A bone marrow transplant (BMT), replaces an affected person’s bone marrow with donor marrow that can produce the missing
enzyme. The procedure is risky, invasive and has had very little success in Tay-Sachs, Sandhoff and GM-1. If done early enough it can be effective in slowing and even stopping the disease in other allied diseases like Krabbe and MPS.
Currently only two centers in the US will consider Tay-Sachs, Sandhoff and GM-1 patients for BMT: Duke University Medical Center and University of Minnesota. The Duke program is not an approved clinical trial (contact June Allison-Thacker at
This e-mail address is being protected from spambots. You need JavaScript enabled to view it
to learn more).
Bone Marrow Transplant is very invasive. Children often succumb to complications of the procedure. The immune system must be completely destroyed before introducing the healthy bone marrow. It is unclear how many of the healthy stem cells cross into the
central nervous system.
Learn more:
http://www.nlm.nih.gov/medlineplus/ency/article/003009.htm
http://www.bmtinfonet.org/
