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Author Topic: Update on Gene Therapy and Debate about Myelosuppression vs Myeloablation  (Read 18018 times)
Andy Battaglia
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« on: March 09, 2013, 12:58:26 PM »

First the update. The first patient that was treated at Sloan Kettering with gene therapy to attempt to cure thalassemia is home and doing well physically. While we won't really know about gene expression for 6-8 months, I can report that the patient has done quite well and has recovered from the chemo regimen used in the procedure. A second patient began during the third week of February and a third will begin in May. In addition, the National Institute of Health has also now come on board and is now involved with SK in this project. That was long sought and is great news.

I want to talk about an issue that is at the heart of this and any other attempt to cure thalassemia via gene therapy, and I want to point out some of the differences between this project and that from another company that is also planning on beginning trials in the US. First, only Sloan Kettering has access to the actual beta globin gene. The patent for this gene has long been held by Errant Gene, and it is now controlled through an arrangement through EG and SK. No other company can use this gene unless it is licensed to them and at this point in time, that seems rather unlikely, so mutated beta globin genes must be used by any other group. Secondly, and to me this is very important. Dr Sadelain, EG and SK decided to use myelosuppression of the bone marrow rather than myeloablation. With myelosuppression chemotherapy, the bone marrow is suppressed but not entirely wiped out, as it is with myeloablation. The thinking is why give the patient more chemo than needed and possibly cause many side effects, including death, if it is not needed? Why not use the approach of a milder chemotherapy to prepare the patient?

This is not a race, although one company may view it that way. Dr Sadelain's team approach is to think about the long term effects on the patient of both the gene therapy and the preparation for the procedure. I totally agree with this approach. If the myelosuppression used is not adequate, then it can be enhanced, but why do this unless necessary. Having had an employee die 4 years after a BMT directly from the damage done to his lungs and kidneys by the preparation for the BMT, was a very sobering experience for me. I don't think we should be approaching gene therapy in the same way as a BMT. I think the lowered chemo regimen is the best approach, until it is proven to not be sufficient. As things stand now, everything looks very good for the first patient and the recovery has gone quickly and gone well. I just cannot see how using a more severe approach of myeloablation is the best decision when we are first starting on this road to a gene therapy cure. I realize that this is also a subject of much disagreement in the world of BMT, as Dr Krishnamurti has been a pioneer of the "kinder, gentler" preparation for a BMT. The big difference here is that we are just starting on this road. Using the milder approach is not an afterthought. As I said, this is not a race. We want to see gene therapy done right and cause as little side effects to patients as possible.

As always, I agree with Dr Sadelain's well thought out methods and I see no reason to use any other approach unless further down the road, we see that it has not been sufficient. My thoughts are always with the patients first and I cannot agree with a more intensive preparation unless it is absolutely necessary. At this point, we do not see that and we should all wait and see what happens as time passes over the next 6 months. I know the other group wants to rush and that they are well financed, but I am not convinced that their methods are what we should be utilizing. Let's stay patient and see how thse first few patients do over the next year.
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Pratik
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« Reply #1 on: March 09, 2013, 01:24:41 PM »

Absolutely agree with you Andy!

I guess we should know how patients reacts and that if it's success or not by end of 2013 I think?

-P.
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« Reply #2 on: March 10, 2013, 12:33:05 PM »

Thank you for sharing this wonderful news with us Andy.  I am definitely in agreement with you about use of a milder preparatory regimen, as the purpose of the therapy is to restore health - not to replace one illness with another.  I whole heartedly appreciate Dr. Sadelaine's cautious approach and hope that in good time we will hear good results from his research. 

Pratik, likely we will hear something good by late fall - and we will have some idea about progress in the first 2 patients by the end of this year.  I think that patience and prayer will get us through the rest of this year while we wait.  We wish this group of researchers and the first few patients the very best - and are thank ful that they are paving the way for a healthier future for us and our loved ones.  We have waited for a very long time - I am glad that the long awaited procedure has evolved into a treatment for patients now. 

Thanks again for being our advocate Andy,


Sharmin
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« Reply #3 on: March 11, 2013, 11:16:31 AM »

Thank You very Andy for this update. In fact all of us have been waiting for this day . The approach ,you mentioned for preparing the patient for gene Theray ,using Myelosuppression seems absolutely better choice and that depicts the mature thought of Dr Sadeline.
As far as the well being of the patients are concerned, our prayers are with them and I can see some light in the tunnel.
Bye and Rgds
Ani's father
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« Reply #4 on: March 13, 2013, 03:18:07 PM »

Andy,

I have heard of both approaches and I totally agree, we should wait and see the results. I really appreciate the update, and I am so happy to hear the patient is doing well.
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« Reply #5 on: March 28, 2013, 10:37:45 AM »

Hey Andy,

I add my voice to yours as well, the approach you proposed sounds more reliable and promising.

I have some questions:
1) How old your first patients are?
2) Is Gene therapy going to be implemented successfully on Adult patients (30+) ?

I would like to hear if their is any research group in this planet that is researching curing Thal for adults.

All the best.
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Pratik
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« Reply #6 on: March 28, 2013, 11:17:53 AM »

Hey Andy,

I add my voice to yours as well, the approach you proposed sounds more reliable and promising.

I have some questions:
1) How old your first patients are?
2) Is Gene therapy going to be implemented successfully on Adult patients (30+) ?

I would like to hear if their is any research group in this planet that is researching curing Thal for adults.

All the best.
The first patient (female) is 27 years old I think.
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Andy Battaglia
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« Reply #7 on: March 28, 2013, 04:55:08 PM »

I do not have any information on the patients, as it is confidential. They are only taking adults who meet the criteria for the trial. It is expected that this will work on all ages, as there is not the rejection that occurs with BMT.
By the end of 2013, we will know a lot about this trial.
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Andy Battaglia
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« Reply #8 on: July 17, 2013, 08:41:37 AM »

I heard from Pat G this week and after meeting with Dr Sadelain, Pat says they feel this debate is over and that myelosuppression is sufficient, making this procedure much easier on patients. This is monumental for patients. The gene expression is happening faster than they thought it would and the chemo regimen is enough without being too much.

Participation in trials by the eligible should be considered.
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All we are saying is give thals a chance.
Sushil
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« Reply #9 on: July 17, 2013, 09:20:53 AM »

Hi Andy,

I agree with the milestone, Myelosuppression of bone marrow is more logical approach, why give chemo more than it require. Btw do you have any update on other people trial also , you mentioned that second patient will be beginning in Feb and another one in May.

we need to see every update carefully as Gene therapy is not a medical practice its a reasearch which will be base for medical practice in future.

Regards
Sushil Thakur
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Andy Battaglia
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« Reply #10 on: July 17, 2013, 09:24:10 AM »

The second patient was started some time ago. Both patients are at home doing well and seeing a steady increase in globin gene expression and Hb level.
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Andy

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Sushil
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« Reply #11 on: July 19, 2013, 09:50:52 AM »

Wow... this is awesome.
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Sharmin
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« Reply #12 on: July 19, 2013, 05:56:07 PM »

This news is getting better each time - what a relief that myelosuppression is working so well.  Myeloablation is very dangerous and has many many serious side effect. 

Thanks again Andy,  I hope that we can all start planning when to sign up for the trial soon!

Sharmin
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« Reply #13 on: September 02, 2013, 12:18:06 PM »



Hello to each and everyone,

My son was recently diagnosed (March 2013 - 8 month at the time) with Beta Thalassemia Major.
Since that time, I have not slept well at nights. He is the only sibling and BMT is out of the question.
Finally, Gene Therapy trials have started I must say that the updates are extremely promising. I totally believe in Dr. Sadelain and his team. May GOD bless them. From a scientific and logical point of view, Myelosuppression makes alot of sense. As Terrance's Dad, I cannot wait to have him cured.

Best regards.
Alicia & Shastri.
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« Reply #14 on: September 04, 2013, 08:33:14 AM »

I am standing at cross roads. We have almost decided to go for BMT for my child who is 8 yrs old. Seeing the good progress of Gene therapy e are confused as to to should we wait or not?
Some decision in life are bigger than the life itself.
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