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Jerry Chipman [email protected] St. Jude Children's Research Hospital 901/495-3306 Kristine Wilber [email protected] Porter Novelli 312/856-8818

EMBARGOED for Release Until August 26, 5:00 PM EST

Milestone Reached in Treatment of Most Common Pediatric Cancer; Trial of Newest Protocol Enrolls First Patients

MEMPHIS, TENN, August 27, 1998 -- A world milestone in the treatment of the most common form of pediatric cancer, acute lymphoblastic leukemia (ALL), has been achieved in the United States and efforts are already underway to test a new treatment protocol that researchers hope will lead to even higher cure rates for this once-deadly disease.

As announced in a paper published in the August 27, 1998 issue of the New England Journal of Medicine, St. Jude Children's Research Hospital in Memphis, Tenn., has reached an 80 percent cure rate for acute lymphoblastic leukemia, a cancer that affects more than 2,500 children every year. Authors of the paper, which reviewed the latest developments in ALL treatment around the world, were Ching-Hon Pui, M.D., director of the leukemia/lymphoma division in the Hematology/Oncology department at St. Jude Children's Research Hospital, and William Evans, Pharm.D., chair of the Pharmaceutical Sciences department and deputy director of St. Jude Children's Research Hospital.

Simultaneously with the announcement of the 80 percent, five-year, event-free cure rate, St. Jude Children's Research Hospital researchers announced that the first patients have been enrolled in the clinical trial of a new treatment that researchers hope will raise the cure rate to 90 percent or more, with an eventual goal of a complete cure for ALL. The new protocol, Total Therapy 14, employs the most comprehensive risk classification system to date, including molecular and immunological studies, which will permit more precise selection of treatment. Patients will also receive more intensive therapy with additional courses of multi-agent chemotherapy, which is designed to increase the efficacy of the treatment. In addition, the therapy is designed to have fewer side effects because, unlike other current treatment methods, no radiation is administered to the patient's central nervous system.

"When St. Jude Children's Research Hospital was opened in 1962, the outlook for children with acute lymphoblastic leukemia was dismal. Only 4 percent survived," said Arthur Nienhuis, M.D., director, St. Jude Children's Research Hospital. "Now we've reached 80 percent, so that parents of children with ALL can be told their child has an excellent chance of survival."

With the new protocol, and others underway at St. Jude Children's Research Hospital, "We are moving even closer to the day when acute lymphoblastic leukemia will be known as a serious, but curable disease," he added.

Study co-author Dr. Evans said, "We are gratified about the remarkable progress being made in fighting this once deadly disease. In the early 1970s, we announced a five-year, 50 percent cure rate and in 1990 we raised that figure to 70 percent. Now it has reached 80 percent, making ALL one of the most curable types of pediatric cancers."

According to Drs. Evans and Pui, a combination of factors has contributed to the increased cure rate:
-- A greater understanding of the biology of leukemic cells and the specific genetic abnormalities that cause them;
-- Increased knowledge about the clinical and biological variables that can be used to optimally assign patients to different treatments to avoid over or under treatment;
-- Improved treatment for opportunistic infections that can weaken the patient's condition, as well as supportive care for psychosocial problems that can affect the patient's health;
-- And, most importantly, the development and testing of more effective multi-drug regimens in well-designed clinical trials.

"Lessons that have helped St. Jude Children's Research Hospital achieve an 80 percent cure rate for acute lymphoblastic leukemia will benefit children around the world through our outreach programs, our involvement in the National Comprehensive Cancer Network and our ongoing commitment to sharing information with colleagues throughout the world," said Dr. Nienhuis. "The progress we've made indicates a much greater understanding of acute lymphoblastic leukemia -- and of cancer in general," he added.

While acute lymphoblastic leukemia is primarily a pediatric cancer, several thousand adults are affected every year. "The cure rate for adults with acute lymphoblastic leukemia is not nearly as high as it is for children," said Dr. Pui. "This discrepancy is caused in part by the higher frequency of high-risk genetic abnormalities in the leukemic cells of adults. However, success we've realized in treating children holds promise for adult patients, too."

"While we are excited about the significant progress we've made," said Dr. Pui, "we're continuing our efforts to identify new antileukemic drugs and new approaches to therapy to achieve even higher cure rates. The challenge now is for St. Jude Children's Research Hospital researchers to find a curative treatment for the remaining 20 percent of children with this disease."

St. Jude Children's Research Hospital's staff includes a winner of the Nobel Prize for Medicine, two Howard Hughes Research Institute Investigators and three department chairs who are members of the National Academy of Sciences.

St. Jude Children's Research Hospital, in Memphis, Tenn., was founded by the late entertainer Danny Thomas. The hospital is an internationally recognized biomedical research center dedicated to finding cures for catastrophic diseases of childhood. The hospital's work is supported through funds raised by the American Lebanese Syrian Associated Charities (ALSAC). All St. Jude Children's Research Hospital patients are treated regardless of their ability to pay. ALSAC covers all costs of treatment beyond those reimbursed by third party insurers, and total costs for families who have no insurance.

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