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World/National News

December 8, 2013

Gene therapy scores big wins against blood cancers

(Continued)

“What we are giving essentially is a living drug” — permanently altered cells that multiply in the body into an army to fight the cancer, said Dr. David Porter, a University of Pennsylvania scientist who led one study.

Several drug and biotech companies are developing these therapies. Penn has patented its method and licensed it to Switzerland-based Novartis AG. The company is building a research center on the Penn campus in Philadelphia and plans a clinical trial next year that could lead to federal approval of the treatment as soon as 2016.

Talking with the researchers, “there is a sense of making history ... a sense of doing something very unique,” said Hervé Hoppenot, president of Novartis Oncology, the division leading the work.

Lee Greenberger, chief scientific officer of the Leukemia and Lymphoma Society, agreed.

“From our vantage point, this looks like a major advance,” he said. “We are seeing powerful responses ... and time will tell how enduring these remissions turn out to be.”

The group has given $15 million to various researchers testing this approach. Nearly 49,000 new cases of leukemia, 70,000 cases of non-Hodgkin lymphoma and 22,000 cases of myeloma are expected to be diagnosed in the United States in 2013.

Many patients are successfully treated with chemotherapy or bone marrow or stem cell transplants, but transplants are risky and donors can’t always be found. So far, gene therapy has been tried on people who were in danger of dying because other treatments failed.

The gene therapy must be made individually for each patient, and lab costs now are about $25,000, without a profit margin. That’s still less than many drugs to treat these diseases and far less than a transplant.

The treatment can cause severe flu-like symptoms and other side effects, but these have been reversible and temporary, doctors say.

Penn doctors have treated the most cases so far — 59. Of the first 14 patients with CLL, four had complete remissions, four had partial ones and the rest did not respond. However, some partial responders continue to see their cancer shrink a year after treatment.

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