Major Announcement Concerning Gene Therapy

I got an email today from Pat Girondi. This is BIG.

"Approved to start clinical trials by FDA!!!!!"

Green light for gene therapy trials in the US! That's the way to celebrate a holiday!

Andy,

Thank you so much for sharing!!  We've been awaiting this announcement for so long!!
Praying now for success in the trials and ultimately a cure for all thalassemia and sickle cell patients!

Sharmin

thank god, some progress is there. I pray that trails are successful and all the patients are cured. God bless all

Hi Andy

Thanks for the good news, but if you do not mind please elaborate on exactly what is Gene Therapy. How is Thal major cured and the effects whether positive or negative. Is Gene Therapy the same as BMT.

Apologies for blasting you with all of the above as i feel we need to know more and you are brilliant

Regards
Mohammed

Hi Andy,

That's fantastic news.

Mohammed,

Look at this reply w.r.t which Andy replied me:

http://www.thalassemiapatientsandfriends.com/index.php/topic,4046.msg43632.html#msg43632

And Andy, I too need even some more info, the positive and negative side effects, like Mohammed asked is it like BMT or just a simple injection like injecting the blood cells and all? What are maybe the risk for dying? Any specific age till it can show good results? (like in BMT 3-8 years phenomena is best, etc).

Thanks.

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.

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.

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

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

Andy,

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

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

I don't want to speculate. There have been so many non-science reasons for delays already, that it is hard to predict what else may arise.