Fifteen months ago, the pain from Pamela Newton's sickle cell disease was excruciating. She spent more time in the hospital than in her Capitol Heights apartment. She was on 15 pain pills a day, all heavy narcotics. She was bleeding regularly and needed daily transfusions of platelets.
She had just months to live.
Today, doctors at Johns Hopkins Hospital say that Newton is one of the first adults in the world to be cured of sickle cell disease - and the first using an experimental bone marrow transplant that could cure thousands like her who have been told they will never get better.
Word of a breakthrough gives hope to the roughly 80,000 Americans - and millions around the world - who suffer from this debilitating and usually fatal disease, which is predominant among African-Americans and Hispanics.
"It could very much open things up," said Dr. John F. Tisdale, a senior investigator at the National Heart Lung and Blood Institute. "Everyone's cautiously optimistic."
What makes the Hopkins procedure different is that it allows patients to receive bone marrow from a donor who is not an exact match - a longtime obstacle to healing large numbers of people.
Sickle cell is an inherited blood disorder that affects red blood cells. Normally round, they become "C" shaped, like sickles, and pile up on one another, keeping them from properly supplying oxygen throughout the body. The patients, prone to infection and serious pain, typically live only into their 40s.
Until now, few have been cured - and there has been little progress in developing new treatments. Only one drug has been approved for treatment of sickle cell. Painkillers and antibiotics help patients live longer, but nothing has addressed the core problem of the genetic defect.
Newton's hematologist, Dr. Robert Brodsky, and his colleagues say they hope to change that. They are trying to enroll 25 patients in a clinical trial.
Still, the procedure is not without risk. When performed on patients with leukemia and other diseases, it has a mortality rate of 16 percent.
"We may shorten people's lives, but this is their only chance for a cure," Brodsky said. "Would you offer this to every sickle cell patient? Absolutely not. But the patient who is ending up in the emergency room three and four times a month and having organ-threatening and life-threatening complications from the disease ... should be able to make that decision."
