updated 8/20/2008 8:02:05 PM ET 2008-08-21T00:02:05

A federal judge on Wednesday ordered a research company to supply an experimental drug to a 16-year-old Minnesota boy who is terminally ill with a rare form of muscular dystrophy.

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The decision offers no immediate relief to Jacob Gunvalson. Due to federal regulations, the teen cannot begin taking the drug immediately.

PTC Therapeutics plans to appeal the ruling by U.S. District Judge William J. Martini, who also denied the company’s request to put his decision on hold while it was appealed.

Jacob’s family contends that PTC, of South Plainfield, led them to believe he could participate in a clinical trial of the drug, which is being investigated as a possible treatment. But they say the company then went back on its word .

The company maintained that no promises were made. It said that allowing the teen to join the clinical trial would not be safe and would set a bad precedent that could hinder research.

PTC also argued that U.S. courts have decided that terminally ill patients have no right to get unapproved drugs.

Martini, who heard arguments on Tuesday, ruled from the bench on Wednesday while Jacob sat in a wheelchair next to one of his lawyers, with his parents sitting nearby.

“It’s clear to me that if the plaintiff, Jacob, was denied this relief, he would suffer irreparable harm,” Martini said. “His condition has already deteriorated significantly in the past year.”

Jacob suffers from Duchenne muscular dystrophy, a genetic, degenerative disease that mostly affects young boys. Typically, those who suffer from it die in their 20s because of weakness in their heart and lung muscles. There is no known cure, but the Gunvalsons believe the experimental drug holds hope.

The dispute centers around a clinical trial of PTC124 that included a 28-day preliminary phase in 2005 and a 96-week phase that is about to begin.

Parents say company promised the the drug
Gunvalson’s parents, John and Cheri, claim that PTC employees — including senior vice president Claudia Hirawat, who once hosted them at her house overnight — assured them that Jacob would have access to the drug even though the medication he was taking at the time excluded him from taking part in the preliminary trial.

The couple says they later discovered that Jacob could not participate in the 96-week trial because he hadn’t participated in the preliminary trial.

The Gunvalsons live in Gonvick, Minn., about 220 miles northwest of Minneapolis.

The judge said that while he has conflicting accounts of what the family was told, he found the company was particularly close to the family.

“They had a special relationship that this court considers more than typical,” Martini said.

PTC president and CEO Stuart Peltz denied that, asserting, “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.”

“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 that described the company as a small startup. It has no products 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 lawyer John G. Hutchinson, in arguing for a stay of the ruling, questioned the urgency of the matter. The family was told in January that Jacob would not get the drug but did not sue until July, he said.

Gunvalson lawyer Marc E. Wolin said the family filed its lawsuit within weeks of when it got a definitive rejection on July 1.

Peltz said allowing individual exceptions while developing drugs is counterproductive and could delay getting approvals for the drugs. “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 provide the drug to Jacob, including making filings with the FDA, which Hutchison told the judge might take at least six to 10 weeks.

Jacob’s prognosis is not clear, Wolin said, adding that teen was last able to walk in March 2007. “It’s clear that he’s getting weaker as it goes on,” he said.

Cheri Gunvalson helped persuade Congress to significantly boost spending to find a cure for the disease. In 2001, she teamed with the late Sen. Paul Wellstone, D-Minn., for passage of the MD CARE Act, which increased federal funding to study the disease. The legislation established several “centers of excellence” — since renamed “Wellstone Centers” — for muscular dystrophy research.

Copyright 2008 The Associated Press. All rights reserved. This material may not be published, broadcast, rewritten or redistributed.

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