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Will Gene Therapy be the Cure for Thalassemia?

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Andy Battaglia:
At the recent TIF conference at Dubai, Dr Michael Sadelain spoke about his work using gene therapy towards finding a cure for thalassemia. His research has the ambitious goal of curing both thalassemia and sickle cell anemia. Some of us managed to get a chance to talk to Dr. Sadelain after his talk. He told us that trials in humans are expected to begin later this year in what he hoped would be three locations around the world. Financing is an issue, but this research holds so much promise, that there is hope that new sources of funding will arise once they become convinced of the potential of this work.

For more information about Dr. Sadelain's work, go to
http://www.asgt.org/member_resources/news/blooddiseases1205.html

This is exciting research and we will be watching as the trials commence.

Andy

Poirot:
Andy,

Do you know which locations?

And, how does one get to apply?

Thanks

Poirot

Danielle:
I want to be so excited about this, but I'm refraining just in case.  The trials are supposedly set for this summer, and I'm on the edge of my seat already.  I can't wait to hear how it goes.  Do you know if Dr. Sadelain is going to be using any Thal patients also, or just using Sickle Cell patients?  I was figuring he'd be more apt to use Sickle patients, since there are many more of them.   :dunno

I would have loved to meet Dr. Sadelain.  I hope to meet him in the future.  Was he easy to talk to, Andy?  I'm so grateful for his work and dedication.  I truly hope that something good comes out of this.  I would love for all of us to be cured some day.  That would truly be a wonderful, wonderful day ... and life after.   :cloud9

Andy Battaglia:
Poirot,

He said that he hoped trials would start in the US and Europe, possibly France, and then a third site, possibily in the mideast if funding became available. I will be trying to find out more about where and how they will be selecting patients.

Andy Battaglia:
Danielle,

Dr Sadelain was probably one of the easiest doctors to talk to that we found in Dubai. He was very friendly and very happy to answer all our questions. He is very excited about the trials and I do believe he does plan on using thals in the trials. He did say that those in better shape would be the best candidates for the trials. I think if you meet him you'll find he comes off as a pretty nice guy.  :smile2

This is exciting but we have to realize that it takes years of trials and study before anything reaches the approval stage, and that's why thals are always so eager to take part in trials, because it's the one way to get access to new treaments.

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