Thalassemia Patients and Friends

Discussion Forums => Announcements => Topic started by: Pratik on December 15, 2014, 11:27:38 PM



Title: New Gene Therapy Trial Results
Post by: Pratik on December 15, 2014, 11:27:38 PM
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:

(http://i.imgur.com/DkwUZuO.png)

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

Best,

-P


Title: Re: New Gene Therapy Trial Results
Post by: Himanshu Kumar on January 01, 2015, 11:10:48 AM
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/


Title: Re: New Gene Therapy Trial Results
Post by: Himanshu Kumar on January 01, 2015, 11:25:38 AM
Another story on Gene Replacement Therapy

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


Title: Re: New Gene Therapy Trial Results
Post by: Andy on January 01, 2015, 12:28:03 PM
Nice to finally see a report from an actual gene therapy patient. Wishing the best for Wanda.



Title: Re: New Gene Therapy Trial Results
Post by: Andy on January 01, 2015, 12:29:56 PM
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.


Title: Re: New Gene Therapy Trial Results
Post by: Himanshu Kumar on January 02, 2015, 11:01:53 AM
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.


Title: Re: New Gene Therapy Trial Results
Post by: jay on January 02, 2015, 05:16:51 PM
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.


Title: Re: New Gene Therapy Trial Results
Post by: Pratik on January 06, 2015, 01:28:18 PM
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


Title: Re: New Gene Therapy Trial Results
Post by: Parin on January 07, 2015, 01:48:09 AM
Thank you for sharing the updates...its makes happy everyone.


Title: Re: New Gene Therapy Trial Results
Post by: nice friend on January 23, 2015, 06:36:20 AM
its a  ray of hope for older thals ... once again feeling hopefull for a bright future ... 


Title: Re: New Gene Therapy Trial Results
Post by: Sharmin on January 25, 2015, 05:34:34 PM
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


Title: Re: New Gene Therapy Trial Results
Post by: nice friend on January 30, 2015, 05:02:13 AM
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 :) ..


Title: Re: New Gene Therapy Trial Results
Post by: Andy on January 30, 2015, 11:49:44 AM
 :thumbsup Umair.


Title: Re: New Gene Therapy Trial Results
Post by: nice friend on January 31, 2015, 05:28:08 AM
thanxx Andy :) u always been a big source of motivation and encrougement fo me :)  .. .


Title: Re: New Gene Therapy Trial Results
Post by: Pratik on February 05, 2015, 12:52:03 AM
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


Title: Re: New Gene Therapy Trial Results
Post by: nice friend on February 10, 2015, 09:29:23 AM
Pratik,
i will try my level's best to never disapear to here again and to stay in-touch always because this forum is my 2nd home and 1st treatment center  and the bouqet of sweet memories .... :) ...


Umair