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Thalassemia Patients and Friends and thalpal Ā© A. Battaglia 2019





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This is Thalassemia Patients and Friends,
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Author Topic: Recruiting patient for Thalgen Finally! Good News about Gene Therapy Trials  (Read 93157 times)
ANI
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« Reply #30 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
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Andy Battaglia
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« Reply #31 on: July 17, 2012, 07:38:53 PM »

Fantastic news! I have posted it on our Facebook groups.
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Andy

All we are saying is give thals a chance.
Canadian_Family
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« Reply #32 on: July 17, 2012, 08:18:43 PM »

Great News, I pray for all the patients and parents......
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JV
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« Reply #33 on: July 18, 2012, 12:08:30 AM »

Hi...can somebody give me some info on Thalgen?
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Andy Battaglia
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« Reply #34 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.
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Andy

All we are saying is give thals a chance.
Andy Battaglia
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« Reply #35 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%.
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Andy

All we are saying is give thals a chance.
catchR
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« Reply #36 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!
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« Reply #37 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.
« Last Edit: July 19, 2012, 03:48:33 PM by Canadian_Family » Logged

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ANI
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« Reply #38 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.
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sagar1
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« Reply #39 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.
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catchR
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« Reply #40 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 
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Pratik
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« Reply #41 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.
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Every child is special.
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« Reply #42 on: July 25, 2012, 08:53:35 AM »

This is great news.


Unfortunately, they exclude Hep C patients. 


Cheers
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Canadian_Family
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« Reply #43 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.....
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Andy Battaglia
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« Reply #44 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.
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Andy

All we are saying is give thals a chance.
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