- Researchers led by Massachusetts General Hospital are testing several different drugs for ALS at the same time.
- They had to delay clinical trials because of the coronavirus pandemic, but ended up finding ways to help people participate from their homes.
- Lead researcher Dr. Merit Cudkowicz expects to have answers for three of the ALS drugs by next summer.
- ALS research got a boost thanks to the "Ice Bucket Challenge," but Cudkowicz said an investment of at least $1 billion will be needed to get a new treatment.
- Because of her work, Business Insider named Cudkowicz to our annual list of the 10 leaders transforming healthcare.
Back in March, a research arm at Massachusetts General Hospital in Boston was getting ready to launch an innovative, faster way to deliver results from testing new drugs.
The trial was targeting treatments for amyotrophic lateral sclerosis, a deadly disease that destroys nerve cells controlling movement. Everything was ready to go.
Then the coronavirus pandemic hit.
Dr. Merit Cudkowicz, director of the Sean M. Healey & AMG Center for ALS at Mass General, said scientists had to recalibrate because they didn't want to risk exposing patients to the virus.
The Healey & AMG Center moved some visits from in-person to video, arranged for a company to visit people's homes to draw blood, and lined up a home device they needed to test people's breathing. The Food and Drug Administration put out new guidance, and ethics review boards became more open to in-home medical research.
It took three months for the Healey & AMG Center to get everything ready.
As it turned out, the changes worked well for patients, Cudkowicz said. Researchers had talked about moving more parts of clinical trials into people's homes before. The pandemic made the switch not only necessary but possible.
For patients with ALS — who gradually lose the ability to walk, speak, and swallow — it was a huge help to be able to cut travel.
"It's almost one of the few positive things out of this pandemic, because I think trials are going to be easier for people with neurological illnesses," said Cudkowicz, who also oversees the neurology department at Mass General. She thinks it'll remain a permanent fixture in medical research.
Now, a dozen sites across the US are enrolling people in the ALS trials the Healey & AMG Center is leading. By the fall, they're hoping to have all 54 planned sites running, with 480 patients taking part.
The ALS drugs are being tested simultaneously, saving time and money
Healey & AMG Center scientists were already preparing to take on a whole new way of conducting their work even before the pandemic hit.
The method they're using is called a "platform trial," which allows them to test several drugs in different people at the same time. It has been used to develop cancer treatments and, according to proponents, cuts the time to find an effective treatment in half and cuts costs by a third.
Doing the study this way means drugs are tested against a single placebo group, and scientists can evaluate results simultaneously. If one drug is working particularly well, then scientists can switch patients to a different drug or do a combination.
Under previous methods, scientists tested one drug at a time. That meant if a drug failed then a lot of time would pass before scientists could start over with another trial.
"The idea was that there's such a big pipeline in ALS and the current approach of testing drugs is so inefficient that we wanted to do a better way," Cudkowicz said.
On top of that, Cudkowicz said it allows for better collaboration.
"We went to the FDA with three people from three companies and all sat in the same room together to talk about this," she said. "And it doesn't maybe sound like much, but that never happens."
The three different drugs they've begun testing were developed by UCB Ra Pharmaceuticals in Massachusetts, Biohaven Pharmaceutical Holding Company in Connecticut, and Clene Nanomedicine in Utah. The medicines are administered through a shot, a pill, and a liquid that's swallowed, respectively.
In a couple of months, researchers expect to test two more drugs, one from Prilenia Therapeutics of Israel and another from Implicit Bioscience of Australia. Researchers selected the handful of medicines after beginning with a list of almost 30 applications.
If successful, the medicines would provide a much-needed reprieve for patients. Four drugs exist to treat or moderate the symptoms of ALS, but there's no cure.
Funding is up, but Cudkowicz says it needs to hit at least $1 billion
In recent years, funding and awareness about ALS has soared, but Cudkowicz said more will be needed.
Back in 2014, a trend called the "Ice Bucket Challenge" went viral on social media. People dunked a bucket of iced water on their heads and challenged others to do the same. The idea was to dunk the bucket within 24 hours or otherwise make a charitable contribution, though many people did both.
The Ice Bucket Challenge raised $220 million across the world. Awareness about ALS soared and journals published more scientific articles about the disease. Pete Frates, who inspired the challenge, was Cudkowicz's patient.
She credited the challenge with funding new projects and bringing more scientists into the field. The Healey & AMG Center was founded in 2018 by a $40 million in donations raised by the late Sean Healey and his company, Affiliated Managers Group Inc.
Cudkowicz thinks at least $1 billion will be needed to find a treatment or two but said she sometimes pegs the amount closer to $2 billion when thinking about a cure or other major breakthrough. She added, however, that much more basic science research about ALS was still needed, which is also costly.
For the platform trial she's leading, they'll consider a drug to be a success if it can slow the illness by 30% or more. The goal is to keep testing drugs to find the ones that have "a huge impact," such as stopping progression of the disease.
Cudkowicz expects to have answers about the first three drugs they're trying by next summer.
"All the drugs could work and they could also not work, or some combination," she said.