(WHDH)– A local family says they’re in a fight for their lives! They want access to unapproved but promising treatments as soon as possible. Hank Investigates.

It was the happiest of days for this Peabody family.

“My little sister is growing up and marrying Ken today,” Mike Moutsoulas said, speaking at his sister Alexandra’s wedding two years ago.

Mike’s sister, Stephenie Dunklee, spoke too, “You guys are awesome together.”

But so much has changed since then. Mike is now one year into his battle with ALS.

“ALS is unimaginable. It strips you of basic things, basic functions,” Mike said.

Stephenie is also fighting ALS. Today she can only speak through an app device on her phone, “I started with slurred speech.”

She needs a feeding tube, a wheelchair, and 24-hour care. “I cannot dress myself, bathe myself, or be left alone,” Stephenie said.

The siblings have a hereditary form of ALS that’s devastating their family.

Mike and his wife, Lisa Moutsoulas, have three kids. She worries their time together is ticking away.

“What hurts my heart is I don’t know how much longer we’re going to have him, and my children aren’t going to see how truly wonderful their father is. So, it’s a struggle; it’s such a struggle. I can’t imagine a life without him,” Lisa said.

ALS weakens muscles and destroys physical function. And it moves quickly. The average time from diagnosis to death is 2 to 5 years.

“Your mind stays sharp, but the rest of you deteriorates,” Mike said.

“Your body is a prison,” Lisa said.

Right now, there is no cure. But there are promising unapproved treatments, including a medication made right here in Cambridge by Amylyx Pharmaceuticals.

“We want to make sure that promising treatments get to patients as quickly and efficiently as possible,” Justin Klee, Co-CEO and Co-Founder of Amylyx Pharmaceuticals, said.

Just last month, two major ALS advocacy groups, “I AM ALS” and The ALS Association sent this letter to the FDA. It asks regulators and the Cambridge company to make its treatment available to patients “as soon as possible.”

“The stakes couldn’t be higher for these families,” Danielle Carnival, Ph.D., and CEO, of I AM ALS said.
Many ALS patients want to try that treatment, and others, before the FDA gives final approval.

“This is why we’re fighting,” Mike said.

“The FDA might say, ‘well, we have to make sure it doesn’t hurt you,” Hank said.

“We would assume any risk that would be involved,” Mike said.

ALS advocates say when COVID hit, Operation Warp Speed showed how quickly drug companies and the government can work together to expedite treatments.

“Why not have an operation warp speed for ALS?” Mike said.

“Right now, there’s no alternative. There’s no alternative. So, let us have a chance.

The FDA tells us it’s committed to expediting treatments for ALS but it must make sure medications are safe and effective. There are bills in Congress right now calling for more access to unapproved therapies.

For information on Accelerating Access to Critical Therapies for ALS (ACT for ALS) click here.

The FDA told 7 Investigates:

The FDA is committed to facilitating and expediting the development of drugs and biologics for serious conditions like ALS where there is a tremendous unmet need while maintaining the FDA’s gold standard for safety and effectiveness. The agency has encouraged sponsors and developers to interact with the agency early on in product development, and has a number of expedited development and review programs available to these sponsors; the main benefit of these programs is increased interactions with the agency that can help expedite development.

The FDA knows that many patients with ALS are seeking access to investigational products for this devastating disease. The FDA remains committed to helping patients and health care professionals evaluate options for accessing investigational products, such as participating in a clinical trial, obtaining access to an unapproved, investigational product outside of a clinical trial through expanded access (compassionate use) or providing information for patients about the Right to Try Act. However, it’s important to note that for a patient to receive treatment under expanded access or Right to Try, the manufacturer or sponsor must be willing to provide it. Additionally, we encourage sponsors to offer access to investigational products after clinical trials are complete when continued access to a promising medicine would be appropriate under the expanded access program.

On September 23, 2019, the FDA published final guidance to help advance the development of novel treatments for ALS. This guidance represents the agency’s current thinking on this topic. FDA issued a statement at the time of publication, which provides information on the contents of the guidance. FDA interacted with numerous ALS organizations prior to publication of the draft and final guidance. There was tremendous interest from the ALS patient community, sponsors, and other stakeholders surrounding the finalization of this guidance.

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