OPINION: Desperate ALS patient angry with FDA vote against new medication: 'We have nothing to lose by taking this drug'

Apr. 8—Bob Palucki feels desperate, and he should.

"The lack of compassion for us by the FDA is unforgivable," said Palucki, a U.S. Navy veteran from Tinley Park, Illinois.

Last week, federal health advisers ruled against a hopeful experimental drug for the debilitating illness amyotrophic lateral sclerosis, or ALS, also known as Lou Gehrig's disease. U.S. Food and Drug Administration advisers voted 6-4 that a single study from Amylyx Pharmaceuticals failed to establish the new drug's effectiveness in treating the deadly neurodegenerative disease.

"I think it would be a disservice to the patients and their families to approve a treatment that is of uncertain benefit," Dr. Kenneth Fischbeck of the National Institutes of Health said in a statement.

The panel's narrow vote of rejection felt like a body blow to many ALS sufferers who've been lobbying and praying for this new drug's approval. One of them is 81-year-old Palucki, who was diagnosed in 2017 and who suspects his military service abroad decades ago played a role with being stricken by ALS.

"I believe something I was exposed to has stayed in my system all these years, then something traumatic triggered the ALS. I don't know where or when or how I contracted ALS, but here I am," he told me last year at this time.

He was hopeful for the new drug's promise, not so much for himself as for other, younger people with ALS. People with young children, jobs, families and plans for the future that have been constricted by the fatal disease.

"People with ALS cannot wait for the full experimental process for AMX0035 to continue," said Gema Ramos, a care services coordinator for the ALS Association of Greater Chicago.

They are now forced to wait longer than they had hoped.

On March 30, the FDA'sPeripheral and Central Nervous System Drugs Advisory Committee (AdCom) voted against granting a new drug application for Amylyx's AMX0035, citing insufficient data. The panel's conflicted decision may have been the correct conclusion from a pharmaceutical standpoint, but the vote wasn't surprising to experts.

"It gets in the way of developing truly effective treatments if it turns out not to be effective," Fischbeck, who's one of the panelists, pointed out.

The FDA released briefing documents prior to the panel's vote, signaling its concerns about results from the new drug's second phase study. The agency is likely planning on using this setback to seek more robust data from Amylyx with a larger size clinical trial. Company officials said the third phase should include 600 ALS patients, with a scheduled release date in 2024.

That date feels like a rumor to people in Palucki's situation.

"The FDA says they want more tests," he told me last week. "We have nothing to lose by taking this drug!"

I would likely feel the same way if this new drug could even potentially extend my life by just a few months. ALS destroys nerve cells in the brain and spinal cord needed to walk, talk, swallow and eventually breathe. Most people die within three to five years of their first symptoms. Palucki is now in his fifth year with ALS.

"This disease is fatal, and the FDA threw us under the bus," he said.

To its defense, the agency has come under fire for approving an expensive medication for Alzheimer's disease with what critics called insufficient evidence of its efficacy. With the new Amylyx medication, the FDA faces more scrutiny from patient groups despite the desperate hopes of ALS patients.

Last month I learned that an old friend from my teenage years has been diagnosed with the disease.

"He's come to grips with it," another friend told me.

Coming to grips with premature death is a fact of life for ALS patients. There's no cure. Only a heightened appreciation for life's fleeting moments.

"Knowing what awaits me in the future, I have become more aware," Palucki said. "I appreciate people and things around me."

Palucki's wife of 57 years, Ceil, is his caregiver. The couple met in 1963 while working together at Sears in the city.

Last week I stumbled onto a social media photo of former Chicago Bears player Steve McMichael, who was diagnosed with ALS in 2020. "Mongo" was in a hospital bed surrounded by former Bears players. He looked even more frail, more helpless, more vulnerable than he did last year at this time when I wrote about "my hero."

The fragility of life and ferocity of disease feels even more poignant when it comes to our heroes from once upon a time.

This is how I first met Palucki, who was desperate for public awareness of what ALS patients are going through. He reached out to me after reading about McMichael. "We need someone to advocate for us," Palucki told me.

They also need empathy from us. This latest "setback," as experts describe it, is so much more than merely a setback. Contrary to many people's beliefs, time is all we have in life. A few additional months, even potentially, can feel like a few more years.

Who among us wouldn't be desperate for a few more years? Or a few more moments?

jdavich@post-trib.com

https://www.facebook.com/JerDavich/

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