‘God allowed me to have this disease’
Dave Siegers lives a full life.
He sells real estate and flies planes for a local charity. He’s an enthusiastic grandfather of four young children. He rides horses and is a strong Christian.
He also has amyotrophic lateral sclerosis, better known as ALS and Lou Gehrig’s disease.
And in September, Dave will become one of the first Americans to try a new drug for treating ALS.
For the first time in more than 20 years, the Food and Drug Administration has approved a new drug to treat the disease. It is only the second time, in fact, the FDA has approved a drug for the treatment of ALS.
The disease’s cause, and cure, remains unknown.
“I feel like I’m contributing,” said Siegers, 61, of Holland, Michigan. “If I can be beneficial to other people’s lives, that brings more meaning and purpose to my life.”
A longer life
ALS is a motor neuron disease that kills the neurons of voluntary muscles, causing twitching, loss of muscle, difficulty swallowing, speaking and breathing, and ultimately death.
About 50 percent of patients live longer than three years.
During a six-month study of 137 patients in Japan, the new drug, Radicava or edaravone, slowed the progression of the disease. The study only looked at a specific subset of ALS patients, but the FDA approved it for all ALS patients.
Paul Twydell, DO, who specializes in neuromuscular disorders for Spectrum Health Medical Group, said he plans to recommend the drug to patients who fit the specific subset studied and he’ll discuss it with his other ALS patients as well.
“We have so few treatment options for the disease, it would be hard not to offer it to everyone in the early stages of ALS,” Dr. Twydell said. “However, patients may have difficulty getting it covered by their insurance company if they do not fall into the specific subset that was studied.”
For people in the study with an ALS function rating score of 24 or higher, the drug slowed the disease’s progression by 33 percent, Dr. Twydell said. The scale runs from 0 to 48.
“Somebody with a score of 30 might have some slight speech disturbance, some early eating problems,” Dr. Twydell said. “They might have some difficulty with motor functions like getting dressed or maybe problems walking, like going up the stairs. They might be short of breath.
“Somebody with a score of greater than 24 will probably have a little of all of that, enough where the disease is obvious,” he said.
“Compared to a placebo, this particular group had a 33 percent less decrease in their FRS-R score over a six-month period. So it’s hard to really quantify, but it looks like edaravone slows the disease down.”
The drug, given intravenously, costs $145,000 a year. But unlike the previous ALS drug, riluzole, it will be covered under Medicare Part D as long as patients get their infusions at home.
“They don’t want to cover the facility bill,” Dr. Twydell said.
Riluzole, a pill taken twice daily, has been shown to extend life anywhere from three to 16 months, according to studies. But patients don’t feel better on the pill and they can’t tell if it’s working, Dr. Twydell said.
And although it’s cheaper than edaravone, riluzole still costs $2,000 for the generic brand and $8,000 for the non-generic version, which adds up to $24,000 or $96,000 a year.
“It may extend life three months,” Dr. Twydell said. “Is it worth spending all your finances, or all the family’s savings? That’s a real conversation we have sometimes with patients.”
Siegers’ diagnosis came in January.
His right arm began twitching last year and then slowly the twitching began to spread to the rest of his body. It doesn’t bother him during the day when he’s active, but at night when he lies down and tries to be still, he notices it.
He decided not to take riluzole because it only extends life—it doesn’t make patients feel better, he said.
“What they had determined was that it extended life expectancy about three months,” Siegers said. “And you know what? That wasn’t worth it to me. It had more to do with quality of life than quantity of life. I would rather live shorter and happier than longer and less happy.”
But the newly approved drug edaravone intrigued Siegers, especially since he gets to be one of the first patients in the U.S. to use it. The hope of finding a successful new drug—and adding to the progress in the war against ALS—overrode his apprehensions. It’s a chance to give others in his situation more hope, he said.
The drug will be available in the U.S. near the end of August.
“God has a strong purpose in my life,” Siegers said. “I don’t understand why God allowed me to have this disease, but I won’t say no to anything. It’s just like this article: If it encourages them to have the courage to get on this treatment, then it’s worth it.
“Faith is important to me. I want to be able to help others in any way God leads me to. I’m willing to be a guinea pig if it’ll help more people down the road.”