Lou Gehrig's Disease: Patients Take Research Into Their Own Hands

People with Lou Gehrig's disease are trying drugs and tracking results.

April 16, 2012, 2:34 PM

April 18, 2012— -- Though his body betrays him, Eric Valor's mind is still strong. Diagnosed with Lou Gehrig's disease in 2004, the 43-year-old relies on machines to move, talk, eat and breathe. But that hasn't stopped him from running his own drug trial.

With help, Valor has been injecting sodium chlorite, a chemical used by water treatment plants, into his paralyzed body through a feeding tube. He's convinced it's the active ingredient in NP001, an experimental drug made by Neuraltus Pharmaceuticals Inc. And other patients are following his lead.

"My original plan was to keep it secret until I could report with confidence that it was safe and even marginally effective," said Valor, who started taking sodium chlorite in October 2011. "But the secret got out, so I made a website to try to capture data as best I could."

From a special bed in his Santa Cruz, Calif. home, Valor uses his eyes to control a ceiling-mounted computer -- a design from his days as a computer specialist. He carefully tracks his disease progression and that of more than two dozen other patients taking sodium chlorite with hopes the makeshift drug will buy them all time until NP001, if proven to work, is approved by the U.S. Food and Drug Administration.

But experts say the DIY approach is dangerous.

"It's pretty frightening," said Dr. Jonathan Glass, neurologist and director of the Emory ALS Center in Atlanta, Ga. "I think it's a cry of desperation for these folks, using something not made to strict standards with no evidence it works."

But people with Lou Gehrig's disease, also known as ALS, are eager to find out whether sodium chlorite works quickly and on their own terms. Frustrated with the pace of clinical research, they've joined forces to tackle the best leads on short order, from off-label drugs to stem cell transplants, and now makeshift NP001. It's a far cry from a properly designed clinical trial, which has a control group to weed out the infamous placebo effect. Still, the DIY design puts patients back in control.

"I don't think anyone should interfere with a patient's right to choose their own destiny," said Jamie Heywood, cofounder of PatientsLikeMe, an online community where patients share their experiences with various treatments, including sodium chlorite. "I look at it from a sort of human rights level, the right to life, liberty and the pursuit of happiness. It's not the right to succeed; it's the right to pursue."

Heywood's brother, Stephen, died from ALS in 2006. He was 37.

"We did a stem cell transplant in Stephen, based on data from Parkinson's rats," said Heywood. "It was an intuitive lead. And in our mind, it was worth doing."

But sodium chlorite takes intuition to a new level. Research papers and patent filings suggest the vaguely-named NP001 could be sodium chlorite, but Neuraltus Pharmaceuticals Inc. has not publicly confirmed the drug's formula. Calls to the company were not immediately returned.

The results of a phase 2 clinical trial of NP001 are expected to come out later this year.

Makeshift Treatments: Too Much Information?

The DIY sodium chlorite trial reflects the shortage of effective treatments amidst a sea of promising preliminary data, data once buried in subscription-only medical journals that can now be found online.

"We have more information now but we don't know what it means," said Heywood. "Broad access to this kind of information, and a lack of control over how people use it, will likely result in a death. Someone's going to die making a mistake because information's coming in fast and we don't have the tools to understand it yet."

Patients often are desperate to slow the relentless disease, which usually kills in five years or less.

But Valor said desperation is best left out of treatment decisions.

"Desperation leads to irrational thought and irrational thought leads to irrational action," said Valor, adding that people with ALS often fall victim to snake oil salesmen and scams.

"Desperate people are prime targets for the unscrupulous,'' he said. "This was one of the reasons for the creation of the FDA and the requirements for companies to prove safety and efficacy before allowing sales to the public. Unfortunately, not all bureaucracy is well-suited to all situations, and people with ALS do not have a lot of time."

Valor said patients should research treatments thoroughly by reading studies and talking to experts, including their own doctors. And to those who think they have nothing to lose, Valor says think again.

"Every day is precious to people with ALS and anything that decreases the number or turns them even more painful or bleak, simply must be avoided," he said. "Ask the family of any current or deceased person with ALS if they would like even one more bright and happy day together."

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