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 TheScientist.com 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
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
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
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.