In 2023, Atlanta philanthropist Bernard “Bernie” Gray endowed a Catalyst Fund at Winship to help support researchers in moving discoveries from bench to bedside. “Winship put out a request for proposals, saying, ‘Do you have a drug that you invented and are you trying to move it into clinical trials?’ And I said, yes, we do,” recalls Edmund K. “Ned” Waller, the Rein Saral, MD, Professor in Cancer Medicine at Winship Cancer Institute of Emory University. Waller also holds appointments at Emory University School of Medicine and is director of the Center for Regenerative Engineering and Medicine at Emory, Georgia Tech and the University of Georgia. 

The drug candidate Waller had in mind works as an antagonist on vasoactive intestinal peptide (VIP), an immunosuppressive protein that cancer cells produce to help them elude the body’s natural defenses. Inhibiting VIP gives more space for a patient’s own immune system to help fight invaders. “What our drug does,” Waller says, “is it gives a little more gas when you’re already driving, and you go a little bit faster. That little bit of increased activity in the immune system is often enough to overcome cancer.”

Fast forward a few years and that drug candidate—imagined at Winship back in 2016 when Waller reviewed a paper with an adjacent mechanism—is being developed practically in-house through Cambium Oncology, the company Waller founded in 2018 to develop first-in-class immunotherapies that improve outcomes for patients with cancer.

The company is currently funded by startup seed money and a Small Business Innovation Research (SBIR) grant from the U.S. Small Business Administration that Waller won along with collaborator Sarwish Rafiq, assistant professor in the Department of Hematology and Medical Oncology at Emory University School of Medicine. Rafiq specializes in chimeric antigen receptor T-cell therapy (CAR-T). 

Together, Waller and Rafiq devised a way to harness CAR-T by using viruses to genetically modify a patient’s immune cells to increase the efficacy of VIP antagonist cells.  The research partners are senior authors of a forthcoming paper describing this novel method’s apparent ability to work against solid tumors, a prior shortcoming of CAR-T. Although CAR-T has been a game-changing innovation in cancer care in the past decade, it has been more effective to date in fighting blood cancers. “We are at a point where we have the funding to try and bring it to the clinic,” Rafiq says. “From an idea, hopefully, we can get it into patients here at Emory.”

Obtaining FDA authorization to start a clinical trial in humans at Winship would come closer to completing the arc of drug development, from idea to pharmacy-ready product. It’s a goal writ large at the institution: Winship is involved in a host of university centers and initiatives aimed at supporting researchers through the process. This also keeps more intellectual property—and profits from patented discoveries—in-house. This cycle then allows for investment in more early-stage work.

These efforts provide varied supportive infrastructure to Winship faculty working on all manner of cancer therapies, including harnessing indigenous botany knowledge, splicing microscopic matter and finding new ways for medicines to interact with each patient’s specific malignancy.

In the quest to discover new medicines—or new uses for older medicines—the journey from bench to bedside tends to happen in iterative increments. One lab discovers evidence that some group of molecules manipulated a certain way might quell some aspect of disease progression. That lab publishes a paper, and another group of scientists, often someplace else, takes the insight and hones it. Their lab in turn—or yet another one—competes to execute clinical trials, often in partnership with a biotech company, which gambles that its investment in the development process will pay off when it gets to sell a blockbuster new drug to the public. 

This piecemeal precedent doesn’t always lead to the most efficiency and collaboration due to the competitive nature of the academic sciences. A drug development holy grail is to own the entire process in one place. “Nobody discovers a drug and takes it through every step of that process, though every cancer center would like to do that,” Waller says. 

Emory’s broader medical center has had great success in recent years doing this with molnupiravir, the first antiviral pill shown to minimize COVID symptoms. The drug, developed through Emory’s Drug Innovation Ventures at Emory (DRIVE) program for early-stage development, received emergency FDA authorization for use in 2021, at the height of the pandemic. Molnupiravir’s success contributed to the university earning $266.7 million in licensing agreement income the following fiscal year, part of some $500 million in profits that medication alone has brought into the institution.   

Molnupiravir was co-developed by George Painter, a professor in the Department of Pharmacology and Chemical Biology at Emory University School of Medicine and director of DRIVE, and by Dennis Liotta, a member of Winship’s Discovery and Developmental Therapeutics Research Program, Samuel Candler Dobbs Professor in Emory University’s Department of Chemistry and chair of the advisory committee for DRIVE. As Liotta, an HIV drug development pioneer, puts it, “There are a lot of great things going on in Winship.” 

Eric Miller, one of Liotta’s former postdoctoral mentees, is another Winship researcher with “great things” in the hopper. A member of Winship’s Discovery and Developmental Therapeutics Research Program and an assistant professor in the Department of Pharmacology and Chemical Biology at Emory University School of Medicine, Miller was another grant recipient of Bernard Gray’s Catalyst Fund. His current cancer research focuses on decreasing chemotherapy toxicity and directing immune cells to migrate toward high-need areas by modifying the DNA building blocks of drug delivery mechanisms to make them easier for the body to absorb. “Bench to bedside is not just unidirectional,” Miller says. “Whatever we learn from the development effort will come back to the laboratory so we can try to understand nuances that will emerge in a clinical trial.”

A key new driver for Winship’s current bench-to-bedside efforts lies in Emory’s Center for New Medicines (CNM), an accelerator formed in 2024 that, from the start, has partnered with Winship to help expedite the pipeline for new cancer therapies. Liotta, the center’s co-leader, explains that, “The Center for New Medicines is kind of positioned to get something where there’s some interesting potential and get it to a proof-of-concept stage, where we understand that there’s much more preclinical work to do, and then clinical work, but we’ve demonstrated safety and efficacy in animal models.” When CNM announced its first cohort of funded research projects, at the end of summer 2024, the majority of recipients were researchers with Winship affiliations. 

The CNM works by adhering to more of a startup incubator ethos than an academic one, according to Haian Fu, who co-leads both CNM and Winship’s Discovery and Developmental Therapeutics Research Program. Fu, a professor and chair of the Department of Pharmacology and Chemical Biology at Emory University School of Medicine and the Winship Partner in Research Endowed Chair, also oversees a lab researching protein-protein interactions, in which cancer’s gene-level mutations form the basis for targeted therapeutics. “CNM projects are evaluated in a milestone-driven fashion,” he says. 

The milestone mentality focuses on keeping intellectual property within the institution. Instead of following the established academic mantra to “publish or perish”—leading researchers to author papers with minute updates in the service of claiming more squares of intellectual turf—CNM-funded researchers might be counseled to publish more conservatively to prevent others from monetizing their innovations first. They are also advised, in consultation with lawyers in Emory’s Office of Technology Transfer, to file patents in tandem, another layer of protection for intellectual property. 

“Each project is building assets for Winship,” Fu says, both financially and in terms of the Center becoming an even more vital local resource. In short, a track record of success via the CNM can potentially increase Winship patients’ access to cutting-edge trials and treatments.

Fu says that the foundation of this growing ability to keep more of the drug development cycle within Winship has been laid, brick by brick, for decades. The CNM uses the school’s Chemical Biology Discovery Center, first created in 2003 and part of the National Cancer Institute’s Chemical Biology Consortium, as an idea source for potential projects.  “All this built up to where we are today,” Fu says. “It didn’t just pop up last year.” He adds, “We believe we are at the forefront of cancer genomics-based drug discovery.”

Another resource for Winship scientists and clinicians is the Discovery and Developmental Therapeutics Research Program (DDT), which Fu co-leads along with Kelly Goldsmith, director of the Neuroblastoma/MIBG Therapy Program, clinical director of Aflac Precision Medicine Program at the Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta, and professor in the Department of Pediatrics and the Curing Kids Cancer Professor of Pediatric Oncology at Emory University School of Medicine. “DDT is a main engine at Winship for drug discovery and development,” Fu says. The DDT Program focuses on discovery, cancer imaging and developmental therapeutics, with a goal of pushing more cancer innovation from bench to bedside to benefit patients in Georgia. In other words, it’s yet another resource dedicated to translational research at Winship.

Cassandra Quave is one of some 200 faculty members affiliated with the DDT Program. Quave holds a joint appointment as a professor of dermatology in the Emory University School of Medicine and Emory Center for the Study of Human Health. She also leads the Emory University Herbarium—those interested can visit it in the former Dental School Building on Clifton Road—full of “medicinal plants that are used in traditional medicine, specifically in infectious and inflammatory disease,” she explains. “I think it’s so important to really support the early stages of the pipeline so we can build that pipeline and keep the toolbox full for physicians to have choices,” Quave says. Her DDT Program-related work involves seeking plant-derived treatments for melanomas and for cutaneous T-cell lymphoma, a rare cancer in which immune cells attack a person’s skin—“applying our drug discovery pipeline to looking for new solutions to deal with these cutaneous cancers.”

Quave sums up what could be called the driving force behind Winship’s bench-to-bedside development of cancer therapies—and how it differs from the usual drive for publication and professional recognition that dominates academic medical research. “I’m at this period in my career where publishing another paper is always great,” she says, “but my real passion is trying to push through some of these discoveries.” Referencing her personal loss of friends and relatives to cancer, she says what really propels her and other Winship scientists is finding answers to the question behind all their research: “How can we move these discoveries from the benchtop into the hands of clinicians to better help patients?”

Story by Kira Goldenberg is a writer and psychotherapist in San Francisco.

Designed by Linda Dobson
Illustration by Christiane Beauregard
Photography by Jenni Girtman