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several years or determined year to year. At the start of 2015, the bill’s backers hoped to have President Obama sign it into law before 2016. That now looks unlikely.
But winds continue to favor biomedical research; the new budget passed earlier this month actually boosted NIH funding by 6.6 percent, the biggest bump in 12 years. I still contend it would take another external shock to grind the biopharma financing gears to a halt.
—“A bigger question, perhaps, is whether the healthcare field starts to pivot from pharmaceutical approaches to more holistic approaches.” Just before 2014 turned into 2015, Danish drug maker Novo Nordisk got FDA approval for a weight-loss injection—a higher-dose version of Novo’s diabetes drug liraglutide, rebranded as Saxenda. It was yet another old drug recast to grab a piece of the anti-obesity market. Previous drugs of recent vintage had not gained much traction. One year later, the pressure to solve problems like epidemic obesity with something other than drugs is even greater. The four drugs on the market still haven’t made much impact. And a possible new entrant in the field, beloranib, has been suspended by the FDA after two patients died in a Phase 3 trial to treat the rare genetic disease Prader-Willi Syndrome, which causes uncontrollable hunger. Even if beloranib is eventually successful in Prader-Willi, the deaths have damaged Zafgen’s chances of expanding it to a more general population.
American obesity rates have recently moved slightly in the right direction among preschoolers, but overall obesity is still a crisis, affecting more than a third of all adults and 17 percent of children. In May, I asked whether we should count on drugs—as we have with HIV and cancer—to make a dent in our massive societal weight problem.
Many say that drugs will remain necessary, which is hard to argue with when it comes to extreme cases like Prader-Willi. But a confluence of factors could continue to push back against the pharmaceutical solution. Backlash against drug prices is one; better diet practices, like getting sodas out of schools, is another; and ever-growing digital health solutions, either without drugs or combined with drugs, is a third.
It wasn’t among my new year’s predictions—shame on me—but gene editing captured the world’s imagination nearly as much as Shkreli. Work continued apace in academic labs to make new and better tools, as well as in small and large companies alike to make new therapies. Nothing is close to becoming an approved medicine; the most advanced is an HIV treatment—immune cells edited with a system called zinc fingers from Richmond, CA-based Sangamo Biosciences (NASDAQ: SGMO)—in Phase 2 trials. The T cells that helped cure Layla Richards were edited with a system called TALEN and will start clinical trials soon.
But the speed at which the the gene editing system called CRISPR-Cas9 has developed and spread means CRISPR-edited therapies could be tested in humans sooner than we think. Editas Medicine CEO Katrine Bosley said her company aims to start a clinical trial for a rare form of blindness in 2017. Will that be the first time humans have their genes edited with CRISPR? Don’t be surprised if someone moves even faster. There are many hurdles to jump to make that happen, but CRISPR developments, such as new “scissors” other than the Cas9 enzyme, or a wider range of guides for those scissors, or more accurate editing, seem to issue forth every day.
The biggest development in the CRISPR world in 2015, however, was the work of Chinese scientists, published in April, that they edited non-viable human embryos—those that would not have led to a live birth—with CRISPR scissors. The scientists reported that the experiment, to change the genetic defect that causes the blood disorder beta-thalassemia, did not go well.
But it underlined the possibility of genetically altered humans passing down their traits, a scenario that some say could lead to parents designing their babies for looks and skills, and others say could wipe out terrible inherited diseases. (These possibilities aren’t mutually exclusive.)
Scientists, bioethicists, and others gathered in Washington, D.C., to debate those scenarios and discuss guidelines. The summit’s organizers, chaired by Nobel Prize winning biologist David Baltimore, issued a strong statement against the modification of human eggs, sperm, and embryos—the germline—in ways that allow genetic traits to be passed from one generation to another, but they declined to call for a ban or moratorium. They also approved of human germline editing in basic research settings, as long as viable fetuses aren’t being produced.
It seems inevitable, though, that someone will flout the recommendation, perhaps in a country where the rules are lax, perhaps not. Will it be 2016? My prediction is yes—or at least a scientist will claim to have done so, even if it lands him or her in hot water. After all, what we’ve seen in 2015 with Shkreli (and in 2005 with Woo Suk Hwang, and many times before and after) is that the ego can help an ambitious person crash through all kinds of boundaries, not the least of which is good judgment.