ALS
(TuckerBlade/Shutterstock)

New Experimental Medication Offers Hope for Thousands of Americans Living With Lou Gehrig’s Disease

The Centers for Disease Control and Prevention estimates that 5,000 Americans are diagnosed with ALS (or amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease) every year. The debilitating and progressive illness robs individuals of their work life and career, causing muscle weakness; stiffness in the hands, feet, legs and ankles; difficulty walking and swallowing; muscle cramps; twitching; and slurred speech. Sadly, most people only live two to three years after being diagnosed with the illness.

While there is no known cure for ALS, a new treatment offers hope for those affected by the disease. The experimental drug, which is still in clinical trials, appears to slow the progression of the disease and improve the lifespan of those affected by the illness. 

Darryl C. Murphy of WBUR News, Boston’s NPR news station, has reported that the Food and Drug Administration is encouraged enough by a new treatment for ALS that they have “opened the door to early approval” for the medication.

“After completing two clinical trials for its drug AMX0035, the Cambridge-based company Amylyx reported earlier this year that the FDA wanted to see results from another round of clinical trials before the company could submit a new drug application, which is a proposal for a drug to be sold in the U.S.,” Murphy said.

However, that plan appears to be changing. 

“The agency typically requires three phases of trials before allowing a company to submit an application,” Murphy explained. “However, months later, after discussions with the agency and lobbying from patient advocacy groups, Amylyx announced its plan to submit the application in the coming months with the FDA’s blessing.”

In a statement praising the FDA’s decision, Amylyx co-founder Justin Klee said, “[I’m] giving them a lot of credit. They are finding the balance, at least in the case of our program, between ‘let’s make sure we have the right data, let’s make sure we run the right studies,’ but [also] understand the unmet need and time.”

Klee said the company intends to apply for approval of the new medication “in the coming months” and will also move ahead with conducting the final phase of clinical trials regardless of their application. He also said that Amylyx may also “create an expanded access program for those who wish to take the drug but do not meet the criteria for the trials.”

“[For] people with ALS, every single day matters,” Klee said. “We need to do all of these things to promote access as quickly and efficiently as we can.”

Encouraged by the preliminary results from the medication, health advocates are also pushing the FDA and Amylyx to move quickly with making the drug more widely available.

Brian Wallach, co-founder of the advocacy group “I AM ALS,” told Murphy that the company’s announcement “shows the power of the ALS advocacy community throughout the nation, making its voice heard and demanding the system do and be better.”

“It also shows what is possible when drug developers include patients in every step of the process,” Wallach said. “We will work relentlessly to make sure this is the first of many steps in changing the system to create more access to therapies for this 100% fatal disease.”

 

Related: For more recent diversity and inclusion news, click here.

Latest News

5 Biggest News Stories of the Week: October 6

As the saying goes, the news never stops — but there’s a lot of it out there, and all of it doesn’t always pertain to our readers. In this weekly news roundup, we’ll cover the top news stories that matter most to our diversity focused audience. 1. The Dollar Divide…

WOCA 2022 Research: How to Improve Representation of Women of Color in the Workplace 

Capital One and DiversityInc shared the results of their collaborative research report on women of color in the workplace at the 2022 annual Women of Color and Their Allies event held in Louisville on Sept. 21 in conjunction with Humana. Dr. Chris Parker, Head of Research & Data, DiversityInc, confirmed…

WOCA 2022: Executive Leaders Share Stories of Allyship for Women of Color

One of the best indicators of an organization’s success around diversity, equity and inclusion (DEI) is leadership accountability for DEI. At our 2022 Women of Color and Their Allies event, DiversityInc’s Head of Strategic Partnerships, Anita Ricketts, was joined on stage by leaders who are holding themselves and their contemporaries…

The Dollar Divide

Too Poor to Die: The Rising Cost of Funerals 

Death is expensive.  For more than two decades, I have extensively written and reported about money-related content and even I was surprised at the cost of my mother’s funeral.  The median cost of a funeral with a viewing and burial is approximately $7,848. That doesn’t include the cost of the…