A federal judge has ordered PTC Therapeutics to supply an experimental drug to a 16-year-old Minnesota boy who is terminally ill with a rare form of muscular dystrophy. However, the decision offers no immediate relief to Jacob Gunvalson, because the teenager can't begin taking the drug immediately, due to federal regulations, theAssociated Press reports.
PTC Therapeutics plans to appeal the ruling by US District Judge William Martini, who also denied the drugmaker's request to put his decision on hold while it was appealed. The boy's family contended that PTC led them to believe he could participate in a clinical trial of the drug that is being investigated as a possible treatment, but that the drugmaker went back on its word.
For its part, PTC maintained no promises were made, and argued that allowing the teen to join the clinical trial would not be safe and would set a bad precedent that could hurt researchers.
"It's clear to me that if the plaintiff, Jacob, was denied this relief, he would suffer irreparable harm," Martini ruled today, while the teenager sat in a wheelchair next to one of his lawyers. "His condition has already deteriorated significantly in the past year."
The teenager 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, according to the AP.
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. Gunvalson's parents, John and Cheri, claim that PTC employees - including senior vp Claudia Hirawat, who once hosted them at her house overnight - assured them their son 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.
Later, they discovered that Jacob could not participate in the 96-week trial because he hadn't participated in the preliminary trial. 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.
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 boy's mother, 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.
Source: Associated Press