Discussion Forums > Working Towards a Cure
Gene Transfer Therapy Stage 1 Clinical Trials at Sloan Kettering
Sharmin:
I agree Canadian_Family, it is great to see the promising and rather ground breaking results bluebird bio posted - but I am also praying for Sloan Kettering's slow and steady progress to yield safe and great results by next year. If the myelosuppression method yields comparable results it would be a better choice for most.
Sharmin
Pratik:
Thanks for the updates. Indeed, it is much preferred to get a concrete information rather than a bit of a hit and miss.
Till then all my fellow thals (including me, definitely), supercharge your bodies and keep chelating your iron and being healthy! :)
Best,
-P
Andy Battaglia:
I heard from Pat G and he had this to say about the current status of gene therapy trials.
--- Quote ---The thal family has been investing in gene therapy for almost 2 decades. Forces have been joined from around the globe all pushing in their way towards a cure.
Til today 3 Beta thals have been treated. They were treated at Mem Sloan Kettering by dr boulad using the sadelain vector. The myelosuppressive pre treatment was easily tolerated. Their ages were from 20-40.
The best result has been in patient number 2 reducing transfusions and all that goes with them by 50%.
This is truly wonderful and if this is the end result, it goes without saying that it is a therapy worth using.
Bluebird has treated 2 E thals from Thailand using myeloablation.
Ski used 8mg/kg and will fortify the dosage of bulsufan on the next 3.
We salute all of the people working so hard to bring this therapy home.
--- End quote ---
Sharmin:
Andy,
Thank you for posting. I hope that as they learn - outcomes will get better and better.
Canadian_Family:
Thanks for the update Andy. I was away on vacation at Disney World creating magic.....
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