A breakthrough drug for a crippling hand disease pioneered by Stony Brook University researchers is now being tested to combat the lumpy skin problem that has long been the beachgoer's nemesis: cellulite.
If approved for cosmetic use, researchers hope the injectable treatment will be a commercial blockbuster that will do for dimpled thighs and backsides what Botox did for the face. Americans spend millions each year on procedures to eliminate cellulite. But medical professionals say existing remedies are ultimately ineffective.
"The commercial potential for a successful treatment is pretty vast," said Marie Badalamente, a professor at Stony Brook University School of Medicine who helped develop the drug.
Approval from the U.S. Food and Drug Administration to treat cellulite with the drug, called Xiaflex, is far from certain. The enzyme-based treatment -- being tested on 63 women in Baltimore -- is in the first of three phases in the FDA approval process, where just 1 percent of all drugs are ultimately sanctioned for use. Final approval will take at least four to five years.
Cellulite is among several conditions for which researchers hope Xiaflex will be used. The drug is in the second-phase of FDA testing to treat "frozen shoulder" syndrome, an agonizing loss of arm motion. And it is in the final stages of the approval process to treat Peyronie's disease, a painful curvature of the penis that can make sex impossible.
The drug was initially developed by Lynbrook-based BioSpecifics Technologies Corp., which worked with researchers at Stony Brook. Expanded use of the drug could be a windfall for Stony Brook and BioSpecifics, which both receive royalties from its sales.
In 2004, BioSpecifics licensed the technology to Auxilium Pharmaceuticals Inc., of Malvern, Pa. The drug's breakthrough came in 2010, when it landed FDA approval to treat Dupuytren's contracture, which contorts fingers and makes it exceedingly difficult to shake hands, grip a pencil or wear a glove. The development allowed a simple injection to treat a condition that long required surgery. More than 20,000 Dupuytren's patients have been treated since.
Xiaflex is an enzyme that dissolves a type of connective tissue called collagen. When it builds up, collagen causes a number of problems. To treat them, doctors inject Xiaflex into the impacted area. "But it isn't like a Pac Man. It doesn't go in and eat everything," said Lawrence Hurst, a Stony Brook professor who has worked on the drug.
Cellulite results from strands of collagen between the skin and muscle. As those strands age, some contract, leaving skin dimpled. Exercise is little help. Laser treatments and creams are no better, most doctors say. So researchers hope Xiaflex will break down those strands, restoring the skin's smoothness. An estimated 85 percent to 98 percent of women have some degree of cellulite, making the potential market for such a drug large.