When a rare disease strikes one of their own, some families take it upon themselves to find a cure. Unsatisfied with treatment options for the genetic disorder cystic fibrosis, the Flatleys have become one of those families.
John Flatley, a Braintree, MA, real estate developer, is spearheading his family’s effort to find a cure for one of his family members and others with cystic fibrosis through a new nonprofit called CFRx. The nonprofit is being funded by the Flatley Foundation, which was founded by Flatley’s late father, Thomas Flatley, the legendary real estate tycoon and philanthropist.
The mission of CFRx is to discover a potential cure for cystic fibrosis, which causes life-threatening lung and digestive problems, John Flatley tells Xconomy. He says he doesn’t want to publicly identify which member of his family has the disease, to protect that person’s privacy. The nonprofit itself has operated under the radar since he launched it in November 2008 with the help of Richard Fitzpatrick, a former scientist from the vaunted Cambridge, MA-based biotech firm Genzyme.
The nonprofit’s operations reflect some of the shrewd business philosophies of Flatley’s father, John Flatley says. Thomas Flatley, whose personal wealth was estimated to be $1.3 billion by Forbes in 2005, was known for being involved in most all details of his real estate empire. John Flatley has applied some of the business lessons he learned from his father at his own real estate firm, John Flatley Company, and now at his drug-discovery nonprofit. Rather than rent expensive labs, he says, he’s built out his own labs with used equipment in the Schrafft Center building in Charlestown.
“The philosophy that I learned from my father is that when it’s your own money, you want to stay involved with the expenses of the business,” Flatley says. “I’m involved, not with the science end of [CFRx], but I’m involved as much as possible with the expenditures.” His family’s strong religious beliefs are also visible in the CFRx’s lab, where a crucifix is mounted where scientists can see it as they do their important work, he says.
Flatley and his family have been long-time financial supporters of the Cystic Fibrosis Foundation, a Bethesda, MD, nonprofit that has helped fund the development of new therapies for patients with the disease, who number some 30,000 in the U.S. alone. The foundation has played a role in prolonging cystic fibrosis patients’ lives, but the median life expectancy for people in the U.S. with the disease is still around 37 years, according to the foundation.
Writing checks to the CF Foundation wasn’t enough for Flatley. In early 2008 he used Monster.com to track down Fitzpatrick, with whom he had played hockey at Milton Academy in the late 1970s, and hired the former Genzyme (NASDAQ:GENZ) chemist to help him explore options to start an organization involved in promoting cystic fibrosis drug research directly.
They considered making venture investments in biotechs developing drugs for the disease, as well as launching a contest with prizes for those who advance potential cures, much like the Cambridge, MA-based nonprofit Prize4Life strives to provide incentives to ALS researchers. (Thomas Flatley died in 2008 after his own battle with ALS, a neurodegenerative disease.) Yet Flatley says he believed he could make the biggest impact with an organization that conducts its own drug research, with a business model more akin to the ALS Therapy Development Institute in Cambridge. He says they talked about the idea with John Heywood, the MIT engineering professor whose family founded the ALS research institute.
The effort to launch CFRx won quick support from Bob Coughlin, the president and CEO of the Massachusetts Biotechnology Council, whose son has cystic fibrosis. Coughlin, who was one of the first people Flatley talked to about the organization, put Flatley in touch with Patrick Marshall, another parent of a child with the disease. Marshall has built deep connections in the cystic fibrosis research community through his work as principal at the Portsmouth, NH-based life science headhunting firm Stratacuity. He played a key role in recruiting scientists for CFRx, which now has a full-time staff of six people, with three more hires in the works, Fitzpatrick says.
Flatley says the late-2008 launch of the nonprofit turned out to be good timing. The financial meltdown was a disaster for biotechs that were both in need of cash and lacked product revenue to support their operations. The crisis was felt at several Boston-area biotech firms with cystic fibrosis drugs in development, including Altus Pharmaceuticals, CombinatoRx, and Epix Pharmaceuticals. Last year Altus and Epix went broke and shut their doors, and the cystic fibrosis program at Cambridge-based CombinatoRx (NASDAQ:CRXX) was a victim of major cutbacks at the company. The CF Foundation had invested millions of dollars in the troubled companies to fund their cystic fibrosis research.
CFRx has picked up scientists, equipment, and technology from both CombinatoRx and Epix. Fitzpatrick says that CFRx has been screening numerous molecules in search of potential cystic fibrosis drugs, but the nonprofit has not acquired any of those specific molecules from CombinatoRx or Epix. (Cambridge-based Alnara Pharmaceuticals inherited Altus’s former cystic fibrosis drug through a deal last year with the CF Foundation, which had taken ownership of the asset from Altus.)
CFRx was “able to acquire some assets and some technology that, due to the distress in the financial market, would have been put on hold,” MassBio’s Coughlin says.
Flatley has big goals for CFRx. The nonprofit wants to find a molecule that addresses a defective protein in cystic fibrosis patients that causes them to produce thick, sticky mucus in their lungs and other organs, leading to lung infections and impeding normal digestion. Cambridge, MA-based Vertex Pharmaceuticals (NASDAQ:VRTX) is in late-stage development of a cystic fibrosis drug called VX-770 that targets this faulty protein, but the drug hasn’t been approved and it’s not a cure for the disease. Flatley wants his nonprofit to advances molecules to a stage at which they are ready to be picked up by drug-development companies with the expertise to bring them to market.
CFRx has formed a close collaboration with the CF Foundation. Robert Beall, the president of the foundation, says that his group is working with CFRx on a molecule that was under development at Epix before the Lexington, MA-based company folded. Some of the foundation’s own researchers are also working at in the nonprofit’s lab in Boston, including Martin Mense, who headed cystic fibrosis research at Epix before he became an employee of the foundation, Beall says.
“It’s a great collaboration,” Beall says. “It’s allowed us a laboratory presence outside the academic environment. And it’s going to allow us to move forward with some drugs.”
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