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ALS Patients Testing Home-Brewed Drugs

Posted By Prucia Buscell, Thursday, March 28, 2013
Updated: Friday, March 29, 2013

Networking and internet research help patients become increasingly involved in treatment of their illnesses, and people with Lou Gehrig’s disease are providing dramatic examples of medical autonomy. Many are making themselves guinea pigs to test unofficial treatments.

Lou Gehrig’s disease, also known as ALS for amyotrophic lateral sclerosis, is a degenerative disease that damages nerve cells in the brain and spinal cord and leads to loss of muscle control and eventual immobility. Life expectancy after diagnosis is only two to five yeas, and there is no known cure.

"Do It Yourself Medicine,” a story in by Jef Akst tells the story of Eric Valor, a 42-year-old with advanced ALS who helped set up an independent drug trial for himself and other ALS sufferers. Although he needs a ventilator to breathe and cannot move any part of his body except his eyes and some facial muscles, he managed to use his eyes to research the web for information about a new drug and set up a website where he and fellow experimenters could report their data. Clinical trials for a new ALS drug NP001 developed by Neuraltus Pharmaceuticals, were to begin in 2010, but Valor’s debilitated physical condition made him ineligible. Developers hope the drug might slow progression of the disease, so to show any impact Valor couldn’t just stop getting worse. He’d have to start getting better, something not proven with any ALS drug.

Based on their research, Valor and others thought the drug contained 50 percent sodium chlorite, a chemical available online for about $50 a quart. He asked his mother to inject a dilute solution into his feeding tube. More than two dozen patients have done the same, and shared their experiences and data. They used a site at PatientsLikeMe, a company cofounded in 2004 by three MIT engineers, Benjamin and James Heywood and Jeff Cole. The Heywood family had spent years searching for anything that would extend and improve the life of a third brother who had ALS, and the experience inspired creation of a health sharing platform. The goal is to help patients manage their own care, and change the way industry conducts research.

According to a Wall Street Journal story by Amy Docker Marcus, many ALS patients concoct their own drugs because they feel they don’t have time to wait for clinical trials and FDA approval. They are also reluctant to risk getting placebos rather than the real thing in a clinical trail. Some medical authorities worry about that approach. Marcus quotes Jonathan D. Glass, professor of neurology at Emory University School of Medicine, who suggests research needs the rigors and controls set by the medical establishment. He worries that guinea pigs could hurt themselves, adding, "Who knows what they’re actually making in their kitchens?”

Neurologist and researcher Richard Bedlack, who directs the Duke University ALS Clinic, thinks greater patient involvement is a good thing. "There’s a new model of medicine, in my opinion,” he told TheScientist. "Once upon a time we had a very paternalistic system where patients would come…and doctors would ask all the questions and give all the answers. In the past decade, things have really shifted, almost to the other side, where a lot of medicine is autonomous now.”

Results from the independent sodium chlorite trials are equivocal. While some self-dosers reported improvement, a report published by PatientsLikeMe investigators found a potentially negative effect. Neuraltus researchers last October announced its drug showed progress, and it seeks a big-pharma partner for a Phase 3 clinical trial. Valor, for one, would like early access to the drug, and he sees no conflict in being both patient and researcher. "I just treat myself as another lab rat,” he said.

Tags:  buscell  complexity matters  medicine  networks  research 

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