A drug under development for 15 years by a team of Long Island researchers has been approved by federal drug regulators for an unusual hand deformity, the first nonsurgical treatment for the condition, doctors said Thursday.
The condition, known interchangeably as Dupuytren's contracture or Dupuytren's disease, contorts the hands and makes even simple tasks - holding a glass, washing up or shaking hands - virtually impossible. In some instances an affected hand may take on the appearance of a claw.
It is caused when collagen - a protein - develops first as a nodule in the palm, then spreads upward in a cord-like structure attaching to one or more fingers, contorting the entire hand.
Drs. Marie Badalamente and Lawrence Hurst of Stony Brook University Medical Center tapped the properties of a natural enzyme they painstakingly isolated in their laboratory, to develop the medication they call Xiaflex. The drug is manufactured by Auxilium Pharmaceuticals Inc. in Malvern, Pa.
With a few injections, they said, the cord breaks apart, freeing the fingers of the affected hand. Historically, the condition has been treated surgically, requiring a long and often painful recuperation.
"Of course we're excited," Hurst said Thursday, referring to the medication's approval by the U.S. Food and Drug Administration. An FDA advisory panel, made up of outside experts, had earlier recommended approval in a unanimous vote. The medication is expected to become standard care for Dupuytren's because it is noninvasive.
"Although it's a natural enzyme, we developed it from our laboratory bench," said Badalamente, adding the grueling work continued beyond the lab, involving "many, many years of clinical trials."
Hurst said Dupuytren's contracture, a genetic condition, generally is not painful but does impinge significantly on a person's quality of life. It usually occurs after age 50 and affects as many as 27 million people in the United States and Europe. About 3 percent to 6 percent of all white people develop the condition, but it is seen to a lesser degree across all ethnic groups.
History - and fiction - are replete with those affected by Dupuytren's. President Ronald Reagan had it, as did British Prime Minister Margaret Thatcher. Children's author J.M. Barrie may have used his own experience with Dupuytren's to develop the character Captain Hook, the villain in "Peter Pan."