Personalized medicine is having its day From made-to-order genetic therapies to model organisms engineered to be ‘patient avatars’, the technology exists right now to save patients with rare diseases
gene , which, when mutated, causes an aggressive form of amyotrophic lateral sclerosis . It’s named for Jaci Hermstad, who in 2019 became the first patient to receive the drug, but it wasn’t designed specifically for her. The drug was already being developed by Ionis and collaborators at Columbia University and had shown efficacy in awhen Hermstad received her diagnosis at age 25.
Yu says the most likely hypothesis is that the fluid buildup is a class effect, but the mechanism responsible for it remains unclear. “It would be really nice if we could simply just get some tominersen and get some valeriasen and do the appropriate experiments to try to identify mechanistic commonalities between the two sequences,” he says. “That isn’t exactly how the world always works.
“A lot of clinicians might have a patient with a rare disease and want to help, but don’t have any idea how to approach the FDA. Where do you find a source of oligonucleotide for under $500,000? For a single patient?” Watts says. “Sharing information and help, guidance and resources and protocols around all of those kinds of questions is another thing that the more clinically minded people in the collaborative do for each other.
Crooke expressed support for the idea of establishing databases, as long as minimum standards for preclinical testing processes and performance are defined and rigorous clinical evaluation protocols with well-defined clinical endpoints are agreed on. “As such, n-Lorem is open to contributing data to the N=1 Collaborative or other data platforms as appropriate,” he says.
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