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
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?
The first report from the Delhi conference is that Dr Sadelain announced that three patients have started!That is worth to cry.
:yaaaaaay
Praying for a grand success.Sure it does, Zahra. :)
Waiting seems harder now it seems so close doesn't it?
Zahra
Please see reply from Memorial Sloan Kettering Cancer Center.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?
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...
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:That's certainly very encouraging.
http://www.youtube.com/watch?v=gPPKvouhLvE&feature=youtu.be
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.
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.