Two researchers from the Perelman School of Medicine at the University of Pennsylvaniawill be honored for their contributions for the burgeoning field of gene therapy by Pennsylvania Bio at their annual dinner this week. Pennsylvania Bio is the statewide bioscience trade organization which works to make the Keystone State a life sciences hub by creating an environment which cultivates progress and success.

Carl June, MD, the Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine and director of Translational Research in Penn’s Abramson Cancer Center, will receive the 2015 Hubert J.P. Schoemaker Leadership Award. The award is presented each year to a Pennsylvania scientist who has shown a “spirit of innovation” throughout his or her career. James M. Wilson, MD, PhD, director of the Orphan Disease Center, professor of Pathology and Laboratory Medicine, and director of the Gene Therapy Program, will receive the Scientific Achievement Award, which is given each year to a Pennsylvania scientist who advanced scientific knowledge, innovation, and/or patient care. 

June is recognized for his decades of work to advance cancer therapies, specifically hiswork as leader of the team responsible for the first successful and sustained demonstration of the use of CAR T cell therapy, an investigational approach in which a patient’s cells are removed through an apheresis process similar to dialysis and modified in Penn's cell and vaccine production facility. Scientists there reprogram the patients’ T cells through a gene modification technique using a viral vector that trains them to recognize specific types of cancer cells. The modified cells – known as chimeric antigen receptor (CAR) T cells –  are then infused back into the patient's body, where they multiply, hunt and attack tumor cells. He has also been a pioneer in the use of gene editing for HIV.

The latest results of clinical trials utilizing CAR T cells to treat blood cancers at Penn showed a response rate of 90 percent among pediatric and adult acute lymphoblastic leukemia patients. Among patients with chronic lymphocytic leukemia, the earliest group the research team began clinical trials with, in 2010, about 50 percent of patients respond to the therapy, and remissions among some of the first patients treated with the approach now exceed four and a half years. Early results in studies of patients with lymphoma and myeloma are also promising, and clinical trials are now underway to test this approach in patients with solid tumors.

Stretching back to the earliest days of work with gene therapy in the 1980s, Wilson’s research focus has been rare inherited diseases, ranging from cystic fibrosis to dyslipidemias to a variety of metabolic disorders. Most recently, Wilson’s laboratory discovered a family of viruses from primates that could be engineered to be very effective gene transfer vehicles. These so called "vectors" have become the technology platform of choice and have set the stage for the recent resurgence of the field of gene therapy.

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