Opinion: Healthcare Nonprofits Drive Positive Change for Patients
16 Jan 2024
Clinical Study
Pictured: Nonprofit groups parachute into a hospital/Nicole Bean for BioSpace
Nonprofit foundations no longer function only as the traditional micro-funders and patient support groups of yesteryear. In 2018, foundations, along with professional societies, collectively devoted $3.8 billion per year to medical research and development in the U.S. That makes them the fourth-largest source of funding, after private-sector biotech firms, the federal government and academic institutions.
In other words, healthcare philanthropy by nonprofit organizations now plays a critical role in life sciences innovation. Nonprofits are uniquely positioned to provide funding, build bridges, make connections and even lead efforts to develop new treatments and cures for a variety of illnesses—including rare ones.
Foundations share the same sense of urgency to find effective cures as the patients they serve. They’re also well-connected to all the stakeholders involved with particular diseases, sitting at a unique intersection between patients, researchers, clinicians, pharmaceutical companies, funders and regulators.
That makes them well-equipped to bridge the “gaps” in drug discovery and development. Consider that basic research leading to medical breakthroughs often begins at university labs, many of which rely on large grants from the NIH or other federal agencies. But to have a shot at winning those grants in the first place, researchers generally need to have preliminary data to show their research is worth funding.
That’s where foundations come in. Nonprofits can, and increasingly do, fund the risky, innovative, early-stage research that yields the preliminary datasets that increase scientists’ odds of securing government funding.
While academic discoveries can lead to high-impact publications in top journals, translating those discoveries into potential future medicines may require a team of cell biologists, animal pharmacologists, chemists, toxicologists and many others. Here, too, modern nonprofit foundations are “filling the gap” by acting as a superconnecter—funding and managing multidisciplinary teams to ensure the potential medical applications of early-stage discoveries are thoroughly investigated.
A Tangible Impact
My organization, the Children's Tumor Foundation, is pursuing this path. We focus on the rare genetic disorders neurofibromatosis (NF) and schwannomatosis, which cause tumors to grow on patients’ nerves. To help find a cure, we started a collaborative research effort called Synodos that assembles scientists, clinicians, researchers and patient representatives to solve complex problems.
Synodos researchers make their data available in real time through a digital portal. This free flow of information—which is atypical of academic publishing—has the potential to accelerate the translation of research into treatments. The Synodos team has already delivered clinical trial-ready drugs for the treatment of one type of NF. That led to a first-of-its-kind clinical trial, co-funded by CTF and Takeda, which is already achieving incredible results.
The Leukemia and Lymphoma Society (LLS) is also playing a key role in bringing drugs to the clinic. LLS brought together world-renowned scientists, drug companies, tech companies and the FDA to launch a precision medicine trial for patients with acute myeloid leukemia, a lethal blood cancer. It was the first time a nonprofit health organization sponsored a clinical cancer trial.
LLS also launched PedAL, a global clinical trial for pediatric acute leukemia designed to test new, safer therapies for children. The goal is to prevent childhood cancer survivors from developing chronic health problems as a result of their treatment—which currently happens about 80% of the time.
Funding Early-Stage Companies
Another gap is in funding for early-stage startups, which has reached an all-time low since 2019. Twenty-eight biotech companies shut down in 2023, compared to just nine in 2021. Often, these companies need investors to fund their R&D and clinical trials, which can cost hundreds of millions for just a single drug. But venture capitalists frequently consider early-stage companies too risky an investment, so many of them go unfunded.
Fortunately, foundations are stepping in. Consider the Cystic Fibrosis Foundation, which has shown that investing in high-risk projects can positively impact patients’ lives. Its “venture philanthropy” strategy led to the FDA approval of KalydecoKalydeco, the first drug to treat the underlying causes of cystic fibrosis.
Or take the groundbreaking CoMMpass study, funded by the nonprofit Multiple Myeloma Research Foundation. CoMpass has over 1,000 patients involved in clinical trials and is delivering its ongoing research to other researchers and biopharmaceutical companies across the country in hopes of accelerating the search for new therapies.
Similarly, the I-SPY series of trials—which aims to change the way new breast cancer treatments are developed—has received funding from several nonprofit organizations, including Quantum Leap Healthcare Collaborative, the Breast Cancer Research Foundation, NIH and the William K. Bowes, Jr. Foundation, among others. Research findings from CoMMpass, I-SPY and similar trials could complement biotech firms’ own R&D efforts, ultimately leading to more success stories like Kalydeco.
And healthcare foundations are doing much more than writing checks. Increasingly, they're hiring executives with biotech experience and setting up R&D infrastructure of their own. In recent years, nonprofits—like the Multiple Myeloma Research Foundation, Tuberculosis Sclerosis Association, LLS and my foundation—are making significant contributions to R&D, from creating new patient registries and patient engagement programs to establishing clinical care and clinical trial center networks.
These sorts of strategic, financial and community-oriented efforts are exactly what patients are counting on. Many life sciences organizations, such as NewYorkBIO (where I am a board member) and the European Federation of Pharmaceutical Industries and Associations, have already recognized as much.
It’s time to encourage more life science organizations to view healthcare nonprofits as changemakers sitting at the intersection of research, community and funding and work with foundations to get more medicines to patients.
Annette Bakker, PhD is president of the Children’s Tumor Foundation, Chair of CTF Europe and board member of NewYorkBIO.
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