The Latest from Pat Girondi on the Progress of Gene Therapy

I posted some of this elsewhere on this site recently, but I want to make sure everyone has a chance to see this.

I recently received this message from Pat Girondi.

Dear Andy,
 
I am sorry that i have not communicated lately.
 
I also want to apologize for the lateness in the project.
 
Michel, Isabelle and team will submit the IND by June 30th. We should be in patients in September.
 
Give my love to all of rocco's brothers and sisters. [Rocco is Pat's son. Rocco has thal major].
 
I am presently in India looking ahead. India has at least 200,000 of our brothers and sisters and certain experts estimate the population over 500,000.
 
I know that all of us live for this dream. A dream that would not have been possible without Michel Sadelain, Isabelle Riviere. Chris Ballas, Norb Weich, Jason Feldman, Sam Salman, kostas Antypas, Joe Feldman, Enrico Colucci, Vito Cirrottola, Giuseppe Schmidt, Paritosh Landahni, Teddy Jimbob Beechcroft, Georgey Hanley, Adrian Herzog, Aurelio Maggio, Angela Iacono, Renzo Galanello, Renzo Arbore, Onofrio Pepe, Mauro Bove, Franco Locatelli, Ron Capano, John Sion, Jonathon Ascoff, Alessandro Contessa, Billy boy Mc Cluskey, Mark Step, the Bucellatos,  Derek Holmes, Derek Persons,  Angela Chen,  Sharon Carsten, Greg Swan, Charles Columbus, Susan Agnelli (late), Steve Petrosky John Walton (late) Mike Ahearn, Hsuan Hargest...
 
Frankly, I could never finish thanking all of the many people and there are no guarantees that the trials will go well. The drug Thalagen looks great in the sick cells treated thus far.
 
As you know we have done the the stem cell mobilization in patients already thanks to the support of Dr George Stam.
 
Please continue to send your love.
 
I'll end on a funny note.
 
Recently we were looking around for some cake. I told Jason to check the Orphan Dream acct. The Orphan Dream acct. Gets the money for the music.
 
Recently, Focaccia Blues the second most popular film from italy used our two songs 'Flim Flam Man' and 'It's Your Time'. The film is now at Cannes and won the Golden Globe Award.
 
Anyway, we needed cake for the final payment of the vector. Jason checked the OD account to find 8,000.
 
Wow!!! The bad taste in music by so many is wonderful.
 
This year at Gateway the Capano family and their fantastic friends hold a golf outing for the Cooleys' Intl (not to be confused with the foundation). To date the Cooly's Intl have invested 100,s of thousands for us.
 
Last year at the even Ron, Flo, Gina and gang dished over 200k.
 
This year I'm going to tell them that I'll do a performance... They may just send a check without telling me the date.
 
Excuse me for all of the people that I did not mention. The list would be 10 times longer.
 
We now have 3 pharma companies looking at the project and as always we need help. My favorite is Sigma Tau. Write gregg Lapointe and thank him for their past support and tell them how much you appreciate them considering to help us further.
 
gregg.lapointe@sigmatau.com
 
Sigma T is our kind of people.
 
Ok my brother Andy. Get the message out!
 
September!!!
 
All we need is a little gene expression of the beta globin gene and they'll be knocking the doors down to help us...
 
Pat g
Sent from my BlackBerry® wireless handheld

I loved that last note about the funds. We do make a difference by purchasing Pat's music. Go to http://www.cdbaby.com/Search/cGF0IGdpcm9uZGk%3d/0 to buy his CDS or download his music. All proceeds go to help fund gene therapy. Tell your friends. It's a cheap and easy way to help out. The only obstacle to success is money. Let's do everything we can to spread the word. For Pat. For my new Facebook friend, his son Rocco. For all of you. Let's get this done.[/size][/quote]

And then yesterday, I received this.

My dear brothers and sisters,

Research proceeds. Clinical Trials are a few months away.

I have been asked again what it is that you can do to help with the gene therapy Clinical Trials for Thalassemia...

My thoughts on this subject;
Michel Sadelain invented this gene 19 years ago. It took all of this time to figure out how to deliver it into the cells. His team now believes that this is possible with some efficiency. I am just a tiny part of his team and not a doctor or scientist.

I know enough to tell you this:

Brothers and sisters, you are precious and special to your parents and friends and to us, the Sadelain team. I personally think about you everyday.

I cherish my time recently spent in India. I felt that I was at home. I felt comfortable in the Taj, on the poorest streets of Delhi and in Krshna's Temple.

I met some of you and for me it was like meeting with dear family members that I had dreamed all of my life existed and was anxious to see.

I sang in a Delhi hospital. I feel that it was the most Important performance of my life.

Brothers, sisters, you can do this for Michel and the team:

Keep yourselves well. Do as your doctor instructs you and learn to be your own doctor.

Exercise, eat correctly. Push yourself to excel in school. Keep your mind fresh and occupied. You have something special that we without your gift don't have.
Your body has learned to do more on less and this does not make you weak... If you open yourself to the wonders of this world it makes you stronger... The mind is infinitely more powerful than the strongest arm...

See the black, the white, the smiles (Chahek) and tears and you will see that you are special and that you have something that the common person does not have.

Of course no one can convince you to use this gift but it's there. Take the time to reflect and find it. Bless us and yourselves.

For gene therapy to work well your body and mind must be in top shape, your iron low etc. If this is the case then it's effect will be enhanced.

I know that many of you are economically poor (you may be the richest person in the world but I'm speaking economically).

For you, let me tell you that only love has gotten us this far. Science is very limited.

Michel's team has done incredible feats with the small amounts of money that we have been able to get to him (small is a relevant term as in the US it costs hundreds of millions of dollars to get the average drug to the patient).

I am a strange man. My friend Paritosh can testify to this.

I am an expert at nothing but I have always been good at doing something with small amounts (many say that it's the italian in me).

I am Rocco's voice and Chahek's voice. In many ways I represent the hopes of Sangita, Edmund, Ron and Bassam.

This is a large responsibility and not easy... especially when your voice is not so good and your mind functions in a strange way.

DO NOT discount the energy created by your good deeds, by your quality work in school or outside the home.

Help me through your kindness to others. A billion dollars without the love and positive energy does nothing. It sits in a bank. If people like Michel don't use their imagination and minds the money is paper or numbers on a bank computer.

The energy and love that you create by your work and your good deeds flow into the same well that Michel takes his water from.  This water nourished his imagination and mind.

When I am feeling like a caged animal I dip into this stream. I can't remember how many times that I have  broken the cage.

We were very poor growing up and my mother taught me the importance of prayer. It remains her most important gift to me...

So dear precious person, do good for others and concentrate on life. If you can then send me your experience explaining your good deed it is more important than money to me. Money feeds research, love feeds the soul. A soul does not need research or money to survive. Research without a soul is worthless and can even be dangerous.. Unfortunately I have witnessed this more than once by small researchers and a large pharma company.

For those of you who have some money you can give it to your president Shobha Tuli (Thalcind@yahoo.co.in) and tell her that your wish is that it goes towards 'gene therapy' work... Thalagen.

The most favorite method has been the downloads of music.

For physical cd's there's;

Patgirondi.com
Cdbaby.com
Amazon.com

For downloads (actually the present method of choice) there's);

Amazon.com
Cdbaby.com
Itunes.com

Also, to promote our cause as people for change you may get enjoyment by seeing the latest Salvatore Centoducati video. My words, voice and the 'Orphan Dream Band' music.

I'll be on radio and television in the Chicago land area. Hit and comment. Our voice grows from this...

http://www.youtube.com/watch?v=qL5z_v69RuY

For those of you who want to invest contact Paritosh Landahni (paritoshl@yahoo.com) or Pawan Goyal (skoyal@sify.com) or Jason Feldman at (errantgene.com).

Many have sent letters to Human BioSciences. I was with them the other day and it helped immensely. Now send your emails to claudio.cavazza@sigma-tau.it.

Dear Don Claudio,

I am from Bangladesh and appreciate your interest in thalassemia etc.

Let him know how supportive you are of Sigma Tau's interest in our cause.

What more can I say?

Love you all and when you give up I cannot go on.

I arrived 1:30 last night in Boston on a bus from Washington. The meetings have gone well up till now and I have a Boston meeting of importance coming up. I am walking and writing to you, praying and thinking...

Since I was a little boy I play 'quarters'. Me and my friends would walk and run for miles with our eyes on the ground looking for money. The one who found the most won and we usually put it in a community fund to buy a beverage or snack.

One of my best friends, Kevin and me still play every morning at 6 AM when I'm in Chicago.

He gets mad if I'm late. He says that I do it on purpose to disturb or throw him off his game (he might not be all wrong).

We have so much fun and we tease each other about needing glasses and distracting the other guy etc. He says I hog the street by taking zigzag steps (can't get anything over on him).

I just found 11 cents... All for the research... (I actually found 12 but the penny is stuck to the street and if you can't put it in your pocket it don't count).

Pat g

The new video, "Without Pity" is outstanding.

We are very close to the future. What happens in the gene therapy trial will have far reaching consequences in the world of genetic disorders. But nothing will happen without adequate funding. In this country, I would like to see a return to government funded medical research, so that we do not have to solely rely on the pharmaceutical companies, many of which are only interested in how much money they can make.

Why the government does not pay for the testing of gene therapy.................?

Under President George Bush, most medical research funding was cut, leaving it to the giant pharmaceutical companies to decide which projects were worthy of investment. Coincidentally, Bush was a recipient of large campaign contributions from Big Pharma. During this same period, hundreds of billions of dollars were spent to wage war. It's all about the priorities of the US and unless people speak up and demand changes, nothing can happen. A shift of 10% of the US defense spending could fund every research project, but there is very little chance that this will happen. Until there is a shift in priorities from killing people to saving their lives, I expect no change.

SO the son of  Pat Girondi is thal major and he is Millionaire Why is it thought of his son with bone marrow in Italy ............?

He's no millionaire. And don't forget that for a BMT one needs a matched donor and most thals do not have a matched donor.

There has been very little testing of gene therapy in humans so far. There is a trial in Europe that has met with some success. The US trials have been slowed by financing issues, but as Pat G mentioned, trials should begin in people this September.

We can not know the success rate until the trails are finished and the results are published. In the last conference that i attended the doctor represnting Thalagen said that she believes patient can go through gene therapy until the age of 35

manal

Baloch,

As Manal said, we can't know the answers to these questions until trials are done in people and the results are known. I will add that Dr Sadelain told us in Dubai that there is no definite age limit, but rather the health of the individual patient will be the determining factor. This is true will BMT also. Being in good health with no organ damage makes one a much better candidate.

I also know that there is no age limit...although I am very pessimistic now that things will develop within my lifetime...as far as I am concerned, best thing is to stay healthy within thalassemia.

Lena.

I agree, Lena. We see how long each development takes to reach approval. Expect gene therapy to take even longer because of concerns about the long term effects. Until it has been tested long term in patients, I do not expect to see approval.

You are right. The tools are there to provide lives for those with thalassemia. Take advantage of what is here now and live a good life.

As long as thals can benefit from tools available to ameliorate their daily lives, they must be happy and without stress, 'cause they must consider themselves to be part of the general population - inspite of thalassemia. Life limits are open now to thals as they are to any other people - but only if they comply with every known rule of how to treat thalassemia.

Lena

there is one think i mentiont...

since the 80`s they speak about the 

gene therapie for thal`s.....

but nothing happend....

and now u says it start september....

i remer somethimk like Pat g. goes  in spring 2010 to italy to start genetherapie....

now september.....

sorry but it sounds the same like the all the other storys forom the 80`s.....

i dont believe in GENETHERAPIE.....NO more

         by  the way ...im doing fine...panos