Before John Wilkinson’s neurologist could diagnose his patient, John spoke up. “I have ALS,” he said. “I have two to five years to live. There is no cure.” (Chris Clark | Spectrum Health Beat)
Months of mysterious symptoms, visits to multiple doctors, and test results led John and – his neurologist – to the diagnosis. (Chris Clark | Spectrum Health Beat)
In February, John became the first patient at Spectrum Health to take part in the HEALEY ALS Platform Trial. He hopes his participation can lead to new, effective treatments. (Chris Clark | Spectrum Health Beat)
John has equipment to adapt to the changes brought about by ALS. Breathing treatments help cleanse his lungs. “You try to make the most of every day,” he said. (Chris Clark | Spectrum Health Beat)
“ALS stands for ‘you always lose something,'” said John. “There is never a good time to diagnose an incurable disease. It’s hard at first. But after a while you get over it. “(Chris Clark | Spectrum Health Beat)
There is no doubt that John loved to be active. On a typical day, he played pickleball for several hours in the morning and then went to the YMCA for training. (Courtesy photos | Barbara Harvey)
In the Healey platform study, one additional patient receives a placebo for every three actively treated patients. (Chris Clark | Spectrum Health Beat)
John has bottles that contain either the investigational drug CNM-Au8 nanocrystalline gold or a placebo. Every morning he pours the liquid into a wine glass and drinks it. (Courtesy photo | John Wilkinson)
“We need a cure or better treatments. Lou Gehrig had that 80 years ago,” said John. “And we’re just as bad off as he was 80 years ago – in terms of healing.” (Chris Clark | Spectrum Health Beat)
When John Wilkinson sat down with his neurologist, he didn’t wait for his diagnosis.
Before the doctor could say anything, John stepped in with the harsh truth.
“I have ALS,” he said. “I still have two to five years to live. There is no cure. “
Months of mysterious symptoms, visits to multiple doctors, and test results led John and – his neurologist – to the diagnosis.
That was 18 months ago.
Amyotrophic lateral sclerosis – known as ALS or Lou Gehrig’s disease – is still not curable.
However, John hopes that an innovative new research program will accelerate the search for effective treatments.
In February, John became the first person to join Spectrum health Participate in the HEALEY ALS Platform Trial, a national study led by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital. Spectrum Health is one of 54 Northeast ALS Consortium sites approved by the US Food and Drug Administration to administer the study drugs.
A platform study allows researchers to test multiple treatments at the same time. This cuts test time in half and reduces costs by 30%.
And people who take part in a platform study are more likely to be given an investigational drug – rather than a placebo – than in a typical clinical trial.
The platform study model has been used in cancer research, but this is the first time researchers have used the approach to testing ALS treatments.
“Because this is an urgent disease, a better way was sought to get promising compounds and other therapies through the pipeline more efficiently,” said Paul Twydell, DO, the director of neuromuscular medicine at Spectrum Health.
“We hope the study will bring us closer to developing advanced treatments for ALS.”
ALS is a fatal disease that affects the nerves that control muscles. It affects approximately 30,000 people in the US and 500,000 worldwide.
Accelerating research is vital for those with this disorder. As John told his doctor, people with ALS live an average of two to five years after they are diagnosed.
Three ALS treatments have been approved by the FDA.
“Only two of them actually slow the disease,” said Dr. Twydell.
The third drug treats the emotional outbursts that sometimes occur with ALS.
“That’s all we have,” he said. “Many drugs have been studied over the years. And many failures. “
“The most active person”
John, 64, grew up in Oklahoma and moved to Michigan in the early 1980s to work in the oil and gas industry. Now retired, he lives in Grand Rapids with his fiancée Barbara Harvey.
“He used to be the most active person I know,” said Barbara. “He took an average of 25,000 steps a day.”
John shook his head and laughed.
“It was only about 19,000 steps a day,” he said.
There is no doubt that John loved to be active. On a typical day, he played pickleball for several hours in the morning and then went to the YMCA for training.
In early 2019, he noticed a weakness in his left hand. He underwent carpal tunnel surgery, but his hand strength did not improve. In fact, it got worse.
After consulting with other specialists, he met with a neurologist and underwent an electromyography test to assess his muscles and the motor neurons that control them.
A few months later he had a second EMG to compare to the first.
This test led to the diagnosis of ALS.
Until then, he knew little about the disease. When he researched it, he was overwhelmed by what he learned.
When his neurologist suggested getting a second opinion, John replied, “No. I just pretend you’re wrong. ‘”
But John continued to learn all about ALS. He is looked after by the ALS Neuromuscular Disease Clinic at Spectrum Health, a multidisciplinary clinic named a Certified Treatment Center for Excellence by the ALS Association.
The clinic brings together specialists in neurology, respiratory therapy, nutrition, social work, nursing and occupational therapy, speech therapy and physiotherapy.
Patients usually meet with the team every three months.
“We take care of things before they become a problem,” said Dr. Twydell. “We think about the disease three to six months in advance.”
John has equipment to adapt to the changes brought about by ALS. Breathing treatments help cleanse his lungs. He has specially designed plates and cutlery that make eating easier as he now has weaknesses in both hands.
He and Barbara recently moved into a one-floor condominium with a kitchen, bedroom, and living area.
He can still walk, but an electric wheelchair is available in his cave for the day when his mobility is more limited.
He also plans to get a feeding tube, knowing the disease will continue to make swallowing difficult.
“We try to live one day at a time and not worry about the future,” said Barbara. “On the other hand, people say to go ahead.”
Maintaining this balance is not easy in a disease that causes progressive deterioration in skills.
“ALS stands for ‘you always lose something,'” said John.
“There is never a good time to diagnose an incurable disease. It’s hard at first. But after a while you get over it.
“You try to make the most of every day.”
An experimental treatment
Through social media, John connected with other people with ALS and their caregivers. This is how he found out about the Healey platform study, into which the first patients were enrolled in August 2020.
The research team is currently testing four drugs while evaluating other experimental treatments for possible inclusion in the study.
Medications include: Zilucoplan, a small macrocyclic peptide inhibitor of the complement component 5 [C5]; Verdiperstat, an oral myeloperoxidase inhibitor; CNM-Au8 nanocrystalline gold, an intracellular nanocatalyst to support cellular bioenergetics; and pridopidine, a highly selective sigma-1 receptor.
The study will include 160 patients in each regimen. Patients will be randomly assigned to either study treatment or placebo for 24 weeks.
In a typical clinical trial, half of the patients will receive a placebo and the other half will receive the study drug.
In the Healey platform study, one additional patient receives a placebo for every three actively treated patients.
For John this means, “I have a 75 percent chance that I have the drug.”
On a table in his kitchen, John has a box with 32 smaller boxes. In each there are two small bottles with a dark liquid – either the test preparation, CNM-Au8 nanocrystalline gold or a placebo.
Every morning he pours the liquid into a wine glass and drinks it.
“It tastes like paper,” he says.
At first, John hoped he was taking the investigational drug – not the placebo. After 14 weeks he is no longer so sure.
“It turns out I’m making progress, which is a bad word at ALS,” he said. “I almost changed my mind. I kind of hope I have the placebo because the real drug could be better. “
At the end of their 24 weeks in the clinical trial, patients have the option to take the experimental drug. And that’s another unusual feature of the Healey process – and a big plus for John.
“We need a cure or better treatments. Lou Gehrig had it 80 years ago, ”he said, referring to the New York Yankees baseball player who died in 1941 of ALS. “And we’re just as bad off as we were 80 years ago – in terms of a cure. ”
It is good that researchers are evaluating other treatments as part of the Healey platform study, he said.
“The Healey process is so much better than any other process,” he said. “We’re lucky that Spectrum Health is involved. It gives people hope. “
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