Stories about: drug pipeline

Duchenne muscular dystrophy: The decade of therapy

A cocktail of approaches is most likely to successfully preserve muscle.
A cocktail of approaches is most likely to successfully preserve muscle.

It’s been 28 years since a missing dystrophin protein was found to be the cause of Duchenne muscular dystrophy (DMD), a disease affecting mostly boys in which muscle progressively deteriorates. Dystrophin helps maintain the structure of muscle cells; without it, muscles weaken and suffer progressive damage, forcing boys into wheelchairs and onto respirators.

Today, a variety of approaches that attempt to either restore dystrophin or compensate for its loss are in the therapeutic pipeline.

“We’re at the point where lots of things are going into clinical trials,” says Louis Kunkel, PhD, who is credited with identifying dystrophin in 1987. “I call it the decade of therapy.”

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Let’s liberate clinical trial data

The Gutenberg press disseminated ideas to a wider society. But in the clinical world, much information is still on "lockdown." (Wikimedia Commons)

The best things in life are free: friends, sunny days, beautiful vistas. Wouldn’t it be nice if knowledge were also free? Historically, libraries promulgated knowledge sharing because it was for the public good. We see this spirit increasingly embraced on the Internet – take the recent announcement of a collaboration between Harvard and MIT to make their courses freely available to users around the world via the edX platform.

But have we made all useful knowledge available in a way that allows for the greatest societal advancement? Not really. According to Ken Mandl, MD, MPH, director of the Intelligent Health Laboratory at the Children’s Hospital Informatics Program (CHIP), one important source of information still on lockdown is clinical trial data. In an article called, “Learning from Hackers: Open-Source Clinical Trials” published this month in Science Translational Medicine (not currently available in full text), Mandl and his coauthors call for making raw, de-identified clinical trial data free to the public.

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Naturally derived medicinals: Where’s the business model?


Kola nut (cola acuminata) from Köhler's medicinal plants, 1887 (via trialsanderrors/Flickr)

It’s widely acknowledged that the pharmaceutical industry is in crisis. Pharma companies are seeing drugs coming off patent, competition from generics, diminished R&D success and increased cost pressures, all of which are contributing to the downfall of the blockbuster drug era. Various solutions have been proposed, including creating shared knowledge repositories to learn from failures and pairing treatments with companion biomarkers.

These solutions and others have merit. But I’d propose more emphasis on what I consider a major failing of the industry: the inability to effectively commercialize naturally derived compounds possessing therapeutic benefit.

Pharmaceutical companies have typically avoided naturally derived compounds because it is difficult to keep competitors out of the territory. Legislation such as the Dietary Supplement Health and Education Act prohibits market exclusivity for natural compounds with known chemical compositions.

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