New U.S. Agency Tasked With "Moonshot" Medical Advances

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Imagine swallowing a surgical nanorobot as a pill rather than being cut open. Or receiving a personalized cancer vaccine for the price of a cup of coffee.

Or having your damaged organ replaced by one 3D-bioprinted in a lab.

These are the types of futuristic health innovations being cooked up at Washington’s newest federal agency, Advanced Research Projects Agency for Health, or ARPA-H. The agency has a $1.5 billion 2023 budget to pursue high-impact, high-reward science.

Reverse osteoarthritis

In May, it just announced its first program, a “moonshot” project designed to reverse osteoarthritis. The proposed non-invasive therapies aim to regenerate tissue, making joints that can heal themselves.

They’ll pioneer injectable bone and cartilage regeneratives, as well as replacement joints made from human cells.

ARPA-H is seeking to build more than 100 additional revolutionary projects over the next several years.

“When you think about a moonshot, we want revolutionary advances that are hundreds or thousands of times better than where we are right now,” said Dr. Amy Jenkins, who leads ARPA-H’s Health Science Futures program.

A proven model

ARPA-H is modeled after the Defense Advanced Research Projects Agency, or DARPA, which is the Pentagon’s innovation arm. It was created in 1958 by President Dwight D. Eisenhower after the Soviets launched the Sputnik-1 satellite.

The agency raced to develop stealth aircraft and anti-tank missiles, ultimately spurring research that brought technologies such as GPS, voice recognition, and the Internet to the public.

Now, ARPA-H seeks to do for healthcare innovation what DARPA did for defense.

Established by the Biden Administration in 2022, it’s the latest in a string of innovation agencies launched over the last two decades. The Department of Energy directs its innovation arm for energy, ARPA-E; for infrastructure, there’s ARPA-I at the Department of Transportation; and the intelligence community has IARPA.

The health innovation factory

The high-risk, high-reward “ARPA” model eschews slow, incremental results in favor of rapid and revolutionary transformation.

In healthcare, that means moving beyond just basic science for science’s sake. Their iterative “fail fast” approach is designed to rapidly move toward treatment trials and commercial applications

With its staff capped at 210 government employees, ARPA-H won’t do any of the research in-house. Rather, it acts purely as a funder, striving to stay small and nimble. It vests much of its power in “program managers,” who act like CEOs of biotech startups with $50 million budgets and broad authority to barnstorm the country to seek out the best teams in academia or industry to accomplish their mission.

Seeking game-changing ideas

“We are really making a push thinking about cost, access to our technologies, and ensuring that the entire U.S. population has access to what we're developing,” Jenkins said.

So far, the agency has hired six program managers, who bring diverse experience including from the National Cancer Institute and the private sector. Their goal is to have 20 by the end of the year, and 60-100 at any given time. They’ll bring on program managers who envision research spanning across the spectrum of disease, including neurodegenerative, autoimmune, and infectious conditions.

“They need to have the right personality and the right, the right approach to motivating people because oftentimes what our program managers are going to ask the community to do is so crazy that people will laugh at them,” Jenkins said. “People will say that's almost impossible.”

Each proposed program is designed to last 2-4 years. With each program manager’s term lasting just three years, the goal is to move boldly with each idea, to get a quick yes or no on its efficacy, and keep cycling through more rapid innovation.

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“We are also going to invest in technologies that we may not see the outcome of for the next five or 10 years,” she said. “It may be 20 years before we realize what our investment has really meant for the field.”

In mimicking DARPA’s culture, would-be program managers at ARPA-H undergo the “Heilmeier Catechism,” a set of eight key questions that former DARPA director George Heilmeier posed to guide agency officials in evaluating proposals. ARPA-H adds additional patient-centered ideas: How to ensure that technology is equitably available to everyone, and how to address its misperceptions and misuse.

Inventing the future

ARPA-H research is disease-agnostic, so rather than narrowing down on one discovering the root cause or treatment for a specific condition, it aims at developing platform technologies with broad-ranging approaches that would power the next generation of science and ultimately reshape our interactions with the medical system as immediately as possible.

“We will invent the future in this office,” Jenkins said.

She cited an example from her previous role at DARPA. In the 2010s, it had a program manager interested in mRNA, and how that could be used to make a vaccine faster. The agency hit the accelerator in using the technology for vaccines and therapeutics, contributing to what are now the COVID-19 vaccines of today.

“Who knows if 20 or 30 years from now, my grandchildren, maybe using devices that have a little widget in them, was a fundamental change that was made at ARPA-H,” she wondered.

First "moonshot" for regenerative medicine

That brings us back to ARPA-H’s first major program announcement for Novel Innovations for Tissue Regeneration in Osteoarthritis, or NITRO. It’s designed to take on the third leading cause of disability in the United States, which affects some 32 million Americans and causes an estimated $136 billion economic impact each year.

Ostensibly it’s focused on one type of disease, but it could have spillover effects across a spectrum of diseases.

“While NITRO focuses on osteoarthritis, it is also a regenerative medicine program,” Jenkins said. “And so we absolutely envision that some of the technologies and the advances that we make in the NITRO program, if we're successful, will be able to be applied to other regenerative tissues or to other tissues that need regenerating as well.”