{"id":4205,"date":"2019-10-11T14:09:01","date_gmt":"2019-10-11T05:09:01","guid":{"rendered":"http:\/\/163.180.4.222\/lab\/?p=4205"},"modified":"2019-10-11T14:09:01","modified_gmt":"2019-10-11T05:09:01","slug":"the-first-drug-designed-for-a-single-patient-scientists-designed-a-drug-for-just-one-patient-her-name-is-mila","status":"publish","type":"post","link":"https:\/\/biochemistry.khu.ac.kr\/lab\/?p=4205","title":{"rendered":"The first drug designed for a single patient – Scientists Designed a Drug for Just One Patient. Her Name Is Mila"},"content":{"rendered":"

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An achievement in ultra-personalized medicine also raises questions about fairness and regulation.<\/p>\n

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Julia Vitarello with her daughter, Mila, 8, who has a rare neurological disorder. Scientists were able to create a drug to treat only the form of the illness she has.<\/span>Credit<\/span>Credit<\/span>Nick Cote for The New York Times<\/span><\/p>\n

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By\u00a0Gina Kolata<\/span><\/a><\/p>\n<\/div>\n<\/div>\n<\/div>\n

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A new drug, created to treat just one patient,\u00a0has pushed the bounds of personalized medicine<\/a>\u00a0and has raised unexplored regulatory and ethical questions, scientists reported on Wednesday.<\/p>\n

The drug, described in The New England Journal of Medicine, is believed to be the first \u201ccustom\u201d treatment for a genetic disease. It is called milasen, named after the only patient who will ever take it: Mila (mee-lah) Makovec, who lives with her mother, Julia Vitarello, in Longmont, Colo.<\/p>\n

Mila, 8, has a rapidly progressing neurological disorder that is fatal. Her symptoms started at age 3. Within a few years, she had gone from an agile, talkative child to one who was blind and unable to stand or hold up her head. She needed a feeding tube and experienced up to 30 seizures a day, each lasting one or two minutes.<\/p>\n

Ms. Vitarello learned in December 2016 that Mila had Batten\u2019s disease. But the girl\u2019s case was puzzling, doctors said. Batten\u2019s disease is recessive \u2014 a patient must inherit two mutated versions of a gene, MFSD8, to develop the disease.<\/p>\n<\/div>\n