News Article on Pat Girondi. Gene Therapy Trial Closer.

This is the latest update from Pat Girondi.

Hi buddy,

All well... The vector is being made as we speak.

Pat also sent a link to a news article about him and his efforts to make it all happen.

http://www.chicagotribune.com/health/chi-orphan-disease-dad-city-zonejul01,0,5516873.story

Businessman a father figure for 'orphan' diseases
Dad champions research for rare, little-known conditions

By Agnes Jasinski | Special to the Tribune
    July 1, 2009

When Patrick Girondi got into trouble with the law as a teenager, he enlisted into the Air Force to get his life on track. When he had few options as a young adult with no high school diploma, he worked his way up from runner to trader at the Chicago Board of Trade. And when his son Rocco was diagnosed with a rare blood disease at age 2, he left trading for the pharmaceutical industry.

Seventeen years later, Girondi believes a breakthrough is near for thalassemia, a disorder afflicting Rocco and about 100,000 newborns worldwide each year, in which insufficient hemoglobin is produced to carry oxygen throughout the body. Thalassemia patients on average survive to their late 20s, Girondi said.

"I promised God a long time ago, 'If you help me with Rocco, I'll do my best to help others,' " he said.

Rocco has a severe form of the disease and must have blood transfusions every three to four weeks and pills that work to deplete the excess iron in his system, a result of the transfusions. A year after his diagnosis, he was hospitalized for 40 days to undergo experimental treatments, a $75,000 cost not covered by insurance, Girondi said.

Girondi, who is from the Bridgeport neighborhood, founded the Chicago-based biotech company Errant Gene Therapeutics (EGT) in 2003, and the company has been working to develop products to treat "orphan diseases" like thalassemia. He splits his time between Chicago and southern Italy, where experimental treatments have been more available as the disease affects more children of Mediterranean descent.

An orphan disease is described by the National Institutes of Health as one with fewer than 200,000 cases nationwide. To promote more funding for treatments for them, Girondi created the Orphans Dream Foundation in 2007. About $35 million has gone into the project, he said, with much of the money coming from parents with children suffering from orphan diseases and his fellow traders.

Girondi has high hopes that the answer to thalassemia lies in a controlled virus developed by Michel Sadelain of Memorial Sloan-Kettering Cancer Center in New York City. The first clinical trials of the resulting drug, which would be produced by EGT, may be tested on the first human patients by the end of this year, Girondi said.

Christopher Ballas, the director of gene therapy with EGT and an assistant research professor at Indiana University's School of Medicine, said the controlled virus will be used as a carrier to deliver what the patients are lacking. If it works, the technique used for thalassemia could be used to treat other orphan diseases, including sickle cell anemia, Ballas said.

"Here we have a chance to really do something that is of major benefit to a lot of folks who might otherwise be overlooked," Ballas said.

Girondi hopes the drug will be available to Rocco within two years, but the process of gaining approval for its use is not easy. Still, he already has passed two major hurdles that exist for orphan diseases: funding and attention for diseases that are so rare that research is often focused elsewhere.

His most rewarding moments come from meeting with parents. Tracy VanHoutan was introduced to Girondi through a mutual friend after VanHoutan found out his son had the rare nervous system disorder Batten disease.

Girondi then introduced VanHoutan to the leading researcher on the disease. While funding has stalled on clinical trials for Batten disease, VanHoutan is grateful.

"In a time when I was lost, after we got the diagnosis, Pat was an incredible resource," VanHoutan said.

Girondi is more modest.

"I'm just the guy in the middle," he said.

With the vector now being produced, we are now very close to the start of Dr Sadelain's gene therapy trial. Unlike much of the nonsense we hear about cures from companies that offer no proof that their methods can do anything other than empty bank accounts, Dr Sadelain's work is very real. I wish him the best as we move towards this historic date. Anyone with 12,000 Euros to spend on untested and unproven therapies would be far better served by donating to this very real work. Please see the link at http://www.thalassemiapatientsandfriends.com/index.php?topic=2559.msg23383#msg23383 if you want to donate any amount.

Best Of Luck PAt , Dr.Sadelian and all the teams working on this project... loking for this to happen in near future ..

Best Regards
Umair

 

Praying......


Zaini.

Andy,

  Is Dr Sadelain  in a different work lab than Dr Stamatoyannopoulos? In which clinical phase are Dr Sadelain's trials?

Lena.

Lena,

Dr Sadelain is working at Sloan Kettering in New York City. Dr Stamatoyannopoulos is working in Seattle, Washington. Dr Stamatoyannopoulos was the moderator at the gene therapy conference in NYC in February, where Dr Sadelain gave his talk. They have worked towards production of the vector for many years. There has been a large team of people responsible for the different phases which have brought them to the point where the vector will be used in a human in trials in the US this year and both doctors have played key roles in this work. I have heard that the trials will eventually take place in more than one location in the US and Washington may be one of these locations.

Thanks Andy,

Dr Stamatoyannopoulos, as you may know, is Greek and he took part in the Panhellenic Conference held in Greece in May for Thalassaemia Day. He announced that phase two clinical trials on a human are to be made by the end of next year here in a hospital in Thessaloniki (the one and only hospital in Greece where his team is working). It is good, though, that there is another team as well on gene therapy projects.
I am sure both scientists excell on their work and we are all holding our breath waiting for results.

Lena.

that's a good news.

 

Hopes are high. Thanks for the update.