Gene Solution (eBook)

I.

Fifteen years later
3 August 2017
New York, New York

Approved.

That was the gist of the email that Dr. Allen Charles ‘Tripp’ Galloway had just read to himself six times. Falling back into his office chair, slumped and numb, he couldn’t believe it. After all these years, he had finally done it.

Tripp convinced the Food and Drug Administration (FDA) to start an international, multi-center, double-blind, Phase III clinical trial that would end sickle cell anemia. What that actually meant is that Tripp was going to wipe that shit stain of a disease off the face of the planet and the FDA was going to let him. The FDA, the goalkeeper to his dream, just let him score.

Tripp wasn’t some head in the clouds, “save the hippos,” type of doctor. He was a realist. It’s what kept his patients from believing that he can do it all. At this moment, Tripp himself was beyond surprised. He had truly outdone himself. The FDA is the arbiter of risk when it comes to clinical life in the United States. This approval was a vote of confidence in the doctor.

Tripp reached under his desk to the mini-fridge. It was the one thing in his practice that wasn’t ornate. Simple, black and filled with Coca-Cola, it fit directly under his desk. The man, although generally healthy, had an immense caffeine addiction.

Next to the stacks of soda was one bottle of Chandon. Taped to the bottle was a bright pink note that stated, “Fuck you sickle cell … Love, The Team.”

Getting excited, Tripp ripped coffee mugs and glasses off the shelf and hurriedly walked into the hallway. If you followed the light blue walls to the right, they would come to clinical grade labs with incubators, water baths, centrifuges and microscopes, gleaming in the artificial light.

A little further, there were rooms to see patients. Tripp hated those rooms. As an OB/GYN and fertility specialist, he always delivered news in those rooms. Parents found out if they could ever raise a child of their own … ever. It really was a crap shoot depending on the day. He told Aiden, his assistant and trial manager, to schedule patients so that it was a healthy mix of shitty and great news. Some days it was rough. Telling three couples in a row they couldn’t have children was never a great time. Finally, at the end of the hall is the waiting room. It was filled with brown leather chairs, a flat screen television and the latest news about the Genetic superstars – Galloway and Stein, Tripp’s private practice.

Tripp turned left and opened the door into the break room. Tony, Tara, Michelle and Aiden were gathered around the table eating the meal of the day. It looked like tacos. Tony Kim, a self-described “bad Korean” had three-quarters of a taco in his mouth when he looked up.

“Doc, what’s up? You want some?” Tony asked with guacamole on his face.

Tony, a short stocky guy with a buzzed head and tattoos, oftentimes forgot his manners. Tripp didn’t mind, though, because Tony was a magician when it came to culturing cells. Without Tony, the business would fail.

“No, where’s Morti?” Tripp asked quickly.

Tara and Michelle were eyeing the champagne. You would think those girls were boozehounds instead of nurses. Aiden, knowing damn well what that bottle meant, stood quickly and said over his shoulder, “His wife called; I’ll get him.”

Tara and Michelle continued to eye the champagne as they pounded down tacos. Nothing got in the way of their food. Tara and Michelle looked like twins except one was from New Jersey and the other was from Vermont. With her curiosity sufficiently riled, Tara managed to squeeze out a “What’s the occasion Doc?” as she polished off her fourth taco.

Tripp ignored the question as Aiden and Morti strode in. Dr. Mortimer Stein was the opposite of Tripp in most ways. While Tripp was tall, white and horribly single, Morti was short, black and horribly married.

It all started when Tripp met Morti at Harvard Medical School. Morti was attending a lecture on Epigenetics when Tripp plopped himself down next to him. Epigenetics, or the study of how the environment can alter your genetic expression, is a little dry. Morti and Tripp commiserated on how boring the talk was and it was history from there. The two things that bothered Tripp was that Morti was a Red Sox fan (Fuck the Sox) and Morti had gotten into Tripp’s dream school, Boston University. So naturally, fuck BU Med. What Tripp liked the most about Morti was that he had a sense of humor. Morti would often use his name and appearance to his advantage. Most people don’t expect a Mortimer Stein to appear as Morti does. Mortimer Stein was adopted by an infertile Jewish couple living on the lower east side, not too far away from their now lower west side office.

Morti, spotting the bottle, looked up. “What’s with the ‘Fuck you bottle’?”

Galloway couldn’t control himself, with a grin he said, “They fucking approved it. We did it!”

Michelle, still chugging along on her tacos said, “Madre de Dios, you’re kidding me right? The FDA is gonna let us do that shit?”

“Are we getting raises?” Tara asked.

Aiden was silently beaming and Tony let out an “Oh Shit”. They fucking did it.

The bottle was popped, champagne was poured and appointments were canceled for the day. Tara and Michelle pulled out a bottle of Smirnoff from some mysterious filing cabinet. Aiden ordered enough dumplings from his favorite place on Mott to feed a small army.

As the festivities continued, Tripp raised a small test tube of Smirnoff and looked at the team. “You all have worked so hard to get to this point and Morti and I can’t thank you enough. With a little luck, we’re gonna be the people who make sickle cell a thing of the past. So with that, fuck you sickle cell. Cheers!”

Hours, and about seven shots later, Tripp was dropped off by an Uber in front of his Upper West Side brownstone. A simple oak door stood in between Tripp and another drink. The hangover was going to be unholy, but he didn’t care. It was a celebratory hangover. It also meant that he would be going to Jack’s BBQ tomorrow morning. A rooster platter was calling his name.

Tripp unlocked the front door and immediately went to the bar cart. While making a vodka and ginger ale, he clicked on the stereo and took a seat. It was nearly an impossible feat, getting the wimpy fuck Myers to approve the trial. That FDA schmuck really had no balls. He understood the hesitation though. An infertility specialist and a geneticist wanted to eliminate the sickle cell mutation from the human race. The trial would take years, if not a decade. The money it would take: astronomical. The process was pretty simple, but in thousands of people it would take time.

The trial itself was a technical wonder, thanks to Aiden. An international, multi-center, double-blind, Phase III clinical trial was serious business. Each word is packed with nuance and added difficulty. International: they were looking at patients from all over the world. Multi-center: they were collecting samples from multiple locations. Double-blind: Nobody knew which patients received the treatment or a placebo. Phase III: It was to be conducted on humans.

The affected patients would come into clinics around the world and give their bone marrow. The bone marrow would be sent to Aiden and randomly selected to be treated or be a placebo. If the marrow is chosen to be a placebo, that sucks for them. All they get back is their marrow and their sickle cell. However, the marrow that is chosen for the procedure is sent to Tony and Stein. They create stem cells and, using the CRISPR/ Cas-9 editing platform, edit the affected patients’ DNA. Voila, no more sickle cell. This means eighty thousand Americans and millions of other people would be able to live happy and healthy lives.

This CRISPR/Cas-9 platform utilizes nature to cut out the nucleotides, or DNA building blocks, and replace them with whatever you want. The Cas-9 does the cutting while the CRISPR does the switching. Tripp had to admit, nature was brilliant sometimes. From there, and this is the cool part, surgeons conduct full bone marrow transfusions, thereby eliminating any sickle cell anemia in the previously affected patient.

Of course, the process of complete cell switch over took some time. Also, women’s eggs were never switched as women are born with all of their eggs. They might have a problem if they decide to have children. It didn’t matter though, within three generations the mutation would be gone except for reversions, or random mutations that turned sickle cell back on. This is due to the way sickle cell is inherited in people, both parents need to pass on the gene to their child, falling on the wrong side of 25 percent. By changing the males, that cannot happen.

Tripp was pleased. After fifteen years of school and hard-work, he finally, at age thirty-five, was the man he thought he should be. His mother would disagree, but honestly, he didn’t give a shit; he was single and successful, not married and miserable. He could do what and whom he wanted.

This was just the beginning too. In a couple of years, if all went to plan, they’d be putting him in the history books. As he tipped his glass back and...