The Latest Update from Pat G about Gene Therapy

This is Pat's latest update. This is very close to being in patients. I trust this group more than any other. Dr Sadelain's team has produced the most thorough research and development of the process. I believe they have taken steps to ensure good results and over the next 6 months, we will finally see these results.

Dear family,
It's pat girondi... We are close. The latest data looks promising. There are no guarantees though I'm confident as well as hopeful that we will see genetic expression of the beta globin gene in our first patients.
Please forgive me for the years of wait. I as well as you would like to have been quicker. I am not an expert at anything and can only say that I did my best to move things along.
Please join the Sadelain team and send them your warmest wishes, prayers and thoughts. The date that we begin is close...
Also for kicks go and hear the preview song of the next CD in the 'Orphan' Series... 'Orphan's http://www.youtube.com/watch?v=qL5z_v69RuYe'.
Love yas.
pat g

Every CD sold by Pat G helps to pay for this work. Go to cdbaby.com and search for Pat G.

Good news but when they will start gene therapy in first patients .....?
gene therapy is tahking long time for cure thal patients .....?

The trials in real patients will commence very shortly. The latest target was September-October. We shall see how things proceed.

Dear Andy
Whe I come to my office in the mornig and open up this site during any opportunity, I always feel very excited of geting some news somewhere. But not finding any  BREAKTHROUGH news , I feel very sorry and pack up and close this site for yet another morning.
I am sure, some day sometime this i bound to come and all of us will be delghted and perhaps this site go further to discuss how all of us are feeling. Me as a father  of a Thal child ,for whom I always feel guilty. The maximum proce i paid for my ignorance.
Love you all.

Andy,
if this is successful, when will the gene therapy be available to us?

No one can give a time line for when to expect this to happen. Long term observation of the patients will be required and more extensive trials done before it would be readily available. However, as with all new drugs and procedures, some patients will be able to take advantage of trials. Healthy patients will get preference in all trials, so it is an incentive to take care of the health now.

Thanks Andy for sharing a great news....

We all are waiting for that day and i hope GOD with us...and soon we will achive.......

Well where these trials are going on? is it the trail conducted all over the world or in US only?

Thanks

They are hoping to eventually run trials in at least 3 or 4 countries. I know that India is under consideration, and I believe Greece will also be, because some of the early work has been done in Greece. Lena has previously mentioned this team and they are working together with Dr Sadelain's team.

Great ,pat have say that after 2 years gene therapy will Be  in India

Thanks for sharing Andy.  Pat, thank you for all of your hard work.  You are in the same boat as us - but you have made such a significant difference.  Your efforts will hopefully change all of our lives for the better. 

Best,

Sharmin

Great! Its my gut feeling InshaAllah(if God wills it )  that this will be successful way of treatment in an year's time for thalassemics.

i hope and pray that InshaAllah it is successful!
ith todays therapies and awareness how old do you think a person with major thalassemia lives?
i know its not a nice question to ask but i'm hoping that it's the same as a person with out it

IN nowdays the person with thalassemia can live long life but they most control ther iron level . i belive that the gene therpy will be successful tretment for thalassemia . im prying for my brother day and night becoz i love my borther so much .

With a good treatment program, thals are now living into their 50's and beyond. This is the first generation with an opportunity to live a long life, so we really don't know what to expect. The younger patients have many advantages in care that the older patients did not have when they were young, so life expectancy for thals is expected to continue to increase.

Andy are there any more gene therapy clinical trials underway?
any news from Pat what day the gene therapy trial will begin?
50 years and beyond is close to the age range for people with out major thal too, isnt it?