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Boston researchers take innovative approach to finding ALS treatments

Boston researchers take innovative approach to finding ALS treatments
TREATMENTS FOR ALS. THEY’RE TESTING THREE DRUGS AT THE SAME TIME, AND IT IS THE FIRST TIME IT’S BEEN DONE IN ALS RESEARCH. >> I REALLY FEEL WE'RE AT A TURNING POINT. EMILY: SINCE THE 1990'S DR. MERIT CUDKOWICZ HAS SEARCHED FOR ANSWERS FOR HER PATIENTS DIAGNOSED WITH ALS. AND NOW, SHE’S LEADING A NOVEL APPROACH AT MASS GENERAL HOSPITAL. WEBSITE, THE HEALEY ALS PLATFORM TRIAL. TESTING THREE DRUGS AT ONCE. >> A PLATFORM TRIAL IS LIKE MANY TRIALS IN ONE. IT'S A WAY TO TEST MANY TREATMENTS IN THE SAME TIME PERIOD EMILY: IN THIS TRIAL, PATIENTS WILL BE SEPARATED INTO 3 ARMS. EACH ARM TESTING A DIFFERENT DRUG. PATIENTS WILL BE RANDOMIZED TO RECEIVE EITHER ACTIVE DRUG OR PLACEBO. THE PLACEBO GROUP WILL BE SHARED AMONG THE 3 ARMS. AND THAT FEATURE MEANS MAJOR BENEFITS FOR EVERYON >> THERE’S A A GREATER CHANCE TO BE ON THE ACTIVE DRUG RATHER THAN THE PLACEBO. WE WILL COMPARE THEIR RESULTS TO ALL THE PLACEBO GROUPS FROM ALL THE TREATMENTS. THAT’S THE EFFICIENCY. YOU CAN GET FEWER PEOPLE ON PLACEBO BUT YOU CAN KEEP LEARNING FROM THAT GROUP. EMILY: WHEN THE HEALEY CENTER OPENED IN 2018, THIS IS THE KIND OF INNOVATION SEAN HEALEY HOPED FOR. THE CENTER IS NAMED FOR THE FORMER BOSTON BUSINESSMAN, DIAGNOSED WITH ALS IN 2018. IN A STATEMENT, HEALEY TOLD US IN PART "THIS IS EXCITING NEWS , FOR THE ALS COMMUNITY. I AM CONVINCED THAT THIS IS THE BEST APPROACH TO FINDING CURES FOR ALS AND THAT THIS IS THE RIGHT TEAM TO LEAD THE EFFORT." FOR DR. CUDKOWICZ, THE TRIAL REPRESENTS HOPE FOR PATIENTS WHO DON’T HAVE TIME TO WASTE >> THE PEOPLE LIVING WITH IT TODAY. THEY DON'T HAVE THE TIME FOR A LONG, INIFFICIENT PROCESS 05:32 INEFFICIENT PROCESS. FINALLY WE HAVE A LOT OF TARGETS, A LOT OF DRUGS, AND IT MAKES SENSE TO DO THIS. EMILY: THEY’RE LOOKING TO ENROLL MORE THAN 500 PATIENTS AT 54 SITES ACROSS THE COUNTRY. THE TRIALS WILL BE SIX MONTHS LONG. FAR SHORTER THAN THE TYPICAL 12 TO 18 MONT
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Boston researchers take innovative approach to finding ALS treatments
Boston researchers are taking a new approach to finding treatments for amyotrophic lateral sclerosis or ALS. They're testing three drugs in the same trial, the first time this approach has been tried in ALS research. Since the 1990s, Dr. Merit Cudkowicz has searched for answers for her patients diagnosed with ALS. She's now leading a novel approach at Massachusetts General Hospital.It's called the HEALEY ALS Platform Trial, testing three drugs at once."A platform trial is like many trials in one. It’s a way to test many treatments in the same time period and at the end get many more answers for people with ALS," Cudkowicz said.In the HEALEY platform trial, patients will be separated into three groups, each testing a different drug. Patients will be randomized to receive either the active drug or placebo. The placebo group will be shared among the three groups and that factor means major benefits for everyone."There's a a greater chance to be on the active drug rather than the placebo. We will compare their results to all the placebo groups from all the treatments. That's the efficiency. You can get fewer people on placebo but you can keep learning from that group," Cudkowicz said.When the Healey Center for ALS opened in 2018, this trial is the kind of innovation Sean Healey hoped for. The center is named for the former Boston businessman, diagnosed with ALS the same year.In a statement, Healey said:"The team at the Sean M Healey & AMG Center for ALS at Mass General brought together a global team to accelerate therapy development for people with ALS. Obtaining FDA approval to move forward with the first three drugs in the HEALEY ALS Platform Trial is a critical step forward to getting access to potential treatments for people with ALS. This is exciting news for the ALS community. I am convinced that this is the best approach to finding cures for ALS and that this is the right team to lead the effort. My friends and colleagues at AMG are proud to support this first platform trial initiative in ALS and grateful for the enthusiasm and input from people with ALS and investigators."For Cudkowicz, the trial represents hope for patients who don't have time to waste."The people living with it today. They don’t have the time for a long, inefficient process. Finally, we have a lot of targets, a lot of drugs and it makes sense to do this," Cudkowicz said.The trial will enroll more than 500 patients at 54 sites across the country. They will be six months long compared to the typical 12 to 18 months in most clinical trials. For more information about the trial or about how to enroll, click here.

Boston researchers are taking a new approach to finding treatments for amyotrophic lateral sclerosis or ALS. They're testing three drugs in the same trial, the first time this approach has been tried in ALS research.

Since the 1990s, Dr. Merit Cudkowicz has searched for answers for her patients diagnosed with ALS. She's now leading a novel approach at Massachusetts General Hospital.

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It's called the HEALEY ALS Platform Trial, testing three drugs at once.

"A platform trial is like many trials in one. It’s a way to test many treatments in the same time period and at the end get many more answers for people with ALS," Cudkowicz said.

In the HEALEY platform trial, patients will be separated into three groups, each testing a different drug. Patients will be randomized to receive either the active drug or placebo. The placebo group will be shared among the three groups and that factor means major benefits for everyone.

"There's a a greater chance to be on the active drug rather than the placebo. We will compare their results to all the placebo groups from all the treatments. That's the efficiency. You can get fewer people on placebo but you can keep learning from that group," Cudkowicz said.

When the Healey Center for ALS opened in 2018, this trial is the kind of innovation Sean Healey hoped for. The center is named for the former Boston businessman, diagnosed with ALS the same year.

In a statement, Healey said:

"The team at the Sean M Healey & AMG Center for ALS at Mass General brought together a global team to accelerate therapy development for people with ALS. Obtaining FDA approval to move forward with the first three drugs in the HEALEY ALS Platform Trial is a critical step forward to getting access to potential treatments for people with ALS. This is exciting news for the ALS community. I am convinced that this is the best approach to finding cures for ALS and that this is the right team to lead the effort. My friends and colleagues at AMG are proud to support this first platform trial initiative in ALS and grateful for the enthusiasm and input from people with ALS and investigators."

For Cudkowicz, the trial represents hope for patients who don't have time to waste.

"The people living with it today. They don’t have the time for a long, inefficient process. Finally, we have a lot of targets, a lot of drugs and it makes sense to do this," Cudkowicz said.

The trial will enroll more than 500 patients at 54 sites across the country. They will be six months long compared to the typical 12 to 18 months in most clinical trials.

For more information about the trial or about how to enroll, click here.