Clinical Trials

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.

When they arrived, Chad helped his mom walk in, and it hurt him to see how difficult it was for her to just to something as basic as walking. Even with a cane she was only able to move at an extremely slow pace as her right leg had to be nearly dragged along. Once it 'caught up' it wasn't strong enough to support her 115-pound weight without the cane, and he knew she would soon need a walker. They eventually made it to the front desk, and as always, she remained pleasant throughout the check-in process.

Much of the paperwork had already been done ahead of time online, and there were only a few forms to fill out. Once a patient was checked in, he or she could go to their assigned room or to the cafeteria until the first event which was a welcome by the doctors conducting the trial at 2pm.

It was just after noon, so the two of them slowly made their way down a long hallway where the smell of food wafted their way. There were wheelchairs available for anyone who needed one, but Chad's mother refused to sit in one until she had no other choice.

"Something smells good," his mom said as she looked up at her son and smiled as they saw the sign for the cafeteria.

"You must be very hungry for cafeteria food to smell good," Chad replied, smiling back at her.

"Oh, I'm sure it won't be all that bad," she cheerfully said as she shuffled along.

Chad grabbed two trays and slid them along as they moved through the buffet. As they were making their way from right to left, he noticed two other people getting in line behind them. One was a woman about his mom's age and the other a woman he assumed was maybe as old as him.

When he glanced her way, she smiled at him, and it took Chad a moment to realize she had. He wasn't a 'pretty boy' but he was definitely considered 'ruggedly handsome' and had never had any trouble meeting women. And while this particular woman was pretty enough, he had other things on his mind, so he only gave her a little nod then continued down the line.

As they sat and ate, several other pairs of people also came in, and Chad realized that each one of them had a story to tell as one of the two undoubtedly had ALS. But it was more than that. They had an entire life story, and now they were facing a death sentence, and it hit him hard just how awful this disease was.

He took a quick look at his mom and realized she'd been talking away and that he hadn't heard a word she was saying. He apologized and admitted he hadn't been listening, and his mother told him it was no big deal.

"I was just rambling on, anyway," she said in that friendly way of hers, just happy to have her son home and with her.

Chad forced himself to focus, but his concentration was broken again when he saw two more people get in line to eat. One was an older man, clearly older than his mother, and the other was a woman who looked to be in her mid-30s. She had shoulder-length blonde hair and a face as attractive as any he'd ever seen. She also had a body to match the face, and he found himself unable to stop looking her until he heard his mother's voice again.

"Sorry, Mom. I don't know what's wrong with me today, but...."

His mom turned around, saw the beautiful woman then told her son, "I'd say there's nothing wrong with you at all, honey. She's very pretty."

"Yeah. She uh, she really is," he said kind of absentmindedly and a with a little embarrassment for being such an...ass.

"You think that's her father?" she asked.

"Probably. Or maybe an uncle?" he replied as he continued looking at the woman who was very nicely dressed, as well.

"I can't imagine doing this without a family member. It'd be...awful."

He broke out of his daydream and looked right at his mom as he put his hand on hers and said, "I'm so glad I can be here for you, Mom."

She put her other hand on her son's and told him she was, too.

As this other pair slowly walked by them, Fran surprised her son when she looked up at them and asked, "Excuse me. Would you like to join us?"

The woman her son had been staring at was even more attractive up close and smiled at Fran.

"Oh, thank you, but we don't want to intrude," she said politely.

"If you're also here for the trial, you're not intruding. I promise. Right, honey?"

Chad was staring again, and it took him another moment to realize he was being spoken to. Again.

"Um. Yes. I mean, no. It wouldn't be an imposition at all," he replied without taking his eyes off of the older, beautiful woman standing just a few feet away.

"Dad? Do you mind?"

"No. Not at all, honey. It'd be nice to meet someone else," he said.

She was carrying both trays and was relieved to be able to set them down. Chad saw her struggling and quickly reached out and took one of them off her hands. Literally.

"Thank you. I didn't have any trouble carrying it, but setting it down proved to be a little more difficult."

She smiled as she said it, and again, Chad couldn't help but notice she had a beautiful smile and also the bright, blue eyes on a face as pretty as any he could recall.

"Sir? May I give you a hand?" Chad asked as the older man struggled with the chair.

His pride didn't want to let him say 'yes', but when he tried to move the chair, he nearly lost his balance. Chad was up and there in an instant and steadied the man.

He pulled the chair out then said, "Here you go, sir."

The older man thanked him, and Chad noticed the beautiful blonde looking at him. He was no stranger to pretty women, but she was disarmingly attractive, so he just smiled quickly then sat back down after making sure her father was seated.

"I'd have helped you with your chair, too, but you were a little too fast," he told her with a smile.

She smiled and thanked him for helping her dad then introduced herself.

"I'm Hallie Dav...Emerson, and this is my father, David Emerson."

Fran then introduced herself but let her son decide what to say, if anything.

"Chad. Decker," he said, looking first at Hallie then her father then back at her.

"Pleasure," Hallie replied. "I just wish it wasn't under these circumstances."

"Life's about change, Hallie. And our happiness depends on how we adapt to it," Fran offered optimistically and in a way that didn't sound rude or pushy.

"I...I guess that's true," she said after thinking about it for a moment.

"Smart and pretty," David said, as he tried to smile at Fran.

"Dad. Are you flirting already?" his daughter teased.

"I don't have any time to lose, honey," he told her.

The 67-year old man tried to laugh, and what he'd said didn't come out quite right as his speech was slightly slurred. It was still understandable, but one had to listen carefully to make sure they got it right.

He looked at Fran again then added, "She's a little too young for me, though, I'm afraid."

He paused then changed his mind.

"Actually, I'm a little too old for her."

Fran laughed and told him age didn't matter much...after a certain age.

"I like this one!" David said as he sort of laughed again.

The two patients hit it off immediately and led the conversation as they discussed their symptoms and diagnoses. David was having difficulty walking, too, but his primary symptom, as of late, had been with swallowing.

Not sure everyone understood, Hallie did her best to explain it. Chad and his mom knew everything she said but let her talk knowing she might possibly say something they hadn't yet heard.

"That that means is that my dad has the 'bulbar' form of ALS," she began before summing it up while carefully avoiding the gory details.

"I gotta be real careful," he said as he started eating. "Gotta cut everything up as small as possible and chew the hell out...sorry. Chew the heck out of it."

He started to try and cut things up, and it was obvious that even that simple chore was difficult for him. But he was managing, so Chad didn't offer to help.

Fran laughed, and so did the 'kids', but it wasn't really funny. It was the kind of dark humor people who knew they were in serious trouble used to minimize their fears.