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Dr. Dave’s Med-Sci Matters

Dr. Dave’s Med-Sci Matters

February 20, 2017 0 Comments

Genetically altering humans? Maybe we Khan!

While advances in gene therapy research continue to accelerate, an enduring redline has been the germline – no tinkering with the DNA of a human embryo, egg or sperm. Doing so would mean not only that the resulting individual would have the altered DNA and the corresponding trait, but also that the genetic modification and trait will be passed down to subsequent generations. Last week, however, the National Academy of Sciences and National Academy of Medicine issued a report on the ethics of human genome editing, and proposed general principles for controlling it. For example, it could be allowed to prevent serious diseases or disabilities, but not for creating “designer babies” smarter, stronger, and/or with longer lifespans than us mortals. Sorry Khan! (OK fellow geeks: what’s the basis for that reference? Give up? Click here.)

Read More: Science, Reuters, NPR

Pharma company cooperation? Precisely!

The Scientist has an interesting article about how, with the growing promise of “precision medicine” (previously “personalized medicine”), health care companies are increasingly collaborating to develop new therapies and identify patients most likely to benefit based on genetics and predictive biomarkers (that’s the “precision” part).  This radical departure from the traditional, one-drug-for-all blockbuster approach is fostering partnerships between, for instance, drug and diagnostics companies. Example: last year, AstraZeneca teamed up with Abbott Diagnostics to identify patients who would respond to the investigational asthma drug, tralokinumab. Precision therapies are an important and growing trend in pharma: In 2015, they accounted for 28% of FDA-approved drugs, and the market for them is predicted to grow to $113 billion by 2025.

The Price of success?

Source: Department of Health and Human Services
On February 10, Tom Price was approved as secretary of Health and Human Services by a narrow margin in Congress, 52 to 47. His confirmation hearing included questions raised about his plan to repeal and replace the Affordable Care Act, opposition to abortion rights, support for cutting Medicare/Medicaid, and financial activities that posed possible conflicts of interest when he was in Congress. Now that he’s confirmed, the Trump administration’s plan for health care is expected soon. Related: Fierce Pharma reported a number of Senators are urging Price to allow cheap drug imports under certain circumstances.

Read More: AP, New York Times, Wall Street Journal, Washington Post, The Scientist

Pharma: Hooray! Cut regulations! Um, wait…

Industry usually loves the idea of reducing regulations, but last week Reuters reported that President Trump’s vow to do so by at least 75% is making some pharma executives anxious. Why? They believe a weakened FDA could make it more difficult to get insurance coverage for expensive new therapies. In a similar vein, STAT reported that WHO Director Dr. Margaret Chan rebuked critics suggesting the FDA be overhauled, saying in a speech at the University of Washington, “We must not let anything, including economic arguments or industry pressure, lower our scientific standards or compromise our integrity. This is an absolute duty.”

Read More: Fortune, New York Times

Orphanedge?

Multiple outlets reported that Sen. Charles Grassley said his staff will investigate whether drugmakers are abusing the decades-old Orphan Drug Act to charge higher prices. The ODA provides incentives to manufactures for developing drugs for rare diseases (defined as affecting less than 200,000 Americans), including 7 years of market exclusivity, tax credits for clinical trial expenses, user fee waivers and federal grants.

Read More: Kaiser Health News, NPR, FiercePharma

Not for geeks only…

It’s ALIVE!!!

Source: NASA
As reported in The Scientist, two algal species survived on the International Space Station (ISS) – not inside, which has been shown before, but outside in space on a panel of the ISS. For 530 days they endured the vacuum of space; extreme temperatures, from -20 °C at night to 47 °C during the day; and ultraviolet radiation strong enough to destroy most forms of life on Earth (luckily, our atmosphere screens most of it). While on the ISS panels, the algae grew protective shells, which they shed after arriving back on Earth – and then started growing and became green again.

Read More: New Scientist, ScienceDaily

What’s in a (drug) name?

Damitol? Havitol? Cellalotovit? Ever wonder how drugs get their names? An opinion piece in STAT by an expert in the field describes the process as “usually the result of intense focus by creative name development professionals coupled with clear-eyed research designed to enhance the prospects of Food and Drug Administration approval.” And if you believe that, maybe you should try the new cognitive-enhancing drug, Skepticaluvitol!

Peddling pedaling

Biking has long been recognized as a heart healthy activity. But a large observational Danish study in Circulation suggests benefits even in those who bike just for recreation or commuting to work. Once-weekly rides of only 30 minutes were associated with a significantly reduced heart attack risk. The findings are consistent with those from another study in the Journal of the American Heart Association.

Read More: NY Daily News, Fox News, AHA Newsroom, Science Daily, Harvard Health Letter

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