Doctors at John Hopkins say Pamela Newton is the first adult worldwide to be cured of Sickle Cell Disease using an experimental bone marrow transplant.

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. 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. Bone marrow transplants have been used to treat sickle cell disease for 20 years - but almost all of the 200 cured have been children. The treatments - high doses of chemicals that knock out the patient's own marrow before the transplant - are so toxic that adults with sickle cell-induced organ damage would be unlikely to survive them.
Brodsky said his team's procedure, developed by Dr. Ephraim Fuchs and Dr. Leo Luznik, is less toxic. They say they no longer believe they have to destroy as much of the patient's marrow as they once did - so they administer just enough chemotherapy to suppress the immune system. That dose keeps patients from rejecting the new marrow without harming their organs. This change allows transplants for adults, as well as children. Because the procedure occurs later in life, it relieves parents of the burden of making the decision for their youngsters (even in children, the sickle-cell transplant mortality rate is 5 percent to 10 percent). Instead, it allows the adult patient to see how severe the disease is before deciding whether to have a transplant.
Another transplant obstacle has been finding a perfect bone marrow match - a full sibling's marrow provides the best chance. But there's only a 25 percent chance that even a full sibling will be a match. And since sickle cell is inherited, siblings may also have the disease. That leaves about a 10 percent chance that a patient will find a suitable donor. Brodsky's procedure requires just a half-match - meaning that children and parents of the patient could be suitable donors.
Three days after the transplant, the patient is given a high dose of a drug called cyclophosphamide. Just as the bone marrow is taking root, the drug kills off the donor's lymphocytes - blood cells that are part of the immune system.
The cyclophosphamide spares the donor's stem cells and allows them to establish new blood cells and a new immune system. The nascent immune system is re-trained to see the patient's body as friend, not foe. This prevents the patient from rejecting the transplanted bone marrow - and prevents the newly developing immune system from attacking the patient.


Information Source: Sickle Cell Information Centre News Update for April 2008:
www.scinfo.org/news.htm
www.baltimoresun.com/news/health/balte. sickle30mar30,0,6112155.story