‘Custom’ Drug Designed For Just One Patient Sparks Questions About The Boundaries Of Personalized Medicine
Although scientists found some success with the highly specialized drug, the path forward to medicines created for just one patient is murky. Questions about fairness and expense put a damper on the possibility of such personalized care.
The New York Times:
Scientists Designed A Drug For Just One Patient. Her Name Is Mila.
A new drug, created to treat just one patient, has pushed the bounds of personalized medicine and has raised unexplored regulatory and ethical questions, scientists reported on Wednesday. The drug, described in the New England Journal of Medicine, 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 (mee-lah) Makovec, who lives with her mother, Julia Vitarello, in Longmont, Colo. (Kolata, 10/9)
After A Bespoke Therapy Rescues A Young Girl, The FDA Considers Advance Of Individualized Treatments
Back in 2016, a 6-year-old named Mila was diagnosed with Batten disease, a progressive and incurable genetic syndrome that would brutally strip away her sight and her ability to walk, and would cause dozens of seizures each day. The condition is fatal. But her parents, Julia Vitarello and Alek Makovec, refused to lose hope. They contacted Dr. Tim Yu, a neurologist at Boston Children’s Hospital, who discovered the precise genetic mutation that caused her disease — and was able to devise an experimental treatment designed exclusively for Mila. His work is one of the first examples of therapies created for the benefit of a single patient — an “N of 1” study. (Keshavan, 10/9)
In other pharmaceutical news —
The Return Of Vioxx: Can A Drug Once Deemed Deadly Be Relaunched?
Vioxx’s days as a widely used treatment for arthritis and chronic pain came to an end when studies revealed that it roughly doubled patients’ risk of heart attack and stroke, leading to an estimated 60,000 deaths. It was a public health disaster, and it led to a congressional investigation, allegations of lapses at the Food and Drug Administration, and agreement by the manufacturer, Merck, to pay a nearly $5 billion settlement. Now, Tremeau Pharmaceuticals, a private Massachusetts company, is developing a generic version of Vioxx as a treatment for severe joint pain in people with hemophilia, a side effect called hemophilic arthropathy. (Garde, 10/9)
2020 Democrats Embrace Aggressive Step On Drug Prices
Democratic presidential candidates are threatening to take a drastic step that even the Obama administration rejected to lower drug prices without congressional approval. The move involves invoking an obscure section of a 1980 law to break the patent on a drug when it is priced too high. The idea, known as "march-in rights," would allow the government to "march in" and break a patent to allow a cheaper version of a drug to be made by another company. (Sullivan, 10/9)