Thalassemia Patients and Friends

Discussion Forums => Working Towards a Cure => Topic started by: Andy Battaglia on June 18, 2011, 12:48:43 AM



Title: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on June 18, 2011, 12:48:43 AM
After many delays, it appears that an agreement between Errant Gene and Sloan Kettering has finally been worked out. Dr Sadelain's trials should begin this year for certain. Stay tuned for more details as they develop.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on June 18, 2011, 01:26:04 AM
Andy,

Thanks for posting this!  I am so glad!  I can't wait to hear more!

Sharmin


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Parin on June 18, 2011, 12:08:33 PM
Thanks Andy for sharing this news...It good news to all.

so do you think by EOD year it will visible to world??

Only my request to you, that please sharing any information releated to trails immedietly......and my advance Thanks for that.


Regards,
AshutoshT


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on June 18, 2011, 07:49:34 PM
Once an agreement is finalized and signed, I will make an announcement. After that, the start of the trials will be completely up to Dr Sadelain and his extraordinarily meticulous and precise methods. It can be frustrating, but the man will not proceed until he is sure every step has been fully investigated and proven. Please remember that his stem cell mobilization produced stem cells at a far higher rate than predicted, so we must allow the doctor to proceed at his own pace and bring us the very best possible results. What I have been told is that this will be in patients by November at the latest. From past experience, I will choose not to hold my breath waiting but I have far more confidence that this will soon begin than I have had in quite some time.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Zaini on June 19, 2011, 12:54:58 PM
 :biggrin good luck to all of us.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Parin on June 19, 2011, 02:37:05 PM
Thanks Andy for sharing the valuable information...you are great.....

My all wishes with Dr Sadelain  and team.

Thanks again and keep updating.

Regards,

Ashutosh T


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Parin on June 21, 2011, 05:08:43 AM
e-mail from Pat


Dear family member,

I know that it has been a while. Michel Sadelain and his staff including EGT and their staff have been working feverishly to proceed on the Thalagen project. Things are moving forward.

I predict that the first patients will be treated with Thalagen by the end of fall.

We all are happily excited. Please keep the prayers and good wishes coming.
 
Pat girondi


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Emby on June 21, 2011, 10:15:44 AM
I guess I'm a really emotional person but I cried when I read this...heres hoping InshaAllah-God Willing.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on June 25, 2011, 01:51:58 AM
The agreement to turn the gene therapy cure for beta thalassemia over to Sloan Kettering, under the supervision of Dr Michel Sadelain, has been finalized and signed. We can expect trials in humans to begin this year. I have complete confidence in Dr Sadelain and his ability to perfect this cure using the true beta globin gene.

On behalf of everyone involved in the world of thalassemia, I want to thank Pat Girondi for the 19 years of work that have brought us to this point. Without Pat's tireless efforts to raise money and support for this incredible endeavor, we would not be where we are today. Pat, from the bottom of our hearts we thank you for everything. You are what I call a "real person" and I am thankful for your friendship and reminders that the road is never easy. As I sit here listening to your song "Lisa" a tear comes to my eye as I think of all those who have come before, are here now and are yet to come and how we each in our own ways have given so much to give some hope to those who need it most, and that we are about to cross into a new frontier, and the tears of sadness over those we have lost turn to tears of joy with the realization just how important this is.

For Rocco, who knows how much his dad has given of himself to try to give his son and every thal on earth a better life, and for everyone who has ever been touched by thalassemia, I salute you, Pat g. And for my little sis

"Lisa forever and ever you."


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on June 25, 2011, 06:33:07 AM
Indeed this is the news we have all been hoping for! I pray that this turns out to be everything that we have been praying for so that millions may benefit from it.   Thank you Andy for posting this, for always speaking up for us and for keeping us updated. 

Pat, I don't have the words to thank you for all that you have done.  May this treatment cure Rocco and bring relief to all of the families who await this cure.  Pat thank you for your tireless efforts. 

Lisa, I wish you were here to benefit from this - but I know that it would make you very happy to know that the future for thal patients is getting brighter everyday.  Thank you for bringing us together so that we can share all of this information and make informed choices for ourselves and our loved ones. 


Sharmin


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: PositiveVibes on July 18, 2011, 01:44:18 PM
A ray of sun !!! :)))) Thank you all for your constant contribution to make thalassemics' life easier.
 
Thank you Andy for presenting such good news !!!

Wish u all luck and can't wait to hear more information !  :veil


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Canadian_Family on October 27, 2011, 06:05:05 PM
Hello Friends,

Not sure if anyone knows any latest news. I am hoping Andy may have some update. I can't wait to see if trials have began.



Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on October 27, 2011, 10:25:43 PM
I expect an announcement on trials beginning very soon.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on October 28, 2011, 12:04:08 AM
Andy, Canadian Family,

I can't wait!!  We all have the same wish - I hope that our prayers will be heard soon,

Sharmin


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Parin on December 05, 2011, 04:42:50 PM
dear all,Andy

Is there any news about the Trials??



Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: joyce on December 13, 2011, 07:16:50 AM
Still waiting for the update news too!!



Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: sagar1 on December 13, 2011, 09:45:39 AM
tired of hearing from so many years, treatment is coming, trials are coming and finally nothing happens ? It is very sad to hear all this. When will the real picture come in front of us.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: joyce on December 13, 2011, 12:10:52 PM
Don't lose hope Sagar.. maybe the time is not coming yet, maybe God put it in delay just to give us the very best later..

Have faith..


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: sagar1 on February 07, 2012, 11:07:49 AM
any update ?


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: sagar1 on February 15, 2012, 04:15:10 AM
bluebird bio to Present at the Leerink Swann 2012 Global Healthcare Conference
February 09, 2012 08:03 AM Eastern Time
CAMBRIDGE, Mass.--(EON: Enhanced Online News)--bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that Nick Leschly, chief executive officer, is scheduled to present at the Leerink Swann 2012 Global Healthcare Conference on Wednesday, February 15, 2012 at 2:15PM EST at the Waldorf Astoria Hotel in New York City.

Mr. Leschly is expected to discuss the company’s vision for developing gene therapies for genetic diseases with high unmet medical needs, including beta-thalassemia, sickle cell disease and childhood cerebral adrenoleukodystrophy (CCALD). The presentation will include updates on the launch of the company’s pivotal CCALD clinical trial, as well as patient data updates and enrollment of the ongoing beta-thalassemia/sickle cell disease trial.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on February 15, 2012, 11:01:45 PM
there may be an update for errant gene next month as well. 


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Canadian_Family on May 01, 2012, 11:37:38 PM
This is a latest reply from Mr. Pat Girondi

Dear Family Member

We just sent the IND to the FDA.
 
We have answered all of there queries and hope that we can begin quickly.
 
My best to you and your family,
 
Pat girondi

Thanks Pat, you are a hero.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on May 02, 2012, 12:31:39 AM
Thank you Canadian Family.  Praying for us all...

Sharmin


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Romy Dese on May 15, 2012, 02:00:31 AM
Dear All,

is it possible to make it happen world wide, because I came from Indonesia.

Thank you.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Parin on June 16, 2012, 04:56:38 PM
Dear All,

Any updates from Pat?? or FDA???





Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on June 17, 2012, 08:09:43 PM
There was supposed to be a response from the FDA this past week but still no word. Hopefully, we will hear something this week.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on June 18, 2012, 02:10:41 AM
Hoping and praying for a positive response so that the trials can commence...


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Pratik on June 19, 2012, 06:42:20 PM
Hi,

Can anyone shed some light on what exactly this is and how would it be beneficiary to us, Majors? Thanks.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on June 19, 2012, 11:48:28 PM
In gene therapy, normal beta globin genes are introduced into the body, so that normal beta globin can be produced. The goal is an Hb of at least 10, and it is believed that Dr Sadelain's process can do this. This would free the patient from transfusions.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Pratik on June 20, 2012, 06:06:20 AM
Brilliant. Please let us know the updates, whenever you have them, Andy. :)

Pratik.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: ANI on July 17, 2012, 03:56:59 PM
Dear All
Afetr many many days got a mail from Pat Girondi saying that for the  FDA  approved "Thalgen" , they have started recruiting the patient between 15 to 50 for this. I am attaching the file with the notification of clinical trial website.
Pl have a look and pray for a  brighter tomorrow.
Rgds


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on July 17, 2012, 07:38:53 PM
Fantastic news! I have posted it on our Facebook groups.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Canadian_Family on July 17, 2012, 08:18:43 PM
Great News, I pray for all the patients and parents......


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: JV on July 18, 2012, 12:08:30 AM
Hi...can somebody give me some info on Thalgen?


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on July 18, 2012, 01:46:01 AM
Thalagen is the name of patented process using the beta globin gene to cure thalassemia. If you search for thalagen on this site, you will find many past posts.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on July 19, 2012, 03:15:11 AM
In reply to some questions about this trial, I have received the following answerr from Dr Fouad. I am very grateful to him for taking the time to explain. Yes, if this trial works as hoped, patients will be cured.

Quote
I am very happy to present the following reply from Dr Fouad. This trial is for real. I will not hesitate to recommend it for those who qualify.

Hello Mr Battaglia

I actually got back to Ms Carbone this evening.

I think there is a little bit of a misunderstanding/miscommunication about yesterday's phone call.
So I will try to answer these concerns and questions.

What we DO KNOW from the laboratory data and from the first mobilization trial for thalassemia patients is that:
- Gene therapy can cure the animal model with beta thalassemia
- On the patients we mobilized - but not reinjected the cells to - we showed that:
- We CAN mobilize stem cells from patients with thalassemia successfully
enough stem cells to be used for this coming trial
- We showed that the cells mobilized and transduced with the vector, do express the gene
and do make the (previously missing) beta chain of the hemoglobin
- We know - from laboratory data and from gene therapy trials for other diseases
that stem cells transduced with the vector and infused WITHOUT chemotherapy do NOT work
and therefore chemotherapy is needed prior to the reinfusion of the transduced cells
- We do NOT know HOW MUCH chemotherapy is needed to do so.
and so we had two choices:
1. Give very high dose chemotherapy - like we use in the standard bone marrow transplant protocol
with a high risk of significant toxicity and, if it works, continue to take that risk
2. Give lower dose chemotherapy (approximately 1/4 of that used in the standard bone marrow transplant protocol)
with a very minimal risk of toxicity, and still a possibility that this is enough for the gene transfer to be successful.
We used the second option. AND, if it does not work, THEN increase the chemotherapy dose
BUT, if it works, then it is the perfect approach with successful gene transfer and minimal toxicity risks.
This was the safest and most ethical option for patients with thalassemia.

SO, in answer to your questions:
1) Will patients be able to expect any change in Hb level from this trial?
We certainly hope so. That IS the goal of this protocol.
We don't know what the percentage/chance of that to work, but THAT certainly IS OUR AIM

2) If not, what will induce patients to participate in light of the fact that they will have to undergo chemo.
See answer to question 1.

3) At the end of this trial, would the same patients be first in line for the cure?
The goal of this trial IS to cure the patients with minimal chemotherapy, successful gene transfer,
normalization of Hemoglobin, and discontinuation of transfusions.

As much as this has been the dream of many ...all patients with thalassemia,
it is also our dream and goal as their physicians.
We have been working very hard for many years to be able to open this trial
and the goal of this trial is to be able to have a successful gene therapy.

I hope this answers your questions.

Regards and thank you for trying to clarify all of this

Farid

The real question in this trial is about the chemo regimen required, not the insertion of the beta globin gene. For patients who meet the eligibility requirements, this is an opportunity to be cured. No one can guarantee anything, but the science involved is valid. I support this 100%.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: catchR on July 19, 2012, 01:51:11 PM
This is an amazing update, I guess it was few weeks back when Thalgen was granted orphan drug status.

Andy,
This trial is open for patients between age of 15 to 50 yrs?? Kindly let me know. Many thanks!


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Canadian_Family on July 19, 2012, 03:38:37 PM
Just to ease some pressure on Andy :)

Yes the trial is open for patients 15 to 50 years with specific criteria (please see the eligibility file attached in the first post).

Thalagen has been granted orphan drug status since November 2006. Trials are starting now.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: ANI on July 19, 2012, 04:31:12 PM
Dear All
lets pray that this time it comes out as successful option . Pehhaps there is an end to presnt  painful journey which makes us feel guilty seing our childs sufer so much.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: sagar1 on July 20, 2012, 03:54:09 AM
I pray to god that let this trial be successful and all of us are saved. Best of luuk to all of you.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: catchR on July 20, 2012, 11:10:04 AM
Its my mistake I think I wanted to refer IND (Investigational New Drug) status from FDA but got it wrong.

Thanks Canadian family for correcting me. Anyways, I am really happy that some progress is being made on this front  :goodluck


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Pratik on July 20, 2012, 12:09:05 PM
Very nice to hear it. I really hope that it turns out to be successful and my prayers are with those undergoing or will undergo the trials.

-Pratik.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Poirot on July 25, 2012, 08:53:35 AM
This is great news.


Unfortunately, they exclude Hep C patients.  :(


Cheers


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Canadian_Family on September 19, 2012, 08:13:50 PM
The study is still recruiting patients. You can save this link in favourites and check back whenever feel like.

http://clinicaltrials.gov/ct2/show/NCT01639690

The study hopes to have the results available in July 2014.....


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on September 19, 2012, 08:20:30 PM
Recruitment in the US has gone slow, possibly because some misinformation about the goal of the trial. The goal is to cure patients. If the procedure works as expected, patients will be cured.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Pratik on September 20, 2012, 10:11:01 AM
Recruitment in the US has gone slow, possibly because some misinformation about the goal of the trial. The goal is to cure patients. If the procedure works as expected, patients will be cured.
Is there any risk? Like if it fails, nothing will happen to the patient right unlike BMT?

Regards.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sahidul on October 19, 2012, 03:29:33 AM
Andy , sharmin,

is thereul   any report of successful gene therapy?

we are all waiting for good thing.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on October 19, 2012, 01:47:58 PM
The latest email from Pat G that came a couple days ago said that two patients have been accepted for the trial and the first will be dosed in December. Results will be known after 40 days.

Patients in the US: Patients are still being accepted for trials. If this works as expected, patients will be cured.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Canadian_Family on October 19, 2012, 04:11:17 PM
Thanks Andy, This is good news.

It is my understanding that total 10 patients are to be recruited at inital stage (Phase 1).


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on October 20, 2012, 04:05:47 AM
They definitely want more patients, but things were slowed by some misinformation that was put out by one of the doctors who is no longer allowed to speak publicly about the trial. Hopefully, we will see more patients apply.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: catchR on November 14, 2012, 05:59:09 AM
Update: Beta-Thalassemia Treatment Approved For FDA's Clinical Phase I Trials - http://www.csrwire.com/press_releases/34862-Beta-Thalassemia-Treatment-Approved-For-FDA-s-Clinical-Phase-I-Trials (http://www.csrwire.com/press_releases/34862-Beta-Thalassemia-Treatment-Approved-For-FDA-s-Clinical-Phase-I-Trials)


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on November 14, 2012, 06:14:10 AM
We can expect the first patient to start soon, possibly in the next two weeks, with results known after about 40 days.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Pratik on November 14, 2012, 09:08:41 AM
Interesting. Let's pray for the best.

-P.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on November 14, 2012, 04:51:34 PM
Praying....



Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Canadian_Family on November 20, 2012, 05:19:51 PM
Praying for the success...


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on November 24, 2012, 07:27:30 PM
Is anyone aware of whether the trials commenced this week?  Hopefully the first patient has been infused...



Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Parin on November 25, 2012, 04:59:55 PM
Yes, Pat said it will be on 23 Nov...I am also waiting for...There was a Thal confer in Delhi where Pat and Dr. Saleden was present....I guess may they have disclosed...if anyone attended the conf ...Please shaare the update..

Thanks You.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on November 26, 2012, 12:56:14 AM
The first report from the Delhi conference is that Dr Sadelain announced that three patients have started!

 :yaaaaaay


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on November 26, 2012, 01:26:32 AM
Tears of joy - I'm so happy for every thalassemia patient today. 

Hoping for good news in 40 days...


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Pratik on November 26, 2012, 01:43:18 PM
The first report from the Delhi conference is that Dr Sadelain announced that three patients have started!

 :yaaaaaay
That is worth to cry.

I really really hope that this surpasses all the boundless limits thalassemia majors had until now.

Praying so hard.

Also are all 3 Indian patients and their treatment is undergoing in India or USA?

Keep us updated Andy.

-P.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Bostonian_04 on November 26, 2012, 07:46:06 PM
What a wonderful news!!!! Best of luck to Dr Sadelain and his team. We are all eagerly waiting for the next update .


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: zahra on November 28, 2012, 03:17:39 PM
Praying for a grand success.
Waiting seems harder now it seems so close doesn't  it?
Zahra


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Pratik on November 28, 2012, 04:03:24 PM
Praying for a grand success.
Waiting seems harder now it seems so close doesn't  it?
Zahra
Sure it does, Zahra. :)

-P.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: ANI on November 28, 2012, 05:05:52 PM
I pray to god for well being of all the patient, who is undergoing this trial . I am sure the day is not far when this therapy will be accessible and affordable to all.
best of luck to all of us.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: catchR on December 18, 2012, 05:35:10 PM
Hello Andy,
Any further updates from Dr. Sadelain on the first few patients selected?? I pray for success in their efforts.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Canadian_Family on December 20, 2012, 12:55:11 PM
Please see reply from Memorial Sloan Kettering Cancer Center.

Question
Understood that it is hard to estimate when stem cell therapy can be widely available. However, if you can provide an estimate time of the phase I results in 2013.

Reply
Submitted by Memorial Sloan-Kettering  |  Wednesday, December 19, 2012 - 11:38 AM.
According to Dr. Boulad, “Although we will be able to estimate the amount of gene in the reinfused stem cells of at least three patients, the time interval for the expression of the gene is not very well known. This information will potentially be available by the end of the 2013.” Thanks for your comment!


Hope it helps...


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Pratik on December 20, 2012, 01:41:56 PM
Please see reply from Memorial Sloan Kettering Cancer Center.

Question
Understood that it is hard to estimate when stem cell therapy can be widely available. However, if you can provide an estimate time of the phase I results in 2013.

Reply
Submitted by Memorial Sloan-Kettering  |  Wednesday, December 19, 2012 - 11:38 AM.
According to Dr. Boulad, “Although we will be able to estimate the amount of gene in the reinfused stem cells of at least three patients, the time interval for the expression of the gene is not very well known. This information will potentially be available by the end of the 2013.” Thanks for your comment!


Hope it helps...
So it will take 1 more year to know the results of those who had undergone trials? That's long. Wasn't 40 days an expected ETA?

-P.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Canadian_Family on December 21, 2012, 09:30:46 PM
Pratik,

Let us hope and pray for the success of the trials, time is irrelevant.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Himanshu Kumar on December 22, 2012, 05:17:33 AM
So does that mean that the genes would take around a year to express themselves? So a patient who would undergo gene therapy now would start producing own blood after a year?


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on December 22, 2012, 06:19:54 AM
They actually expect to see expression within 40 days, but it will take much longer observation to see if it continues to express over time and to measure the safety and efficiency of the chemo regimen used.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: zahra on December 22, 2012, 06:43:00 AM
Hi, on clinical trials.gov it says they estimate the study will last 2 years. Patience required.
Zahra


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on January 09, 2013, 10:44:05 PM
Praying for good news soon.........


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on January 15, 2013, 04:33:21 PM
This is extremely preliminary, but word is that the first patient is doing fine so far. This does not imply anything other than the patient's health is good after the chemo and introduction of the gene. We will find out more soon.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Canadian_Family on January 15, 2013, 08:46:27 PM
Encouraging signs.

Thanks for the update.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on February 21, 2013, 10:43:24 PM
The timing of the release of this video is encouraging - as of yet however we do not know anything for certain about the progress of the gene therapy trials:

http://www.youtube.com/watch?v=gPPKvouhLvE&feature=youtu.be


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Pratik on February 22, 2013, 06:44:28 AM
The timing of the release of this video is encouraging - as of yet however we do not know anything for certain about the progress of the gene therapy trials:

http://www.youtube.com/watch?v=gPPKvouhLvE&feature=youtu.be
That's certainly very encouraging.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: ANI on February 23, 2013, 05:17:08 PM
Really it was an amazing video. Vey Very encouraging . I just pray to God that the resuts come shortly and it becomes  equally encoraging for all of us.
God bless all of us
Bye


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Canadian_Family on February 25, 2013, 09:00:09 PM
Keeping you in the loop.

Update February 19, 2013

Dr. Boulad and Dr. Sadelain provided answers to your questions. They said the protocol is currently open until July 2014 and patients are still being enrolled. The timing of the results will depend on patient accrual numbers and the levels of gene expression that are seen. They hope these issues will both be solved by July 2014. If it moves forward, this therapy would be for all types of Beta-thalassemia major independent of the Beta-globin gene mutation. In order to start treating patients affected by Beta-thalassemia, all (or at least the majority) of patients would need to engraft with the gene and become transfusion independent without any major side effects. The next steps would be production of the vector in larger scale, FDA approval for the vector, and extending the trial to a Phase II study. Licensing the vector to a biotech company may be necessary to broaden access to this therapy, just as it is with any new medication, chemotherapy, or biologic agent.

As mentioned in the youtube video posted by Sharmin, it takes about $1million to prepare the vector for small number of patients. Licensing to a biotech compnay should bring the cost down.

Best of luck to everyone.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on February 25, 2013, 09:20:26 PM
Canadian Family,

Thank you kindly for the update.  It seems like we have taken the first steps in the right direction - and a lot is pending on gene expression in the patients and subsequently financing and manufacturing of the vector.  I hope that more patients will enroll in the study and be cured of thalassemia. 

I hope we begin to see excellent results from the first few patients!

Sharmin


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on May 07, 2013, 02:27:14 PM
I received this note from Pat G today.

Quote
Dearest family,

The 2 treated patients continue to do fantastically. A third patient has already done the stem cell mobilization and will do the 'Gene Transplant' in July. The team will decide whether an increase in chemotherapy is necessary for the next 3 patients.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sushil on May 07, 2013, 03:07:58 PM
Thanks for Great News Andy sir, Millions of Thalassemia patient,their family member and their well wishers prayers are finally heard. I am waiting for a day in my life when i will read that now thalassemia is 100% cure.   


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: zahra on May 07, 2013, 07:14:50 PM
I am so glad to hear this.
Zahra


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on May 08, 2013, 03:58:18 AM
We are all waiting for a day when thalassemia and related hemoglobinopathies are
completely and safely curable.  What this
preliminary news is telling us is that the conditioning and treatment appears
to be safe and there do not appear to be significant side effects
at this time.  This is major, major and wonderful news - because the only
other known cure - BMT - is a grueling procedure plagued with major side effects and
life threatening risks.  To know that this form of gene therapy - were it to be successful - seems
to be safe - is exactly what we need to hear.

I pray that the trials are successful with the low chemo
preparation presently being used - and if additional prep is required
I pray that it won't require high doses and certainly not myeloablation.

Praying for the best


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: sagar1 on May 08, 2013, 04:25:42 AM
thanks andy for providing the information. I pray that the treatment is successful and all the patients are cured. God bless all


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Pratik on May 08, 2013, 10:07:38 AM
It cannot be any other great than May 08 (World Thal Day) to hear this news. :)

Thanks Andy and I hope we all shall see the ray of light, the ray of hope, soon.

-P.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on May 11, 2013, 03:06:31 PM
Another update from Pat G.

Quote
Yesterday it was reported that the first treated patient made it 40 days before transfusing again and that the second made it 60 days. This is not proof that Thalagen works, as chemo therapeutic agents can also stimulate higher hemoglobin.

We do however know that there is 'Gene Expression' and that the patients have tolerated the treatment without incidence.

As always, we have to be patient as this unfolds, but the fact that gene expression is observed is is a major development.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: JV on May 12, 2013, 12:34:52 AM
Amazing, thanks for the update


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sushil on May 28, 2013, 01:56:45 PM
It seems gene therapy really fascinating corporate to invest..... future for Thalassemia is bright, keep faith and patience

http://itsoftware.pharmaceutical-business-review.com/news/sangamo-receives-grant-to-develop-zfp-therapeutic-for-beta-thalassemia-270513





Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on May 28, 2013, 11:28:15 PM
That is fascinating. I am glad to see stem cell research advancing.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on June 04, 2013, 12:21:44 AM
wow, this looks great!


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on June 04, 2013, 12:23:24 AM
Andy,

Do we have any updates on the trials?

thanks,

Sharmin


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on June 04, 2013, 02:59:43 PM
Nothing new but I hope to learn more at the Philly conference at the end of this month.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on June 04, 2013, 04:18:31 PM
Thanks Andy,

I hope that more encouraging news will be relayed.  Even small successes are important as they lead to the fine tuning that will bring about the bigger ones.  I am very confident that this study will yield a complete and safe cure for thalassemia and sickle cell anemia.

Perhaps we can get the information from you in person as well will likely see you first week of July:)

Sharmin


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: elegantkunal on July 20, 2013, 05:38:00 PM
Hi,

i have a dear friend of mine who is suffering from thallasaemia major.She is 25 years old and goes for blood transfusion at AIIMS(india) after every 21 days.Is there any chance she could be well treated, to cure her so that she could live a much healthier life.Which hospitals in US could provide her a better treatment so taht she could live a normal life.Looking at her no one could say she isa thal patient.Please help me in getting her a better life.

Thanks in advance,
Kunal


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on July 22, 2013, 07:14:31 PM
Dear Kunal,

The most important thing that your friend can do at this time is to maintain good hemoglobin levels and low iron levels.  This requires adherence to a good transfusion regimine, keeping hemoglobin level above 95 at all times, and keeping iron levels as though as possible - preferably below 1000 (the lower the better).  We can help her in selecting a good chelator and chelation schedule. 

We also recommend many supplements along with a good diet, rich in plant food sources.  We recommend IP6, L-carnitine, vitamin D, vitamin e, zinc, calcium and magnesium as well as others to maintain good health.  Drinking tea with meals is also very helpful

The comprehensive clinic in Oakland, Dr. Vichinisky is wonderful and would give you the best information. 

Sharmin


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Luana on October 06, 2013, 09:54:42 PM
What is the latest news about Thalgen therapy? Let's all pray for a better life x


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on October 07, 2013, 02:07:18 PM
Three patients have been treated with another about to begin. One of the first two patients is now on a reduced transfusion schedule. Each month shows improvement in expression of new hemoglobin. Optimism is warranted.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Canadian_Family on October 07, 2013, 05:46:40 PM
Great news.

Andy,

Thanks for sharing.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Pratik on October 07, 2013, 06:51:11 PM
Absolutely wonderful news.

Thanks for sharing Andy!

Best,

-P.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on October 09, 2013, 02:03:44 AM
Thank you for the wonderful news Andy.  Praying for more good news!

Sharmin


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Luana on October 09, 2013, 07:23:10 AM
Let's all pray for more wonderful news! 


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: catchR on October 11, 2013, 01:39:30 PM
This indeed is good news!! Praying for early success, as it would help thals worldover....

Andy,
Any initial details (vital counts, other changes in patients) of how well the treatment is picking up???


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on October 29, 2013, 06:42:00 PM
We're approaching one year since these trials began... has anyone heard anything new regarding the trials?


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Canadian_Family on October 30, 2013, 06:12:00 PM
Hi Sharmin,

It seems that progress is slow and Memorial Sloan-Kettering Cancer Center is very careful to announce any news. I am guessing that an official word would not come until first half of next year. However, Andy may be able to get some news (now and then) as he has always been able to update us. Thanks Andy.

Lets keep our fingers crossed.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on October 30, 2013, 07:05:29 PM
Canadian Family,

You're quite right.  When the trials began we were warned that likely we would not know much for about two years... I guess all we can hope for is for some positive preliminary results.  I am glad that things have come this far because until now nothing had been approved for human study.  Now we have potentially two sets of clinical trials on the go. 

I look forward to hearing more good news over the next few months. 


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Canadian_Family on November 12, 2013, 09:29:53 PM
The trial update as of November 7, 2013 is provided in the link below.

http://clinicaltrials.gov/ct2/show/study/NCT01639690

Hope to see some primary results in July 2014 as stated in the link, please see below:

Estimated Enrollment : 10
Study Start Date: July 2012
Estimated Study Completion Date: July 2014



 Estimated Primary Completion Date:

July 2014 (Final data collection date for primary outcome measure)


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on November 12, 2013, 10:07:32 PM
Canadian Family,

Thank you for sharing.  Praying for good news for our children. 

Greetings from Western Canada :)


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Canadian_Family on November 13, 2013, 04:39:00 PM
Greetings back from Eastern Canada. However, I still hope Toronto Maple Leafs win over Calgary Flames on April 1, 2014.   :smiley





Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on November 14, 2013, 04:25:01 PM
ha ha Canadian Family -we'll have to see about that  :wink



Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on January 17, 2014, 03:09:54 AM
Has anyone heard anything new regarding the trials?  Hoping and praying that the first few patients are transfusion free - or at least close to being transfusion free....


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Baby_boy on January 21, 2014, 02:56:02 AM
Hello, I have not seen or heard of any new updates thus far. As I type this message, I hope that the trials are progressing with positive results, not just for the brave patients, but everyone who is affected by this disease. My son is now 18 months and my heart hurts for him everyday. May GOD continue to bless and guide the dedicated team during these trials.

Regards.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Canadian_Family on January 21, 2014, 02:21:37 PM
Hi Sharmin,

Memorial Sloan-Kettering Cancer Center is ofcourse tight lipped in this matter. Lets hope for the best results. I expect some news by June or July 2014.



Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on January 21, 2014, 09:53:06 PM
Canadian Family,

Yes, let's all keep praying for a safe and complete cure.  There is a conference in June in Chicago - perhaps there will be news released at that time.  My best wishes to all of you and your children.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Canadian_Family on January 22, 2014, 05:49:48 PM
Thanks Sharmin.

Best wishes to you and your family.

Just wondering if Andy has inside scoop in this matter  :huh


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on February 04, 2014, 05:02:07 AM
It is a good idea to contact Cooley's anemia for more information about the trials and to discuss enrolling in the trial.



Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Pratik on February 16, 2014, 07:23:41 AM
Wow, I really hope we would finally see a cure that works. Eagerly awaiting for June, roughly 4 months from now. I heartily hope all the patients who underwent would come out thal-free. It would be the most wonderful plight to see that happen.

Praying everyday for them, and as well as for all of us who waited years to listen to a positive outcome.

Best,

-P.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on February 20, 2014, 05:30:58 PM
I am currently looking into enrolling Lil A into the trials.  He is only 15 years 5 months right now - but I am guessing by the time the work up is done and everything is set up - he will be close to the age requirement for the trials.  I am also hoping to hear good news about the trials over the next few months.  I hope that a cure is close and that it is a complete cure.

Asking for prayers as we pursue this…

Sharmin


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: catchR on February 20, 2014, 06:00:33 PM
Way to go.... hope we get to hear some really good news come this June...eagerly waiting for the results.

All the very best wishes for Lil A, will surely pray for Lil A!!!


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on February 21, 2014, 07:39:07 PM
Thank you catchR  :heartpink


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Andy Battaglia on February 23, 2014, 06:46:13 PM
Pat G's son also wants to go for this but has stated he will wait until after 10 patients have been treated. I think little A has this same luxury of waiting. By the time any decision has to be made, we will have a good idea of how well this works.


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on February 23, 2014, 10:14:29 PM
Thanks Andy,
You're right - by the time lil A is old enough - likely 10 patients will have been through the procedure and we will have a good idea of how things are going.  Things sound really positive already - I can't wait to hear more updates about the patients in June!



Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Canadian_Family on March 06, 2014, 09:04:51 PM
Memorial Sloan-Kettering Cancer Center has updated the clinicaltrials.gov website on March 4, 2014.

Following are the highlights:

1)   The primary outcome measure data collection is still July 2014 (this is good given it was postponed in the past).
2)   The safety issue should be addressed by July 2014 (removing the main hurdle)

Excerpts from the site as follows:

Further study details as provided by Memorial Sloan-Kettering Cancer Center:

Primary Outcome Measures:
•safety [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
of transplanted autologous CD34+ hematopoietic cells that are transduced ex vivo with TNS9.3.55 and transplanted in subjects with ß-thalassemia major conditioned with a reduced-intensity non-myeloablative preparative regimen.

a.The occurrence of insertional oncogenesis, which will be investigated by monitoring peripheral blood cell counts and leukocyte clonality using FACS analysis, qPCR for vector. copy number, LAM-PCR and/or 454 sequencing;
b.The generation of a replication-competent lentivirus (RCL).
c.The safety of a low dose non-myeloablative conditioning regimen

•tolerability [ Time Frame: 2 years ] [ Designated as safety issue: No ]
of transplanted autologous CD34+ hematopoietic cells that are transduced ex vivo with TNS9.3.55 & transplanted in subjects with ß-thalassemia major conditioned with a reduced-intensity non-myeloablative preparative regimen. monitoring the following: 1. The occurrence of insertional oncogenesis, which will be investigated by monitoring peripheral blood cell counts & leukocyte clonality using FACS analysis, qPCR for vector. copy number, LAM-PCR and/or 454 sequencing; 2.The generation of a replication-competent lentivirus (RCL). 3.The safety of a low dose non-myeloablative conditioning regimen

Secondary Outcome Measures:

•the level of engraftment [ Time Frame: 2 years ] [ Designated as safety issue: No ]
of the genetically modified autologous CD34+ cells and expression of the transduced ß-globin gene as measured by: Blood for measurement of vector copy number and transgene expression in blood cells at months 1, 2 and 3.
- Bone Marrow Samples for evaluation of CFU, BFU-E gene+ colonies and Q-PCR will be tested at month 1.

•The frequency of post transplant palliative transfusions [ Time Frame: 2 years ] [ Designated as safety issue: No ]
The number of red blood cell transfusions after engraftment will be quantitatively evaluated for efficacy purposes.


Estimated Enrollment:   10
Study Start Date:   July 2012
Estimated Study Completion Date:   July 2014
Estimated Primary Completion Date: July 2014 (Final data collection date for primary outcome measure)


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Rajiv on March 07, 2014, 03:17:11 PM
Dear Canadian family,
thanks a lot for the update.
Valuable info.  Always pray to GOD to give us a permanent solution to this problem.
May GOD bless all patients and friends by giving this cure.
Best regards,


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: Sharmin on March 08, 2014, 12:08:08 AM
Canadian Family,

Thank you kindly for summarizing the information and sharing it with us. 

Best,

Sharmin


Title: Re: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials
Post by: FatherViaan on April 22, 2014, 08:25:41 AM
Any inside news on these trials...please share.