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Patient-Led Studies Emerge For ALS

Posted on: Friday, 28 November 2008, 12:25 CST

Once Alan Felzer, a 64-year-old grandfather, was diagnosed with Lou Gehrig's disease (ALS), he sits strapped to a wheelchair, only moves a little, and his voice has been restricted to just a whisper.

The fatal disease steals the ability to move, speak and ultimately breath.  However, rather than succumb to despair with the illness, Felzer turned into his own medical researcher and his own guinea pig.

There have been dozens of ALS patients testing treatments on their own instead of waiting for the slow pace of medical research, according to an Associated Press report. These people are part of an emerging group willing to share intimate health details on the Web in hopes of making their own medical discoveries.

Doctors warn that such patient-led research lacks rigor and may lead to unreliable results, false hopes and harm to patients.

"The Internet is a wonderful tool, but you know, its buyer beware," said Dr. Edward Langston, immediate past chairman of the American Medical Association's board.

In Felzer's case, the drug he tried did no good, however he and his family felt they had little time and little to lose trying.

"ALS is such a short illness," said Felzer's wife, Laura. She helps her husband communicate using sign language with his one good hand when his slow, halting words become difficult to understand. "You want to do what you can as fast as you can."

Only one drug has been approved by the U.S. Food and Drug Administration to combat ALS symptoms.  It only works on some patients and its effects are limited.

Italian scientists reported that every ALS patient given the drug lithium, commonly used to treat bipolar disorder, saw the disease's progress slowly.

Many ALS patients persuaded their doctors to prescribe lithium "off-label," an unapproved practice by the federal drug regulators, but a common one when patients face terminal illness.

Despite the risks, Langston of the AMA pointed out that doctors often stumble upon treatments, and patients could possibly do the same. "If patients are willing to share their experiences, that may in fact occur," he said.

Felzer began taking lithium in January, and his scientifically minded family reached out to other ALS patients. "All those people are taking it anyway," said Alan Felzer, whose smile remains bright and his gaze sharp even as the rest of his body fails him. "So it only made sense to keep track of what was happening."

Felzer's daughter Karen, a U.S. Geological Survey earthquake researcher, lead the ALS lithium project.  Her partner in the effort was Humberto Macedo, a 42-year-old computer systems analyst, father of six and and ALS patient in Brasilia, Brazil.

Macedo said the study grew naturally out of the strong reliance of ALS patients on one another for information.

Felzer and Macedo worked online to recruit nearly 200 patients worldwide to take a specific lithium dosage and answer standard surveys to gauge their symptoms. They then began running their study through a website called Patientslikeme.com, using it to attract volunteers and track their progress.

Patients share information on the site about their symptoms and the drugs they are taking.  The site focuses on conditions that have stubbornly resisted medical science, such as ALS, Parkinson's and multiple sclerosis.

The hope for the site is that professional and amateur researchers alike will dip into the resulting pool of data and emerge with insights that lead to better information.

"My ultimate frustration that drove this site into existence was an overall feeling that there was a lack of transparency and speed or urgency" by the medical system, said Jamie Heywood, who co-founded PatientsLikeMe months before his own brother died of ALS.

Heywood also hoped lithium was the answer he had been seeking.

However, after six months, none of the 87 people who stuck with the study showed any sign of relief in the disease's progress.  Karen Felzer now doubts the Italian study's results.

"It's obvious to everyone it's not the miracle drug we thought at first," she said. She also thinks other tests of lithium for ALS should be halted to spare patients the drug's possible side effects, such as tremors, weakness and difficulty breathing. Her father stopped taking the drug, though Macedo is continuing.

A Harvard Medical School professor, Dr. Merit Cudkowicz, is going to begin a research with lithium in December with 84 patients.  She will stick to the gold standard of research, in which each patient will be randomly chosen to take the drug in question or a placebo, and neither patient or researchers will know who got the drug to avoid introducing bias.

Because the patient-led lithium study lacked those tight controls, it is unreliable as a measure of safety and effectiveness, Cudkowicz said. With an incurable disease, she said, "You don't want to be throwing something away that works because of a bad study."

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On the Net:

• American Medical Association
• Patientslikeme.com

Source: redOrbit Staff & Wire Reports

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