2003 Young Investigator

Robert Vonderheide, MD, PhD

Abramson Family Cancer Research Institute, University of Pennsylvania School of Medicine, Philadelphia, PA
Professor of Medicine, Hanna Wise Professor in Cancer Research, Associate Director for Translational Research


Research Focus: Immunotherapy & Cancer Vaccines
Cancer Type: Gene Therapy for Neuroblastoma


2003-2006 Research Grant: Creating A Vaccine For Children With Cancer
A promising approach in cancer treatment is to vaccinate patients against molecules expressed by cancer in hopes of starting or awakening an immune response that will kill tumor cells. One leading strategy bases the center of such vaccines on the patient’s own immune cells, which are removed from the body and re-engineered outside the body before re-injection. With this grant, we will evaluate a novel gene therapy vaccine specifically designed for children with cancer, for whom very few attempts at cancer vaccination have been undertaken. This work will establish the scientific and manufacturing rationale for translating this gene therapy technology to the clinic, especially for children with cancer.

Current Research:

Dr. Vonderheide’s research has expanded beyond the treatment of just neuroblastoma to that of pancreatic cancer, lymphoma, breast cancer, and ovarian cancer. His lab is working on identifying universal tumor antigens for cancer immunotherapy, focusing mainly on a specific telomerase antigen. Telomerase, an enzyme, is expressed greatly in all human cancer cells but not nearly as much in normal cells. With this research, Dr. Vonderheide is working on a vaccine against telomerase, in which a certain type of white blood cell will target the telomerase in cancer cells and kill the tumor. Dr. Vonderheide has also worked on the development of another cancer vaccine which deals with a specific protein, CD40, and its role in activating the immune system. This vaccine has been used to privately owned dogs with large cell lymphoma and has worked with great success in treating the disease in the dogs. Now, the focus is to develop a similar vaccine that works as well in humans. Dr. Vonderheide’s research continues to focus on the treatment of various types of cancer through the use of his vaccines and immunotherapy, both of which will provide the potential for many types of cancer treatments if the results from clinical trials prove successful.

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