Researchers developed a custom genetic drug just for one patient, eight-year-old Mila Makovec, who has a progressive neurological disorder, the New York Times reports. It adds, though, that this type of super personalized medicine opens up questions of how to assess the safety and efficacy of the treatment and how to pay for the treatment.
Boston Children Hospital's Timothy Yu and his colleagues developed milasen, a splice-modulating antisense oligonucleotide drug tailored for Mila, who had a novel mutation in the MFSD8 (also known as CLN7) gene. As they report in the New England Journal of Medicine this week, Yu and his colleagues have been treating Mila with the drug and it appears to be have reduced the frequency and duration of the seizures she was experiencing.
The researchers add that they were able to treat Mila with this drug designed for her within a year and that their approach could serve as a framework for other patient-centered studies.
In a related editorial in NEJM, Janet Woodcock and Peter Marks from the US Food and Drug Administration write that N-of-one studies like this raise questions about what sort of evidence is needed before a patient can be exposed to such a tailored drug, how drug dosage should be determined, and how the drug's efficacy should be gauged. They add that the agency plans to address these issues in coming months.
The Times further notes that such super personalized treatments are likely to be expensive — Yu and Mila's mother, Julia Vitarello, declined to say how much was spent in this case — and not covered by the government, drug companies, or insurers, leaving treatments like these available only to the wealthy, those who can raise large sums of money, and those, like Mila, who get support from foundations.
To change this, Yu tells MIT's Technology Review that he and Vitarello hope to develop a network of academic medical centers that are working on personalized drugs to share their results, though he says that too will likely have to rely in part on support from foundations.