Happy New Year! This edition of Medical-Science Moments begins with a wrap up of the top 10 science stories of 2018, as designated by Science News. Not surprisingly, a number of the stories relate to gene technology, but health effects of alcohol and progress on spinal cord injury treatment also made the list. The wrap up is followed by more recent Medical-Science Moments from the last few weeks, including more on gene technology, “pharmajuana,” the pharma industry, FDA, dairy data, and interesting findings on exercise.

Please enjoy and, as always, I welcome any feedback or suggestions of items to consider for the next issue! – Dr. Dave

Science News’ Top Stories of 2018 

Along with news about climate change (the #1 story), neutrinos (#4) and a possible large body of liquid water on Mars (#8), SN’s top stories a bit closer to home for us pharma pholks included:

  • First gene-edited babies (#2). Many expressed outrage when the Chinese scientist He Jiankui announced in November that he had created the first gene-edited babies – but with all the progress in CRISPR/Cas9 technology, it was probably only a matter of time. In this case, gene editing conferred a lower risk for contracting HIV. Critics expressed concern that the children may be exposed to other health risks, and that this may encourage the creation of “designer babies.” On November 28, he announced he’s edited an embryo of a second pregnancy and, about a week thereafter, went missing . His status remains unclear, but it’s recently been reported he’s in a government-owned apartment, possibly under house arrest and, according to the U.K.'s The Telegraph, one of He’s colleagues said He may face the death penalty.

Read more:  Washington Post, STAT, Newsweek, Harvard Crimson 

  • Bye-bye mosquitoes (#6)? In another use of CRISPR/Cas9, for the first time, scientists inserted a destructive “gene drive” into genetic material of male mosquitoes, pushing a lab-housed population of the pests to extinction. Since gene drives are passed on to offspring and are highly expressed, this approach could theoretically be used to wipe out mosquitoes. However, given more than 3000 species and genetic diversity that might have some genetic work-arounds, we should probably keep the DEET handy for the time being.

Read more: The Scientist, New York Times, NPR

  • Health risks of alcohol (#7). Two major meta-analyses had sobering findings showing that alcohol – in any amount – is bad for health, countering current recommendations that moderate drinking confers a benefit over abstention. One report, based on data in 28 million people in 195 countries, concluded that if everyone in the world was a teetotaler, there would have been 2.8 million fewer deaths in 2016 alone. The other analysis, noted in MSM In June and based on data from nearly 600,000 drinkers in Europe, showed the lowest risk of all-cause death was associated with about “one drink” per day or less. However, experts pointed out that, while excessive drinking is clearly unhealthy, the studies weren’t really designed to tease out the risks or benefits of light-to-moderate drinking from other potentially confounding factors.

Read more: Washington Post, CNN, Science Daily

  • Spinal Paralysis Progress (#9). Three small studies showed that intensive rehabilitation along with electrical stimulation enabled people who were paralyzed for years to walk. The findings are good news for the approximately 1.5 million people in the United States with some degree of paralysis from a spinal cord injury.

Read more: ABC.net, Discover Magazine, SpinalCord.com 

And recently…

Gene-Editing Drug Trial in Humans

Just days after He Jiankui’s announcement of gene-edited babies, FDA approved the second company-sponsored trial of a CRISPR/Cas9 drug: Editas Medicine’ therapy for the most common cause of childhood blindness, Leber congenital amaurosis 10 (LCA10), resulting from a mutation in the gene CEP290. In contrast to He’s work, which involved genetically altering human embryos and making genetic changes that would be passed on to future children, Editas’ therapy targets cells in the eye and have no impact on reproduction.

Read more: Editas Medicine press release, Boston Business Journal, STAT, Foundation Fighting Blindness 


The Canadian marijuana company Tilray recently announced a “joint” venture (sorry, couldn’t resist!) with Novartis’ subsidiary Sandoz to sell medical marijuana in countries where it is legal (35 currently). The marketing agreement, which includes other products as well, appears to be a first for marijuana and a major pharmaceutical company, and…uh, I forgot what I was going to say…

Read more: Bloomberg, CNBC, Marketwatch, STAT

Pfizer and Glaxo Combine Consumer-Health Businesses

Pfizer and GlaxoSmithKline plan to merge their consumer-health units to create a joint venture which, the companies said, would be the world’s largest maker of OTC products, from toothpastes to pain relievers. Glaxo will own 68% and Pfizer 32%. The two units had combined sales of $12.7 billion last year.

Read more: Wall Street Journal, The New York Times, Reuters, Bloomberg, Associated Press

FDA Highlights 2018 as Banner Year for Drug Development and Approvals

As of December 20, 2018, FDA approved 56 new molecular entities (NMEs), surpassing 1996, previously the busiest year with 53 approvals. For the first time, the majority of NMEs approved were orphan drugs to treat rare diseases. 

Read more: Fierce Biotech, FDA CDER presentation, FDA website

FDA Lauds Real-World Data for Drug Development

FDA Commissioner Scott Gottlieb, MD, announced a 2019 framework to advance collection and use of real-world data (RWD) across drug and biologic development efforts. He noted that use of RWD is a “top strategic priority for the FDA” that “provides us with a potential source of information that can complement, augment, and expand our understanding of how best to use medical products – improving what we know about our medical care.” He noted that RWD can come from a diverse array of sources, such as electronic health records, medical claims, product and disease registries, laboratory test results and even cutting-edge technology paired with consumer mobile devices. Somewhat related: JAMA recently had a good review on interventions to improve medication adherence, an important element of therapeutic success for most all patient-administered therapies.

Read more: FDA statement, Healthcare Analytics News, BioCentury

Got Milk?

For more than 40 years, nutrition guidelines have recommended low/no-fat dairy products over full-fat versions. Little evidence, however, actually support s those recommendations, and may even run counter to them. Consider the Prospective Urban Rural Epidemiology (PURE) study, recently reported in The Lancet.  That large cohort study included more than 136,000 adults from 21 countries in five continents. The researchers found that, from 2003 to 2018, a higher self-reported intake of total dairy (>2 servings per day vs. no intake) was associated with lower risks of the composite outcome of death or major cardiovascular (CV) events (by 16%), total mortality (by 17%), non-CV mortality (by 14%), CV mortality (by 23%), major CV disease (by 22%), and stroke (by 34%). No significant association with heart attack was found. Higher intake of milk and yogurt (>1 serving vs no intake) was associated with lower risk of the composite outcome, but cheese and butter intake were not. So, get moo-ving!

Read more: JAMA, Skeptical Cardiologist

Best Exercise for Living Longer?

It’s not surprising that “get more exercise” was the most common New Year’s resolution in 2018 (tied with “eat healthier” and “save more money”). Sure, we all know exercise is good for us, but what kind is best for living longer? Some answers emerged from the first study to compare the effects of different types of training on telomere length and activity of telomerase (an enzyme that lengthens telomeres), both of which are associated with improved cell survival and healthy aging.  For the six-month trial, researchers randomly assigned inactive adults to three 45-minute sessions per week of supervised aerobic, high-intensity interval or resistance training, or to a control group. By the end of the study, completed by 124 men and women, aerobic and interval training, but not resistance training, increased telomerase activity two-to-three fold and lengthened telomeres compared to the control group.

Read more: New York Times, Newsweek, MSN

About the Author:

David Canty, PhD, (“Dr. Dave,” as colleagues call him) has more than 30 years of experience in health care communications, including 20 years at the Chandler Chicco Agency (CCA), now part of Syneos Health. He serves as a resource across Syneos Health group companies, including CCA, Chamberlain Healthcare PR, Biosector 2, Cadent Medical Communications and Allidura. He helps develop communication messages and materials; “translate” data and science for media, health care professionals (HCPs) and consumers; write HCP peer- and non-peer-reviewed journal articles, press releases and direct-to-consumer materials; create scientific presentations; advise HCP spokespeople on message delivery; and address issues. He has a broad base of therapeutic experience including diabetes, obesity, cardiovascular disease, nutrition, oncology, psychiatric and neurological disorders, various rare diseases, and many others. He has also been an adjunct assistant professor at New York University for 30 years.