Jacob is doing awesome. There’s no question of that. He has amazing doctors, a wonderful nutritionist, and his condition is well managed. But let’s face it… “well-managed” is not the ideal. “Cured” is the ideal. And there may be hope on the horizon!
Yesterday, I ran across this article: “Ex-Pfizer Exec Gutierrez-Ramos to Lead Synlogic’s ‘Smart Bug’ Plan.” It sounds like science fiction. The concept is that scientists can engineer bacteria to find a disease or infection, treat it, and then self-destruct. You would think they would start going after big fish first – breast cancer, heart disease – but instead they are first going to target UCDs!
“Given this is a new, unproven technology, Synlogic’s strategy is to start out going after a few rare diseases, where the ‘benefit-to-risk ratio is extreme,’ and the company can know quickly whether it’s onto something, [CEO Gutierrez-Ramos] says. Its first two candidates will target urea cycle disorders (UCD), the name for a group of genetic diseases that trip up the body’s waste-disposal system; and phenylketonuria (PKU), an inherited disease characterized by the abnormal buildup of an animo acid called phenylaline…”
Can you imagine? It’s like a real-life magic schoolbus filled with citrullinemia-fighting ninjas. What if Jacob could be cured – really cured, not liver-transplant cured – with something as simple as a shot?