Streetwise Father Takes On Sloan Kettering to Save His Sick Son

This is an article about the father of a Thalassemia patient who fights for his son. From his days as a troubled teenager on the gritty South Side of Chicago, Patrick Girondi has never shirked a fight. Although he was fired from one job for “socking someone” on the trading floor. He made a fortune as a commodities trader. Mr. Girondi, a high school dropout, is in a fight of a different kind — against the august Memorial Sloan Kettering Cancer Center over the rarefied field of gene therapy. It is a fight, he said, to save his son.

Mr. Girondi accuses the cancer center. Their dawdling on developing a gene therapy delayed a potential cure for his son. The beta thalassemia, or Cooley’s anemia often kills people by their late 20s — an age his son will reach in a few years.

Ten years ago, when few companies were interested in gene therapy, Sloan Kettering licensed the rights to an experimental treatment to Errant Gene Therapeutics, a tiny firm started by Mr. Girondi. But after being accused by the cancer center of not fulfilling its obligations to move the therapy toward the market, Errant Gene ceded its rights in 2011.

Now, because of technological progress, gene therapy is considered highly promising. A company called Bluebird Bio has a market valuation exceeding $3 billion, largely on the basis of a very similar gene therapy for beta thalassemia and sickle cell anemia, which has had strong results in early clinical trials.

But while the well-financed Bluebird races ahead, the project at Sloan Kettering appears to have languished. In the absence of other therapy, Mr. Girondi’s son gets blood transfusions every 18 days and takes 10 pills daily to reduce the toxic iron that builds up in his blood and organs.

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