I am finally home from travelling and speaking on behalf of people living with Sickle Cell Disease. My conclusion is not good. We (people with Sickle Cell) have the same concerns....and it's not looking good.
I will address these issues soon, but I wanted to finish my series on "Stem Cell Cure For Sickle Cell (Or Not?) Part II"
We hear in the news "Sickle Cell Cured!!!!" then.....nothing. We don't hear about complications, infections, secondary diseases caused by "treatment" or the true life-span of those "cured" children AND adults.
This is why I needed to finish exploring this "cure" subject. Here goes:
Stem Cell Cure For Sickle Cell (Or Not?) Part II
1. CHILDREN - Today, in children 250-300 stem cell bone marrow transplants are done in US. Doctors report a 90% success rate “IF AND ONLY IF” done on children with “perfectly matched sibling stem cell bone marrow donor”.
(Personally, I have NOT seen any long-term results data on these children’s quality of life, future complications from the aggressive chemotherapy treatment or immune suppressing medications, or the mortality rate after their “successful” stem cell bone marrow transplant)
I’m just saying.
In children, when there is a non-sibling stem cell donors (from the limited supply of National Bone Marrow Donor Program or Cord Blood Bank) there is a higher risk of immune diseases and transplant rejection which causes the body to attack itself. Over 5% reject the graft (transplant) & sickle cell comes back. National bone marrow donor register is also limited.
Cord blood stem cell donors are usually from a family member. This is very expensive, does not always cure sickle cell, and some patients die in the process with immune diseases caused by the aggressive chemotherapy and immune suppressing drugs.
My conclusion: Parents (and) patients must weight the benefits vs. risk with a FULLY INFORMED decision.
2. ADULTS - In adults, they have higher risk of side effects (immune diseases) and existing complications like previous strokes, eye damage, kidney damage, and bone decay issues like Avascular Necrosis will not be reversed if stem cell transplant is offered.
Answer: Cure in Adults has not proven effective, however future research & clinical trials will include (Genetically) Modified Stem Cell Bone Marrow Transplants.
According to a presentation (2012) by Dr. Donald Kohn at the Human Genome Medicine Program at UCLA, a recent $9 million dollar grant to California Institute of Regenerative Medicine (CIRM) was issued for stem cell “gene therapy” for curing sickle cell disease (in adults). Preparation for clinical trials (getting volunteer subjects) will begin soon.
(The requirements for this clinical trial are: 18 years or older, SCD (SS SThal), have few existing complications (that offset the risks of treatments), no existing matched donor, no existing matched sibling, pass state of health criteria.)
Stem cell “gene therapy” means bone marrow is removed (taken from hip), then “genetically modified” while patient is given high doses of chemotherapy to kill their existing bone marrow, then the modified bone marrow is given back to patient through transfusion directly into blood stream.
MY CONCLUSION:Sickle cell patients are being poked and prodded, clinical trialed and drug tested. In the meantime, we have to LIVE with sickle cell disease. I wish someone would fund Comprehensive SCD Care Centers again…..and help us “live” while you search for a “cure”.
Dr. Donald Kohn at the Human Genome Medicine Program at University California at Los Angeles (UCLA):
Sickle Cell Anemia Stem Cell Gene Therapy
and Bone Marrow Stem Cells