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

Errant Gene Therapeutics, LLC™, hopes to begin Thalagen™: Gene Therapy Treatment for Thalassemia trials by June 2008.


I contacted EGT and received the following responses from Pat Girondi, CEO of EGT.

Dear Andy,
 
Thanks for your interest. Actually there are a lot of things going on. We hope to be in our first patients by June of '08'. 5 patients will be treated at Sloan Kettering and  5 at NIH.
 
There is a link being put up to view the last FDA-NIH meeting. It was called RAC. WE got through it and are beginning to write the IND.
 
Thanks for all that you do for the people with Thalassemia.
 
The link will be posted on myspace.com/patgirondi shortly.
 
Thanks.
 
pat girondi CEO of EGT

and

Dear Andy,
 
We will need about 3,000,000 USD this year.
 
You could do me a big favor by sending people to the www.Myspace.com/patgirondi.
 
If for example they buy a CD for 13 bucks we see all 13 for research thanks to the kindness of Street Factory Music Records in Chicago.
 
Thanks again.
 
pat girondi

$3,000,000 will be needed to get this going. Some funding will surely come from government and also private foundations and donors, but much more will be needed, so think about ordering the cd. 100% of the money raised will go towards this effort. Ask your friends and family to order the cd.

http://www.myspace.com/patgirondi

Latest update from pat g:-

The project is within 6 months of dosing the first thalassemic patient.

wish the entire team working on this..best wishes for all the success..it will change many lives...

Thanks & Regards,
Ashutosh Thaokar On the behalf of my son PARIN

may it come's to sucessful launching  and  will start treating thalassemia very soon .....

Please advise if there is any update on the trials

Andy,
Please inform if there is any progress/update on these trials.

Thanks
Meena

Hi everyone, it's Rocco's father, pat girondi. I believe that it is correct that a thal patient can live longer than ever. There are doctors that have said to me that a thal patient treated effectively can live a normal life span.

Unfortunately this is good news that often has bad ramifications. I remember the terror of transfusing Rocco in the early '90's. I'm sure that many of you can.

I will not accept that at anytime a new virus (known or unknown) can enter into our blood supplies and devestate our family much as hepatitis and in some cases HIV has done. I will also not accept that the attitudes of many, including our own doctors has relegated the search for a cure to some unimportant position because in their opinions thal patients are well off compared to people in other situations.

When a doctor commented to me that I was exaggeratiing in my aggressiveness to find a solution, I asked him how many transfusions had he had in his life. The answer was 0.

If anyone wants a cd (for free) just let me know. The new one, 'Orphan's Hope' will be out in less than 60 days. In September we actually raised almost 5,000 dollars. This pays a vector consultant for a month.

I apologize if any of my views or statements may have caused any problem. I as you want to see this whole project work well. I, as you will then dedicate the rest of my life in helping others with 'Orphan Diseases'. Sickle Cell is the next planned target.

Again, I apologize if my enthusiasm or statements hurt or disturb. I love all of you.

pat g

Pat, I'm looking forward to meeting you in NYC in February.

Dear Pat,

I completely understand how you feel and I back you up 100%.  I also cannot wait for their to be a cure for this disease because the worry that we live with every day is very stressful.  Thank you for all of your efforts that will benefit all of us one day. 

Sharmin 

Hi Pat,

 

It's good to see you posting here,people like you are an inspiration to us all,we are proud of you,every one of us is waiting for a final cure,we do say that thalassemia is a manageable disorder,but sometimes it hurts to see our little kids managing so much when they are so young themselves,of course you can understand it better.

  and 

Zaini.

Pat,

Your commitment and passion has helped us cope with this situation. These are our children and there is nothing more precious for all of us.
When things were the darkest, your work offered us hope, a possible light at the end of the tunnel.

Know that our prayers are with you and your work.

It's great to have you post here.

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

Hi Pat,

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

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.

Hi Pat,
  , 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

[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