From his days as a troubled teenager in Chicago, Patrick Girondi has never shirked a fight. Even on his way to making a fortune as a commodities trader, he said, he was fired from one job for “socking someone” on the trading floor. Now Girondi 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.
Girondi accuses the cancer center of dawdling on developing a gene therapy that could potentially cure his son of an inherited blood disease called beta thalassemia, or Cooley’s anemia. The disease 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 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 .
But while the well-financed Bluebird races ahead, the project at Sloan Kettering, in New York City, appears to have languished. In the absence of other therapy, 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.
In a lawsuit in federal court in Manhattan, Errant Gene is demanding to regain control of the project. It accuses Sloan Kettering of sitting on the therapy because the cancer center’s president, Dr. Craig Thompson, has ties to Third Rock Ventures, the venture capital firm that bankrolled Bluebird. Third Rock also financed Agios Pharmaceuticals, a company of which Thompson is a co-founder.
The main evidence offered by Errant Gene is its relationship with Sloan Kettering started to sour just as Thompson took office in late 2010.
Jeanne D’Agostino, a spokeswoman for Sloan Kettering, said the accusations regarding Thompson were “utterly without foundation.’’ She said the cancer center has been moving the therapy forward, with a few patients receiving treatments in a clinical trial. Sloan Kettering is asking the judge to throw out the case, saying the 2011 agreement states that Errant Gene irrevocably surrendered its rights.
Andrew Maslow, who was the director of industrial affairs at Sloan Kettering until early 2012, said the cancer center had to wrest back control of the project from Girondi because he lacked the necessary money and the expertise.
“He’s kind and well-meaning — and in way over his head,” Maslow said. He said the narrative in Errant Gene’s lawsuit was often “distorted and not true.”
Girondi said he was being unfairly written off. “You’ve got an uneducated father of a son who has the disease who does rock concerts,” he said. (Girondi also writes and performs his own music.) “How easy is that to discredit?”
Girondi has supporters, particularly among patient advocacy groups, who wonder why Sloan Kettering has not announced a deal with a different company in the four years since it took back the rights to the therapy.
“He’s a great guy, very driven, as I am, and passionate about this,” said Ronald Capano, who heads Cooley’s Anemia International, which contributed money to Sloan Kettering for the gene therapy project. “Unfortunately we have no idea what’s going on at Memorial Sloan Kettering anymore.”
Starts with a diagnosis
In 1992, Girondi learned his young son had beta thalassemia. (He spoke on the condition his son’s name not be used because the young man has not told many people he has the disease.)
Because of a genetic mutation, people with beta thalassemia do not properly produce hemoglobin, the protein in red blood cells that transports oxygen to the body’s tissues. The disease is extremely rare in the United States but somewhat more common in the Mediterranean region and Asia.
Girondi began scouring the world for drugs. For a time he ended up working with John Walton, a son of Wal-Mart’s founder, who had a child with a different disease.
In 2000, Girondi contacted Dr. Michel Sadelain, a young researcher at Sloan Kettering who had published a paper on his experiments with gene therapy for beta thalassemia.
What is gene therapy?
Gene therapy involves putting a healthy gene into the cells of the body to correct a defective one. Only a year earlier, in 1999, a teenager with a different disease had died in a gene therapy experiment at the University of Pennsylvania, casting a pall over the field.
With no other company interested, the cancer center struck a deal with Errant Gene in 2005. Girondi sought funding from disease foundations and colleagues from the commodities industry, and scientific help from various researchers, some of whom worked pro bono.
“He’s very genuine and generous,” said Norbert Wiech, a biochemist and pharmaceutical executive who crossed paths with Girondi and was won over, agreeing to be a co-founder of Errant Gene.
But Girondi never raised a huge amount of money. And it took until 2010 for Errant Gene to manufacture enough of the therapy to treat several patients in a clinical trial.
One reason things went slowly was the therapy had to be improved and Sadelain was diverted by work on cancer. He helped develop a way to engineer immune system cells to attack tumors. That work contributed to the founding of Juno Therapeutics, an extremely hot biotech company attracting a lot of money and attention, which could bring more than $100 million to Sloan Kettering.
Sadelain declined to comment for this article. He has told people, however, he is frustrated the beta thalassemia project has progressed so slowly.
By 2010, gene therapy was becoming more attractive, and Sloan Kettering apparently realized it might obtain a more lucrative deal with a more attractive partner. Bluebird itself discussed licensing the technology but an agreement was not reached.
Late that year, Sloan Kettering accused Errant Gene of defaulting on the 2005 licensing agreement. To bring in more money, Errant Gene tried to sublicense the technology to Sigma Tau Pharmaceuticals, an Italian company, but Sloan Kettering would not allow it.
“I didn’t want Pat or Sigma Tau because I had better people in mind,” Maslow, the former head of industrial affairs, said.
In a letter that is part of the court record, a lawyer for the cancer center said that Errant Gene’s behavior, including “personal attacks on Sloan Kettering personnel” made further collaboration impossible. It did not specify the attacks, but Errant Gene once sent letters to every member of the cancer center’s board accusing Thompson of having a conflict of interest.
Maslow declined to say what happened with the company he was talking to. But four years later, no partner has come to light. BioMarin Pharmaceuticals, which specializes in rare diseases, tried to buy the rights last year but was turned down, according to the lawsuit. BioMarin would not comment.
Girondi said he had spent tens of millions of dollars, mostly his own, on his quest to cure his son since 1992, including $6 million for the gene therapy. He said he nearly faced bankruptcy in 2012 after some real estate investments soured. To save money, he sleeps in his lawyer’s office when he is in New York.
“I’m just a little guy trying to get my son cured,” he said. “I can’t sit by and watch the world go by and not do all I can to make sure what I believe is the better product pushes ahead.”