Four new clinical trials for breast cancer, using very high-dose chemotherapy with bone marrow transplantation, are expected to be fully under way within a month in Baltimore and at 28 other medical centers across the country.
In going public for the first time about its plans yesterday, the National Cancer Institute said the studies will involve between 1,200 and 1,500 women whose cancers are advanced, have spread, and who for other reasons have a poor chance of recovery.
The trials, offering new hope to women, will be run by clusters of oncology centers and will last for five years.
Half of the patients will receive standard chemotherapy and half standard chemotherapy plus an autologous bone marrow transplant. Random assignment of patients by computer will determine which types of patients will truly benefit from the new treatment.
Both the Johns Hopkins Oncology Center and the University of Maryland Oncology Center will participate in the trials, which experts term significant.
"People once wondered if these randomized trials could ever be done and whether women would ever accept them," said Dr. Jeffrey Abrams, who heads UM's breast cancer program and is one of the investigators. "To have someone say, 'I'll let fate or chance be my guide,' that requires some very courageous people."
Autologous bone marrow transplantation, or ABMT, which has been described as a radical new approach to chemotherapy, involves three steps:
* Removing the patient's bone marrow, the gel-like tissue inside of bones that produces immune system cells, which is then purged of any remnants of cancer.
* Administering very high doses of chemotherapy.
* Re-introducing the healthy bone marrow cells to regenerate the patient's marrow.
"Studies have demonstrated that the use of high-dose chemotherapy with autologous bone marrow support can result in higher frequency of complete remission in patients with metastatic breast cancer," said William Peters, a Duke University physician and a principal investigator.
"It can also result in some even very poor prognosis patients remaining continuously disease-free for extended periods of time."
Peters made his comments at a one-day forum on emerging breast cancer treatments at the National Institutes of Health in Bethesda yesterday that drew insurers as well as doctors, researchers and key personnel from many medical institutions.
The funding of bone marrow transplants has been a difficult issue in Maryland, resulting in many court cases, some of which have been successful.
The national Blue Cross-Blue Shield has said that it would be willing to support the random trials and several of the individual "Blues" across the country have agreed.
"To my knowledge, however, the Maryland 'Blues' have not gone along with that," Abrams said. "But, I think there's a movement towards trying to find a meeting of the minds. Until we know how effective ABMT is, I think the costs will have to be fought out on an individual patient-to-patient basis."
Hopkins has been doing its own high-dose chemotherapy and bone marrow transplantation studies on breast cancer patients for the past two years. "Our data show that if they have advanced disease, 20 percent of the transplant patients have prolonged remissions -- between two and four years -- without doing a further thing," said Dr. M. John Kennedy, an investigator who treats breast cancer patients.
"We have a lot of hope that these new very high-dose chemotherapy approaches with bone marrow transplant will truly be an advance," said Abrams. "But, they certainly have more side effects. They even have the risk of death during the treatment, although we've managed to reduce that risk considerably.
"The way to prove whether these very high personal costs and treatment costs are worth it, are these randomized trials."
The average cost of treatment is $250,000.
The Hopkins Oncology Center is part of a group seeking to enroll several hundred women who have had a malignant lump removed but are considered at high risk for a recurrence of the cancer, Kennedy said.
Hopkins, with its long experience in the field of bone marrow transplantion, probably will serve as a referral site for many centers that do not have that expertise.