Well, the 2013 MDA Scientific Conference is over — and what a conference it was. On Tuesday and Wednesday, we heard about different stages of the drug development process — often referred to as “bench to bedside.”
But today’s laboratory benches are a long way from the Bunsen burners and test tubes of previous decades. They’re filled with computers that do everything from sequencing DNA to telling scientists where they are in their experiments. Electron microscopes capture images of cellular structures previous generations only dreamed of, and video cameras allow scientists around the world to instantaneously share their images of how lab animals move or muscle fibers contract.
And thankfully, today’s “bedsides” aren’t what they used to be either. For one thing, most people with neuromuscular disorders aren’t in bed, except at night. Thanks to greatly improved treatments for the cardiac and respiratory problems associated with neuromuscular disease, along with changing social attitudes and advances in disability technology, people with certain neuromuscular disorders are highly likely to be spouses, parents, students, artists, professionals or business people.
Wednesday’s sessions, for instance, were opened by Vance Taylor, a man with limb-girdle muscular dystrophy who describes himself as a husband, father and homeowner. Taylor, who lives in Washington, D.C., is a specialist in national security, a field in which he holds a master’s degree.
When he was 9 years old, a neurologist told Vance that his muscles would weaken and that he would start falling a lot and soon need a wheelchair. With the startled child still in the room, the doctor turned to Vance’s mother, telling her that her son would be dependent on her for the rest of his life.
The doctor was right about one thing: Taylor uses a power chair to get around. But he was wrong about everything else.
Lab benches may have gone beyond Bunsen burners, and people with neuromuscular disorders are reaching adulthood and independence more often than ever before. But that doesn’t mean that it’s smooth sailing for anyone.
Taylor told the audience a story about being trapped in a hole in a sidewalk because he didn’t take the time to go around it. He had the good fortune to be rescued by a strong and helpful pedestrian who happened to be in the neighborhood, despite the lateness of the hour (it was midnight). The man easily lifted the 250-pound chair, with Taylor seated in it, and pulled them out of the hole.
“In retrospect,” Taylor said, “it’s clear that in the events leading up to getting stuck, I had been my own worst enemy. I had let impatience cloud my judgment. I didn’t want to go 3 feet around out of my way. I crashed, and it hurt. That’s how life works. Sometimes we drive ourselves into a mud pit, and sometimes we’re pushed in there.”
John Porter, a program director at the National Institute of Neurological Disorders and Stroke (NINDS) at the National Institutes of Health, echoed these sentiments, but in reference to the challenges and pitfalls facing researchers.
“I try to facilitate things happening,” he told the conferees. “This is hard. There’s lots of effort but, to date, there are very few approved drugs for all neuromuscular disorders combined. The stars need to align. We all have to facilitate that, and today we can see the hope and the promise. Even more with this conference than with the one two years ago, hope and promise are starting to emerge. But we must increase the stringency of the work, and the data sharing.”
Porter continued, “While research is incredibly rewarding, if we are to get neuromuscular disease therapies, drug discovery must begin and end with the patient. Identifying the gene or the polymorphism [gene variant] does not immediately lead to knowledge and therapies.”
He cautioned the audience to beware of quick and easy answers, which can be tempting but unfortunately are wrong all too often. Taking what appears to be the shortest route can often land you in an unexpected hole.
He urged the researchers in the audience to determine carefully the target they want their drug to hit and then to make sure that it was hit and appropriately changed by the treatment; to be aware of safety concerns throughout the drug development process; to make good use of technology, such as new types of tissue imaging and new approaches to protein measurement; and to make use of knowledgeable people and other resources available to them.
In neuromuscular disease research, Porter said, no one has the resources to go from drug discovery all the way to a marketed product on his or her own, and he urged the conferees to be good communicators and good collaborators.
“No researcher is an island,” Porter said. “We need each other.”
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