Thalagen™: Gene Therapy Treatment for Thalassemia to Begin Trials in 2008

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Offline Sharmin

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Pat,

Thank you for the update and thank you very much for everything that you are doing to help us.  We will all do everything that we can to help.  Please keep us posted and continue to let us know how we can contribute to this project.

Our warmest wishes to you and to Rocco,

Sharmin
Sharmin

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Offline Zaini

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Pat,

You can not imagine how your post has effected me positively,i am sorry i can't contribute financially,due to the huge difference of currency,but all my prayers are with you,give our love to Rocco  :hugfriend .

Zaini.
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Offline nice friend

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 :thankyou2 Pat ,
for wat you are doing for all the thals is greater than anything else .. i hope n wish that this project run successfuly in trials and it work to cure thals . you're doing a great job and i appreciate it to the bottom of my heart . i wish to hear the good news in soon future to you ...  my wishes and my prayers are with you .... May God Bless you with sucess in finding a cure for thals ....  lots of love ad lots of prayers for you  nad your son Rocco ...

Best Regards
Take Care
Umair
Sometimes , God breaks our spirit to save our soul.
Sometimes , He breaks our heart to make us whole.
Sometimes , He sends us pain so we can be stronger.
Sometimes , He sends us failure so we can be humble.
Sometimes , He sends us illness so we can take better care of our selves.
Sometimes , He takes everything away from us so we can learn the value of everything we have.

===========
Umair

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Offline Manal

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Thanks a million :hugfriend

Any news when the first phase will start??

manal

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Offline Andy Battaglia

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That will be announced at the Symposium.
Andy

All we are saying is give thals a chance.

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Offline Sharmin

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Re: Thalagen™: Gene Therapy Treatment for Thalassemia to Begin Trials in 2008
« Reply #20 on: February 04, 2009, 10:04:12 PM »
Andy,

Have a good trip!  I feel very fortunate to know that you will be at the symposium tomorrow to hear first hand about the trials.  Thank you for being there for us Andy.


Sharmin

Sharmin

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Offline nice friend

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Re: Thalagen™: Gene Therapy Treatment for Thalassemia to Begin Trials in 2008
« Reply #21 on: February 04, 2009, 10:12:23 PM »
[bgcolor=#0000ff]Thank You Andy[/bgcolor][/glow] for being there for us >>>>> :)

Umair
Sometimes , God breaks our spirit to save our soul.
Sometimes , He breaks our heart to make us whole.
Sometimes , He sends us pain so we can be stronger.
Sometimes , He sends us failure so we can be humble.
Sometimes , He sends us illness so we can take better care of our selves.
Sometimes , He takes everything away from us so we can learn the value of everything we have.

===========
Umair

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Offline Manal

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Re: Thalagen™: Gene Therapy Treatment for Thalassemia to Begin Trials in 2008
« Reply #22 on: February 04, 2009, 11:00:08 PM »
Best of luck Andy :hugfriend
manal

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Offline Sharmin

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Re: Thalagen™: Gene Therapy Treatment for Thalassemia to Begin Trials in 2008
« Reply #23 on: February 05, 2009, 04:21:42 AM »
February 5th, 2009 is a special day for all thals.  I hope that this turns out to be what we all wish for, a safe and effective cure for thalassemia that cures all of our thal pals


Sharmin
« Last Edit: February 05, 2009, 04:27:06 AM by Sharmin »
Sharmin

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Offline Zaini

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Re: Thalagen™: Gene Therapy Treatment for Thalassemia to Begin Trials in 2008
« Reply #24 on: February 05, 2009, 08:59:22 AM »


                       

Zaini.
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Offline Sharmin

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Re: Thalagen™: Gene Therapy Treatment for Thalassemia to Begin Trials in 2008
« Reply #25 on: February 05, 2009, 06:18:59 PM »
Andy,

Our energy and thoughts are with you today.  I hope you receive lots of good news today! 

Sharmin

Sharmin

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Offline Zaini

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Re: Thalagen™: Gene Therapy Treatment for Thalassemia to Begin Trials in 2008
« Reply #26 on: February 06, 2009, 03:20:26 AM »
There is a big time difference between here and US or Canada,it's already 6th feb here,it's hard to wait for any news :) .

Zaini.
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Offline Andy Battaglia

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Re: Thalagen™: Gene Therapy Treatment for Thalassemia to Begin Trials in 2008
« Reply #27 on: February 06, 2009, 07:13:18 AM »
Just a quick update. I got back home at midnight and am exhausted from walking in the cold. I also want to review my notes and the photos of the slide presentations before going into more depth. There was a lot more to this than gene therapy, so I will have to post some updates on chelation (L1 still can't be beat), BMT and also try to give some idea of the scope of what is involved in getting to this point with gene therapy. Everyone needs to remember that this research has been going on for years and that we cannot expect anything soon in terms of general use in patients.

The final information has been filed with the FDA. If they do not hear back from the FDA within 60 days, they are free to proceed. The important things have all already been approved by the FDA and this should be routine. The first vector has been ordered and is expected in June. It then has to be tested for 60 days (if my foggy late night memory is correct) and the hopes are that the first patient will be started sometime around September/October. If hemoglobin is expressed by the new genes, more patients will be added to the trial, with a goal of ten patients. Interestingly, no patient with an HLA matching sibling will be allowed in the trial, as bone marrow transplants are already a proven cure, although this varies greatly depending on where it is done. Sloan Kettering does do some BMT's, and while it is not a large number compared to other centers, all 16 patients who have had BMT's at Sloan Kettering since 1995 are still doing well today, making their own blood and cured of thalassemia. So, BMT is considered the first option, disallowing any patient with a matching sibling from these trials. This will be exciting to watch unfold. The one drawback of the gene therapy trials in mice was that mice don't have long lives, so the long term effect on hemoglobin production could not be measured. However, the mice did live normal length lives after the gene therapy and were able to produce their own hemoglobin for the duration of their lives.

I was cautioned by several people that we should be realistic and not expect anything for real world use too soon. But we need to remind ourselves how many years it has taken to develop new iron chelators. Exjade was in the works back in the 1980's. Starch DFO has been in development for 20 years and I hope they can secure funding, because in some ways it is better than any other chelator. New drugs and new therapies take many years to develop  ad long term efficacy and safety will have to be proven.

One observation. A large part of the focus today was on convincing investors that this will pay off. I did get a hint from a couple different potential investors that they are seriously considering putting money into Errant Gene. If this comes through, this may be as important as the announcement that trials will be happening this year. I wish Pat G much success in getting the funding to make this all happen. It was a pleasure to finally meet him.
Andy

All we are saying is give thals a chance.

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Offline Zaini

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Re: Thalagen™: Gene Therapy Treatment for Thalassemia to Begin Trials in 2008
« Reply #28 on: February 06, 2009, 07:36:29 AM »
Andy,

Thanks a lot for the update  :thankyou2 .Every new invention takes time,but when gene therapy will be available after successful trials,it will be worth waiting.

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One observation. A large part of the focus today was on convincing investors that this will pay off. I did get a hint from a couple different potential investors that they are seriously considering putting money into Errant Gene.

This indeed is a good news,I hope they'll find the required funding,mean while we can keep praying  :pray :pray :pray.

Zaini.

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Offline Manal

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Re: Thalagen™: Gene Therapy Treatment for Thalassemia to Begin Trials in 2008
« Reply #29 on: February 06, 2009, 10:48:04 AM »
Many thanks Andy and i wish you had a good WARM sleep after the hectic day. I hope that they will really start at September and have nothing to delay them anymore.

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Interestingly, no patient with an HLA matching sibling will be allowed in the trial, as bone marrow transplants are already a proven cure, although this varies greatly depending on where it is done.

hope this is only in the trails because the side effects (GVHD) have hindered many families to go through BMT even when they have the HLA match, so having another cure is their only hope.

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I was cautioned by several people that we should be realistic and not expect anything for real world use too soon. But we need to remind ourselves how many years it has taken to develop new iron chelators

What i know is that researches in gene therapy had started in the 70s, isn't this a long time to see a cure, sometimes i am afraid those scientist who work on the cure would die before it is available and we have to start all over again. Definetly i hope for them all the best and luck and for time to pass as quick as possible

Thanks again Andy
manal
« Last Edit: February 06, 2009, 11:42:06 AM by Manal »

 

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