The specialised custom created drug market has some new news. A new drug, created to treat just one patient, has pushed the bounds of personalized medicine and has raised unexplored regulatory and ethical questions whether this is okay to do as there are many patients around the world suffering form the same issue is this not curtailment of their right to have proper medication irrespective of their social or economic status.
The drug is believed to be the first “custom” treatment for a genetic disease. It is called milasen, named after the only patient who will ever take it: Mila Makovec, residing with her mother, Julia Vitarello, in Longmont, Colo. Mila, aged 8 has a rapidly progressing neurological disorder that is fatal. Her symptoms started to show 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. Ms Vitarello learned in December 2016 that Mila had Batten’s disease in a diagnosis which was also weird as she had one just mutated gene, and the other copy seemed normal. That should have been sufficient to prevent the disease. Dr Timothy Yu and his colleagues at Boston Children’s Hospital discovered that the problem with the intact gene lay in an extraneous bit of DNA that had scrambled the manufacturing of an important protein. He then made a custom piece of RNA to block the effects of the extraneous DNA which was expensive as there is no cure for this his team developed the drug and tested it.
In January 2018, the Food and Drug Administration granted permission to give the drug to Mila. She got her first dose on Jan. 31, 2018. Mila’s condition has been improving with continuous treatment and her seizures have been reduced to none or six in a day. Now able to eat pureed foods but remains largely disabled.
Tags : Julia Vitarello, in Longmont, Colo, DNA, RNA ,