INDIANAPOLIS — Mark R. Kelley, PhD, Betty and Earl Herr Chair of Pediatric Oncology Research at the Indiana University School of Medicine, has been named a 2025 fellow of the prestigious National Academy of Inventors for his many contributions in pediatrics and cancer research.
The fellowship is the highest professional distinction awarded to academic inventors. Kelley will receive his recognition at the academy’s annual conference in June. The fellowship recognizes cutting-edge thinkers across all fields of scientific development, from medical research to artificial intelligence, quantum computing and beyond.
Kelley's research focuses on developing new drug treatments for cancer and a variety of other ailments.
"Being recognized by the National Academy of Inventors affirms that my team’s decades-long dedication to turning basic science into tangible therapies is making an impact," Kelley said. "It's not just about the patents; it's about the patients. Every discovery, every compound we create and every clinical trial we conduct offers hope to those fighting aggressive cancers, vision-threatening retinal diseases and severe inflammatory conditions."
In addition to several professorships, he co-manages the Cancer Drug Discovery & Development Accelerator at the IU Melvin and Bren Simon Comprehensive Cancer Center and directs the Molecular Oncology and Experimental Therapeutics program for the Herman B Wells Center for Pediatric Research.
He holds 19 U.S. and 35 foreign patents. His distinguished academic career at IU, which also includes more than 200 peer-reviewed journal article publications, stretches back to the early '90s.
Much of Kelley's work revolves around APX3330, a drug compound that has demonstrated effectiveness in targeting aggressive solid tumors in cancer patients during clinical trials. Kelley’s invention is also in the early testing phases for treating other ailments.
"The development of APX3330 represents the culmination of decades of work," Kelley said. "Taking a compound from our academic lab all the way through phase I clinical trials in cancer patients, seeing it licensed first to Apexian Pharmaceuticals, a company I am a founder of, to Opus Genetics for diabetic retinopathy, and now watching it advance toward phase III trials, that's the kind of translational impact every researcher dreams of achieving."
Some of Kelley's accomplishments include:
- Discovering the dual function of apurinic/apyrimidinic endonuclease 1/redox effector factor 1, an enzyme that can be targeted with drugs to block cancer-promoting pathways.
- Discovering the above enzyme was more prevalent in cancer cells and contributes to treatment resistance.
- Developing APX3330, a first-of-its-kind drug that targeted part of the enzyme in clinical trials as a way to stop cancer growth.
- In addition to cancer treatment, the drug has also shown potential in preventing chemotherapy-induced peripheral neuropathy, a side effect of current cancer treatments.
Developing APX2009 and APX2014, stronger versions of APX3330. - Discovering the enzyme’s role in creating new blood cells, which will allow Kelley’s drug to target other ailments including macular degeneration and diabetic retinopathy.
- Preclinical demonstration of APX3330 reducing inflammation, leading to its potential as a treatment for Crohn’s disease and ulcerative colitis.
- Kelley’s drugs have shown in preclinical data to be effective against necrotizing enterocolitis, a disease that affects premature infants that currently has no known treatment.
"This recognition highlights the collaborative spirit of innovation at Indiana University," Kelley said. "I've been fortunate to collaborate with talented colleagues, mentor dedicated students and postdocs and partner with industry leaders. The NAI fellowship celebrates not just my achievements but the entire ecosystem of innovation we've cultivated here at the Wells Center for Pediatric Research, IU School of Medicine and the IU Simon Comprehensive Cancer Center."
About the Indiana University School of Medicine
The IU School of Medicine is the largest medical school in the U.S. and is annually ranked among the top medical schools in the nation by U.S. News & World Report. The school offers high-quality medical education, access to leading medical research and rich campus life in nine Indiana cities, including rural and urban locations consistently recognized for livability. According to the Blue Ridge Institute for Medical Research, the IU School of Medicine ranks No. 13 in 2024 National Institutes of Health funding among all public medical schools in the country.
Writer: Rory Appleton rapplet@iu.edu
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