The pandemic crisis has illuminated a stark reality that innovators, particularly those in biotech, have long been aware of: The world is filled with great ideas that don’t have the impact they might because how to develop and scale them isn’t considered until it is either absolutely essential — or too late — to do so.
The mRNA vaccines against SARS-CoV-2 represent a perfect example of this principle. While the technology behind mRNA vaccines was developed more than 40 years ago, it took the emergency of a global pandemic for researchers to receive the support needed to bring their concept through Food and Drug Administration authorization and approval and to market. Imagine how many people might have benefited from this technology — how many lives might have been saved — had it been developed and applied earlier.
Now, as the U.S. finds itself at the cusp of so many other innovative approaches to developing solutions to treat diseases and improve lives — including new advancements and applications in my field of nanomedicine — government regulators and other crucial gatekeepers face the challenge of building a better on-ramp for innovation. Successful examples of such efforts are already underway, such as the Cancer Moonshot initiative, supported by the passage of the
21st Century Cures Act in December 2016. To date, the National Cancer Institute’s substantial investment in the Moonshot has benefited more than 240 research projects across more than 70 distinct areas of cancer study.
Yet challenges persist, even in such advanced initiatives. According to Norman Sharpless, who directs the National Cancer Institute (NCI), the Cancer Moonshot program focuses on “investigator-initiated science” but, as Sharpless noted in a presentation session of the Cancer Moonshot Seminar Series in September, “What’s been challenging in recent years to the NCI is that there has been a huge influx of applications.”
Initiatives to support the worthy goals of cancer research must go deeper to ensure that the projects that have the best opportunity to make a difference are identified, incubated, and accelerated. And a stronger system must exist to support these processes. Fortunately, seeds of such efforts in other areas of biotech innovation are starting to take root.
Standardizing a process
In 2004, the Nanotechnology Characterization Laboratory was founded in collaboration with the NCI, the FDA, and the National Institute of Standards and Technology to provide preclinical characterization of cancer nanomedicines to support their translation into clinical use.
As a member of the first generation of scientists pioneering medical applications for nanotechnology, I realized that advancing the discipline in Europe, where I was working at the time, was not simply about developing great product candidates. While many of us working in the nascent nanotechnology space in Europe viewed the NCL as a part of a wider ecosystem for supporting innovation in medicine — indeed, some of us were working with the lab or modeling our early endeavors after it — we also saw an additional need for a standardized process that enabled the best projects to move from idea to human trials, which is where we saw the highest failure rate. This meant that the few ideas with the most promise were left to flounder alongside thousands of other ideas, many of which were given support despite their inability to profoundly improve patients’ lives.
My colleagues and I decided we needed to transform the ETPN from a think tank into a do tank. We reached out to the NCI, FDA, and NIST to start establishing nanomedicine as a discrete priority for advancement in Europe. This helped us begin to lay the groundwork for building a better on-ramp for others working in nanomedicine who would come after us. But we wanted to go a step further than the U.S. model to ensure the best ideas were making their way into human trials and toward the market. To support these efforts, we founded the ETPN’s Translation HUB.
The Translation HUB was groundbreaking because, while it included a characterization lab similar to the NCL’s that develops tests to assess the reproducibility, safety, and efficacy of nanomedicines, as well as a selection process similar to the one later used by the Cancer Moonshot, it also included a HealthTech Translation Advisory Board that gathered industry leaders who had already experienced the type of success that applicants were aiming for as a way to ensure they were getting holistic counsel. We also included a process for the development of manufacturing pilot lines because we saw that an inability to manufacture products at the scale required for human trials was a stumbling blockfor entrepreneurs in the space.
With this new model, we created a one-stop shop for the identification, selection, and acceleration of promising nanomedicine projects.
Selecting the best projects and giving them an on-ramp
When my colleagues and I first began our work with the Translation HUB, we needed to figure out how to choose from the thousands of ideas that crossed ourdesks, since it was impossible to focus on them all.
We developed a two-step process for project selection. First, we set threshold criteria that would provide us a basis to assess projects’ viability and the strength of their science, designed to target those that would be unique and truly make a difference. Second, from the thousands of entries we received, we evaluated those that achieved these high standards in order to select just 200 of the best of the best projects.
Building a stronger system to support innovation
But designing an effective process to select the best ideas is only part of the solution. More than ever, allowing novel approaches to realize their full potential in improving health and expanding life depends on additional support from government initiatives aimed at solving the major health issues of our time. The successful application of scientific breakthroughs is helping see us through this global health crisis, but the door to innovation must remain open long after it is over.
Currently, there exist only a precious few remaining years of the 21st CenturyCures Act. Signed into law in 2016, the legislation was designed to drive medical innovations and help accelerate product development. But additional support from the U.S. Congress is necessary. In June of 2021, U.S. Representatives Diana DeGette (D-Colo.) and Fred Upton (R-Mich.) released a draft of 21st CenturyCures 2.0, a piece of legislation meant to build on the successes of the original Cures Act.
The legislation would create a new agency, the Advanced Research Projects Agency for Health (ARPA-H), which would be tasked with finding cures for diseases such as cancer, Alzheimer’s, diabetes, and others. The agency would be modeled after the Defense Advanced Research Projects Agency (DARPA), which has designed some of the most thrilling and substantial innovations of our time, including the internet, self-driving cars, the Global Positioning System, and even cyborg insects. Notably, it was DARPA that funded Moderna’s mRNA vaccine technology back in the early days of the technology when other agencies were wary of throwing their weight behind potentially disruptive innovations.
The process exists to help more great ideas reach their potential. Now it’s time to build the system to support it. As I write this article, the draft of 21st Century Cures 2.0 has yet to gain traction in Congress. The U.S. simply cannot afford towait until the next emerging crisis to build the infrastructure that will allow governments, regulators, researchers, and companies to apply promising ideas and innovations to the myriad health crises society already faces.