UAMS Researchers’ Cure of Metastatic Skin Cancer Published in New England Journal of Medicine

Newswise — LITTLE ROCK – Identifying a patient’s genetic mutation led University of Arkansas for Medical Sciences (UAMS) physician-researcher Ling Gao, M.D., Ph.D., to an existing drug that eliminated the patient’s stage IV Merkel-cell carcinoma. Gao’s findings, made in collaboration with two other UAMS researchers, were published today in The New England Journal of Medicine.

Metastatic Merkel-cell carcinoma is often fatal and there is no effective treatment. Gao’s 86-year-old female patient was diagnosed in 2013 with stage IIIB Merkel-cell carcinoma of the right temple. She had surgery and received radiation therapy in May 2013 and additional surgery in July 2014. In November 2014, doctors confirmed that the cancer had metastasized.

Gao, a dermatologist who treats Merkel-cell carcinoma patients from Arkansas and surrounding states, performed genetics tests on the tumor that revealed multiple mutations, including PI3Kδ.

Gao was aware that the drug idelalisib is a novel PI3K pathway inhibitor approved by the Food and Drug Administration (FDA) for treatment of B-cell lymphoma, a cancer of the blood. She was also aware of recent studies showing that disruption of the PI3Kδ mutation allows the body to mount an effective antitumor immune response.

On the basis of her laboratory work and knowledge of idelalisb, Gao began treatment of the patient with the drug on Feb. 6, 2015. Her findings were published in the Journal in a letter to the editor.

Three months after administering the idelalisib, there was no sign of the tumor in the patient’s liver, Gao said

Based on what she’s learned, Gao said the case can be made for further study of PI3Kδ inhibitors like idelalisib in solid tumors, not just blood cancers.

“The efficacy of idelalisib in our patient provides initial clinical evidence that the targeting of PI3Kδ in Merkel-cell carcinoma is warranted,” Gao said in the letter, which was also signed by UAMS’ Fade Mahmoud, M.D., and Mallory B. Shiver, M.D.

Gao also credits support she received from Peter Emanuel, M.D., director of the UAMS Winthrop P. Rockefeller Cancer Institute, and James Suen, M.D., chair of the Department of Otolaryngology-Head and Neck Surgery in the UAMS College of Medicine.

View the article and accompanying PET-CT findings here.

http://tri.uams.edu/files/2015/10/NEJM-Letter-Gao.pdf

Gao’s research has been supported by the UAMS Translational Research Institute through the National Institutes of Health National Center for Advancing Translational Sciences, the Department of Dermatology in the UAMS College of Medicine, UAMS Winthrop P. Rockefeller Cancer Institute, and the Arkansas Biosciences Institute.

UAMS is the state’s only comprehensive academic health center, with colleges of Medicine, Nursing, Pharmacy, Health Professions and Public Health; a graduate school; a hospital; a northwest Arkansas regional campus; a statewide network of regional centers; and seven institutes: the Winthrop P. Rockefeller Cancer Institute, the Jackson T. Stephens Spine & Neurosciences Institute, the Myeloma Institute, the Harvey & Bernice Jones Eye Institute, the Psychiatric Research Institute, the Donald W. Reynolds Institute on Aging and the Translational Research Institute. It is the only adult Level 1 trauma center in the state. UAMS has 3,021 students, 789 medical residents and two dental residents. It is the state’s largest public employer with more than 10,000 employees, including about 1,000 physicians and other professionals who provide care to patients at UAMS, Arkansas Children’s Hospital, the VA Medical Center and UAMS regional centers throughout the state. Visit https://www.uams.edu or https//www.uamshealth.com. Find us on Facebook, Twitter, YouTube or Instagram.