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Jun 14

The Mystery of ALS Patients Who See Improvement

ALS Headlines
Researchers hope studying so-called reversals could yield new understanding and even treatments

Richard Bedlack has treated more than 2,000 patients with ALS, the neurological condition also known as Lou Gehrig’s disease. Almost without exception, his patients get worse over time and eventually die.

Now, Dr. Bedlack, longtime head of Duke University’s ALS clinic, is focusing on a different kind of patient: someone who seems to be getting better.

ALS, or amyotrophic lateral sclerosis, progressively robs people of the ability to move their muscles and is fatal, usually within two to five years.

But in a small number of cases—Dr. Bedlack says he has verified 23 so far—patients report unexpectedly regaining lost motor functions for at least a year. Some attribute their improvement to supplements or experimental therapies, but acknowledge they can’t be certain why they started to improve.

Dr. Bedlack believes that studying these so-called “ALS reversals” and trying to determine what, if anything, separates these individuals from the overwhelming majority of others may lead to new understanding of the disease and, potentially, new therapies.

For over a decade, a multi-institution research consortium has followed so-called “elite controllers,” people infected with HIV who somehow naturally control the virus without anti-retroviral medications and don’t develop AIDS. Studies of patients by the International HIV Controllers Consortium and other groups helped identify a genetic signature associated with controllers, and experimental therapies are now being tested.

“At the beginning, HIV was this incredible black box,” says Bruce Walker, director of the Ragon Institute of MGH, MIT and Harvard, which operates the HIV study. “The difficult thing is, how do you turn other people into elite controllers?”

Dr. Bedlack says he would like to do the same for ALS.

Earlier this year, researchers, including Dr. Bedlack, published a paper in Neurology based on a database of more than 10,000 patients stripped of any identifiable information who are taking part in clinical trials for ALS treatments.

The researchers found a small subset—less than 1% of patients in the database—they consider ALS reversals, who had significant improvement and regained lost function for a year or more.

Even if the numbers of these patients are small, “I think they are worth studying,” says Merit Cudkowicz, director of the ALS clinic at Massachusetts General Hospital in Boston and one of the authors of the Neurology paper.

Dr. Bedlack says a number of theories may explain ALS reversals. Perhaps, he says, these patients didn’t really have ALS but an unknown mimic syndrome. They might have genetic traits that help resist the disease. An environmental factor that helped drive the disease could be removed, he says, or an unusual treatment the patient tried worked.

To study the phenomenon, Dr. Bedlack is running two programs. For his Study of ALS Reversals, he is collecting verified cases of reversals. When he hears of a case, usually through word-of-mouth or social media, Dr. Bedlack gets in touch with the patient. He asks to review medical records and speak to the doctor. This summer he will start collecting blood samples of verified cases to be used for whole genome sequencing and testing for any unusual antibodies. 

One participant, Kim Cherry of Boise, Idaho, was diagnosed with ALS in 2011. His lowest point was in 2012, but since then his function has dramatically improved. The 68-year-old says he tried a variety of approaches, including treatment in a hyperbaric oxygen tank, a gluten-free diet and deepening spirituality. He thinks his reversal may be due to a combination of factors. “ALS is a puzzle,” says his wife, Kay Cherry.

In cases where an unusual treatment seems to be involved, Dr. Bedlack’s second program—Replication of ALS Reversals—is trying to reproduce the reversal using the patient’s regimen. The first such trial, based on the experience of Michael McDuff, a 64-year-old former machinist from Westport, Mass., has enrolled 16 patients and is expected to enroll 34 more.

Mr. McDuff says he first noticed unusual weakness in his arms in 2010 and was diagnosed with ALS. By the spring of 2013, he couldn’t dress or feed himself. He had trouble speaking and had a feeding tube placed in order to eat. “I was declining fast,” he says. His five adult children all came home to say goodbye.

At the suggestion of a friend, he started taking a supplement called lunasin—a protein found in soy and grains that is claimed to have a range of potential health benefits. After three months, Mr. McDuff and his wife noticed improvements. Today, Mr. McDuff can swallow again and has gained weight. His arms are still weak, but he can walk short distances. He is the first to say he isn’t cured. But now, “I have a better quality of life,” he says.

Participants in the lunasin trial will report data and monthly function scores to PatientsLikeMe, a company that aggregates and analyzes health data. When it comes to potential ALS reversals, “anecdotes are all over the place, but if you really want to make a decision on a treatment, we need better and more powerful data,” says Jamie Heywood, co-founder and chairman of PatientsLikeMe.

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