Within the past 10 years, the quest for cancer treatments that genetically alter cells in a patient’s immune system has attracted millions in grants and private investments, generated fierce competition in research labs and drug companies, and are now becoming the subject of headlines.
The race to bring such therapies to market hit a major milestone when an FDA panel this month unanimously recommended approval of such a treatment for a type of leukemia, a so-called CAR-T therapy submitted by pharmaceutical giant Novartis. The results from trials are fairly astounding, and doctors are uncharacteristically enthusiastic about the potential for this and similar treatments that may follow for other diseases.
Efforts to alter the immune system to fight cancer have been in the works for decades, but it wasn’t until 2011 when a breakthrough in a small clinical trial led by Dr. Carl June at the University of Pennsylvania accelerated progress, followed by a flurry of trials, research partnerships, and several million in government and industry funding. Penn would go on to license a resulting treatment to Novartis and establish an R&D partnership with the firm.
But years before the technique became so prominent, now pursued by most major drug companies, June’s work was funded by some philanthropic sources open to supporting very specific, unproven areas of research.
In particular, the Alliance for Cancer Gene Therapy (ACGT) was an early backer of June’s gene therapy work. ACGT is a research grantmaker started by therapist Barbara Netter and Edward Netter, an investment banker who has since passed. The couple’s daughter-in-law died of breast cancer, and not long after, in 2001, the couple founded the charity after seeing a talk on the potential of gene therapy.
It's the kind of story we hear about all the time in giving for medical research: a family loss, wealthy donors who want to make a difference, and a willingness to take risks in the search for a breakthrough.
ACGT has a very narrow focus, solely backing work in gene therapy and cell treatments for cancer, a field that always showed exciting potential, but had failed to deliver until relatively recently. ACGT is quite small, having granted $27 million since it launched, including fellowship support for Carl June in 2004 and again in 2008.
Now a superstar in the cancer research world, June said in a recent release from ACGT, “When other organizations, including the NIH, considered gene therapy too risky, ACGT believed in the science and funded us when no one else would. ACGT really kept us going and kept the research alive. Without them, we wouldn’t have had a clinical trial and I don’t think we’d be where we are today.”
Another specialized, early supporter of June and the University of Pennsylvania’s work is the Leukemia & Lymphoma Society, a charity founded in 1949 by a family that lost a teenage son to leukemia. The LLS has given $21 million to June’s lab since 1998. In 2012, when Penn announced its partnership with Novartis to the tune of $20 million to advance its immunotherapy work, the university released a statement acknowledging the contribution of the nonprofit.
The case of June’s work and the evolution of its funding sources is a useful example of the impact science philanthropy can make. Long before this field of cancer research exploded, it was funded by these small, focused backers motivated by personal experience and willing to give highly focused attention to one area. Once a particular approach struck oil (reducing the risk and increasing likelihood of profit), other, larger sources of funding in government and industry stepped in at higher levels to take it across the finish line.
The other thing that’s apparent from the backstory of this breakthrough is the competitive and collaborative nature of science research, for both the scientists doing the work and the funding sources behind them. It might seem like a straight line from grants to industry to FDA, but that’s nowhere near the case. June’s work at Penn received backing from a number of sources over its many stages. And ACGT has made dozens of grants, with no way of knowing which if any would produce results. It can be quite difficult for science donors to know what impact their giving is making.
Meanwhile, researchers all over the world are pursuing related research that may yield comparable or more significant advances, and each lab has its own patchwork of funding sources. Even one lab’s work doesn’t happen in isolation. In fact, there was a dispute over some of June’s publications in which a key contributor was not given credit, prompting corrections.
Of course, funding also has a way of building on itself. Just last year, tech billionaire Sean Parker entered this space in a big way, committing $250 million for a new institute devoted to immunotherapy treatments for cancer across six universities and research centers, including Penn. Stanford just landed a $10 million gift from tech donor Jeffrey Rothschild for related work.
Behind such a big breakthrough, there’s a complex web of investigators and funding sources supporting it. At the end of the day, science philanthropy (even something that seems as big as Parker's initiative) is just one piece of what it takes to allow progress to unfold. But this particular development shows how even small, targeted support can contribute to something groundbreaking.