Gene therapy is a revolutionary new form of treatment. It has emerged in recent years with the remarkable potential not only to deliver a cure for many types of cancers, but also to improve the quality of life with reduced side effects for patients undergoing treatments. Every year, over a half million people die from cancer and another million are diagnosed with some form of the disease. Conventional therapies, including chemotherapy, surgery, and radiation, while having produced a measure of success with some cancers, have their limitations and can generate adverse side effects for patients.
The research efforts of these Young Investigators, pursuing newly emerging strategies within the cancer gene therapy field, represent significant potential applications for all forms of cancer and an improved quality of life for those being treated for cancer.
LAURENCE COOPER, MD, PhD, City of Hope National Medical Center, Duarte CA
Using The Immune System's T Cells To Treat Leukemia And Lymphoma More Effectively (2003 grant recipient)
Relapsed or drug-resistant leukemia and lymphoma are very difficult to cure with surgery, radiation and chemotherapy, the traditional workhorses of cancer treatment. Dr. Cooper and his lab are developing a new approach that uses the patient's own immune system to directly attack the disease. The investigators are harnessing the power of a type of immune cell called T cells, using gene therapy to redirect the function of the T cell so that it can target most leukemias or lymphomas.
TIMOTHY F. LANE, PhD, Jonsson Comprehensive Cancer Center, The David Geffen School of Medicine, University of California Los Angeles, Los Angeles CA
Seeking A New Target For Cancer Treatment of Breast Cancer (2003 grant recipient) A tumor cannot grow larger than a pinhead unless it establishes an independent blood supply through a process called angiogenesis. Cancer researchers are seeking new and better molecular targets to halt the growth of this independent blood supply that cancers need. A type of stem cell called an endothelial cell precursor (ECP) that plays a role in the formation of new blood vessels has recently been identified. Dr. Lane and his lab have identified a genetic element expressed in ECPs and created a virus that uses this element to target gene expression directly to these blood vessel progenitors.
RICHARD REILLY, PhD, Johns Hopkins University School of Medicine, Baltimore MD
Developing A Vaccine To Protect Against Breast Cancer (2003 grant recipient)
This project is focused on developing a vaccine that harnesses the body's immune system to seek out and eliminate minimal residual disease in women with breast cancer, and to wipe out any microscopic cancer deposits that may have spread to other parts of the body. Dr. Reilly and his lab are using a genetically modified tumor cell as the vaccine. The target of the vaccine is called HER-2/neu (neu), a cancer-related gene that is overexpressed in about one-out-of-three breast cancers. Using a specially developed laboratory animal model, this group has demonstrated that the combination of neu-targeted vaccination and neu-specific monoclonal antibodies eradicates neu-expressing tumors in nearly half the animals, while the individual treatment components used singly are ineffective.
KATHERINE D. RYMAN, PhD, Louisiana State University Health Sciences Center, Shreveport, LA
Infecting Prostate Cancer Cells With A Killer Virus (2003 grant recipient)
Prostate cancer currently afflicts more than nine million men in this country. More than 220,000 new cases will be diagnosed this year, and over 31,000 men will die of the disease. Virotherapy, which employs viruses that specifically target and destroy cancer cells, has recently gained favor as a means of delivering cancer gene therapy. This project is developing virotherapy for prostate cancer, using a virus called Sindbis (SB).
ROBERT VONDERHEIDE, MD, PhD, Abramson Cancer Center, University of Pennsylvania School of Medicine, Philadelphia PA
Creating A Vaccine For Children With Cancer (2003 grant recipient)
Vaccinating cancer patients against molecules expressed by their cancer, with the hope of starting or awakening an immune response that kills tumor cells, is a highly promising treatment approach. One such strategy involves generating the vaccine on cells from the patient's own immune system by removing the cells from the body, re-engineering them in the laboratory and re-injecting them. However, very few attempts to vaccinate children with cancer have been made. Dr. Vonderheide and his lab are evaluating a novel gene therapy vaccine specifically designed for children and based on the child's own B lymphocytes, a particular type of white blood cell.
For more detailed information, including an update on the research of the 2002 recipients, please visit www.acgtfoundation.org
The mission of Alliance for Cancer Gene Therapy, Inc., Stamford, CT, is to increase private funding to support basic research, technology innovation, pre-clinical validation and clinical translation to expedite development of effective and safe therapies for cancer. In 2002, ACGT awarded $1.5 million in grants and in 2003 awarded $2.5 million in grants. In May 2004, ACGT will award its first $1 million grants for gene therapy for lymphoma and leukemia. The primary recipients of ACGT Awards and Grants are Young Investigators and Investigators - scientific researchers at academic institutions throughout the United States who have obtained Assistant and Full Professor status.
ACGT is a 501 (c) (3) non-profit organization founded in 2001 by Edward Netter, a Greenwich, CT philanthropist and chairman of Geneve Corporation, and his wife Barbara, in collaboration with Dr. Savio Woo. ACGT is governed by a Board of Directors and advised by a Scientific Advisory Council of 12 pre-eminent physicians and researchers, all national leaders in cancer gene therapy research. Members of both groups serve without financial remuneration. 100% of all funds raised go directly to research, with separate funding for administrative expenses. For additional information regarding ACGT, please call 203-358-8000 or visit www.acgtfoundation.org.