In addition, laminin-111 could potentially benefit any patient with Duchenne, which is not only the most common type of muscular dystrophy, but also the most common lethal genetic disease among children. By comparison, the most promising drug in the pipeline for DMD patients is probably
PTC124, which is aimed at a type of mutation that only accounts for 10 to 15 percent of cases. "Theoretically, laminin-111 protein therapy should be able to treat all patients with Duchenne muscular dystrophy," Burkin says.
Of course, there are still some big "ifs." Since the mice in this study were treated before they began to show symptoms, the next step is to find out if the therapy can halt disease progression after it starts. "Most of the patients who are diagnosed with Duchenne MD, they're diagnosed at periods between three and five years of age, and muscular dystrophy has already begun," Burkin says.
The University of Nevada has licensed its patent for laminin-111 to a Boston biotech startup called Prothelia, which is making arrangements to produce a human version of the protein for safety testing and possible clinical trials.
The small company's founder, Brad Hodges, was a fellow researcher with Burkin at the University of Illinois early in both their careers. Hodges worked at a large biotech company from 2000 until 2007 until he decided that "working at a big company was too safe." He wanted to develop therapies for muscular dystrophy patients. "Large companies typically don't develop drugs," Hodges says. "They typically buy them from small companies that take the early-stage risks.
"I set myself a goal," he says. "I'm going to get at least one drug approved for muscular dystrophy. I'm going to do it; it needs to be done. Patients deserve it and there's a critical need." Hodges cashed in his 401k and began "living on the edge."
As he looked around for candidates, he kept abreast of Burkin's research, and laminin-111 is now the first product in the company's "pipeline." Hodges credits the Parent Project Muscular Dystrophy, or PPMD, for supporting him as he wrote grant proposals under NIH's Small Business Innovation Research awards program. "I wake up every morning so excited — and a little bit frightened at the same time," Hodges says.
In addition to Burkin's latest results, Hodges recently received the good news that Prothelia will receive $250 thousand from NIH in June that will support its efforts for six months. He says it will take twice that just to get safety studies started.
But most importantly, he says, "I will have to demonstrate to myself, as well as to everyone else, that this drug is for real."
This research was published in the Proceedings of the National Academy of Sciences early online edition the week of April 20 - 24, 2009; American Journal of Pathology, January 2009, and was funded by the National Institutes of Health (NIAMS & NINDS).
Elsewhere on the Web:
Parent Project Muscular Dystrophy
Muscular Dystrophy Association: "Helping Jerry's Kids"
Limb-Girdle Muscular Dystrophy Gene Therapy First to Show Promise Beyond Safety
Update on Clinical Trial of Lab-Engineered molecule for Duchenne treatment