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Thalagen™: Gene Therapy Treatment for Thalassemia to Begin Trials in 2008

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Manal:
Dear Pat

Your work and efforts are the things that give me hope to continue everything i am going through. We endlessly back and support you. I am happy that you joined. We are all here for you, thanks

manal

Canadian_Family:
Hi Pat,

Thank you for you comments. Your work and enthusiasm is much appreciated. Rocco is in our prayers.

Narendra:
Hello and Welcome to the forum Pat,

We have been hearing your name and the tireless efforts you are putting to get a cure for this orphan disease, some from Andy and some from your site itself. With US not having as many thalassemia cases as the rest of the world is having (Do you know - there are around 10000 thalassemics born each year in India??), Those denying the funding - don't understand the significance of a cure for thalassemia. With the immigration pattern and with more and more cases showing up, I hope they realize they need to help more to people like you who are dedicating your time and efforts to find a cure for thousands on the earth. Funding has been a huge problem for thalassemia in US and I hope that changes sooner than later.

We are all with you in the fight and would love to hear soon of a cure. Rocco is lucky to have a dad like you. We salute your efforts and hope for the best.

nice friend:
Hi Pat,
 :welcome2 , its realy nice to see you heere and to hear from you , you are an inspiration for the others to do the strugle for good .. i wish that you got to met succes in wat you are doing ..
Best of luck buddy please stay in-touch with us on this forum it encourage's us  and it feels like the ray of hope is still there for us ...

Best Regards for you buddy
Take Care
Umair

Parin:
[Latest  Update]

Hi everyone,
 
It's pat girondi. As Feb 5th gets closer my mind is more and more focused on the imminent gene therapy trials for thalassemia. My mind is focused on you.
 
I ask myself daily how I can make the trip a little less bumpy. It's critical that we ask ourselves how we can be more efficient. We are our brother's keeper.
 
My mother always taught me to be kind and generous. My youth was spent with my mother's brother 'Uncle Frank' who never had enough to cover the rent but never said no to anyone. He literally would give anyone the shirt off his back and gladly go without. His wife was not always happy about this but he just couldn't help himself.
 
I remember, about 2 years ago. He was succumbing to lung cancer and living with one of his daughters. My family were immigrants to the US in the 40's. Money has never been my family's companion... We are about 30 cousins and out of the 30 there are no university degrees and only two high school diplomas.
 
I brought Rocco, Ciccio and Giancarlo to see their dear uncle Frank. We made it up the stairs and one by one my sons kissed him and handed him a hundred dollar bill that they said they had found on the stairs on the way up to the apartment.
 
My uncle looked at me with those eyes that only an 'Uncle Frank' can have and said... "Aw Pat, you shouldn't do this... you've done so much for me already.'
 
For the first time he had omitted the part about paying me back every cent and it was sad for me to know that he had given up hope... He was the eternal optimist.
 
He, my grandfather Rocco and my mother planted the seed of love in me. I feel it today more than ever. We need to be realistic as well as loving or we can unintentionally hurt the ones that we love. I would never want to do that and for that reason I am communicating to you my ideas about this trial. I don't want false expectations.
 
The goal of this Phase 1 Trial is the same goal as any other drug going into Clinical Trials.
 
Phase 1 is about safety. Is it safe for human use?
 
Phase 2 is about efficacy. Does it work?
 
Phase 3 is about dosaging... What is the most effective dosage per kilo etc.
 
Now I know that a few of you are grumbling. I must be completely candid.
 
I am confident that we will do no harm and that we will see 'Gene Expression' in Phase 1.
 
This is lucky for us as usually efficacy is not seen until Phase 2.
 
Phase 3 will be long and difficult. You see, a thalassemic patient is like the car on the side of the road without gas...
 
Michel Sadelain invented the gas... It's the same natural beta globin gene that you need to start your engine...
 
So we are able to arrive with a tanker truck full of gas (beta globin gene). The difficult part is to get it into your gas tank (stem cells).
 
At the moment we have this tiny cup with holes in it. So delivering the gas is tough. We can only get small amounts of gas into your tank that will not likely be significant enough to get the car started and running smoothly.
 
If you remember I told you that I was speaking with Doctors Gray and Persons about a tube.
 
There are dozens of centers all over the world working on a tube as an efficient delivery system (vector) will help people with many genetic diseases.
 
So let's all pray for our first patient of demonstrate 'Gene Expression'. This is our goal for Phase 1.
 
Now that this is out of the way...
 
Many of you have written and asked... How can we help... First of all, the most important thing that you can do is to take good care of yourself so that when the therapy is available it will be right for you. High iron is an eventual problem for the therapy.
 
The second thing that you can do is to concentrate daily for a minute or two on our project. Weekly, I need to make decisions. I need all of your positive energy, prayers, good Karma or whatever it is that you call it to make the proper choices.
 
The third thing that you can do is to download my music. Last month there were 2410 downloads. The favorite song was 'Divorce Blues' from the album ' Orphan's Soul'.
 
We have also put a link for paypal on the myspace.com/patgirondi. It should work with most credit cards. Please let me know if you run into any problems. It seems to be functioning... but you never know.
 
I realize that most of you can not afford and this is fine. I realize that the economy, especially in the US is in real danger.
 
I appreciate whatever you can do.
 
I need to do some things and must go.
 
pat g, The 'g' is for gonna cure thalassemia

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