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A new approach to ALS drug trials gives patients hope

Published in the Boston Globe August 12, 2020, written by Jonathan Saltzman

When Dr. Marc Litt was diagnosed with ALS in March 2019 at Massachusetts General Hospital, he was dismayed to discover how little medicine had to offer him.JAMES GILBERT/FOR THE BOSTON GLOBE

Mass. General will lead the simultaneous testing of multiple potential treatments for the deadly disease.

As a cardiologist, Dr. Marc Litt has plenty of arrows in his quiver for patients with heart disease: Medications that thin blood. Drug-coated stents that widen blocked arteries. Implants that replace damaged valves in minimally invasive surgery.

But when the 63-year-old physician was diagnosed with ALS in March 2019 at Massachusetts General Hospital, he was dismayed to see how little medicine had to offer him. Despite at least 50 clinical trials of potential drugs since 1995, only two medications have been approved to slow the progression of the deadly degenerative neurological disease, and neither is all that effective.

So when he learned last fall that Mass. General was going to lead the world’s first clinical trial to simultaneously test multiple potential ALS drugs, he felt a glimmer of hope. Later this month, the interventional cardiologist plans to temporarily move from his home in Jacksonville, Fla., to an apartment in the Fenway to participate in the study.

“It’s like being given an opportunity to slow this [disease] down a little bit so I can enjoy my family and have a better quality of life,” he said as he fought back tears during a Zoom call. “I’ve taken care of patients for 30 years. I always had the opportunity to offer them something.”

Mass. General had planned to launch the first so-called platform trial for amyotrophic lateral sclerosis in March, but it was delayed by the COVID-19 pandemic. Like most other hospitals in the United States, the renowned Harvard-affiliated teaching hospital postponed clinical drug trials to focus on treating people with the coronavirus.

In the past few weeks, however, MGH and six other hospitals in Connecticut, Florida, Pennsylvania, and Texas have started the study. It will initially enroll 480 volunteers at 54 clinical sites, with researchers testing one of three experimental drugs on groups of 160 patients.

The drugs come from UCB, a Belgian company that recently acquired Cambridge-based Ra Pharmaceuticals; Biohaven Pharmaceuticals Holding Co. of New Haven; and Clene Nanomedicine of Salt Lake City.

Researchers in the next several months plan to add two more groups, each with 160 volunteers, to test drugs made by Prilenia Therapeutics of Israel and Implicit Bioscience of Australia, according to Dr. Merit Cudkowicz, the neurologist who directs the Sean M. Healey & AMG Center for ALS at Mass. General, which is running the trial. Healey, once head of the investment company Affiliated Managers Group, helped found the center in 2018 with about $40 million in donations after he was diagnosed with ALS. Healey died in May.

There’s no limit on how long the trial will run or how many patients can ultimately participate.

“We’re going to keep going until we find effective treatments,” said Cudkowicz. “I expect to have results of the first three [drugs] in about a year.”

ALS, also known as Lou Gehrig’s disease, is a rare disorder diagnosed in about 6,000 people a year in the United States, according to the Massachusetts chapter of the ALS Association. The disease affects the nerve cells in the brain and spinal cord. It gradually robs patients of the ability to speak, eat, and, finally, breathe. The disorder kills most patients in three to five years.

RELATED: For families of ALS patients, providing home care can be an infinite challenge

The only drugs prescribed to slow the progression are Radicava, approved in 2017, and riluzole, the generic name of Rilutek, which was OK’d in 1995. A third drug, Nuedexta, is prescribed to treat unpredictable episodes of crying and laughter that sometimes occur in ALS, but it doesn’t affect the underlying disease.

Until now, a clinical trial of a potential drug for ALS typically took about a year to set up and another year to run, according to Cudkowicz. When the trial failed to produce a beneficial drug, researchers had to start over.

A platform trial cuts the time to find an effective treatment in half and decreases costs by about a third, proponents say. Multiple drugs are tested and evaluated simultaneously. New treatments are added as they become available, and others are dropped if they don’t work. The setup also increases the chance that patients will receive an experimental drug instead of a placebo.

RELATED: Mass. General to begin ALS trials that test several drugs at once

Such trials have proven particularly effective in the testing of drugs to treat cancer. Champions of the approach include Janet Woodcock, director of the Food and Drug Administration’s Center for Drug Evaluation and Research.

“We didn’t have enough [experimental] drugs in neurology for this to make sense,” said Cudkowicz, but now there are at least 10 other potential ALS medicines that researchers are considering testing in addition to the first five in the platform trial.

Cathy Soucy, 66, a retired medical office worker who lives in Amesbury, recently began participating in the trial at Mass. General. She was diagnosed with ALS two years ago and takes Radicava and riluzole. She used to run 5 miles a day and hike in the White Mountains, but now needs an electric wheelchair to accompany her family on long walks.

On July 28, she began participating in a study of verdiperstat, a compound developed by Biohaven. Soucy said she’s not sure whether the new pills she takes daily contain the drug or a placebo, but she’s thrilled to be in the study.

“Without these trials, there’s no hope for cures or more treatments,” she said.

Litt, the Florida cardiologist, has a similar view.

In 2017, he developed a limp on his right side and soon began struggling to walk. For a while, he thought it might be attributable to spine problems, perhaps exacerbated by the 15-pound lead apron he wears at work to protect him from radiation used in medical imaging. He underwent back surgery, but the problem persisted. When his own patients began remarking that he was losing weight, Litt got alarmed.

After he was diagnosed with ALS last year, he began taking Radicava and riluzole, but his health has continued to deteriorate. He uses a wheelchair, needs his wife to help him get in and out of the shower, and has fallen several times.

“You can see the writing on the wall,” said Litt, who continues to see patients in telehealth appointments three days a week.

Litt and his wife plan to move this month to an apartment in the Fenway, where they have a six-month lease, so he can participate in the study. (One of their two sons, Dr. Michael Litt, a resident at Brigham and Women’s Hospital, lives two blocks away.) There’s a 75 percent chance he will get a drug instead of a placebo, according to Cudkowicz.

All the participants in the study, whether they receive an experimental medicine or a placebo, will get the actual drug under an open label provision after six months.

Litt said he’s not looking for a cure, just something that would turn ALS into a manageable disease.

“You can’t cure diabetes, but at least they have insulin for treatment,” he said. “Find something like insulin to treat it. Give me something.”

ALS patients who want to participate in the trial can contact HEALEYALSPlatform@mgh.harvard.edu.

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