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Author Topic: Help Fund the Cure for Thalassemia. 100% Goes to Fund Gene Therapy Trials  (Read 64406 times)
Andy Battaglia
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« on: July 24, 2007, 10:12:14 PM »

Thalagen™: Gene Therapy Treatment for Thalassemia trials will begin in 2008. $3,000,000 will be needed to fund this in its first year. Help fund this by going to http://www.myspace.com/patgirondi
where you can purchase Pat Girondi's CD for $13. All $13 for each CD will go into the fund to finance these trials. Spread the word and ask people to buy the CD. I just bought mine. 
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Andy

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Manal
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« Reply #1 on: July 26, 2007, 09:02:38 AM »

Andy i will too and will spread the word

Thanks Andy

Manal
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Canadian_Family
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« Reply #2 on: July 26, 2007, 11:18:53 AM »

Hello,

I just ordered mine, I think it is best when odering to send comments to the musician (there is a place for it) for great cause, I did the same.

Thanks

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Andy Battaglia
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« Reply #3 on: July 28, 2007, 01:10:21 AM »

Pat Girondi's, Orphan's Soul CD arrived today and I just finished listening to it.    I enjoyed the cool vocal jazz and was happy to think my thirteen bucks was going to the gene therapy trial fund. I pay a lot of taxes. A lot. What are the chances that $13 from those taxes will go to fund the trials? My guess is slim to none. This really needs to change. I'm paying thousands yearly to finance wars. Those are my tax dollars! If taxpayers were actually given a choice where the taxes went, would we really be spending so much money on endless war? This has to change folks. We have to demand better of the people we elect.

Check out the CD and tell your friends. It for a good cause and cheap enough for people to consider.
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Andy

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rutuja2000
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« Reply #4 on: September 18, 2007, 11:21:31 PM »

hi
im rutuja's mom
andy sir i also want to order that cd for fund but im staying in india.pl tell me how should i order from india.thanx.also guide me if i want to go for gene transplant theropy in trial base .is there any chance to me if i go fot that.rutuja is beta thal major of 7.8yrs old,getting 99transfusion till now,and serrum ferritine is near about 1250 microg/gl.i was trying for BT but i had no donor at all.
but i really want to operate my daughter.i give desferal as wel as kelfer also.now she is in gud condition as doctor says.
if u want any center for stem cell trans.or CBT with unrelated donor then pl tell me.
i have 4/6 matched donor from Relience Life Sciences,Mumbai.
i hope u get my problem and do the needfull.
have a nice day.bye
reply soon.
i heard abt Singapore General hospital.but huide me in this regard.
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KHALIFA
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« Reply #5 on: September 19, 2007, 12:44:38 AM »

HI
   Dear all i read all what is written and i really felt what do you feel ..don't forget am a father of    X-thalassemia child am really felt that do you want to know and find out one day there is new transplant for thalassemia PpL like what you discuse a bout GENE THARAPY  ...
   Dear all i don't want any body get down a bout what i said but every body know there is always A HOPE  also me i hope one day i hear somebody find out good treatment for thalassemia ...

   am personally ask PROFF. LUCARALLY (i like to call him FATHER OF THALASSEMIA)  a bout the gene tharapy last april when i was in italy to make rotin check up for ABDULWAHAB i ask him what a bout the gene tharapy .. he just close his computer and start to talk with me a bout it he told me they start to make reserch since 1975 and every five years they say we are going to find out the result after five years more...
     and the reserch still there is no good news ... also he replay he told me the only treatment for thalassemia availeble now is BMT or stem celles like what i did for my son .. 
       Dear Rutuja  mother i know what do you feel  because as i said am afather of X-thalassemia child who i made for him 2 operations one in 2003 and 2005 .
    first one was BMT and the seconde is STEAM CELLSE from the same donor who was is his sister .. i pray every day for those children who they suffering from thalassemia and i fight for them right her in my country ,,, but we still i don't know how much and how long to find out the prober result of the GENE THARAPY ...
        Dear RUtaje and Rutaja mother my heart with you and belive me i got a feeling you talk a bout me also and my son ...
     in the end i would like to say
                 DON'T LOOSE YOUR HOPE  
                                      khalifa
                                 state of kuwait
                        ONE FOR ALL &ALL FOR ONE 
         
« Last Edit: September 19, 2007, 06:05:20 PM by KHALIFA » Logged

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rutuja2000
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« Reply #6 on: September 22, 2007, 12:00:01 AM »

gud morning
Mr.khalif
i really want 2 cure rutuja.but iam helpless.
b'coz i cannt found any donor for transplant.i just want 2 go for stem cell transplant.
pl.pl. help in this regard.
thanks for reply.
waiting 4 ur reply.
from where i will did the stem cell transplant.
can u send the addresses of that centers who are did that transplantation
bye.
god bless you
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Zaini
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« Reply #7 on: September 22, 2007, 05:47:15 AM »

Hi Rutuja,

just a little note,that wether you go for a BMT or stem cell transplant,in both cases you'll need HLA matched donor for your kid,and transplantation is not an easy process.it's a big decision.

ZAINI.
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^*^Xaini^*^
Andy Battaglia
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« Reply #8 on: September 22, 2007, 12:57:43 PM »

Hi Rutuja's mom,

BMT is both costly and risky. I think if you go to Singapore Dr Tan will advise you to continue with your child's current treatment since your daughter is doing well with her health. Deciding to do a BMT is a huge decision and you need to be aware of the dangers. I do not want to mention any names but a member of this group recently lost their child after a BMT and several of the Maldivian children who have undergone BMT's have also passed away. This is very sad and makes me think we need to work very hard to make sure parents are well aware of the risks before proceeding. Some do succeed as with Omega and Khalifa's children, but it is not an easy path to follow. I spoke to Dr Lucarelli at the Dubai thal conference in 2006 and he told me that Khalifa's son was very fortunate to have survived, as most patients with severe graft vs host disease do not survive.

If gene therapy does prove to be a cure it will be many years before it is widely available as long term studies will be needed before it is ever approved. However, once more trials are underway, the patients chosen as subjects, will be those in the best of health. At this time there is no process for applying to be a participant in trials but if early trials show some success the trials will be expanded to include more patients. As with many drugs, it may be that the only way to get gene therapy for many years to come, will be to be part of a trial.

I have emailed Pat Girondi to inquire about shipping his cds to Asia. I will update when I get a response.
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Andy Battaglia
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« Reply #9 on: September 22, 2007, 01:47:51 PM »

Pat Girondi replied that the cds can be sent internationally.

Quote
We can ship it.
Sent wirelessly via BlackBerry from T-Mobile.

http://cdbaby.com/cd/patg
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Andy

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KHALIFA
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« Reply #10 on: September 26, 2007, 10:59:38 AM »

Hi Rutuja,

just a little note,that wether you go for a BMT or stem cell transplant,in both cases you'll need HLA matched donor for your kid,and transplantation is not an easy process.it's a big decision.

ZAINI.
           Dear some body answer you before i did ..
      you must have 100% match donor ...
                  khalifa
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Andy Battaglia
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« Reply #11 on: October 07, 2007, 09:52:14 PM »

I received the following update from Pat Girondi on the current progress towards the gene therapy trials and also about the fund raising efforts. If anyone knows any investors please let them know about this. It is an exciting investment opportunity that may eventually lead to cures for thalassemia and other orphan diseases and disorders. Check out the link to see Pat's new video.

Quote
"Dear Andy,
 
The vector is being tested. If it is a good batch we will then commence the making of the batch for the patients.
 
June 15th looks to be a good starting date for the first patient.  After that we can expect to see hemoglobin expression by the end of summer. Then it's tinkering to get the most effective scheme.
 
We need all the help that we can get. We have just come out with a PPM with the hope of raising 5 million dollars.
 
I am coming out with my second album and have added a link to a song that will be used in a movie that will be at Cannes, Berlin, Venice and hopefully out by January.

Here is the link to the updated "It's Your Time:"
 
http://www.youtube.com/watch?v=7A8TCsnnChA
 
Quote
I appreciate all the support. Right now we could use investors. The units are 50 k a piece, they of course are highly speculative like all biotech. the kicker is that they come with the 'Orphan Drug Tax Credit' which if you do the math really is a reduced price.
 
It works like this,
 
50k invested
20k returned in the form of the ODTC
12k returned if the company fails
----------------------------
 
Real risk is about 18k. besides it's an incredible company.
 
Thanks again.
 
pat
"



Anyone know any big investors?

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Andy

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Sharmin
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Little A


« Reply #12 on: November 06, 2008, 12:00:50 AM »

Andy,

Do you know how things are going with this project? 
Sometimes it seems like this can't happen fast enough - yet I know the obstacles that need to be overcome before the trials can even start.  I hope that a new government means a new, more liberal mentality and more support for causes like this.

Sharmin
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Andy Battaglia
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« Reply #13 on: November 06, 2008, 09:17:38 AM »

Good timing Sharmin. Here is the latest from Pat Girondi.

Quote
There is a lot of mis-communication about the 'Thalagen' project. I guess this is normal and natural for a project that is so significant for so many. This is not my project. This is the project of every person who has suffered from thalassemia, living and deceased.
 
I am but an infinitely tiny toggle on this 'Hope for a Cure' ship.
 
The invention of 'Thalagen' is a guarantee of a cure for the 1,000,000 or so people affected by thalassemia in the world (notice I did not or avoid using the word thalassemic as we are not thalassemics but people affected by thalassemia).
 
Thalagen once inserted into the patients stem cells will make the 'beta globin gene'.
Thalagen has cured 5 generations of Thalassemic mice and created 'genetic expression' (tiny amounts) in two monkeys of the 'human beta globin gene'.
 
Our greatest challenge is called 'titre'. It basically means getting into enough of the patients cells with the right copy of titre.
 
The Italian Telethon has recently donated 500,000 to help this problem. Cooley's Anemia International of Ron Capano in New York has donated 250,000 towards this challenge. The Giambrone Foundation of Italy (National Foundation) has donated hundreds of thousands towards the project.
 
Presently there are people collaborating on this project from Singapore on the east to Los Angeles on the west. Michel Sadelain and his open honest way of approaching life has made it possible to assemble an 'All Star' team.
 
We have a dozen researchers in New York, Farid Boulad, Lila in Thessaloniki, Franco, Aurelio in Italy, doctors in Los Angeles, Washington and Singapore who are working on this project.
 
We see the shore!! We see the shore!!
 
I over the years have been contacted by hundreds of hopeful patients. Recently I have been contacted by one who is very sad and down. He is suspicious of the project's possibility to succeed. He is afraid that funding is insufficient and that we will sit here in the bay without ever docking.
 
I can only say to him and all of us that there are no guarantees in life.
 
My son Rocco is now 18. I have been working towards the goal of a 'Cure' for 16 years. October was my anniversary as he was diagnosed in October of '92'.
 
Literally millions have gone into this project. We have all participated. I have tried to shore the project up with a company and thanks to this effort we have seen significant support.
 
Investors in Bio-tech are rare and extraordinary people of vision, hope and willingness to put their resources where their heart is.
 
Thousands have now bought music from my 'myspace' site, Amazon, CD Baby, ITunes etc. In fact September saw over 7,000 dollars raised for our mission from the music alone with almost 2,000 actual purchases.
 
We are presently writing the IND. It should be done by February. As soon as the FDA gives us the go ahead, which I envision being April we will begin to make the 'Thalagen' needed for the first 5 patients. My son WILL NOT be in the first 5 patients.
 
The patients will be picked by Doctors Bouland and Tisdale at Sloan Kettering and at the NIH. We envision no problem in finding patients as the initial therapy will be very 'friendly' with little or no side effects.
 
The first patients should begin in the summer of '09'.
 
Our goal is 'Genetic Expression' of Thalagen, the beta globin gene.
 
If we achieve this it will be literally earth shattering as it will mean hope not only for us but for all people who suffer from 'Genetic Disease'.
 
Clinical Trials usually go in 3 phases, PHASE 1, 2 and 3.
 
Phase one is about 'doing no harm' to the patient.
Phase two is about 'seeing if it works'.
Phase three is about 'dosage'.
 
Please forgive me for my simple language. I was forced into the military at 16 by a judge in Chicago and I never received a high school diploma.
 
We believe that we will go from Phase one and then immediately into Phase three. We believe this because we are convinced that the first patients will see 'Gene Expression' of Thalagen the beta globin gene. This is great news.
 
People have asked about centers and cost. Well, initially the cost will be that of a Bone Marrow Transplant, since in essence it is an autologous BMT.
 
The centers will initially be in the US but then quickly spread out to Singapore, Italy and Greece.
 
We have helped and will continue to help anyone that we can. We recently assisted a Romanian woman and her son who was feared to have G6PD. Doctor Luzzato of Genova resolved the problem.
 
I will personally do what I can to help all patients who hope for the cure. I like to think of myself as a sensible man with the interest of my large family (Orphan Disease) at heart.
 
Rumors that we are out of cash (could always use some) to continue... Rumors that in France (where there was some sort of experimentation going on in no way affiliated with us) that things have failed... Rumors that the treatment will be given only to a select few to people on some remote island in the South Pacific... Rumors that this is all a 'get rich quick' money making scheme... are all that people, rumors.
 
I can not control what people think or say. (The Bush Regime tried and failed). Nor do I want to.
 
To these rumors, I say.
 
Funding
 
We need funding. I will continue to look under every rock with my management and investment team. We will accept donations and investments. If I have to hold a cup on the freeway, accept a sheik from  ABU DABI, as an investor who has 30 wives (be nice if he had a few extra for me) or rob a cheese store in Italy... WE WILL GET INTO PATIENTS. Case closed. I hope that this is clear.
 
France
 
For years I have heard of and about a project reportedly in France. I have known researchers who have worked on this project. I have known the CEO of the company that claims to support the project. It makes little sense for me to speak about things that are not crystal clear and open. This experiment was NOT done in a way that I would have done things. Yet all experience can be beneficial. By the way 'Michel Sadelain' is French.
 
 
Accessibility
 
When and IF the treatment is available, I will do my utmost as I believe everyone on the team will do to get it to as many people, wherever possible. If some sheik  has a child affected by thalassemia and some extra wives, I will also work with him. Realistically, not all is always possible. We are already collaborating with people in Los Angeles, Minnesota, Washington, Singapore etc... By the way, I've never been to an island in the South Pacific.
 
Get Rich Quick Scheme
 
According to the Wall Street Journal it takes 18 years and 800,000,000 USD average for a drug to get to market. Michel invented this project 19 years ago. If the project is successful, people who invested may see profit. God bless them. We couldn't have done it without them and their willingness to risk. By the way, I don't see anything quick about this. I personally will invest my life in 'Orphan Disease Research'. If any profit is made by anyone it will also profit research.
 
Bad news... My third CD, 'Orphan's Hope' will be out in the beginning of 2009.
 
This is all.
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Andy

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Sharmin
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« Reply #14 on: November 06, 2008, 09:36:03 AM »

Andy,

Thanks for putting this up - this is after all what we all want and what we should be supporting. 

Sharmin
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Sharmin
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