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Drug Trial for Blood Cancers Shows Great Promise


Image: Edward Sadowski is back to his weekly fishing trips thanks to his participation in a Temple clinical trial.

It began with a palm tree and a string of Christmas lights. Or so 84-year-old Edward Sadowski thought.

"I was visiting a friend in Florida and she asked if I would string some lights around a palm tree. I ended up getting all cut up. But everything seemed fine until a week later when I woke up back at home with a bunch of red and purple blotches all over my chest and side. I thought it was some sort of reaction to the palm tree and didn’t pay much attention to it. Then the next day I felt like a truck had hit me."

Unfortunately for Sadowski, his symptoms had nothing to do with the palm tree. After undergoing several tests, the diagnosis was far worse – acute myeloid leukemia (AML), a cancer of the blood and bone marrow.

"It was a shock. I'm very active and had been in great shape my whole life…I guess I got to thinking I was King Kong," says Sadowski.

AML is typically treated with high doses of chemotherapy. For some patients, however, traditional chemotherapy doesn’t work. In other patients, older age or weak physical condition rules out its use. This was the case with Sadowski.

"But my doctor said there was a new drug trial that had just opened up at Temple, and he thought I’d be a good candidate for it," says Sadowski. "So I volunteered to participate."

The clinical trial Sadowski's doctor referred him to is testing a drug called SGI-110, a small molecule agent that is delivered by injection. The drug is designed to work by removing abnormal tags within the DNA of leukemic cells. These tags work like bookmarks in the cell and are needed for normal protein expression and cell function.

Prior work in the research laboratory of Jean-Pierre Issa, MD, Director of Temple's Fels Institute for Cancer Research and Molecular Biology, showed that the activity of this drug could be directly related to its ability to correct these bookmarks in DNA. Temple is the only local hospital participating in this trial and has enrolled the second-highest number of patients in the country.

"The trial opened in February of this year and so far we are seeing very promising results," says Patricia Kropf, MD, Assistant Director of the Temple Bone Marrow Transplant Program and lead investigator of the trial at Temple.

Sadowski was one of the first to enroll in the trial at Temple. For five consecutive days he received injections of the drug, followed by four weeks off and then another five injections. After two rounds on this schedule, new blood work and a bone marrow biopsy revealed some very good news.

"The doctor told me I was in remission!" says Sadowski, who continues to receive injections and is currently on his ninth round.

"Now I'm back to doing everything I did before I got sick – taking my boat out for weekly fishing trips, taking care of my yard, tending to my 50 tomato plants. I feel almost like Superman."

In late August, Astex Pharmaceuticals announced that top-line results from the trial showed "clear activity" in patients with AML. The detailed results have been submitted for presentation to the American Society of Hematology meeting to be held in December.

Date Published: Monday, November 04, 2013