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.
His mother, Fran Decker, was only 59 years old, and he was having a hard time understanding how this could happen to anyone that young.
Chad had been in the US Marine Corps for nearly ten years. He was an infantryman, or as 'Gunny' Hartmann said in Full Metal Jacket, "Oh-three hundred. Infantry."
The Marine Corps designator for infantry was 0300 with 0311 being 'Rifleman' and the first zero pronounced as an "O" as in "Oh-three-eleven." There were others like 0331 for a Machine gunner or 0341 or Mortarman along with several others. Since making staff sergeant, he was technically an oh-three sixty-nine which meant 'infantry unit leader.'
He'd initially done four years on the West Coast at Camp Pendleton after boot camp and the School of Infantry and spent one of them in Afghanistan. Having made sergeant/E-5 in just three years, he left Pendleton and headed a few miles south to Marine Corps Recruit Depot San Diego where he completed Drill Instructor School then spent three years pushing recruits through boot camp, taking them from civilians and turning them into basically-qualified Marines.
He was now at Camp Lejeune, North Carolina, on the East Coast, and as a staff sergeant/E-6, he was the platoon sergeant for Marine rifle platoon of 40 Marines, give or take. A new second lieutenant was in charge and called the platoon commander (platoon leader in the Army), but this young officer was smart enough to know he didn't know a lot yet and leaned on SSgt Decker to help him learn the ropes. Chad had previously worked for a know-it-all 'butter bar' (pejorative nickname for a 2ndLt who wears gold bars), and it was still the worst experience of his career. Fortunately, it was during peacetime and no one got hurt or killed.
At the urging of his company commander and first sergeant, Chad had recently applied for the Marine Corps's warrant officer program. He had no desire to be a regular, commissioned officer, and even if he did, that would require a bachelor's degree to get there. He was now 27 years old and had cobbled together nearly two years worth of credits and was on the verge of getting an associate's degree but needed one more core class. So even if he could go to college full-time, he'd be too old to go to Officer's Candidate School unless the Marine Corps gave him a waiver for age.
He didn't feel like he had much of a chance to be selected for warrant officer on his first try, but his first sergeant and company CO were adamant that he was exactly what the Marine Corps needed, so he applied.
He felt better about his chances but still wasn't excited about his chances, and now, after learning about his mother's health issues, it was way down his list.
In fact, his concern was great enough that he'd asked for something he never imagined doing. He put in for a humanitarian transfer to be closer to his mom.
Again, his chain of command came through with strong endorsements recommending his request be approved, and yet he was still shocked when it happened in record time. It wasn't exactly a humanitarian transfer but rather a six-month temporary duty assignment or TAD in the Navy and Marine Corps but TDY in the Army and Air Force given for reasons of hardship.
It seemed to most people as a distinction without a difference, but it meant he had to pay his way there which was just some gas money, and it wasn't an actual PCS or permanent change of station move. Six months later he would return to Camp Lejeune and finish his tour there.
That had been just one week ago, and he was now living back home and assigned to Hanscom Air Force Base in Bedford, just a few minutes from his childhood home where his mother still lived.
Because there were no Marine or even Navy facilities nearby, he'd been assigned to the closest military base to his mother's residence and ended up working for an Air Force Chief Master Sergeant/E-9.
Then again, SSgt Decker wasn't exactly 'working'. The Chief, as the Air Force called their E9s, was very sympathetic to what the only Marine he'd ever had assigned to him was going through and told him to call in once a day and let him know he was alive. It was too good to be true, but it really was true.
So Chad gave the older man a call to let him know where they were going and what they'd be doing.
"Got it. Now go take care of your mom," the 45-year old, 27-year veteran told him.
"Will do, Chief," Chad replied before hanging up.
Chad knew after doing some very basic, initial research, he'd be lucky to even have a mom two years down the road. The good news was that her doctor knew another doctor who was conducting a new ALS drug trial in nearby Needham, and he'd been instrumental in getting her enrolled in it before he arrived back home.
His mom would have to stay there overnight off and on, but could be home most of the time. The clinic was well funded and even had visitor's rooms for family members who needed a place to stay. They paid a minimal fee to offset costs, but it was something around $20 a day, and they always filled every room.
