Update on Gene Therapy Trials

I received this update from Pat Girondi.

Pat Girondi and Doctor Onofrio Pepe will hit Rome Thursday March 6th, for a meeting with RAI to draw up plans for an eventual film on the life of Pat Griondi.
 
Friday March 7th The two will be off to Thessaloniki to speak with Evangelia Yannaki,MD
at the G.Papanicolaou Hospital, Gene and Cell Therapy Center, Hematology-BMT Unit in Greece about preparation for the Clinical Trials.
 
Saturday March 8th will be spent in Palermo at the Cervello Hospital. Onofrio and Pat girondi will be meeting with Doctors Maggio and Acuto preparing for the Clinical Trials.
 
We hope to be in the first patients by the end of the year. Send your prayers and well wishes. Those of you who can afford a CD remenber that every cent goes to Orphan Disease research.
 
For updates further refer to Myspace.com/patgirondi
 
 
pat g

Let's hope for the best and keep buying those Cds.

Thank you for the information Andy, I so hope that the trials go well.

 

Sharmin

My all prayers are with them.

ZAINI.

Praying for the success and an authentic cure for Thalassemia.

Few months back I posted this gene therapy trial story, this is an update on Parker posted today.

http://www.thestar.com/News/Ontario/article/308771

Thanks

Hi C.F,

Thanks for sharing!

It really boosts our hope for Gene therapy!

Here's the latest update. It's really exciting to know that 40 days after they dose the first patients they will know if it's working. If things go well, parents in the future will be hearing the words "your child has thalassemia...but the good news is, there's a cure." Let's all pray for success.

Dear family,
 
Today is March 11th. My son Rocco will be 18 years old on March 31st. I remember the day that he was diagnosed. I remember the tears of his mother. I remember the confusion that it created in my mind. We are all linked by the tears and the fears for our loved ones. In this life you are as close to me as any distant relative and closer than those in my blood line who have never gone to bed with the same thought for 15 years.
 
I apologize for my lack of communications. I can guarantee to you that you are constantly in my prayers and make up particles of my every breath. I don't take a sip of coffee or stretch my leg to kick a soccer ball, sing a song or reach for the phone without all of you.
 
Our lives as well as our deaths are linked forever. A day when our existence has vanquished here to join eternity we will be together as we are now, brothers and sisters drawn together by the hope of health and the love of others.
 
This is what is going on:
 
 I have added the George Papanicolaou Hospital of Thessaloniki, Greece to the collaborating centers of New York, Cagliari, Palermo and Pavia to our group.
 
I am in talks with the government of Italy to facilitate the building of a Vector Production Site. This can be critical. We presently are held hostage to pay whatever price asked by the Vector Producer in the US. We need to make it feasible, affordable, and efficient.
 
The biggest new, news is that after an unfortunate delay we have our final scheduled meeting with FDA on April 28th at 8 pm Rome time. This means that within 120 days we should know the rough date, within a week of when we dose our first patients. The vector takes 2-3 months to produce and 2-3 months to test after production.
 
You all know me. I don’t want to raise any false hopes but we are getting there. Within 40 days after dosing our first patients we will announce to the world that we have ‘GENE EXPRESSION’ of the beta globin gene. This will be a significant event. Investors should arrive.
 
Challenges are many. Resources are scarce and with the American down turn things are even worse. There are many things that I would change about my country. I am an American as well as an Italian. But one thing that is true of us Americans (regardless of what it sometimes appears) is that we will put our money where our mouth is, we will buckle up to the bar.
 
I have mentioned before my friends who are traders in Chicago who wrote checks without a piece of documentation. They wanted to help my son, me, us.
 
I’ve raised some cash in NY from some professional people. There are special tax credits which reduce the risk. Europe and other countries should adopt the ‘Orphan Drug Tax Credit’ of the US.
 
I have put my own resources into the project. It has been my pleasure, my obligation and my joy to do so.
 
When the first CD ‘Orphan’s Soul’ was released it was done as a fun thing, as a way to blow off steam, as a way to communicate with people. I love people.
 
We released ‘Orphan’s Journey’. We are printing the second 5,000 now. My music can be downloaded on 40 internet sites. There are an average of 25 downloads a day. Sales of the CD’s have been good.
 
I open the act in Chicago on April 6th for an Italian performer named Renzo Arbore.
 
Two of my songs are going to be in an Italian movie soon to be presented at the Venice film festival, ‘Focaccia Blues’. It’s a film turned into a small love story around the closing of a Mc Donald’s in a small Italian town. It has been the subject of countless articles including France’s Liberation and the New York Times.
 
The actors (including Michele Placido) have agreed to release a CD of the whole film soundtrack and donate the money to ‘The Orphan’s Dream Foundation’.
 
The Orphan’s Dream Foundation is our foundation. I founded Robin Hood in ‘83’ to help the disadvantaged in Chicago. I closed it last year and opened the ‘Orphan’s Dream Foundation’.
 
I have recently been the subject of newspaper articles. I was on Italian Television and RAI (Italian Television) liked my character so much that they have signed an option with me to do a film of my life.
 
I am a nobody. I finished school at 16, got lucky in the stock and cereal markets and am running EGT because no one else will run it for the same price. If a dime comes out of the entertainment it will go for our goal.
 
Why am I telling you all of this today?
 
I don’t really know. But I really do know that I need to find 700,000 dollars for vector production. In the current economic client it is unlikely and maybe unfair to think that it will come from America.
 
Don’t worry, I will find it. Music has actually begun to help put gas in the car.
 
So brothers and sisters send your WARMEST most POSITIVE thoughts. Visit Myspace.com/patgirondi.
 
Send people to buy CD’s or download songs. Use your intelligence to figure out how to push this wonderful project. Maybe I could do a concert in Dubai or in Mumbai. I could add them to Chicago, New York, Palermo and Bologna.
 
PRAYERS people. PRAYERS will bring ideas and strength.
 
I’m at a moment where it would come in handy. Please send me your thoughts, ideas and jokes. I like them too.
 
Love, pat g
 
PS. I’m not sure if any of you like any of the stuff that I write and perform but my mother loves it.
 

Aamen Andy!

A BIG to Pat

You CAN DO IT!!

My all prayers are with Pat and his team,i hope that day would come soon Andy.

ZAINI.

Indeed very good news. This is a hope for all of us.

 

I have followed thalagen since I came across it through this very website.
I remember watching on their company site the extremely interesting video regarding the RAC approval they received and the encouragement my wife and I received from this news.

I only wish work had not kept me away from the boards. I would have loved driving up to Thessaloniki just for the chance of speaking to Pat.

George Papanicolaou hospital is well thought of and is experienced with clinical studies.

Good luck to them!
(the music is great as well)

These trials should be starting soon - I hope they go well!! 

Sharmin