Kim Irwin ([email protected])
(310) 206-2805
Kambra McConnel ([email protected])
(310) 206-3769
Note to Editors: Patients quoted in the news release are available for interviews
DRUG FOR ADULT LEUKEMIA SHOWS PROMISE IN
EARLY STUDIES; PATIENTS BEING SOUGHT BY
RESEARCHERS AT UCLA'S JONSSON CANCER CENTER
FOR SECOND PHASE OF CLINICAL TRIALS
Researchers at UCLA's Jonsson Cancer Center are seeking patients with chronic myelogenous leukemia (CML) to participate in a study of a new experimental drug that has resulted in dramatic responses in some patients in early testing.
Dr. Charles Sawyers, a co-investigator who led the phase one trials at UCLA's Jonsson Cancer Center, said he is "tremendously encouraged" by results from early testing of the experimental drug STI-571, being developed by Novartis Pharmaceuticals. The drug, taken once a day in pill form, is a targeted therapy, meaning it attacks only cancer cells and leaves healthy cells alone. Targeted therapies often result in few, if any, side effects for patients, Sawyers said.
Results of phase one testing of STI-571 will be announced Sunday (Dec. 5) at the American Society of Hematology conference in New Orleans by Dr. Brian Druker of Oregon Health Sciences University, who served as lead investigator for the phase one study. The three institutions that tested STI-571 include UCLA's Jonsson Cancer Center, Oregon Health Sciences University and the M.D. Anderson Cancer Center in Houston. Dr. Moshe Talpaz led clinical trials at M.D. Anderson.
Novartis officials are discussing with the U.S. Food & Drug Administration the possibility of accelerating the testing process of STI-571 because of the encouraging early results. Second phase testing of STI-571, to take place at UCLA and 18 other sites in the United States and Europe, is expected to begin this month.
At UCLA's Jonsson Cancer Center, 24 patients in the chronic stage of CML are enrolled in the phase one testing of the experimental drug. Sawyers said nearly all have done well so far.
"I think this is tremendously exciting, a confirmation of the rationale of targeted therapies in drug design," said Sawyers, associate chief for basic research at UCLA's Jonsson Cancer Center and an associate professor in the Division of Hematology-Oncology at the UCLA School of Medicine. "This drug attacks a mutant protein without harming surrounding normal cells."
Specifically, STI-571 attacks a mutant protein in a cancer-causing gene linked to CML, which strikes more than 4,300 adults in the United States every year. Much of the pioneering work done to link the gene and its mutant protein with CML was done at UCLA's Jonsson Cancer Center by researcher Owen Witte, a professor of microbiology and a renowned investigator with the Howard Hughes Medical Institute. Sawyers, who did his postdoctoral training with Dr. Witte, participated in the research linking the mutant gene with CML.
In the chronic stage of CML, patients experience skyrocketing white blood cell counts, but generally exhibit no symptoms for three to five years. The disease can then advance to the accelerated stage, during which white blood cell counts increase even more. The end stage of the disease, known as blast crisis, can be fatal within several months.
Early testing of STI-571 at UCLA focused on patients in the chronic stage of the disease who did not respond to standard treatments with interferon. At doses of around 200 mg of STI-571, nearly all the patients had their white blood cell counts return to normal levels. At higher doses, the molecular cause of the disease - the genetic mutation - disappeared in some of the cases, Sawyers said.
Seth Shapiro, a 35-year-old designer of computer games for children, was among those UCLA patients whose white blood cell counts returned to normal levels after taking the experimental drug. Shapiro said his white blood cell counts were normal within three weeks of taking STI-571.
Shapiro was diagnosed in April of 1998 with CML. After interferon failed to help him, instead prompting a host of debilitating side effects such as extreme fatigue and depression, Shapiro began taking STI-571 in February of this year.
"I feel like I've gotten my life back," said Shapiro, who lives in Los Angeles. "For the first time in more than a year, I don't worry about being sick every day. When I got my diagnosis, I felt like I was out of the race. Now I'm back in the game. I believe this drug saved my life."
Shapiro said he has experienced few side effects from the experimental drug. Better still, he's back "at full speed," working at the job he loves and enjoying his busy life - something he never thought he'd be able to do when he was diagnosed with CML.
"I can do just about anything I want now. It's almost as if my leukemia is now a chronic disease like diabetes," he said. "That's the greatest gift you can ever get. Life is not over for me, and that's pretty amazing."
Virginia Garner, a 54-year-old high school English teacher from Claremont, also participated in the phase one testing of STI-571 at UCLA's Jonsson Cancer Center. Diagnosed with CML in August of 1997, Garner underwent interferon treatments and experienced what she characterized as "extreme side effects."
She began taking STI-571 last April and saw a measurable response in her white blood cell count within five months. She's back in the classroom and living a full life, including taking a recent dream vacation with her husband to Viet Nam.
"I have a feeling about this new drug, that it's going to turn out to be something everyone is going to be amazed at," Garner said. "Those are precious pills."
With interferon, the current standard therapy for CML, a small fraction of patients are cured, Sawyers said. Most relapse. A bone marrow transplant also can help, but Sawyers said fewer than 20 percent of CML patients qualify.
"I believe that in five years or sooner this drug will be the front-line therapy for CML if the results hold up as they have in the first phase of testing," Sawyers said. "I want to stress that this validates a whole concept of drug development: That through basic science we can understand what's wrong with a cancer cell and actually fix it."
Dr. Harmon Eyre of the American Cancer Society said it's rare to see a response in phase one testing of experimental drugs. But in this case, the testing has resulted in very good responses early on, which he calls "very exciting."
Each year, more than 4,300 new cases of CML are diagnosed in the United States, according to the American Cancer Society. Of those cases, about 2,400 will result in death. Because the chronic stage of the disease can last up to five years, the population of adults living with CML continues to increase, officials from the American Cancer Society said.
For more information about the phase two testing of STI-571 at UCLA's Jonsson Cancer Center, or to volunteer to participate in the study, please call 310-794-4928.
-UCLA-
For more information about UCLA's Jonsson Cancer Center, its people and resources, visit our site on the World Wide Web at http://www.cancer.mednet.ucla.edu.
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