Sidney: You actually corrected all of those conditions? I’m beginning to feel a bit uneasy here. Did you correct the patient’s height too? What about looks?
Joshua: No, dad. Only real diseases are corrected, things that doctors will treat. Things that will kill or hurt you. If it wasn’t an inherited genetic cause for death or deterioration, I didn’t touch it. I mean dad, I would never want all the people to have blond hair and blue eyes. That would be boorr-ring.
David: I must say, short-guy, no one would ever say that you thought small. Can I get everyone’s idea on whether this project should proceed or not?
Martin: I have to agree with Josh, if we can do a proof of concept here with LPH Deficiency, then do a phase IIa trial with it, if it reduced progression of the deficiency in the slightest, it would be the greatest and most revolutionary breakthrough since penicillin, no since cleaning wounds.
Corey: I agree with Martin, we need to proceed slowly, but the virus vectoring mechanism and the DNA snipper were very well thought out.
Janet: I can see the logic of devising a global mechanism, but I agree we should proceed slowly. We would need to test it one disease at a time. The FDA will demand that anyway.
Sidney: Hmm, ‘solving all of mankind’s genetic diseases, one disease at a time’. That has a really nice ring to it.
Martin: I get the feeling that we all agree that this project is a go? OK, I think the next step is to start our plans on testing it. Sidney and I, will start writing up a study synopsis and then we can write up the complete study procedures, line up investigators, I think we’ll also need a full hospital setting for this one.
Sidney: Brown at the University of Saint Louis Medical Center would be perfect. He seems to be a regional magnet for LPH patients.
***
Roderick Coombs came into the basement garage of the building where Dr. Lester Brown had his clinic. He wheeled his son Malcolm into the elevator. It has been a three-hour drive from their home, but with less traffic on the return trip, would only take two-hours to go home. The boy was no longer able to stand or even feel his feet. Malcolm would be confined to his wheelchair for the rest of his life. Roderick wondered if there was a way to donate the chair to someone then. Malcolm’s hands and arms also had very limited movement. Fortunately, the muscles in his face and torso were still functioning, but not for long. Roderick looked at the reflection of his son in the mirror smooth door of the elevator rising to the fourth floor office. Malcolm’s face looked like his brown skin was stretched over a potato, with the characteristic swelling of his entire head and barrel chest on his 23-year old frame. The other thing that marred his son’s face was a very thick pair of glasses. Roderick let his glance move up to his own reflection. Roderick’s 51-year old dark brown face was well lined. He looked like he was in his late 60s. It was a combination of all the stress from the imminent death of Malcolm, the ever-increasing medical bills, and the two full time jobs to pay the bills. Life was taking a tremendous toll on Roderick’s body. Roderick couldn’t remember when he last had five hours of uninterrupted sleep. Ever since the Franklin incident, his life, once so full of promise, had collapsed. Roderick looked in the mirror at his practically bald head, with the small tufts of curly grey hair by his temples. Malcolm on the other hand, was in need of a haircut. Should he get Malcolm a haircut before or after dies? After a brief moment he felt that immediately before would be best. The money was very tight. Roderick stifled the tears coming out of his eyes. The elevator door opened and they exited.
Roderick wheeled the chair to the clinic door, pressed the automatic door opener and rolled the chair in. One bad thing about Dr. Brown is that his 10 AM appointments rarely started at 10 AM. Often the doctor was more than a half-hour late in seeing patients. On the other hand, Dr. Brown never did the HMO 10 minute checkup. He was as likely to talk about how Roderick’s jobs were going, as testing Malcolm. Roderick had arrived in the waiting room at 9:30. Perhaps it was the engineer in him to have a built in safety fudge-factor for the long ride in. On the other hand, it gave Malcolm a chance to talk to the other LPH kids. Malcolm’s voice was barely a whisper at this stage. These were the only real friends the boy had. He glanced around and saw some of the other ‘regulars’ there. Roderick parked the wheel chair near one of Malcolm’s friends, while he read the Scientific Frontiers magazine. Dr. Brown had gotten a subscription just for Roderick. Wednesday was the day the doctor scheduled his LPH Deficiency patients. For the last two years, all the LPH patients were younger than Malcolm - all of them. Roderick stifled back a tear. He had to be strong. Virginia was falling apart. It was Rod’s duty to be strong. It was Rod’s duty to hold everything together. A man has to do, what a man has to do. He had to be a rock.
At 10:15, Anita, the nurse, called Malcolm and Rod in. Everyone was on a first name basis at this point. Doctor Brown was unusually quiet today. He did a brief check-up, made some comment about a progressive deterioration in Malcolm’s arm and hand strength. He checked Malcolm’s ability to exhale into a device. He said, “It’s down to 28%.” The doctor no longer tested Mal’s grip strength by squeezing on the partially inflated blood pressure cuff. Malcolm’s grip strength was zero three months ago. Nerves do not grow back. Roderick saw the frown on the doctor’s face. The doctor did some additional testing. Roderick no longer listened to the words, but focused on the doctor’s expression, which was completely dour.
Finally, the doctor spoke to both, “I’m noticing some more progressive deterioration in Malcolm. I think you should start making some plans now for the next stage.” Dr. Brown didn’t need to explain what the next phase was.
Roderick was quiet. He thought, ‘I know the boy is dying, Whitey. No, Brown was always kind and helpful. Yes, I’m bitter. Life just gave me the dregs. But Brown was one of the good ones.’ What Roderick finally said was “Dr. Brown, thanks for giving it to us straight. How long?” The words were hard to say, but he had to.
Dr. Brown said, “Three weeks, maybe twice that. However, I’d like you to talk to someone; I have them in my office, about a possible study.”
Roderick perked up, hope? “I thought Malcolm wasn’t able to get into the LPHase study?” Roderick had known about that study since its inception. Most of the Wednesday LPH crowd were participating, and the parents all talked about it. It had been their only hope.
Brown sighed, “He isn’t. The company who is doing the study had certain physical restrictions that Malcolm didn’t fit. He’s just too sick. However, they have a new treatment, a very radical treatment, which might eventually cure the deficiency – genetically. It’s all very, very preliminary.”
Roderick perked up more, “Cure the disease? Make Malcolm better?”
Brown’s frown increased, “No, too many of Malcolm’s nerves are destroyed. Nothing we know about can repair nerve cells. Perhaps we can go into my office and you can talk to them.”
They wheeled Malcolm into the office. Two Caucasians sat in chairs, a man and a woman. The man was thin, in his mid forties, and looked fit for his age. He was not wearing a tie, but a ‘business casual’ blue shirt and a dark pair of pants. The woman wore a pants suit and a necklace with what looked like American Indian jewels on them. Roderick noticed that the wedding bands were identical. In fact, the man’s shirt buttons were miniature versions of the woman’s black jewels, an odd couple. ‘But what is this all about, Mal can’t do the LPHase study.’
Dr. Brown introduced the pair, “Malcolm Coombs, Roderick Coombs, I’d like to introduce you to Janet and Sidney Ryan of Organic Biotechnology. The company is the only one doing research in LPH Deficiency disease. They are the chief scientists there and the ones who developed LPHase. While their Phase III study on LPHase is still on-going and blinded, they and I have no idea how well the results are officially. Unofficially, I can tell you that many of my patients have not gotten any worse. For those in th
e study after six months and crossed over to the open-label active treatment I’m definitely seeing any less progression.” Dr. Brown smiled.
The man called Sidney Ryan got up, put Malcolm’s hand in his own and gave it a firm handshake. He then went to Roderick and shook his hand, while looking him straight in the eyes. Roderick took an instant liking to this man. The woman moved closer, put her hand on top of Malcolm’s, knelt beside him and introduced herself to him. ‘Where did I hear their names before? They sounded familiar.’ Roderick thought. ‘No matter, is there really a chance to hope?’
They then sat down. Sidney started, “I don’t know what Dr. Brown had said, but we are the only company doing research in LPH Deficiency. We’d like your son to be a subject in a completely new type of therapy.” Sidney cleared his throat. “It’s a completely revolutionary untried approach where we actually correct the defective DNA which causes the disease. On the other hand, as it’s geared only for humans, and we’ve never tested it outside a test tube. Your son, if he agrees would be the very first living creature we test this on.”
Roderick sat up, “Do you think it could cure him?”
Sidney initially looked down at the floor, held it there for a couple of seconds, and then he looked Roderick in the eye. Sidney slowly said, “No. My personal belief is that the product will kill him.” His voice tone then perked up, “I’m trying to be as honest as I can with you Mister Coombs. Have you ever read about the early airplanes?”
Roderick just finished a library book on early flight recently in his Midnight to 8 AM shift as a watchman. “Yes.”
Sidney had a slight smile and continued, “The first trial of anything typically fails. In fact, many dozen initial attempts failed. Even the first attempt by the Wright brothers failed. We are actually changing the DNA and there are 6 billion base pairs in the human chromosomes. Even with the best computers at our disposal, I personally think there will be major errors in our approach. In addition, your son already has irreparable nerve damage. Do I think it will work? Honestly, no. But your son could be instrumental in saving countless lives. He will be the first test pilot.”
Roderick looked down and angrily said, “We won’t get any benefit at all. What about you? What do you gain?”
Janet looked startled then spoke, “Us? It’s not for the money. There are too few patients with LPH Deficiency for us or the company to make a profit. Fame, Sid and I don’t care about that either. Mr. Coombs, we also have a boy with LPH deficiency. He’s 6 ½ years old. We want him to be able to reach Malcolm’s age and live.” Janet last words were barely above a whisper.
Roderick then said, “I’m going to have to think about it.”
Janet said, “Dr. Brown, how long does Malcolm have?”
Dr. Brown looked at Roderick, who gave a nod. “Three weeks, on the outside six. On the other hand maybe less.”
Sidney looked at Roderick and Malcolm, and then said, “Given the state of your son, we can give you a week to decide, before he gets too frail for us to attempt it on him.”
Malcolm moved his head, a signal that he wanted to talk. Roderick came close to his son’s mouth. The boy whispered, “Would it hurt?” Roderick repeated his son’s question aloud.
Janet put her hand on his again, “I have no idea. It’s never been tried. It could be a very painful death. However, if it gets too bad we will do everything we can to ease the pain. You will have round the clock medical supervision, the very best care.”
Roderick thought now was the time to be practical too. “Would Malcolm be paid for this?”
Janet looked up into space then at Roderick, “No, the FDA would allow us to pay only for medical expenses. It is against ethical procedure for patients to enroll for profit. However, I know of a charity that can make a donation to your family, so you could spend more time with him and pay for the other expenses.” She had a small smile. “This donation would be whether you agreed to be a patient or not. You have been through enough. Call them any time 24/7, like tonight.”
Janet opened her pocketbook and wrote down a phone number. “It’s C H Electricity charitable donations department. Then just give your name. There are no stings attached. None. Whatever your decision.”
Roderick said, “I’m going to need to think about it with Malcolm and his mother.”
Janet nodded, “Yes, of course.”
Roderick stood and left the office with Malcolm. He wheeled Malcolm outside near the elevator when Malcolm moved his head again.
“Yes, Mal?” Roderick walked around to the front of the wheelchair and bent down to hear his son’s whisper-soft voice.
Malcolm said, “Dad, I want to do this. I want to do this. I’ve never done anything useful in my entire life. Let my death give me meaning. Please, let me do this.”
“But Mal, I don’t believe them. They’re looking to profit from your death. They’re in it for the money. They’re in it for the fame. No one will remember Malcolm Coombs. No one.”
“Dad, Che Che and Louise might. Let me do this, please. I’m going to die anyway. Does it matter if it’s two weeks with this or three with no treatment?” Malcolm’s plaintive voice was barely a whisper. Che Che and Louise were two of the younger LFH patients. Hell, they all are younger than Malcolm.
***
“Hello, C H Electricity? Charitable donations department, please. … Hello, charitable donations department? My name is Roderick Coombs. … My address? 37 Wayview Avenue in Westphalia Missouri 65457. … Yes, that’s correct. I was told that there is some provision for helping a family with a dying child. … A check will be over-nighted by tomorrow? For $30,000? … Don’t I need to give you more information, like my son’s name? … Yes, he is Malcolm. That’s it? … A personal note from Charlie Haines himself? … Well, God bless you too, Miss. God does succor those in need.”
Roderick sat staring at the wall in his living room for thirty minutes. He was sitting alone in the darkness. Tears welled in his eyes. He would take a leave of absence from his jobs tomorrow, until … until …
***
Roderick and Virginia were with Malcolm as they wheeled him into the empty hospital room. Bright blue light bathed the large room, with a view of the Saint Louis Arch through the large picture window. Lamps, which looked more at place in a fancy living room, lit the room and a painting adorned one wall of a sailboat racing along with billowing sails straining against the ropes on choppy seas. There were other, extra lights, giving off a bright blue light. Roderick remembered about that ultra-violet light killed the virus.
A small table was in the corner, near a small cube refrigerator. A second bed, a standard double bed, was alongside the hospital bed. Roderick noticed a small cabinet. It looked like a hotel, not hospital, cabinet. He opened it. The bottom shelf contained sealed snacks, paper plates and cups. The top shelf contained toothbrushes, razor blades and other bathroom items. Curious, Roderick opened the refrigerator; it contained a small freezer with ice cubes, fruit, a few sodas, juices, and bottled waters. Above and behind the patient’s head was a blank 36” flat-screen monitor. Another flat-screen 54” TV faced the bed. Roderick guessed that the hospital had suites like this, but now he knew. Roderick thought, ‘this room reminds me of the Hilton rooms I stayed at when I was working at Franklin Industries. No this is nicer.’
Dr. Brown again told Roderick, Virginia and Malcolm all about the nature of the DNA snipper and the virus. How the virus would behave, how it would disintegrate in two days, how light and alcohol would destroy it, and, finally, how the snipper would manipulate every gene in Malcolm’s body. Virginia had only heard this second hand.
Brown gave Roderick and Virginia the hazmet suits, just like the doctors. The suits looked like what firemen used to fight chemical fires. Rod and Virginia drank a glass of wine and told they had to have a half glass or more every four hours they were in the room or a full glass if they wan
ted to lie down. A bottle of wine, from some vineyard in Italy, was on top of the cabinet. They could hold the boy’s tin foil covered hand and talk with him, but he told them that the boy must remain in the dark for the next few days.
They all knew this was the end, and were trying to keep a brave face on for the others. They were told that probably nothing would occur for two days, but Malcolm needed to stay under a aluminum foil tent. Malcolm was set up with an IV, and given a pair of goggles/earphones that allowed him to watch TV.
A nurse, also wearing a hazmat suit, came by and put various leads on the boy’s chest, abdomen, and skull. Then she attached the leads to a machine behind the bed. She turned on the monitor out of Malcolm’s line of sight that displayed his breathing rate, pulse, blood pressure, temperature, and electroencephalograph. She sat in a chair facing the monitor displaying Malcolm’s vitals signs, opened a thick book of fiction, and started to read. She then set up the tin foil tent that would be Malcolm’s home for the remaining time.
Doctor Brown came in and injected the virus into the foil covered IV. He then sat at Malcolm’s side, “You know Malcolm, medical history will be made today. You are as much of an adventurer as Lindberg and Columbus, maybe even greater.” After waiting ten minutes, talking to Malcolm, Roderick and Virginia, Dr. Brown left. The long wait began. It was Monday at 9 AM.
***
Virginia was the first to notice that the temperature, which had been 36.9 increased to 37.3 degrees Celsius. The nurse checked the leads, she said that is very normal and nothing to worry about. Roderick did a quick mental calculation; the temperature is slightly higher than 99 degrees Fahrenheit. The nurse sat back down again and went back to her book. It was Wednesday at 5:45 AM. Over the space of the next two hours, the temperature rose to 38.4. Roderick did some more arithmetic and realized that it was 101 degrees Fahrenheit. He didn’t tell Virginia of the Fahrenheit temperature. Roderick noticed that the nurse, under her hazmet helmet, was still smiling, but her book was closed. The temperature rose another half degree. A resident appeared. He removed the struts of the tent and had sealed ice water bottles placed along the side and on top of Malcolm. The aluminum covering still covered Malcolm, but his breathing became more labored. Doctor Brown appeared twenty minutes later, it was 10 AM. An oxygen mask was placed on Malcolm’s face. In the space of 10 minutes, Malcolm’s temperature increased 3 more degree Celsius. The ice water was replaced by ice.