Thousands of patients won't take it: Online drug studies using PatientsLikeMe

Karen Felzer's father has ALS (amytotrophic lateral sclerosis), or Lou Gehrig's Disease, so she read with special interest an article about the results of a new drug. Italian researchers had published a small study in February saying that lithium, a drug used to treat bipolar disorder, seemed to slow the progress of ALS.

A scientist herself – she's a seismologist - Karen helped to launch an online study by patients of the effects of the drug. Participating patients use the web site PatientsLikeMe.com. Karen says, "We want to keep track of how people are doing on [lithium]. It's important for the whole community."

Lithium didn't seem to help her father, and he experienced some side effects, so he stopped taking the drug. That is consistent with the reports of other patients, which indicate that lithium seems much less effective than reported in the Italian study in the Proceedings of the National Academy of Sciences.

As a result, says James Heywood, the co-founder of PatientsLikeMe, less than a year after the Italian study was published, "we now have data on over 100 patients who have been on a drug long enough to demonstrate the hope of that drug was not what was originally assumed – and that means thousands of patients won't take it."

Advice to patients interested in clinical drug trials: Look into PatientsLikeMe.

Read another ALS story.

Thanks to Carolyn Johnson for the source article in the Nov. 17 issue of the Boston Globe.

He's hiking 2,000 miles: Lou Gehrig's Disease

Ben Davis became committed to fighting ALS (amytrophic lateral sclerosis, Lou Gehrig's Disease) after watching Bill Jackson, a chef at the restaurant he manages, die from the disease. An endurance athlete, Ben is hiking the length of the 2,175-mile Appalachian Trail to raise money to fight ALS.

He is going south toward the end of the trail in Georgia. You can follow his progress at www.2175forALS.com

Advice: Get friends like Ben Davis.

Read another ALS story.

Thanks to George Vecsey for the source article in the Nov. 8 issue of the NY Times.

What do I have to lose?: Experimental stem cell therapy

I have been tossing and turning all night wondering where I get my strength, but I dig deep and find the answer, always, somewhere in my mind's abyss. A friend told me yesterday [now a year ago] that this will be my calling when I get better to breathe life back into people and talk about my experience with this illness and my journey to get better.

Drew Schemera, 35, wrote this in his blog, The Journey, a year ago before going from his Connecticut home to Beike Biotechnology in China for experimental stem cell therapy, six months after being diagnosed with amyotrophic lateral sclerosis (ALS, or Lou Gehrig's Disease), and being given a life expectancy of 2.5 years.

He feels he did the right thing. There were "no clinical trials in the USA of any significance and no cure on the horizon," he wrote. "I made a choice to come to China and work my butt off in therapy and change my diet, and I'm slowing it [ALS] down. I probably will die from ALS, but I'll be damned if I'm not going down swinging! What do I have to lose?"

However, experts at the International Society for Stem Cell Research and elsewhere caution that professional standards for stem cell therapy have not yet been set. Dr. George Daley, the president of the Society, decries a "misconception by some patients that the cure is already here." He adds, "We need to be clear that the path to cures is a long and arduous one."

Advice: Make your own critical decisions, after careful thought.

Thanks to Neil Munshi for the source story in the Boston Globe of June 13.

A second opinion for Lou Gehrig's Disease

Gaetan Guertin, a native of Quebec, Canada, sought a subspecialist's opinion after his first neurologist suggested the diagnosis of amyotrophic lateral sclerosis (ALS, or Lou Gehrig's Disease). Gaetan began to experience leg fatigue when he was 55 and soon developed foot weakness and muscle atrophy. In an interview with Neurology Now, Gaetan said the expert's approach was more confident. "She had a lot of experience and knowledge and was more open,” he said. He added that since the neurologist worked at a research and training hospital, the diagnostic testing could proceed more promptly.

Advice: If you're unclear about a diagnosis, consider getting a second opinion.

Browse for related stories in the index at the very bottom of this page, or read a story about a man with Lou Gehrig's disease.

Thanks to Dr. Orly Avitzur for the source article in the November/December, 2007 issue of Neurology Now.

Curt was able to get rid of one curse – let’s see if he can get rid of another: ALS patient advocate

Bruce Statham wasn't dying of Lou Gehrig's Disease, he was living with it. For more than a decade after his diagnosis, he lived on, and became a spokesman abut the disease. He helped raise funds for research on ALS, also called Lou Gehrig's Disease, and helped Harvard Medical Students understand what is was like to live with the disease. He died this week at age 39, of complications from ALS.

He worked with the Angel Fund, a nonprofit that raises money for research at the Cecil B. Day Lab for Neuromuscular Research at Mass General Hospital. There, soon after the Boston Red Sox won the World Series, he was invited to introduce pitcher Curt Schilling, who raises money for the National ALS Association. He said, "Curt was able to get rid of one curse; let's see if he can get rid of another."

Advice for survivors: See if you can help others with your disease by being a patient advocate

Read another ALS story, or read more from Bryan Marquard's story in the October 9 Boston Globe.

ALS Advocate Avi Kremer Wins Award

Avi Kremer, the Harvard Business School graduate who is using his management skills to battle his dread disease, received a top prize from the ALS Association last week.

Avi received the Lawrence A. Rand Prize for "raising awareness about the disease as well as millions of dollars for research in Israel and the U.S." Avi, age 32, was diagnosed with the condition, also called Lou Gehrig’s Disease, during his first semester at Harvard.

Advice: Appreciate Avi’s courage.

Read our earlier blog post on Avi.

Source: Stephen Smith’s article in the July 23 Boston Globe.

He’s turning his disease into a business plan: ALS patient advocacy

He was a platoon leader in the Israeli army, and later was accepted to Harvard Business School. Then came an odd tremor in his right forearm. It persisted, and once in Boston, he was diagnosed with ALS. Now, almost three years after his diagnosis of ALS (Lou Gehrig’s Disease, amytotrophic lateral sclerosis), his wheelchair has a Borat sticker.

"Avi moved very quickly from the anger stage after diagnosis to a very applied, entrepreneurial stage when he said, 'Let’s get on with it. Let’s find the solution. Let’s put the resources on the table to empower research,'" said his neurologist, Dr. Robert Brown of Mass General Hospital. He raised money with a guarantee to donors: If there’s no solution to the problem, they would get their money back. With the help of friends from the business school and neurologists, Avi started a scientific competition called Prize4Life. Scientists will win prize money only after solving crucial questions about ALS that could speed the discovery of drugs to slow the disease. The first question, with a $1 million prize and deadline in November 2008, asks researchers to identify markers of the disease’s progression. So far, Prize4Life’s scientific advisors have selected the five winners of small cash awards in the first round.

Learn more at Prize4Life.

Read another of our hero stories, or read Stephen Smith’s source story in Monday’s Boston Globe.