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“You all knew my dad, Dr. Garret Phillips. He died at sixty with the cardiovascular, immune, and metabolic profile of someone in their mid-to-late eighties. If this treatment had been available to him then, according to our data, he should have aged in the way all of you are aging.”
Nope, saying that was a mistake.
The offense was palpable.
“He should have aged the way a typical body ages, not you all. You all are doing super well.”
“So you’ve found the fountain of youth. How soon until it’s a deliverable?” The man to Brandon’s left asked.
Piper put her hand to her heart. “I … no.”
“Twenty years. Isn’t that something?” he asked.
“For Runker-Bailey Syndrome patients, yes it is. It’s a very exciting time,” Piper started.
“Let’s talk about off-label usage.” Brandon flapped his tie, then swept his palm down its length to settle it in place.
“We need to get it through the FDA to begin with,” Piper said. “What off-label are you considering? I … there’s no off-label use.”
“We financed your father from the very start and continued with this project because your parents were friends of Lionel.” Lionel’s sister, Ria Harcourt-Swain, was the Alabaster CEO and Brandon’s mom. They often tag-teamed.
“I’m very grateful.” Piper tried to find the right way to hold her face, somewhere between a science professional on the brink of introducing a major new life-saving therapy and a supplicant kissing the hem of the royal robe, hoping for alms.
“But in the end, we’re a business,” Ria concluded.
“I appreciate the ongoing support. We’re so very close. While this doesn’t affect large swaths of the populace, it has the potential to save lives and spare families incredible pain. And I see it as a wonderful way to let your consumers feel good about using the Alabaster label—beyond the benefit of the beauty product, of course. Without an ulterior motive, you’re helping save lives. It’s wonderful PR.”
“My understanding is that you’re here today to tell us about getting the FDA’s thumbs up and get this product out the door to the public,” Brandon asked. “And once it has FDA approval, doctors can write prescriptions. That’s your aim.”
“Yes.”
“And once a drug is available via prescription, doctors can write that prescription for off-label use. So this medicine might do more than just help people with Runker-Bailey Syndrome. There might be a broader market.” He walked her through the chain of future events like someone holding a child’s hand while crossing the street.
“Oh, I think I understand what you’re asking me. Yes, I am here to talk about the FDA. But no, it isn’t possible to use this drug off-label. While this therapy is safe and effective in treating the SSR1030P mutation with limited side effects in mice and rats, that doesn't hold true for the broader population. My lab did extensive testing on a mouse population without the SSR1030P mutation, and we discovered a cancer risk associated with use. That’s why it’s imperative to have genetic testing in advance to verify that the mutation is present.”
“A high cancer risk?” Brandon asked.
Piper scowled. “Any risk is too high when a medication isn’t needed. My main concern with even suggesting an off-label use was something we talked about last year when I gave you my updated timeline. This formulation has specific negative outcomes for offspring of someone exposed to this therapy. So the individuals must—"
“How many doses does someone need?” Ria asked, “Do they continue to take them for life? Is it a daily dose so they have that ongoing expense?”
“It’s a one-time shot,” Piper said, thinking they’d appreciate the ease of treating Runker-Bailey Syndrome. But then, she caught the calculating look on Ria’s face, and Piper realized she misjudged the situation. Maybe they wanted patients to depend on daily doses and the corresponding costs.
“Twenty years dissolved with one dose.” Ria’s mouth twitched with what threatened to be a smile as she turned to Brandon, then rounded back to Piper. “And what happens with the second dose?”
“Yes, the effects …” Obviously, there was some unexpected undercurrent she needed to navigate. Piper centered herself. “This is not the scientific explanation. It’s the vibes.”
They blinked.
“It’s a stick-figure-picture kind of explanation. Basically, why people ‘get younger,’” Piper made extra sure to trap that phrase in air quotes, “is that it removes the twenty years of accumulation that happened too quickly in people with Runker-Bailey Syndrome. In essence, it’s prolonging the disease for twenty years. By the time twenty years have passed, their systems are about the same as their peers, and they will die on the normal life expectancy timeframe.”
Brandon lifted his eyebrows. “But with the second dose twenty years later …”
“Patients can’t get a second dose because it makes the cells reproduce with mutations causing fast-growing cancers. Typically, it starts in the pancreas and quicky metastasizes to the liver and other organs. One can’t think of this as a cure. It is only a prolonger of life.”
That wasn’t even English, Piper scolded herself. She picked up her water and sipped.
“So let me dive into next steps for this intervention drug,” Piper said. “As you all know, Runker-Bailey Syndrome is rare. We cannot go through the normal trials to bring this to the FDA because there isn’t a large enough population that needs it. Therefore, there isn’t a large enough population to test the formula in the typical phases. When my dad was alive, this was a major issue that he wasn’t sure how to overcome. However, since then, the U.S. signed into law something called the ‘Right to Try,’ which developed a separate pathway to drug approval. These guidelines say that a terminal patient—and that’s anyone with signs of Runker-Bailey Syndrome—and their medical team can request a drug from a manufacturer even if the manufacturer hadn’t submitted an application to the FDA and didn’t have FDA permissions. And that is what I want to do. Make this available to those who choose to try it.”