• Welcome, Guest. Please login or register.
    July 03, 2020, 07:34:03 AM

  • Login with username, password and session length

Sajid's dove

Tell everyone they can now find this site by typing this into their browser:

thalpal.com

Click to visit us on Facebook


If you have any problems registering or signing in, please send an email to: andythalpal@yahoo.com
Please do not send questions about thalassemia to this address.


Administrators
Andy
Danielle

Thalassemia Patients and Friends and thalpal © A. Battaglia 2019





55311 Posts in 5912 Topics by 6212 Members
Latest Member: Pocatju

Forum Tip: 
You can now simply type thalpal.com into your browser and it will take you to this site. Tell your friends about this easy-to-remember name.
www.thalpal.com
« previous next »
Pages: « 1 2 3 » Go Down Print
Author Topic: Thalagen™: Gene Therapy Treatment for Thalassemia to Begin Trials in 2008  (Read 29646 times)
Sharmin
Global Moderator
Supreme Member
*****
Offline Offline

Location: Canada

Gender: Female
Posts: 4155


Little A


« Reply #15 on: January 26, 2009, 10:25:45 AM »

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
Logged

Sharmin
Zaini
Global Moderator
Supreme Member
*****
Offline Offline

Location: Karachi,Pakistan

Gender: Female
Posts: 3448


Life is too short to be perfect.


« Reply #16 on: January 26, 2009, 11:37:27 AM »

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  .

Zaini.
Logged

^*^Xaini^*^
nice friend
Thalassemia Major
Greeter
Supreme Member
*
Offline Offline

Location: Faisalabad, Pakistan.

Gender: Male
Posts: 2834


If I Can, Why Not You??... If I Can U TOO !!!...


« Reply #17 on: January 26, 2009, 03:34:07 PM »

  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
Logged

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
Manal
Global Moderator
Supreme Member
*****
Offline Offline

Location: Cairo,Egypt

Gender: Female
Posts: 3100

mother of thal intermedia child


« Reply #18 on: January 27, 2009, 07:40:28 PM »

Thanks a million

Any news when the first phase will start??

manal
Logged
Andy Battaglia
Administrator
Supreme Member
*****
Offline Offline

Location: In my heart, Maldives

Gender: Male
Posts: 8661


Will thal rule you or will you rule thal?


« Reply #19 on: January 27, 2009, 07:42:23 PM »

That will be announced at the Symposium.
Logged

Andy

All we are saying is give thals a chance.
Sharmin
Global Moderator
Supreme Member
*****
Offline Offline

Location: Canada

Gender: Female
Posts: 4155


Little A


« Reply #20 on: February 04, 2009, 04:04:12 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

Logged

Sharmin
nice friend
Thalassemia Major
Greeter
Supreme Member
*
Offline Offline

Location: Faisalabad, Pakistan.

Gender: Male
Posts: 2834


If I Can, Why Not You??... If I Can U TOO !!!...


« Reply #21 on: February 04, 2009, 04:12:23 PM »

Thank You Andy for being there for us >>>>>

Umair
Logged

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
Manal
Global Moderator
Supreme Member
*****
Offline Offline

Location: Cairo,Egypt

Gender: Female
Posts: 3100

mother of thal intermedia child


« Reply #22 on: February 04, 2009, 05:00:08 PM »

Best of luck Andy
manal
Logged
Sharmin
Global Moderator
Supreme Member
*****
Offline Offline

Location: Canada

Gender: Female
Posts: 4155


Little A


« Reply #23 on: February 04, 2009, 10:21:42 PM »

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 04, 2009, 10:27:06 PM by Sharmin » Logged

Sharmin
Zaini
Global Moderator
Supreme Member
*****
Offline Offline

Location: Karachi,Pakistan

Gender: Female
Posts: 3448


Life is too short to be perfect.


« Reply #24 on: February 05, 2009, 02:59:22 AM »



                       

Zaini.
Logged

^*^Xaini^*^
Sharmin
Global Moderator
Supreme Member
*****
Offline Offline

Location: Canada

Gender: Female
Posts: 4155


Little A


« Reply #25 on: February 05, 2009, 12:18:59 PM »

Andy,

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

Sharmin

Logged

Sharmin
Zaini
Global Moderator
Supreme Member
*****
Offline Offline

Location: Karachi,Pakistan

Gender: Female
Posts: 3448


Life is too short to be perfect.


« Reply #26 on: February 05, 2009, 09:20:26 PM »

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.
Logged

^*^Xaini^*^
Andy Battaglia
Administrator
Supreme Member
*****
Offline Offline

Location: In my heart, Maldives

Gender: Male
Posts: 8661


Will thal rule you or will you rule thal?


« Reply #27 on: February 06, 2009, 01:13:18 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.
Logged

Andy

All we are saying is give thals a chance.
Zaini
Global Moderator
Supreme Member
*****
Offline Offline

Location: Karachi,Pakistan

Gender: Female
Posts: 3448


Life is too short to be perfect.


« Reply #28 on: February 06, 2009, 01:36:29 AM »

Andy,

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

Quote
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  .

Zaini.

Logged

^*^Xaini^*^
Manal
Global Moderator
Supreme Member
*****
Offline Offline

Location: Cairo,Egypt

Gender: Female
Posts: 3100

mother of thal intermedia child


« Reply #29 on: February 06, 2009, 04:48:04 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.

Quote
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.

Quote
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, 05:42:06 AM by Manal » Logged
Pages: « 1 2 3 » Go Up Print 
« previous next »
Jump to:  

Powered by MySQL Powered by PHP Powered by SMF 1.1.21 | SMF © 2015, Simple Machines Valid XHTML 1.0! Valid CSS!