11.5 Hours: New Record Speed For Rare Illness Diagnostic DNA Sequencing

Stat: Speed Record For Diagnosing Rare Genetic Diseases Is Shattered 
About a year ago, Matthew Kunzman’s heart was failing, despite doctors’ best attempts to bolster it with every pump and gadget they could think of. But the 14-year-old has bounced back in large part due to super-speedy genetic sequencing that pinpointed the cause of his disease and helped doctors decide how to treat it — in just 11 and a half hours. That speedy diagnosis — faster than any other medical team has previously reported — resulted from a new approach to DNA sequencing to help patients with deadly and rare diseases. On Wednesday, a team of Stanford researchers and collaborators published a letter in the New England Journal of Medicine reporting that they had sequenced 12 seriously ill patients and successfully diagnosed five of them (including Matthew). In all five cases, the information led to tangible changes in how patients were treated. (Wosen, 1/12)

In updates on the nominee to lead the FDA —

Axios: Biden's FDA Nominee Faces Uncertain Senate Vote Amid Abortion, Prescription Drug And Opioid Epidemic Concerns
Robert Califf, President Biden's nominee to lead the FDA, is facing a surprisingly tight confirmation vote in the Senate. A handful of Democrats have already announced their opposition to him over concerns about his drug industry ties or the FDA's record on the opioid epidemic, and several others may be on the fence. But abortion politics — not to mention the opportunity to sink a Biden nominee — are complicating efforts to make up for these defectors with Republican votes. (Owens, 1/13)

In other pharmaceutical industry news —

Stat: Medicare's Alzheimer's Decision May Hurt Biogen's Competitors, Too
Medicare may have released a plan to restrict access to Biogen’s controversial Alzheimer’s drug, but the biggest losers may actually be other drug makers readying treatments for the debilitating disease. The question is whether they encounter such difficulties and, if so, to what extent? Under a draft policy announced by Medicare on Tuesday, any company developing a monoclonal antibody — the same type of injectable medicine as the troubled Biogen drug — would have to satisfy the same stiff requirements in order to win Medicare reimbursement. Coverage would be limited to patients enrolled in Phase 3, randomized clinical trials that are approved by the agency. (Silverman, Cohrs and Florko, 1/12)

CBS News: An Experimental Vaccine Could Help Fight The Opioid Epidemic
Tackling the opioid crisis requires changing strategies and the way we think about addiction, says Columbia University professor Sandra Comer. ... "One of the mistakes that people make when they think about drug users, 'Oh, it's somebody's choice to have this disorder.' That's not true," she said. "It's a medical disease and we need to treat it." Medically assisted treatments can be effective but have a relapse rate of about 50%, Comer said. "That's why we're continuing to look for new medications," Comer said. That search led to a new type of treatment — a vaccine that targets the chemical makeup of oxycodone. Comer and her research colleague, Marco Pravetoni, are testing the vaccine on volunteers with substance use disorder. (Lapook, 1/12)

The Wall Street Journal: Theranos Founder Elizabeth Holmes Faces September Sentencing 
A federal judge has scheduled a late-September sentencing date for Theranos Inc. founder Elizabeth Holmes, when she could be handed prison time after her fraud conviction. The Wednesday order from U.S. District Judge Edward Davila comes just over a week after a jury convicted Ms. Holmes of one count of conspiracy to commit wire fraud and three counts of wire fraud for intentionally deceiving investors about her now-defunct blood-testing startup. (Randazzo and Somerville, 1/12)