Steve Sprague has had more than his fair share of medical problems. After surviving diabetes, a heart attack and bypass surgery in 1993, Sprague learned in 1995 that he had chronic myelogenous leukemia (CML). “It was discovered during a routine check-up,” the 52-year-old labor union official recalls.

For the first two years after his diagnosis, Sprague managed the disease with chemotherapy. But in April 1997, while on a business trip, he woke up with unbearable pain. His disease had progressed to blast crisis—the most advanced stage of CML. “Everything I read said that CML progresses over 3 to 5 years,” says Sprague. “It felt as if I had been robbed of three of the five years I had been counting on.”

Sprague’s hematologist referred him to the Blood & Marrow Stem Cell Transplantation Program at Hackensack University Medical Center in New Jersey. After six weeks of intensive chemotherapy, he was back in remission, but the remission would be temporary, he was told.

“They said I needed a bone marrow transplant,” explains Sprague, “but it took awhile before I made up my mind to do it. Everything I read about transplants was so harrowing.”

Once Sprague decided to proceed with the transplant he quickly ran into a major obstacle: a donor with a matching marrow type could not be located. It was then that the director of Hackensack’s Stem Cell Transplant Program, Dr. Andrew Pecora, offered Sprague another alternative – an umbilical cord blood transplant.

Cord blood transplantation is similar to bone marrow transplantation. The stem cells in bone marrow that repopulate a patient’s body with healthy blood cells following a transplant are also found in umbilical cord blood, but in smaller quantities. For this reason, cord blood transplants are usually only offered to pediatric patients.

Pecora proposed using a new medical device called the RepliCell™ System that could increase the number of stem cells in a cord blood sample. Left with no other viable option, Sprague agreed to be Pecora’s first patient in the clinical trial.

A cord blood donor was quickly found. “All I know is that it was a female donor,” says Sprague.

After receiving high-dose busulfan, ATG and cyclophosphamide, he received his first dose of expanded cord blood cells. His daughter, Lisa, was with him throughout the treatment. Seventy-four days later, there was no evidence of leukemia in his bone marrow.

“The transplant gave me a whole new life,” says Sprague who has recovered from the procedure and a subsequent two-year struggle with graft-versus-host disease. I’ve lived to see both of my children marry and will soon be a first-time grandfather.”

“What was remarkable was that Steve had CML in blast crisis and had no available allogeneic donors,” recalls Pecora. “The cord unit was approximately one-third of the recommended dose, so a regular cord blood transplant was not optimal. Hence, the only reasonable option was to proceed with the RepliCell System study. The rest is history.”

Like many transplant survivors, Sprague has adopted a new lifestyle. He’s retired, is writing about his experience, and is taking some time to “smell the roses.”

He’s hopeful that other patients without a matching bone marrow donor will experience similar successes with this new therapy. “I want them to be able to follow me down this exciting new medical breakthrough trail,” he says.

Sprague may soon get his wish. Makers of the cell expansion device, Aastrom Biosciences, plan to open a clinical trial this Fall that will offer the therapy to patients with leukemia and lymphoma who lack a matching bone marrow donor. Although details have not yet been finalized, at least a half-dozen medical centers in the US are expected to participate in the trial.

Not a Guaranteed Cure

Not all patients who have been transplanted with expanded cord blood cells have been as fortunate as Sprague. More than half of those who participated in early clinical trials died either as a result of graft-rejection, graft-versus-host disease, infection, or malignant hypertension.

“These were all patients with very advanced disease who lacked a suitable bone marrow donor, and for whom the likelihood of a cure even with a matched bone marrow donor was in the 10-20 percent range,” says Patrick Stiff MD, Director of Transplantation at Loyola University Medical Center in Maywood Illinois, and the principal investigator on the study.

“In the new study, we will be treating patients who are in a less advanced stage of their disease,” says Stiff. “We expect to see a higher survival rate because their risk of developing a life-threatening infection will be less.”

“We will also use more sophisticated tests to determine if donors are a good match for the patient,” he said. “While this may decrease the number of patients eligible to enroll in the trial, it should also lower the incidence of graft-versus-host disease.”

Other centers are testing different methods of expanding the number of cells in bone marrow, stem cell and cord blood patients, and are achieving similar results, said Stiff. “Our goal is to mimic the excellent results that we are seeing in unrelated donor marrow matches facilitated by the National Marrow Donor Program,” says Stiff.

Steve Sprague’s success story may be early evidence that they will, indeed, succeed.