Dr. Leor Weinberger earns NIH Director’s Pioneer Award to tackle evolving cancers.
Oct. 6, 2026 — Leor Weinberger, Ph.D., virologist and professor with Sylvester Comprehensive Cancer Center, part of the University of Miami Miller School of Medicine, has been awarded the National Institutes of Health (NIH) most prestigious award—the NIH Director’s Pioneer Award—for his “High-Risk, High-Reward Research” to develop a novel cancer therapy with the ability to mutate, or evolve, over time.
His research will focus on three different types of cancer with poor survival rates, due in part to their broad genetic diversity: pancreatic cancer, glioblastoma and acute myeloid leukemia.
Dr. Weinberger calls the novel therapy they’re building a neoplasm emergent oncolytic virus, or a neovirus, based on existing tumor-busting viruses that only infect tumor cells and with a nod to the movie, “The Matrix.” The cancer work is early, but his lab has already shown proof of concept for a therapy that can evolve with a tumor.
This is the second time Dr. Weinberger has received this award, which provides five years of funding and goes to a handful of scientists who have outstanding records of creativity.
“Like cancers, viruses mutate,” said Dr. Weinberger, who is also the founding chair of the Department of Cell and Systems Biology at the Miller School. “But for over 100 years, we’ve been using static chemistry-based technologies to treat these dynamic, evolving biological systems.”
After pancreatic cancer claimed the life of a close family friend in his 40s, who had tried every therapy available, Dr. Weinberger set out to build a new class of anti-cancer medicines based on an antiviral therapy he had developed.
As a young graduate student studying the biophysics of HIV, Dr. Weinberger was consumed by a simple yet profound insight: A fundamental mismatch exists between viruses and the therapies developed to treat them.
Dr. Weinberger has dedicated his career to solving this problem.
He figured the most rational solution was to build a therapy that can also mutate, so the drug could not be circumvented by mutation. Instead, the therapy would evolve together with the virus.
For more than 20 years, few believed it could work. “In very polite and sophisticated ways, almost everyone told me the idea was ridiculous –– it does not exist, you won’t find it, stop working on it,” Dr. Weinberger said.
Yet, Dr. Weinberger forged ahead anyway, sometimes spending years working in a direction that amounted to nothing. The first time he won an NIH Director’s Pioneer Award in 2013, he spent years engineering 150,000 therapeutic candidates, all of which failed.
Until one didn’t.
One day, a postdoc in Dr. Weinberger’s lab brought him a picture of the amputated genetic material they had been pursuing for 20 years. It had evolved by itself in a flask in the back of the lab. That foothold led to pre-clinical trials that worked exactly as Dr. Weinberger had imagined 20 years earlier, protecting cells from HIV.
“It’s not perfectly sane to follow something for decades when it keeps failing, but when success is a medicine that could help millions of people, you keep going,” says Dr. Weinberger.
The HIV therapy is now in the final stages of FDA review for clinical trials.
Dr. Weinberger has now turned his attention to what many consider an even more pressing problem: cancer.
“Like viruses, cancer cells evolve, resulting in resistant tumors that escape therapy and are not susceptible to treatment,” he said. “It’s a fundamental problem across broad domains of biology.”
In biology, there’s an evolutionary concept called the Red Queen Hypothesis, taken from the character’s quote in Lewis Carroll’s Through the Looking-Glass, to Alice, “It takes all the running you can do to keep in the same place.”
This co-evolutionary arms race means a biological system like a virus continually counter-evolves while the host tries to evolve resistance and mutate away or “escape.” Both end up running in place.
“We are developing a therapy that can establish a scenario analogous to Lewis Carroll’s Red Queen, a therapy that can run after the tumor as it tries to run away,” said Dr. Weinberger.
Now that Dr. Weinberger has a precedent for a therapeutic that can keep up with the rapid evolution of HIV, it seems likely that pivoting to cancer will be much faster.
“In 2020, we were able to produce a COVID therapeutic candidate based on this concept in about 18 months. We believe the same concepts can be used to rapidly build a new class of therapeutics across all of these domains, including deadly cancers like pancreatic cancer,” he said.
Dr. Weinberger moved to the Miller School in 2025 to pursue this new cancer therapy through its Phase 1 clinical trial unit for experimental therapeutics.
“I can think of no better place than Sylvester to take this concept to clinical trial. It’s a place designed to propel therapeutic concepts to the clinic. In some cases, clinical colleagues have indicated that if the medical need is dire enough, they will move directly to testing in patients, not to delay a potentially life-saving medicine,” he said.
The possibility of successfully building a novel therapy to treat cancer is Dr. Weinberger’s all-consuming goal, building on his HIV viral treatment that’s entering clinical trials.
“If the reward is significant enough, for example overcoming pancreatic cancer, it’s worth the possibility of failure,” he said.
With the Miller School’s integrated bench-to-bedside model, and the region’s rapidly expanding aging population – the dominant risk factor for cancer – this award places Weinberger and his team in the ideal environment to build a new class of anti-cancer medicines for those who need it most.
