Will Gene Therapy be the Cure for Thalassemia?

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

Andy,

Do you know which locations?

And, how does one get to apply?

Thanks

Poirot

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.   

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.   

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.

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.

Wow, Andy.  I'm so glad to hear that Dr. Sadelain was so friendly.  You don't hear much of doctors being *friendly* and *easy to talk to* anymore.   

Now I'd really love to meet him.   

I know that the trials are going to be a long journey, so that is why I'm not trying to get so excited about it.  I'd like to know what the trials entail, though ... and how *healthy* you need to be.  I wonder what the criteria is.   

Thanks, Andy, for the replies.

That is really good to know. Maybe we can lure him to do a trial in India or Singapore as well.

Is there any easy way to contact him? Or do you reckon that he will consider that intrusive?

Yeah, Danielle, wouldn't it really be great if this line of research leads to something "lasting"!!
Well fingers and toes crossed .....

Cheers

Yeah, Danielle, wouldn't it really be great if this line of research leads to something "lasting"!!
Well fingers and toes crossed .....

Yes, it would be the best thing ever!  A dream come true.   

What if the Modded HIV virus stays in our system, then all we have to do is to donate our Gene corrected HIV infested blood to the patients of the same blood group(type) and have them cured too?!?! Or do we get banned for life and not being able to donte blood to anyone (Even in Emergency!) as the virus might mess the genetic code.  :d'oh

The whole thing seems quite scary to me.   

I don't know how safe I'd feel with a manipulated HIV retrovirus running rampant inside my already messed up body.   

I hope it works, though.   

 

Hi Andy
Read Dr. Sadelain's research. Sounds promising. Thank you for the information. Also, do you know of anymore research going on with wheatgrass.?

Regards
Bharat

Thanks Andy.......................Nice piece of Information.

do you know of anymore research going on with wheatgrass.?

Bharat, I don't know if this will help, but this is the only site that I know of that's dedicated to wheatgrass that also has info about its effect on Thalassemia.

I hope this helps a bit. 

http://www.wheatgrassprofessional.info/

Dr. Chris (the owner of that site) was a member over at our MSN forum, so hopefully he will join here also and tell a little bit more about it.   

Guess it's time for me to email Chris.

Is it possible to donate directly to fund this trial?

If yes, Andy/Manal/Sharmin, do you have the link? I can try and talk to a few corporates here.

Poirot