Clinical trial begins with drug developed at IU

By Mary Hardin

Monday, March 12, 2018

Enrollment has begun at the IU Simon Cancer Center for a phase I clinical study of a drug designed to have an anti-cancer affect while protecting against chemotherapy-induced peripheral nerve damage, a common side effect when patients are exposed to certain types of chemotherapy.

The clinical study of the orally-administered drug, APX3330, is the first to explore its use in patients with advanced cancer. Preclinical data indicates that APX3330 inhibits the cancer-promoting activity of a dual function protein called APE1/Ref-1 without interfering with the nerve cell protection activity of the protein.

By inhibiting the cancer-promoting activity of APE1/Ref-1, APX3330 appears to not only protect nerve cells from platinum-related damage, but preclinical studies suggest that it repairs existing damage. Chemotherapy-induced peripheral nerve damage can be so severe in patients receiving cancer treatment that it causes patients to receive less chemotherapy than is optimal for treating cancer.

The phase I study will determine whether APX3330 is safe for administration to cancer patients, and subsequent studies will determine whether the drug is effective at preventing CIPN in patients receiving platinum chemotherapy. The study is also being conducted at two START Center for Cancer Care locations in San Antonio, Texas, and Grand Rapids, Mich.

Dr. Mark Kelley Dr. Kelley

The clinical study is the outgrowth of nearly 30 years of research by Mark Kelley, Ph.D., associate director of basic science research at the cancer center. Dr. Kelley’s work has earned significant research grants from the National Cancer Institute to explore the drug’s mechanism of action and its potential to benefit cancer patients.

The clinical study is being funded by Apexian Pharmaceuticals, an Indianapolis-based biopharmaceutical company. Dr. Kelley is the chief scientific officer at Apexian.

Details of the study, including eligibility criteria, can be found at clinicaltrials.gov. Search for “APX3330.”



Learn more about clinical trials Clinical trials are research studies in which people with either a high risk for cancer or diagnosis of the disease help physicians find ways to improve health and cancer care. Each study tries to answer scientific questions and to find better ways to prevent, diagnose or treat cancer as well as improve quality of life of cancer survivors. It is important that men and women of all ages and backgrounds take part in clinical trials. Each trial has eligibility criteria or rules about who can and cannot participate. You should talk to your doctor about whether you are eligible for a clinical trial. Learn more about clinical trials at the IU Simon Cancer Center.