Company Ordered To Give Experimental Drug To Teen
A federal judge has ordered PTC Therapeutics to provide an experimental drug to a 16-year old Minnesota boy who has Duchenne muscular dystrophy, a rare terminal form of the genetic, degenerative disease that mostly affects young boys.
U.S. District Judge William J. Martini heard arguments on Tuesday, and ruled Wednesday as Jacob Gunvalson sat in a wheelchair next to one of his lawyers, with his parents sitting nearby.
Those who suffer from Duchenne muscular dystrophy typically die in their 20s because of weakness in their heart and lung muscles. There is no known cure for the disease, but the PTC drug is being investigated as a possible treatment.
The South Plainfield research company plans to appeal the ruling by U.S. District Judge William J. Martini, but was denied putting the process on hold during the appeal.
Federal regulations prevent the teen to begin taking the drug immediately, so the ruling offers no immediate help for Jacob.Â
Gunvalson’s parents, John and Cheri, claim that PTC led them to believe the teen could take part in a clinical trial of the drug, known as PTC 124, and then later went back on its word. However, PTC said that no promises were made, and that allowing the teen to participate in the clinical trial would be unsafe and would set a bad precedent that could hamper research.
"It’s clear to me that if the plaintiff, Jacob, was denied this relief, he would suffer irreparable harm," Judge Martini said.
The Gunvalsons, who live in Gonvick, MN, believe PTC’s experimental drug holds hope as a potential treatment for Jacob.Â
"His condition has already deteriorated significantly in the past year."
At the center of the dispute is a clinical trial of PTC124 that included a 28-day preliminary phase in 2005 and a 96-week phase that will begin shortly.
The Gunvalsons claim that PTC employees, including senior vice president Claudia Hirawat, who once hosted them at her house overnight, promised them their son would have access to PT 124 despite the fact that the medication he was taking at the time excluded him from participating in the preliminary trial.
They later learned that Jacob would be permitted to take part in the 96-week trial because he hadn’t participated in the preliminary trial.
The judge said that despite conflicting accounts of what was said to the Gunvalsons, he found the company was particularly close to the family.
"They had a special relationship that this court considers more than typical," Martini said.
Stuart Peltz, PTC’s president and CEO Stuart Peltz, flatly denied the claim.
"In fact, on the very night Mrs. Gunvalson and her son were staying at the home of a PTC employee, another patient’s parent was staying with her as well," he said.
"In contrast to big pharmaceutical concerns, it is quite natural for our team to form close relationships with patients and other members of the rare disease community," Peltz said in a statement.
The statement described the company as a small startup with no products currently being marketed.
"Our relationship with the parents of patients should be taken as evidence of our commitment to the community, rather than as evidence of some promise of special treatment," Peltz said.
PTC also maintained that U.S. courts have already decided that terminally ill patients have no right to receive unapproved drugs.
Questioning the urgency of the matter, PTC lawyer John G. Hutchinson argued for a stay of the ruling.  The Gunvalson’s were told in January that Jacob would not get PTC 124, but did not sue until July, Hutchinson said.
However, Gunvalson lawyer Marc E. Wolin said the family filed its lawsuit within weeks of receiving a definitive rejection on July 1.
Peltz said allowing individual exceptions while developing drugs is counterproductive and could delay the eventual approval of the drug.
"If we do, there is serious risk no patients will want to participate in our current clinical trials, in which they might receive a placebo rather than PTC124," he said.
The company would have to take a number of steps to add Jacob to the trial, including making filings with the FDA, something Hutchison said could take at least six to 10 weeks.
Wolin said Jacob’s prognosis is unclear, but noted that March 2007 was the last time the teen was able to walk.
"It’s clear that he’s getting weaker as it goes on," he said.
Cheri Gunvalson was part of a successful initiative that persuaded Congress to boost funding to find a cure for the disease. She partnered with the late Sen. Paul Wellstone (D-MN) in 2001 for passage of the MD CARE Act, which increased federal funding to study the disease.
The legislation established several "centers of excellence", now renamed "Wellstone Centers", for conducting muscular dystrophy research.
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Image 2: Translation of an mRNA into protein: comparison of normal translation, premature translation termination, and treatment with PTC124 restoring synthesis of full-length protein.
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