Mark Kelley, PhD
Phone: (317) 274-2755
Herman B Wells Center for Pediatric Research
1044 W. Walnut St., R4 302
Indianapolis, IN 46202
- Betty and Earl Herr Professor of Pediatric Oncology Research, Department of Pediatrics, IU School of Medicine
- Associate Director of Basic Science Research, IU Simon Comprehensive Cancer Center
- Professor of Pediatrics, Biochemistry & Molecular Biology, Pharmacology & Toxicology and Ophthalmology
- Adjunct Professor, Eugene and Marilyn Glick Eye Institute
- Director, Program in Pediatric Molecular Oncology & Experimental Therapeutics, Herman B Wells Center for Pediatric Research
- Bantz-Petrino Translating Research into Practice Scholar
- Co-leader, Cancer Drug Discovery and Developmental Therapeutics
- Full member
, Experimental and Developmental Therapeutics
Headlines & highlights
- Blog post - NIH grant (August 2020)
- News Release June 2019
- News Release (November 2018)
- Clinical trial begins with drug developed at IU (March 2018)
- News Release (May 2017)
- IU doctor develops drug that could effectively treat pancreatic cancer (Nov. 2016)
- 2016 IU Simon Cancer Center Scientific Report
- 2014 IU Simon Cancer Center Scientific Report
Our highly collaborative research environment allows for amazing teamwork to advance our state-of-the-art science and research from our labs to the clinic.Mark Kelley, PhD
Dr. Kelley was appointed associate director of basic science research in 2005. Dr. Kelley, the Earl and Betty Herr Chair in Pediatric Oncology Research, also serves as a director of the Program in Pediatric Molecular Oncology & Experimental Therapeutics and co-leader of the IUSCCC Cancer Drug Discovery and Development Program. As associate director of basic science research, Dr. Kelley oversees all basic science activities of the IU Simon Comprehensive Cancer Center, including stimulating interdisciplinary research collaboration, evaluating new research opportunities, and overseeing cancer center shared facilities.
In broader terms, all the academic positions he has held and the program leader and director positions he currently holds are dedicated to fast-tracking basic science, collaboration, and translational research to find more effective cancer treatments. Additionally, he helps equip the next generation of researchers by training and mentoring graduate students, MD and MSTP students, junior faculty, postdoctorates, fellows and others. He is also committed to diversity, equity and inclusion in his lab and IUSCCC/IUSM. He strongly supports women in science and mentors scientists from many different backgrounds.
Dr. Kelley’s work has focused on translational research in DNA damage and repair, specifically to determine how those activities can be exploited therapeutically to treat cancers and protect normal cells from oxidative and DNA base damage. He has focused on the enzyme apurinic/apyrimidinic endonuclease 1/ Redox effector factor-1 (APE1/Ref-1)—mechanistically as well as a therapeutic target in cancers and other diseases that manifest cancer-like properties.
APE1/Ref-1 is unique to the Base Excision Repair Pathway (BER), with dual repair and redox signaling functions that are crucial to cellular viability. He has concentrated on teasing apart these functions and in the process, he has discovered and has been developing redox-specific inhibitors of Ref-1. This original work was the impetus for becoming Chief Scientific Founder and Officer of Apexian Pharmaceutical targeting Ref-1 to produce new therapeutics for some of the deadliest and hardest-to-treat cancers.
Apexian recently completed a phase 1 clinical trial using oral APX3330 in solid tumor patients (NCT03375086). This trial established safety, expected PK, target engagement, and responses in patients in the trial. Phase 2 trials are being developed in cancer and other indications, including ocular diseases. A phase 2 trial using APX3330 in diabetic retinopathy (DR) is underway using a drug he developed and licensed to Ocuphire Pharma (NCT04692688).
Dr. Kelley’s laboratory currently studies DNA repair genes involved in repairing base damage that occurs from oxidative and alkylation events in normal and tumor cells and following cancer treatments. Ongoing projects include:
Studying the multifunctional APE1/Ref-1 protein structure/function in order to determine its redox and repair functions in normal and cancer cells
Molecular targeted therapeutic approaches targeting APE1/Ref-1 with a focus on pediatric cancers as well as adult solid tumor cancers
Role of APE1/Ref-1 in chemotherapy-induced peripheral neuropathy (CIPN) and developing therapeutic mechanisms to combat this awful side effect of chemotherapy.
Dr. Kelley has more than 19 patents relating to the targeting the enzyme apurinic/apyrimidinic endonuclease 1/ Redox effector factor-1 (APE1/Ref-1) in cancer, ophthalmology diseases as well as other indications such as inflammatory bowel disease (IBD) and chemotherapy induced peripheral neuropathy (CIPN).
Dr. Kelley has been consistently funded by NIH for over 29 years (since 1993) and is currently funded by 3 NCI grants as well as a NEI grant from NIH in addition to a number of other foundation grants. He directs the IUSCCC American Cancer Society (ACS) Institutional grant, which has been in place for over 50 years.
Dr. Kelley earned his bachelor’s degree from DePauw University and received his doctoral degree in genetics from Louisiana State University in Baton Rouge, La. He completed an ACS postdoctoral fellowship in molecular biology at Rockefeller University in New York in the laboratory of Dr. Michael Young, the 2017 Nobel Prize Winner in Physiology or Medicine, and was an assistant professor at Loyola University Medical School in Chicago prior to joining the Indiana University School of Medicine faculty in 1993. In 2021 he was selected as a Fellow to the prestigious AAAS Science Fellow association.
Dr. Kelley and his wife are the parents of two children who have graduated from college and are pursuing their careers. They are at home with the most adorable lab in the world, Ziggy (for Ziggy Stardust).