Huntington’s disease is one of the cruelest genetic afflictions a family can endure. The condition, which affects around 41,000 Americans, is the result of an error in a person’s DNA that causes their brain to deteriorate, typically starting in their 30s or 40s. Symptoms include dramatic personality changes, Alzheimer’s-like forgetfulness and an eventual breakdown of motor and speech skills. Families are forced to watch as their loved ones slowly lose their sense of self in the prime years of their life, with no hope of recovery.
There is hope, however, that this devastating disease might one day be eradicated, thanks to rapid advances in gene therapy.
A new treatment has shown remarkable promise in a clinical trial, slowing down symptoms by an average of 75 percent among patients who received it. The results, released in September and not yet peer reviewed, come with caveats: The trial in England consisted of just 29 patients, and long-term effects remain unclear. Still, scientists believe this might lead to therapies that can address the genetic glitch before someone becomes sick.
That is just one of many recent breakthroughs achieved in gene therapy. In May, a team of researchers at Children’s Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania published a study detailing the successful treatment of an infant born with a rare and life-threatening genetic liver deficiency. The baby has since reduced the medications he needs to take, and his parents have been enjoying the thrill of watching his childhood milestones — eating solid foods and learning to walk.
The scientists used CRISPR, the gene-editing tool that won the Nobel Prize in 2020, to develop the therapy in a span of just six months. That made it the first-ever treatment specifically designed to correct a single patient’s genetic code. While such “precision” medicine is just beginning (and still extremely expensive), scientists are optimistic it might unlock personalized treatments that could help millions of people worldwide with rare genetic diseases whose conditions are not profitable enough for drug manufacturers to confront.
The Food and Drug Administration has approved dozens of such medicines in recent years. And many more are on their way: One has shown impressive results this year to address a disease that causes blindness in children. Another harnesses a harmless virus that can deliver a genetic “Trojan horse” that has shown promise to kill certain cancers.
The field of gene therapy remains in its infancy. The FDA approved the first CRISPR-based treatment in 2023: Casgevy, to treat sickle cell disease. There’s no telling what once unimaginable cures this technology might unleash.
Americans gathering for Thanksgiving might take a moment to contemplate the extraordinary power of these innovations. The scientists who spend countless hours poring over humankind’s genetic code in endless battle against disease deserve a hearty toast.
The post Thankful for breakthroughs in gene therapy appeared first on Washington Post.
