Clinical Trials
*Synthalaxate is an imaginary drug and not something that exists.
*****
Bedford, Massachusetts
"Honey? I appreciate you doing this for me. I...I just don't think I can stand to ask you to...."
"Dad? It's okay," his grown daughter told him before he could apologize yet again as though helping him out was a burden.
It wasn't easy, but she'd never thought of it as a hardship from the day she learned his diagnosis of ALS was a death sentence. That news had come just over a two months ago, when, after several months of her 67-year old father stumbling, tripping, and later on, dropping things, she convinced him to see a doctor.
Most recently, he was having difficulty swallowing, and that was the most disturbing symptom of all because it indicated the possibility of the most serious form of ALS in which 'bulbar onset' or difficulty swallowing, was a symptom. Sadly, all cases degenerated into the bulbar variety, but people tended to live the longest when that was one of the last symptoms to arise.
Initially they were told it could be any number of things to include the worst-case scenario--ALS or as it was more commonly known--Lou Gehrig's disease. Two weeks and a ton of tests later, their worst fears were realized when the internist gave them the news.
Hallie Emerson began learning everything she could about this hideous, dreaded disease that left one's mind fully in tact as it ravaged the body's muscles until one could no longer even swallow, causing a kind of death-by-drowning end to life. That had been one of the first things she learned.
The second was that it was 100% fatal. Yes, there were extremely unlikely scenarios in which people survived for decades after a diagnosis with the late Stephen Hawking being the most well known example. He'd survived and published numerous papers, but he'd been confined to a wheelchair or a bed all those years, a fate Hallie thought might be worse than death as one lay there fully alert yet unable to move or call for help.
Once she'd learned as much as any layman possibly could she began looking for some way, any way, to prolong her father's life or make it more comfortable for as long as possible.
There were drugs that were FDA approved, but none of them seemed all that great.
The first was called Rilutek. Approved in 1995, Rilutek inhibits glutamate release and prolongs life by approximately three months. Not exactly a cure.
Nuedexta had been approved in 2011 and is a combination of dextromethorphan and quinidine. The quinidine inhibits a liver enzyme that would otherwise rapidly break down the dextromethorphan in most patients. The technical explanation was baffling, but she read it anyway. "DM is a selective, noncompetitive antagonist of the N-methyl-D-aspartate subtype of the glutamate receptor."
"Thanks for nothing," she remembered reading before dutifully reading more to understand the technical nature of what it did and why it, too, wasn't enough.
Another was called Radicava, the first new treatment specifically for ALS in 22 years. The drug was approved by the FDA in May 2017, less than a year after Mitsubishi Tanabe Pharma America submitted a New Drug Application to the FDA.
Lastly, there was something called Riluzole which delays the onset of ventilator-dependence or tracheostomy in some people and may increase survival by two to three months. In other words, it helped patients avoid drowning on their own spit a little longer.
Feeling depressed but not hopeless, Hallie began a vigorous search for clinical trials; anything her father might be able to get into and try before it was too late to help.
Because ALS affects such a relatively small number of people each year, it didn't get the kind of funding cancer or heart disease or something new and deadly like COVID-19 did. But there were trials, and when she learned of one less than 50 miles from their home in Needham, Massachusetts, she felt hopeful for the first time and began making phone calls.
The technical explanation of the trial was over her head. "This study seeks to enroll people living with ALS caused by a mutation in the superoxide dismutase-1 gene. SOD-1." The layman's explanation was much simpler and gave her hope and caused her to pick up the phone and call.
After answering a long series of questions, Hallie nearly cried when the woman on the other end of the line told her that her father qualified for the trials. She cautioned her that although he was now in the database, he would still require the approval of two of three doctors running the trial. She couldn't guarantee anything, but she told Hallie to remain optimistic in a way that gave her even more hope.
Just four days later, her father was enrolled, and they were getting ready to make the first drive to Needham for his initial intake to begin the clinical trials for a promising new drug called Synthalaxate.
There was a 50% chance her father would be given a placebo, but doing nothing was a 100% guarantee of death in what would almost certainly be in less than 18-24 more months which could end up being as little as twelve due to this new bulbar issue.
*****
Lincoln, Massachusetts, about 10 miles southeast of Bedford
"You ready, Mom?" he asked with a smile.
"Ready or not, here we go, right?" she replied, trying to smile back and being more cheerful than he thought he could ever be in her situation.
"I guess," her son said with a chuckle as he handed her a cane, something she hadn't needed just a month ago when the clinic confirmed her diagnosis.
He thought about the phone call that evening. He'd been in the field for four days and had only slept ten or twelve hours. He'd just taken a hot shower around 8pm and was trying to eat a little something without falling asleep.
"Mom? Hey. What's up?" he asked when he saw who was calling being careful not to let his utter fatigue show through.
Not one to mince words she answered him directly.
"Chad? I'm calling because I have ALS."
It took his foggy brain a few seconds to understand what she'd just said.
"Wait. ALS as in...Lou Gehrig's Disease?"
"That's the one. I've had tests run and rerun, and I just saw the doctor today."
She paused then said, "I guess that explains why I've felt like my right foot keeps getting caught on things like rugs or sometimes it just sort of...drags itself along."
Too stunned to speak, Chad Decker sat in silence as his mother explained as much as she knew. His dad had passed away four years earlier from cancer, and his mom had been alone since then with occasional visits from him and his older sister who also lived out of state. Frankly speaking, she was too self-absorbed to care.
