Lawsuit Accuses Sloan Kettering of Delaying Gene Therapy for Rare Blood Disease By ANDREW POLLACK OCT. 15, 2015From his days as a troubled teenager on the gritty South Side of 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 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 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 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.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 B. Thompson, has ties to Third Rock Ventures, the venture capital firm that bankrolled Bluebird. Third Rock also financed Agios Pharmaceuticals, a company of which Dr. Thompson is a co-founder.The main evidence offered by Errant Gene is that its relationship with Sloan Kettering started to sour just as Dr. Thompson took office in late 2010.Continue reading the main storyRELATED COVERAGEEye Treatment Closes In on Being First Gene Therapy Approved in U.S. OCT. 5, 2015Huso Yi, the director of research at the Chinese University of Hong Kong Center for Bioethics, said of China’s gene modification work, “The consensus among the scientific community is, ‘not for now.’ ”A Scientific Ethical Divide Between China and WestJUNE 29, 2015Michael Miller, Gilead’s director of clinical virology, in the company’s antiviral lab in Foster City, Calif. Money is flowing into the biotech industry as never before.Riding High, Biotech Firms Remain WaryJAN. 18, 2015Jeanne A. D’Agostino, a spokeswoman for Sloan Kettering, said the accusations regarding Dr. Thompson were “utterly without foundation.” She said the cancer center had 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 clearly 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 Mr. Girondi because he lacked the necessary money and the expertise.“He’s kind and well-meaning — and in way over his head,” Mr. Maslow said. He said the narrative in Errant Gene’s lawsuit was often “distorted and not true.”Mr. 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. (Mr. Girondi also writes and performs his own music.) “How easy is that to discredit?”Mr. 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 F. 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,” Mr. Capano said.Mr. Girondi’s rags-to-riches story seems meant for television — and he told it years ago on Oprah Winfrey’s show. He said he was arrested numerous times as a youth but avoided prison by joining the Air Force. He entered commodities trading almost by accident, eventually starting a successful company called Bridgeport Securities.He bought a Rolls-Royce, ran for clerk of Cook County in 1986, losing badly, and was named a most eligible bachelor by Playgirl magazine.In 1989, Mr. Girondi said, he abruptly moved to Italy to escape a federal investigation of the commodities exchange that led to some convictions, including of one of his employees. Mr. Girondi, who was never accused of wrongdoing, still lives much of the time in Altamura, Italy.In 1992, he learned that 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.Mr. Girondi began scouring the world for drugs. For a time he ended up working with John Walton, a son of Walmart’s founder, who had a child with a different disease.In 2000, Mr. 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.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. Mr. 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 L. Wiech, a biochemist and pharmaceutical executive who crossed paths with Mr. Girondi and was won over, agreeing to be a co-founder of Errant Gene.But Mr. 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 that the therapy had to be improved and Dr. 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.Dr. Sadelain declined to comment for this article. He has told people, however, that he is frustrated that 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,” Mr. Maslow, the former head of industrial affairs.Mr. Girondi’s personal style did not help. Mr. Wiech, who retired from Errant Gene several years ago, said that while Mr. Girondi could have scientists “rolling in the aisles” with stories from his old neighborhood, he also dressed too informally and could be impolite at times.Flashes of the old neighborhood do come out. In one email to a reporter, Mr. Girondi said of Sloan Kettering, “When I grew up, this kind of behavior would have got someone baseball batted.”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 what the attacks were, but Errant Gene once sent letters to every member of the cancer center’s board accusing Dr. Thompson of having a conflict of interest.Mr. 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.Sloan Kettering has treated three or four patients, with treatments starting in late 2012. In one patient the need for transfusions was reduced by 50 percent, it reported.Bluebird, by contrast, has reported on the treatments for seven patients with beta thalassemia and one with the more common sickle cell anemia, which can also potentially be treated with the same therapy. Some of the patients seemed to be freed of the need for transfusions.Mr. Girondi argued that Bluebird treated less sick patients and used a harsher regimen, one he would not want his son to undergo. Bluebird’s chief executive, Nick Leschly, said that some of the patients treated by Bluebird were suffering from the most severe type of the disease.Bluebird’s stock has lost nearly half its value in the last few months, partly on investor concerns that it will face new competition.Mr. Girondi says he is close to raising money to make more of the therapy and treat more patients. In its court filings, Sloan Kettering suggests it will not stop him, as long as he does not use the cancer center’s money, property or personnel. However, the fact that Errant Gene does not control the rights is a deterrent to potential partners and investors.The cancer center seems to be trying to find a corporate partner itself, according to industry executives. Under the 2011 agreement, Errant Gene will be entitled to half of what Sloan Kettering earns from any licensing deal. So if his lawsuit prods the cancer center to make a deal, Mr. Girondi will profit.Mr. 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.”