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Gene Therapy Experiment in Milan

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Sharmin:
I pray for the family involved - and for the sake of all others who will benefit when doors are opened - if these therapies are a success.

§ãJ¡Ð ساجد:
:banghead

Blogspot is banned on my ISP :mad

even Pkblogs and Inblogs are not working to override the ban

Is there another way to view it?

Zaini:
Posting here for you Sajid !!


--- Quote ---I do apologise, as I normally do when I do not write an article in a long time. We have run out of Internet time so we have to purchase a card for every 30 minutes of Internet we use now. So, we try not to use up the card too quickly. In addition, I have tried to use as much of my free time to review for my exam that I have to write a couple weeks from returning to Canada. That said, I am writing now and we do have some good news.

I had mentioned that there was a good possibility that we would be returning home with Parker very soon. I am happy to say that we have gotten confirmation from Parker's team of doctors in Italy that we may return to Canada. Thus, in the next several days we will once again get to sleep in our own bed and Parker in his own room.

My wife mentioned last night at dinner that we have spent a lot of time in our battle against Parker's immunodeficiency. Now, that we are going home I can say that the first half of the battle is complete.

Parker still remains immunodeficient. Actually, he is much more immunodeficient leaving Italy than he was coming. Without giving away too much information about the team's work I will give you a couple of numbers to ponder. Eight months ago Parker had about 2500 neutrophils per unit of blood and the results of some tests in the months prior to then Parker had over 3000. Since completing the gene therapy and after giving his bone marrow some time to recuperate from chemotherapy Parker's most recent blood results have his neutrophils just beginning to reach 2000. And even still, there will definitely be some fluctuations in these level over the next several weeks.

If you have been reading the blog for a while now you may remember how well Parker responded to PEG-ADA (the injections of ADA he received twice a week to give him the enzyme he was unable to produce himself). The greatest number of lymphocytes that I have recored for Parker are 2600 cells/unit of blood. Now, this is a relatively high number of cells for a child on PEG-ADA but as I had mentioned Parker did respond well on enzyme replacement therapy. Now, before I mention the number of lymphocytes that Parker has three months post gene-therapy I have to mention a couple things about PEG-ADA.

First, PEG-ADA is not a long term solution. Although Parker had normal levels of lymphocytes (low but relatively still normal) while on PEG-ADA the effectiveness of the drug after only a few years of being on it decreases significantly. It decreases enough after three years that Parker would have very little few lymphocytes left by the time he was to enter kindergarten.

Secondly, Parker would have to have the twice weekly injection of PEG-ADA for the rest of his life. With each injection costing $2500US it becomes very expensive and there is no guarentee that the government would pay for the drug indefinitely.

It was for those reasons we chose to take part in the gene therapy trial rather than having Parker stay on enzyme replacement therapy for his lifetime.

As I was saying, Parker had quite a few lymphocytes while on PEG-ADA (2600 in August 2006). Shortly, when we leave Italy, Parker's lymphocyte level will be somewhere in the range of 100 to 200 cells/unit. Yes, this is significantly less that what he had on PEG-ADA but we knew right from the start that it would take quite a bit of time for Parker's lymphocyte level to increase after gene therapy. They may never reach the level they were while Parker was on PEG-ADA but at least a portion of his cells produce ADA by themselves. For that reason these modified cells will be able to rid Parker's body of the metabolites that cause serious damage to his organs in addition to putting his immune system into a state where it is unable to fight infection.

We are still unsure how well the therapy will work for Parker since there was some difficulty with the therapy over the past months. Remember, there was a time that we thought we were going to have to repeat the therapy because it did not work. Now, only a couple of months later we are happy to know that although Parker may not be their most successful patient to date the therapy does seem to be working.

What do these numbers mean for us and Parker? We must continue to keep him protected so that he does not catch a serious infection. My wife will remain at home for at least another year before returning to work and we both will do everything possible to keep ourselves from bringing anything that can harm him inside the walls of our home.

We are extremely excited about returning home to see our family and friends. There are so many people that we want to thank and I know that I am going to miss a bunch of people but I want to try anyways.
 
--- End quote ---

Written by Parker's father on 25th september 2007,want me to post more?

ZAINI.

§ãJ¡Ð ساجد:
Thanks Zaini,

I think I get the picture. I know he is a difference case than ours and his low lymphocytes are not enough for him to stay outside his protected environment, but the good news is that he is off from getting the hormone replacement therapy. So, it is still good.

I hope even a partial success in case for Thal. would mean a low Hb but it should be enough to keep us Thals off the Tx. and Desferal which is good enough for me.

Sharmin:
My understanding of gene therapy for thalassemia is that some amount of expression is required - it does not need to be complete.  heck - many of us are thal minor and we function just fine.  This is all very tricky and very different, depending on the disease being treated. 

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