A Growing Force in Drug Development,” which she co-authored.
The Milken Institute is a nonprofit, nonpartisan think tank with offices in Santa Monica, Ca., Washington, D.C., New York City and Singapore. To date, she said, it has influenced more than $1 billion in philanthropic funds.
“This is not ladies who lunch anymore,” said Keller, describing the changing landscape of philanthropy.
Instead, she calls it “thoughtful philanthropy,” involving conducting “deep due diligence” on behalf of clients and individually tailored services by staff Ph.D.-level specialists to assist them in their giving decisions. She noted the importance of these services due to the high risk involved – an estimated 5,000-10,000 compounds are developed for each drug approved by the FDA.
Still, there have been successes, she said. For example, the Cystic Fibrosis Foundation provided seed funding to Vertex Pharmaceuticals and earned a $3.3 billion payout from the drug Kalydeco, which it recycled back into support other CF R&D efforts.
The report contains a list of six unique ways nonprofits can support drug development: academic support, as supporter or matchmaker to resources for developers, setting up incubators, awards to or creation of nonprofit clinical research organizations, conducting in-house R&D or providing milestone-driven support to for-profit companies. It also outlines which funding models are appropriate for which gap they identify in a drug development process. Finally, she presented a decision tree they use to guide clients toward the best approach.
Foundations use creative approaches
The final panel, moderated by Tara Britt, associate chair of the NC Rare Disease Advisory Council, discussed creative ways three foundations are trying to speed drug discovery. The panelists were: Sonya Dumanis, Ph.D., senior director innovation, Epilepsy Foundation; Amy Gray, CEO, Charcot-Marie Tooth Association; and Rich Horgan, founder and chairman, Cure Rare Disease.
Dumanis described the Epilepsy Therapy Project, which provides seed funding to companies via a competition akin to the Shark Tank television show. Entrants submit a 4-page proposal. The five finalists chosen receive coaching to perfect their 30-minute pitches. Then the audience, as well as the judges, vote. To date, she said, nine projects have already reached the market. In addition, even the unfunded entrants receive valuable feedback from the business and scientific advisory boards.
In addition, Dumanis said the Epilepsy Innovation Institute takes a hands-on project management approach with the teams it funds. Because the funding is milestone-driven, she said they are on the conference calls. They also give the teams access to a consortia of retired industry experts, such as intellectual property attorneys.
Gray of CMT described her organization’s Strategy to Accelerate Research (STAR). This includes forming alliances with pharmaceutical and biotechnology companies, nonprofits and government into a consortium to complement a network of academic researchers. Results have included development of licensed animal models and patient-derived human stem cell lines.
Finally, Hogan of CRD emphasized the importance of collaboration and eliminating bottlenecks in research.
“We’ve incentivized and mandated that researchers share data downstream so as not to waste resources and have researchers conduct experiments that don’t matter and cost time,” he said. Furthermore, his organization is trying to establish a new regulatory pathway with FDA for clinical trials containing only one patient.