Personalized, one-off medicine is becoming a reality
And for a few lucky patients, it's entirely free
Longevity Hound
March 30, 2023
In 2018, a girl named Mila became the first person ever to receive a drug designed for just one patient
What's the news: Personalized, "N-of-1" (as in, just for one patient) medicine is becoming a reality, at least for a few lucky individuals.
Why should we believe it: This news comes from an article published earlier this month in the science journal Nature Biotechnology. The article summarized a few recent highlights in the field of personalized medicine:
In 2018, a girl named Mila, who suffered from a rare genetic disease, became the first patient ever to receive a custom medicine using antisense oligonucleotide (ASO) technology. Remarkable, the treatment was developed and then administered in just one year.
In 2020, the n-Lorem Foundation was founded. This organization funds the development of experimental ASO treatments for genetic diseases that affect fewer than 30 patients worldwide.
Last October, an 8-year old girl named Susannah received her first dose of a custom-made ASO medicine to block a rare but devastating neurodegenerative disease. Susannah got the treatment for free, courtesy of the n-Lorem Foundation.
Why this is a big deal: Much long research naturally goes to treatments that can benefit the greatest number of people. But revolutionary research is quietly going on to treat very unique diseases as well, and real people are benefiting from those.
What's more, because these treatments are applied to only one person, who is often at risk of imminent death, the FDA has been allowing treatments it would never allow on a broad scale without many more years of testing.
"You can make a really strongly ethical case that if something is safe in rats, and a patient is going to die, or is going to lose neurons, it’s a better move for that patient to proceed than to wait for a monkey study, because the risk of not treating outweighs the risk of treating."
The long-term view: Ultimately, many of us might benefit from personalized medicine, even if we are not at risk of imminent death. In time, the technologies and insights gained from treating patients with rare and deadly genetic disease might benefit many of us directly. As one researcher in the field, Ethan Perelstein, put it, "What were these few N-of-1s is going to become a tide of N-of-1s."