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GREENWICH, Conn. When Greenwich resident Barbara Netter founded the Stamford-based Alliance for Cancer Gene Therapy in 2001 alongside her husband Edward, no one had heard of gene therapy as a treatment for cancer. Recently, the group funded a leukemia study that found a breakthrough treatment using gene therapy.
Theyre making a lot of progress taking what they know in the laboratory and transferring that to the pharmacists and to the bedside, said Netter. In the future, gene therapy will take center stage, and we hope it will help mankind.
Dr. Carl June, an alliance fellow, and his team at the University of Pennsylvanias Abramson Cancer Center and Perelman School of Medicine made the breakthrough this year. The study, published in the Aug. 10 issue of the New England Journal of Medicine and in Science Translational Medicine, was 20 years in the making. The alliance, not the National Cancer Institute or a pharmaceutical company, funded the recent clinical trial.
About five years ago we funded Dr. June, who was a young investigator, and he evidently has come forth with a big breakthrough in terms of immunotherapy treatments, said Netter. Two of his people seem to be totally free of cancer and another one of them was no longer in need of treatment.
The treatment uses genetically modified versions of the patients own T-cells and has shown remission for up to a year.
The protocol provides a tumor-attack roadmap for the treatment of leukemia and other cancers, including those of the lung and ovaries as well as myeloma and melanoma. To make this roadmap, patients white blood cells must be removed and modified at University of Pennsylvanias vaccine production facility, then infused back into the patient's body after chemotherapy. This is the first demonstration of the use of gene transfer therapy to create serial killer T cells aimed at cancerous tumors.
"Within three weeks, the tumors, which were several pounds each, had been obliterated in a way that was much more complete than we ever expected," said June, director of translational research and a professor of pathology and laboratory medicine in the Abramson Cancer Center, who led the work. "The results exceeded our expectations quite a bit; our entire team is really excited, and as well, the patients are excited."
Although the study size was just three patients, the results are outstanding. These are patients that had few treatment options, said Dr. David Porter, professor of medicine and director of blood and marrow transplantation.
The Alliance for Cancer Gene Therapy is the nations only nonprofit dedicated exclusively to cell and gene cancer therapy research. It was started by Barbara and Edward Netter of Greenwich in 2001 after their daughter-in-law, Kimberly Lawrence-Netter, battled breast cancer for 11 years.
After my daughter-in-law passed away, and we just happened to meet two wonderful scientist and were so impressed that we wanted to put our energies into this, said Netter, whose husband died in February. Ed was so visionary and he felt that [gene therapy] had to be the answer and thought that this was worth enough work and time.
Also, 100 percent of all contributions made to the alliance goes directly to research and funds grants. In the past 10 years, the alliance has funded more than 37 research fellows and currently has 17 human clinical trials under way. This is a clinical study trial one, and he still wants to get into clinical phase trial two. He will need more funding, but we are still funding and its important for people to contribute to us, said Netter.
For more information visit the Alliance for Cancer Gene Therapy website.
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