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Stem-Cell Transplantation for Sickle Cell Disease

Posted Dec 15 2009 12:00am

Anyone Want a Bone Marrow Transplant or
maybe a Stem Cell Transplant?

The airways are popping. The media blitz is in full affect. Massive editorial posts have been sent all over the internet. The word is out. There MAY be a cure for Sickle Cell Disease.

According to a New England Journal of Medicine report dated December 10, 2009, researchers at the U.S. National Institutes of Health say that a new method of bone marrow transplantation cured nine out of 10 adult patients with sickle cell disease.

NEW…hmmmm. I’ll take a big mac, fries and a bone marrow transplant…. please.

Here’s what I’ve gathered from reading the media stuff.

Old Bone Marrow Transplant Method - In conventional bone marrow transplants, high doses of chemotherapy drugs and radiation were given. Chemotherapy and radiation is used to wipe out the person's own bone marrow, which makes the faulty red blood cells. There are many complications including destroying fertility

Adults were usually not good candidates for bone marrow transplants because they were thought to be too sick to handle the high doses of chemotherapy and radiation needed to prep the body for the procedure.

New Bone (Stem Cell) Marrow Method - Senior study author, Dr. John Tisdale, a senior investigator at the U.S. National Institutes of Health explained the new method allows for less grueling pre-transplant routines, which adults with severe sickle cell can tolerate.

10 patients ages 16 to 45 with severe sickle cell disease received bone marrow from donors that were siblings with matched HLA (human leukocyte antigen) in their blood.

Lower levels of radiation were used in the new method which does not seem to destroy fertility. The bone marrow is replaced with stem cells from a donor's marrow, which then takes over and begins to produce new, healthy red blood cells.

NOTE: When doing the new bone marrow transplants, the researchers noted that not all of the patient's own marrow was wiped out. Some remained and seemed to co-exist with the donor marrow without causing problems. Dr. Tisdale said, "That meant we didn't necessarily have to kill the entire bone marrow of the patient to make this work." Tisdale said, this opens the possibility of using an even less toxic means of preparing the body for transplant.

Patients in the new method study were also given:

Alemtuzumab, a drug used to suppress immune system T-cells
Side Effects of Alemtuzumab - events include hypotension, rigors, fever, shortness of breath, bronchospasm, chills, rash, syncope, pulmonary infiltrates, ARDS, respiratory arrest, cardiac arrhythmias, myocardial infarction, and cardiac arrest. Some cases of cardiac adverse events have resulted in death. http://www.drugs.com/sfx/alemtuzumab-side-effects.html

Sirolimus, an immune suppressant to fight rejection
Side Effects of Sirolimus – acne, back pain, constipation, diarrhea, headache, joint pain, nausea, trouble sleeping, vomiting and weakness. http://www.drugs.com/cdi/sirolimus.html#side-effects

After 30 months, all 10 patients are alive, and nine of the patients had successful grafts where none of the patients experienced graft-versus-host disease (where the body rejects the new bone marrow). They are also considered cured of sickle cell disease, according to the study. Though most patients in the study are still taking immune-suppressant drugs, researchers hope to eventually wean them off the medications.

The new procedure doctor’s say is promising, especially since it could eventually include those who don't have an HLA-matched sibling. Dr. Tisdale said, "These were the sickest of the sick patients. Some were in the hospital every other week for pain or other crises. Today, some have gone back to school and to work. One patient had a baby."

In the past, these older/sicker patients were excluded from transplant studies as they are very poor candidates for high dose chemotherapy regimens. This study makes it possible to offer patients with severe sickle cell disease stem cell transplants."

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My conclusion: This is a “study”….also know as a “trial”. We won’t see this stem cell treatment given to “us” for a while. But, I’m hopeful.
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Links related to this report:

Bone Marrow Transplants May Cure Sickle Cell in Adults http://www.dreddyclinic.com/forum/viewtopic.php?f=25&t=22080&p=34580#p34580

Bone Marrow Transplants May Cure Sickle Cell in Adults - http://www.nlm.nih.gov/medlineplus/news/fullstory_92818.html

Stem Cell Researcher Dr. MM Hsieh – Co-author of report http://www.stemcellscience.org/showauthor.php?surname=Hsieh&initials=MM

New England Journal of Medicine report dated December 10, 2009
“Allogeneic Hematopoietic Stem-Cell Transplantation for Sickle Cell Disease”
http://content.nejm.org/current.dtl

National Institute of Health

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