Gene Therapy Update From The Philadelphia Conference.

Dr Michel Sadelain was a surprise speaker at the Philly conference, and as always, it as a pleasure to meet him. David Davidson from Bluebird also spoke on his company's upcoming trials and future endeavors. I must admit, I was impressed by both speakers.

I want to give an update on something we have all been waiting for. The future for gene therapy is looking more promising than ever. The French patient is still well and transfusion independent. The trials currently being run at Sloan Kettering are still very early in the going, but the results are outpacing those of the French patient at this point in the trial, even though a low intensity chemo program was utilized. It was expected that the results would come at a slower pace than what has been seen so far. It is early on, and we should remember that results for the French patient were not released until 3 years into the trial.
I will also mention that the other company that will be running trials, Bluebird, will be recruiting patients internationally and will be paying expenses to the US for the trial. I was impressed by both gene therapy presentations. Over the next 25 years, we will be seeing a revolution in the treatment of genetic disorders.

It's A WOOOOOOOOOOW

But 25 yrs is not much long??

I am expecting it to be 10 yrs

I just fail to understand that if the french patient is doin well after 3 years, what is the harm of calling it a success. S/ He must be undergoing various test and is that not enough to understand his condition.
Also, one more thought cramps in my mind that instead of different companies working in isolated manner, can there not be a synchronized effeort t make this success. We are ar certain strage and many experimentation has been done of diff paatients. Its the matter of consolidating the journey and proceeed from there.
Ha. I feel the US governemnt has many other jobs and may be it is just another problem for them.
We as parnet and patients are waiting and waiting. may be another 10-15 more years.

I dont know how i should react, but to wait for this treatment for another 25 years is not what i was expecting. Anyhow we do not have any other option as this is the only ray of hope.

Regards
Sushil Thakur

I think Andy was saying 25 years for the treatment of OTHER genetic disorders not thal

JV you are correct. The time frame for gene therapy issued by CAF is on a ten year plan. JV, I wish you could have been at Philly this weekend. I got to meet Amy C who knows you.

I know, very upset about missing the conference but had family stuff. Amy C and Anthony Viola said so many great things about the weekend. The North Jersey Chapter, along with New York and Long Island are very active and we hope to see you at some of our future events.

It is truly amazing....we are on the cusp of history...we have the opportunity to wipe out a major disorder (don't call it a disease anymore) in the very near future. This WILL happen....

I felt honored when Tony Viola thanked me and mentioned my work with thalpal to the audience. It was unexpected but greatly appreciated. For me personally, it was wonderful to see the acceptance given me by the people at CAF. And it paid off imediately, as doctors were quite happy to talk to me at length about the problems of some of our members. It isn't for myself that I want the recognition. It is because it means more access to the people that can help the most.

10 Year is fine to me. My daughter is 4 year old so I am happy that her youth age will be thal free  . Touchwood knock knock.

I felt honored when Tony Viola thanked me and mentioned my work with thalpal to the audience. It was unexpected but greatly appreciated. For me personally, it was wonderful to see the acceptance given me by the people at CAF. And it paid off imediately, as doctors were quite happy to talk to me at length about the problems of some of our members. It isn't for myself that I want the recognition. It is because it means more access to the people that can help the most.

Congratulationsss Dear Andy

10 Year is fine to me. My daughter is 4 year old so I am happy that her youth age will be thal free  . Touchwood knock knock.

Cheers and a big congratulations to you Andy.

So the trials on the French patient was running since 3 years? I thought the trials just started recently though?

10 years are sort of big too. I hope in 5 years or so, we will see a working cure for thal. Not that I'm impatient, but it feels to great to live without any disorder or I should ask "How it feels to live without any problem", without any transfusion, daily 2 doses of chelators, basically the change in whole day-to-day routine. Haha.

-P.

MashaAllah (what God willed (for))! Andy......THANK YOU

From the data reviewed at the conference, I would say that the full effect is not seen for at least 2-3 years as the gains in Hb occur over a long period. I think that after two years, we will have an excellent idea if it works. The key for patients is to stay in good shape so they can benefit from this advancement.

 My husband and I unfortunately could no make this, Thank you so much for the update!!! =)