New Gene Therapy Trial Results

Hey friends,

I have been away for a while but I couldn't help but to step in and announce what I read yesterday in CAF's (Cooley's Anemia Foundation) newsletter.

There has been some exciting and heart soothing results in regards to the gene therapy trials currently undergoing at Sloan Kettering.

Here is what it said:



This has made me very optimistic and let us hope that we have a fully functional treatment available soon!

Best,

-P

Read this on my facebook wall. This is a story of 33 year old patient who successfully received bone marrow transplant.

http://josephinebila.com/2014/12/bone-marrow-transplant/

Another story on Gene Replacement Therapy

http://wgntv.com/2014/12/30/gene-replacement-could-replace-need-for-life-long-blood-transfusions/

Nice to finally see a report from an actual gene therapy patient. Wishing the best for Wanda.

The person who received the BMT and was interviewed by Josephine is David Levy, a long time member of thalpal and founder of the thalassemia support group on Facebook, which I also administer. It is so exciting to see an older patient cured.

Absolutely, it is so heartening to see older patients getting cured. Gives hope to many patients. Great to hear about successful cases on Gene Therapy. If the early results are so encouraging, why aren't they recruiting many more patients. I sincerely hope the treatment is made available across the world at a low cost.

Himanshu,
We have clinical trials -> FDA regulatory process ->Price skimming
and then we can see it near to us ... loooong way to come to gene therapy.
Regarding recruiting more patients: clinical trials are normally the most costly affair in the process.
By the way Himanshu not to confuse with David Levy's BMT with gene therapy, he gone through a unrelated full match BMT, not gene therapy.
Regarding low price BMT, assuming you are from India let me tell you if you have complete HLA related match (not just siblings it is also highly possible to find a related donor in cousins) then it could be done in five lac (RS 5,00,000).
Regarding BMT in older patients it has been there for a while we are excited here as he is one of us we talk.

Another story on Gene Replacement Therapy

http://wgntv.com/2014/12/30/gene-replacement-could-replace-need-for-life-long-blood-transfusions/

I just came to post this and saw you posted it. Great!

I just received newsletter from CAF which contained link to it and thought on sharing here.

Yes, its amazing to see the testaments of new treatment. Couldn't be more excited for all of us!

Best,

-P

Thank you for sharing the updates...its makes happy everyone.

its a  ray of hope for older thals ... once again feeling hopefull for a bright future ... 

You have every reason to be hopeful Umair.  You have come a long way with chelation etc.  Continue to take good care of yourself so that you can take advantage of the cure when it becomes widely available.

Sharmin

sharmin ,
i m trying my level's best to keep everything in normal range and fine and its all wat i can do ...
but sometimes you dont know when n why/how something wrong happen's  to you and takes you off track again....   :/

anyways still am not gonna loose hope ,, i will keep trying my levels best to keep everything in normal ranges ..

  Umair.

thanxx Andy u always been a big source of motivation and encrougement fo me  .. .

Umair,

It is so good to see you after long! I feel members should keep turning in at least every few months so we can know what's going on with everyone's lives and how well they are doing.

Keep it up, my friend!

Best,

-P