Funding Small Studies — and Life-Changing Cures

Snakebites kill tens of thousands of people a year in low-income countries — mostly children in rural areas, far from the antivenom that could save them. Standard antivenom is expensive and species-specific and must be administered intravenously under medical supervision. For a child bitten in a remote village, it is frequently out of reach.

A few years ago, researchers began exploring whether an oral drug approved for treating heavy metal poisoning could reduce the harm of snake venom. It had shown promise in mice, and because it was already approved, it was deemed safe. Cures Within Reach, a Chicago-based nonprofit, provided $50,000 for the first small human study, which showed the compound was tolerable at the doses needed. Coefficient Giving, formerly known as Open Philanthropy, subsequently committed $5.4 million to run a larger efficacy trial, which is now underway in Brazil and Ghana.

That sequence is the model that Cures Within Reach has spent 20 years refining: a small proof-of-concept clinical trial, a clear signal about whether the idea is worth pursuing, a much larger investment unlocked, and movement toward a potentially life-saving intervention for those who need it.

The nonprofit backs early clinical trials for drugs that are already approved by regulators, testing whether they might work for diseases beyond their original purpose. The approach, known as drug repurposing, moves faster and costs less than developing new compounds from scratch. But big pharmaceutical companies have little incentive to invest in them either because the drug’s patent has expired and any manufacturer can sell it or because the new use simply isn’t lucrative enough.

“Patients don’t really care if a drug is on- or off-label or on or off-patent,” says Barbara Goodman, the group’s CEO. “They just want treatments for their disease. They want them to work.”

Even when the science is compelling, the studies often don’t happen because no one will pay for them.

The result is that good ideas fall through multiple gaps at once — too commercially unattractive for industry, too preliminary for most government funders, too far along for the academic grants that favor basic laboratory research. In philanthropy, this is sometimes called the “missing middle,” where promising ideas stall not because they’ve failed but because no one has stepped in to help test their readiness for larger investment. For Cures Within Reach, that gap is its sweet spot. It’s a problem that any organization working in a neglected field is likely to recognize.

Founded in 2005 as an offshoot of Goldman Philanthropic Partnerships, a Chicago family foundation, Cures Within Reach has since grown into a $3.8 million operation that channels funding to researchers at hospitals and universities. Its grants are relatively small — averaging $100,000 per study — because the drugs involved are already approved with established safety profiles. Its studies move faster and cost far less than traditional trials because they are just determining whether a drug, for example, works on a new condition. The goal is not necessarily to secure FDA approval but to generate enough credible human data to justify larger trials, attract additional funding, or in some cases support off-label use. 

Cures Within Reach

Funders including Coefficient Giving, the Burroughs Wellcome Fund, and Mark Zuckerberg and Priscilla Chan’s science philanthropy, Biohub, have been drawn to Cures within Reach’s unique place in the research ecosystem. 

The nonprofit’s leaders see its role as taking a risk that gives others more confidence to take the next step, Goodman says. “Industry, the NIH, larger foundations, even investors, they’re waiting for these really intriguing opportunities to be de-risked.” Since its founding, the organization has granted $12 million in clinical trial funding. That has resulted in $145 million in follow-on investments for larger clinical trials that those early studies made possible. 

That massive follow-up investment illustrates the group’s guiding strategy, one that Goodman often emphasizes to supporters: Success rarely looks like a cure. It looks like a door opening for the next, larger funder. After two decades of persuading donors to fund work that the market won’t touch, navigating partnerships with industry, and measuring impact in follow-on dollars rather than patients helped, she has come to believe the lessons extend far beyond the world of medicine. They are about what it takes to be effective in a neglected field.

Funders who have supported the organization say its approach has broader implications for nonprofits trying to effect change. “Identifying the right gap matters as much as the size of the gift,” says Tania Simoncelli, vice president of translational impact and engagement at Biohub. Eliminating the right bottleneck, she adds, can create momentum across an entire field.

Born of Frustration

Cures Within Reach traces its origins to a family confronting cancer and a funding landscape that frustrated them at every turn. After Judy Goldman was diagnosed with multiple myeloma in 1990, she and her husband George — who built a real estate investment company in Chicago — began funding medical research through their family foundation, Goldman Philanthropic Partnerships.

More Results From Big Philanthropy

Read more deep reporting on major philanthropic investments — what worked, what didn’t, and what it takes to drive real change.

One of their earliest bets was on thalidomide, a drug with a troubled history of causing birth defects when prescribed as a remedy for morning sickness. But early research suggested it might help starve myeloma cells. Supporting that work crystallized a philosophy: Sometimes the breakthrough is not a new drug but a new use for an existing one. Judy Goldman ultimately survived 25 years after her initial diagnosis, in part because of treatments the family helped bring to clinical testing.

By 2008, the family looked back across roughly a decade of funding and noticed something: Their only investments that had actually reached patients were repurposing projects. At the time, there were virtually no other nonprofits focused on this approach. In that same year, the organization, initially called Partnership for Cures, narrowed its focus and changed its name to Cures Within Reach — a charity designed to fund the first proof-of-concept clinical trials that major funders were not yet willing to support. The Goldman family’s children now run the foundation and continue to fund Cures Within Reach.

Today the model is spreading. Newer nonprofits including Every Cure and Reboot Rx use machine learning platforms to identify repurposing candidates and have attracted support from funders like Biohub, Lyda Hill Philanthropies, and Arnold Ventures.

“It’s amazing that we’ve got these platforms and these players that are continuing to hammer away at the problem,” says Kristin Schneeman, a senior director of FasterCures, a Milken Institute project focused on understanding bottlenecks in biomedical innovation. What distinguishes Cures Within Reach, she says, is its longevity and its deliberately disease-agnostic model — the ability to take its repeatable funding structure and apply it across conditions, geographies, and donor interests rather than advocating for a single disease or technology approach. Twenty years in, that flexibility has proved to be both its defining feature and its most replicable lesson.

“Their work helped show that this was possible,” says Elizabeth Weiss, principal director of philanthropic advising at the Science Philanthropy Alliance, whose member organizations include several supporters of Cures Within Reach. As technology evolves and helps speed the cycle along, there’s even more opportunity.

Why the Gap Persists

The challenge with drug repurposing runs deeper than any single funder’s priorities. The National Institutes of Health supports repurposing work through its National Center for Advancing Translational Sciences, but federal grants tend to require preliminary data before committing — the very data that doesn’t yet exist. Academic grant programs tend to favor basic laboratory research over applied clinical trials. And many large philanthropic foundations prefer to fund work at a later stage where outcomes are more visible to donors.

“You can’t make money off repurposing generic medicines,” says Ray Kennedy of Coefficient Giving, which has backed two follow-on trials built on Cures Within Reach proof-of-concept studies. “These trials don’t happen, even though there are lots of plausible hypotheses out there.”

In biotech and science policy circles, this is known as the “valley of death” — the funding gap between promising lab results and the first human trials that would prove whether they work. In the social sector more broadly, there’s a similar phenomenon: the missing middle, where organizations and ideas are too far along for seed funding but too early for major institutional investment.

“This goes beyond just drug repurposing,” whether that’s areas like innovations in health care delivery or policy research, says Andrea Noda, a vice president of health care and drug policy at Arnold Ventures. “Philanthropic capital is often what enables experimentation and risk-taking in spaces where market failures exist but where successful outcomes generate large spillover benefits for society.” 

Doing More With Less

Cures Within Reach has built an organization designed to do more with less and to make every dollar it grants unlock several more. The group raises philanthropic dollars and directs them to researchers at hospitals and universities, which run the trials themselves. In about 40 percent of the trials it has supported to date, Cures Within Reach has joined with other external funders. Last year, the organization directed  $775,000 in grants for 11 new clinical trials. This year, Goodman estimates grant making will increase to between $1.3 million and $1.5 million. Patient size varies widely, from as few as five people to 200.

A 2025 study in Frontiers in Pharmacology found that repurposing an existing drug can cost as little as one-fifth of what traditional drug development requires and move into human trials in roughly half the time — yet most repurposing efforts never generate the evidence needed to change clinical practice at scale.

With a core staff of four full-time employees plus a handful of part-time staff and contractors, the organization manages to fund far more than its staff size would suggest. It relies on a network of volunteer grant reviewers: clinicians, researchers, patients, industry scientists, and government experts who evaluate submissions. Last year, more than 140 volunteers pitched in.

Rather than making ad hoc bets, the organization runs structured requests for proposals, inviting researchers to apply within specific focus areas — a particular disease, a population such as veterans, early-career investigators, or a methodological approach like AI validation. Each funded study is designed to answer one clear question: Does this work in people? If it does, the evidence it generates is meant to unlock larger investments from NIH, industry, or major foundations.

Through these trials, Cures Within Reach aims to generate data or a publication that can lead to the next step, not necessarily to secure FDA approval. If the researcher or university they fund does get an approval that leads to commercialization, Cures Within Reach gets a small share of that money, which it then reinvests in more research. That payback is typically fixed to the amount of the grant rather than an ongoing royalty stream.

Making the Table Bigger

One of the most transferable lessons from Cures Within Reach is about the mix of funders needed to sustain work that no single donor wants to carry alone. That means making room not just for a diversity of donors but for industry as well.

The team at Biohub viewed their investment in Cures Within Reach as a way to encourage broader participation rather than operate in isolation, says Jeanie Kim, a senior program manager at Biohub. “Our mission to cure or prevent all disease requires working across institutions, disciplines, and funding partners,” she says. “No single organization can generate the scale of data, breadth of expertise, or clinical connections this kind of science demands.”

Early in her tenure, Goodman found herself frequently explaining to donors why a nonprofit working in this gap should welcome, rather than resist, industry involvement.

“There are a lot of people who have a beef with the pharma industry,” she says. “But industry has to be at the table. They’re the ones who hold the market authorization. You can’t succeed by keeping them out.”

The organization’s approach to donor relationships is deliberately flexible. Goodman compares it to Mad Libs: every donor fills in a different set of blanks — type of disease, geography, level of involvement, interest in a particular population. One donor wants to focus exclusively on blood cancers in the United States; another wants to fund whatever is most neglected globally. 

One recently completed pediatric oncology clinical trial at the University of Texas MD Anderson Cancer Center drew on pooled funding from donors focused on oncology, health equity, and rare diseases — interests that might not have come together without an organization to connect them. The trial came to Cures Within Reach through a request for proposals backed by a pediatric-focused foundation.

Different funders contribute based on their own priorities. Coefficient Giving focuses its support on high-burden diseases in lower- and lower-middle-income countries. The Burroughs Wellcome Fund has supported trials led by underrepresented researchers and partnerships with trusted local organizations that help reduce barriers to enrollment for communities of color. Biohub and Lyda Hill Philanthropies have backed the organization’s newest effort, testing whether AI-identified drug repurposing predictions actually work in patients.

What holds this coalition together, Goodman says, is a shared understanding that success at this stage doesn’t look like a cure — it looks like the next door opening. “It’s not just ‘number of patients served’ or ‘number of lives touched,’” she says. “We measure it in catalytic funding raised or publications that are changing patients’ lives everywhere.”

That way of framing the potential power of a donation “really resonates with individuals and families who come from the business world and are used to that mindset of making investments that they’ve been able to leverage,” says Weiss, with Science Philanthropy Alliance. “What are the opportunities to seed something or start something that then could have significant effects in the long run? A small amount of money could have a significant impact.”

Many nonprofit leaders prefer to share good news and evidence of a big impact with donors. But Goodman says being explicit about the risk of failure builds donor relationships. She is clear that many early-stage studies will not work, and equally clear about what “risk” actually means in context: By the time a drug candidate reaches a clinical trial, it has already cleared years of preclinical safety work. To a researcher who has spent a decade getting to that stage, it represents extraordinary progress. To a philanthropist unfamiliar with the pipeline, it can look uncertain. Helping donors calibrate that difference, Goodman says, is part of what keeps them engaged.

When the Bet Pays Off

Cures Within Reach-backed studies sometimes open doors that no amount of advocacy could. In one case, the organization funded a proof-of-concept trial at Children’s Hospital of Philadelphia for a rare pediatric immune disorder called ALPS, a condition that was often fatal. The drug being tested was a generic transplant drug that carried no patent protection and attracted no commercial interest. But the trial worked. Within a year of the first publication, the lead researcher had heard from 300 physicians around the world, Goodman says. The treatment is now a first-line option reimbursed by insurers in multiple countries based entirely on the evidence that a single early study made possible.

Kennedy says Coefficient Giving is considering expanding its work in drug repurposing. He sees Cures Within Reach as a way to vet work worthy of continued investment. 

“They do these small proof-of-concept trials. And then if it works, I can come in and fund the next stage, or someone else can,” he says. Since 2023, after providing the group with two grants totaling $2.3 million, he has now funded two follow-on trials built on their work — $5 million connected to the snakebite remedy and $400,000 for a trial to test a therapy to improve long-term outcomes for tuberculosis patients.

“Some of the ideas that we’ve uncovered have been sort of a happy coincidence,” he says. “It makes us think that there could just be lots of other ideas in this shape out there.”

Reporting for this article was underwritten by a Lilly Endowment grant to enhance public understanding of philanthropy. The Chronicle is solely responsible for the content. See more about the Chronicle, the grant, how our foundation-supported journalism works, and our gift-acceptance policy