PREMEDINFO-L Archives

November 2017, Week 3

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Wednesday, November 15, 2017





[Morning Rounds by Megan Thielking]





Sponsored by

 [https://gallery.mailchimp.com/f8609630ae206654824f897b6/images/ef8c678a-32b7-4438-9d6f-29221f480534.png]







Good morning, and welcome to Morning Rounds. Rebecca Robbins here, filling in for Megan. On to today's news:





A landmark moment: Scientists try to edit a patient's DNA in his body



Exciting news this morning from California, where scientists have for the first time tried to cure a genetic disorder by editing the patient's DNA in his body, the Associated Press reports<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=3461b81777&e=4aad33fd68>. Brian Madeaux, 44, received an infusion this week of billions of copies of a corrective gene meant to produce an enzyme that breaks down certain carbohydrates. He lacks that enzyme — a condition called Hunter syndrome — and as a result has suffered a wide array of medical challenges. Along with the corrective gene, Madeaux received an infusion of two "molecular scissors" — not CRISPR, but zinc finger proteins. If all goes well, they'll travel to his liver, where the fingers will cut the DNA and let the new gene slip in. The gene should then direct the cell to start making the crucial enzyme. Scientists working on the case say they should get early signs in a month and know for sure in three months whether it's working.



The therapy is made by Sangamo Therapeutics, a California company. Dr. Sandy Macrae, the company's president, calls it "invisible mending," the AP reported. But there are, of course, risks of unforseen side effects. "How bulletproof is the technology?" said Dr. Carl June, a scientist at the University of Pennsylvania who has worked on other types of gene therapy. "We're just learning."





Obamacare's individual mandate back in the crosshairs



The GOP quest to undo a key tenet of the Affordable Care Act is back on. Senate Republicans said yesterday<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=b831efbdb7&e=4aad33fd68> that they will include in their proposal to overhaul the tax code a repeal of the individual mandate, Obamacare's requirement that most people have health insurance. Eliminating the mandate would enable tax cuts by freeing up money that the government spends subsidizing health plans, but it would also likely disrupt the health insurance market, driving up premiums and leaving more Americans uninsured. Already, the plan has rekindled the fight over the fate over the ACA: Groups representing doctors, insurers, and hospitals yesterday came out with a letter<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=be4ba00a61&e=4aad33fd68> urging Congress to keep the individual mandate in place.



In other happenings on Capitol Hill, recently minted Surgeon General Dr. Jerome Adams will be testifying this morning at a Senate committee hearing on building healthy communities. You can watch the hearing<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=3d944e0a91&e=4aad33fd68> live at 10 a.m. ET.





Top addiction treatments deemed roughly equivalent



With the opioid crisis ravaging communities, there's lots of debate about which treatment for opioid addiction has the best chance at leading users to long-term recovery. Now, the largest head-to-head study<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=e7302c7520&e=4aad33fd68> to date between two leading drugs has found them roughly equivalent — a finding that could boost the fortunes of the newer drug, Vivitrol. Researchers found that about half of people with opioid addiction relapsed within six months, regardless of whether they took either a monthly shot of Vivitrol or the daily pill of buprenorphine and naloxone sold as Suboxone. But there's also a big caveat, in favor of Suboxone: a large number for people were unable to even start treatment with Vivitrol, because of the need to wean themselves off of opioids before initiating treatment.











Sponsor content by Merck



Taking the mystery out of selecting the right health insurance



“Open-enrollment season can bring a wave of anxiety for many Americans, but knowing how to choose the right health insurance should never feel like a mystery,” said Julie Gerberding, M.D., M.P.H., Executive Vice President and Chief Patient Officer, Merck. “To help address this problem, Merck collaborated with health literacy experts to develop a simple online guide to help families dealing with these decisions.” Learn more by visiting the Know Your Health Insurance<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=3a9720ae7e&e=4aad33fd68> guide.













Inside STAT: 3-D printing could revamp MRIs for infants



[https://gallery.mailchimp.com/f8609630ae206654824f897b6/images/3683f64d-65ff-4464-815f-53a5f45c8980.jpg]



THIS BLANKIE CONTAINS PRINTED MRI COILS, RATHER THAN THE USUAL CHILDHOOD MEMORIES. (Usha Lee McFarling/STAT)



MRI imaging of children has long been a challenge: It's hard for them to stay still during the long exams. Sometimes they need a tube inserted down their windpipe to ensure that they can keep breathing under the heavy weight of coils. And the simpler CT scans that physicians sometimes opt for with their youngest patients are sub-optimal. In these problems, UC Berkeley physicist Ana Claudia Arias sees an opportunity. An expert in the field of low-cost printable electronics, she's developing lightweight, flexible, plastic MRI coils — and wrapping them in a soft, fuzzy "blankie" to appeal to pediatric patients. Along the way, Arias and her collaborators at their spin-off company, called InkSpace Imaging, have had to think about details down to the level of patterns and colors to use. STAT correspondent Usha Lee McFarling has the story<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=8c605bffbb&e=4aad33fd68>.





Mining experts gather after health study scuttled



The scientists and industry experts who advise HHS on mine safety and health research are meeting today<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=de10064bbc&e=4aad33fd68> outside of Denver. Included on their agenda: How best to manage fatigue among miners working long hours. The meeting comes in the wake of the Trump administration's decision to cancel a new federal study<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=a2a1de3d02&e=4aad33fd68> that was supposed to provide the most comprehensive review to date of the short- and long-term health effects of mining, focusing on Appalachia. (The administration, a strong backer of the mining industry, pointed to budget reasons to explain the study's suspension.) Among other health problems, breathing coal dust can cause the respiratory illness known as black lung<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=1651868b7b&e=4aad33fd68>.





Is it time for triple-blinded studies?



Double blinding, where both researchers and clinical trial participants don't know who's getting which treatment, is the gold standard for scientific research. But a new study<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=576b9cce09&e=4aad33fd68> suggests there may be a need for a third blind — of the peer reviewers vetting research before it's published. Researchers at Google analyzed 500 papers submitted to a computer science conference. The upshot: When reviewers knew the name and affiliations of a paper author, they expressed greater interest in reviewing papers from top universities and companies, and were more likely to recommend those papers for acceptance, compared to reviewers who were blinded to author information.



Critics say that triple-blinded study design makes it hard to suss out conflicts of interest and to evaluate work in the context of a group's research direction over time. But the Google researchers said their findings should prompt conference organizers to consider a triple-blinded model.





U.S. unprepared for future Alzheimer's breakthrough



Alzheimer's researchers have been humbled by 15 years of failure in which not a single new drug has made it to market, much less a disease-modifying one. Still, that hasn't quenched hope that the next slate of big data readouts, coming in 2019<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=6720068293&e=4aad33fd68>, might finally deliver. But even if a breakthrough materializes, the U.S. health care system lacks the infrastructure to deliver such a transformative treatment on a widespread scale, a new study warns. The problems are many: There are too few medical specialists to evaluate millions of patients. Too few diagnostic scanners. And too few infusion centers to deliver treatment. If a therapy were to be approved for use in 2020, wait times for diagnosis and treatment would begin at 18.6 months, according to the projection from researchers at the RAND Corporation. Biogen, one of the companies working on an Alzheimer's drug, sponsored the new research<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=f9dd6d9964&e=4aad33fd68>.



And as my colleagues write in STAT's biotech newsletter The Readout<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=57d937672f&e=4aad33fd68>, there's an odd omission from the RAND report: any analysis of the sky-high costs anticipated for such a therapy, perhaps the biggest thing that the U.S. health care system is unprepared to handle.





What to read around the web today

§  When dentists criticize this online braces company, it takes them to court. Buzzfeed<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=d2177929cb&e=4aad33fd68>

§  California's public health department investigating free DNA tests given out at 49ers game. SFGATE<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=45b6144518&e=4aad33fd68>

§  Several states roll back 'retroactive Medicaid,' a buffer for the poor. Kaiser Health News<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=6d15e024cc&e=4aad33fd68>





More reads from STAT

§  FDA issues strong warning<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=da75acef82&e=4aad33fd68> against kratom, an herbal supplement used to treat pain and other conditions.

§  Dear Alex Azar: The next HHS secretary must redefine<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=806dd96dc9&e=4aad33fd68> ‘affordable’ health care.





The latest from STAT Plus

§  On drug importation, Trump and his pick to lead HHS are on opposing sides<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=ea7e5f991c&e=4aad33fd68>.

§  Lawmakers try to catch up<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=90f248e218&e=4aad33fd68> with genetic engineering and its potential.

§  Janet Woodcock wants you to know<https://statnews.us11.list-manage.com/track/click?u=f8609630ae206654824f897b6&id=5c82857741&e=4aad33fd68> the FDA is not picking speed over safety in drug approvals.









Thanks for reading! Megan will be back with more tomorrow.

[Megan]















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