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Major Announcement Concerning Gene Therapy

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Andy Battaglia:
Chemo preparation is used for both BMT and gene therapy, but with gene therapy there is no chance for graft vs host disease, GVHD, which is the biggest danger of BMT.

Pratik:
I understand your last reply less Andy, hehe. So in basic words, just as BMT has chances of life being endangered, gene therapy/injection has no side effects or risk at all? At the most it would have is it would go fail on a person but the person wouldn't be affected at all, is it?

-Pratik.

Sharmin:
Pratik,

Gene therapy has yet to go through trials, so risks and side effects are yet to be determined.  But like anything, there may be risks yet not known to us.  All of the research done to date has been to create a cure that is effective and safe - and the main purpose of some of the early trials is to determine safety more so than efficacy.  

Gene therapy should eliminate many of the risks inherent in bone marrow transplant because the cells come from the patient (the donor and recipient are one and the same - aka autologous).  The risk of gvhd - which is the greatest risk in bmt will be eliminated.  Also, harsh anti rejection drugs will not be required.  

Chemotherapy however, will be required - although it is yet to be determined how much.  Chemotherapy does have side effects and how big these effects are depends on what type of chemo preparation is found to be necessary by these trials.  Our hope is that very minimal amounts are required.  

Other risks may be do to how well the gene fragment is incorporated in the stem cells - and whether it may have unwanted effects - but Dr. Sadelain has worked long and hard to avoid these risks.  It is yet to be seen if all possible effects have been eliminated.  

All of this being said, transfusions, chelation - long term anemia all have side effects and for some people very detrimental effects - so our hope is that the side effects of the gene terapy will be very minimal compared to a lifetime of thalassemia - or the dangers of bone marrow transplant.

Let's all pray that these trials begin soon and that the results are ones we are hoping for.  Gene therapy, and in particular Dr Sadalain's protocol is the biggest and best chance thalassemia and sickle cell anemia patients have for a cure. 

Sharmin

Canadian_Family:
Great News, I am happy for all of the thals.

Andy,

Do you know, once trials begin how long before we know the results....

Parin:
All the Best Dr. Sadelain and hoping soon we will receive a good news from you.

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