It’s been an exciting year for pediatric health care. As Thanksgiving draws near, Vector is taking a pause to acknowledge the inspiring people and ideas that are helping set the table for a better future.
What are we thankful for?
The growing cadre of citizen scientists — passionate parents pushing for answers for their kids, helping to move rare disease research forward through their own investigations and initiatives. They’re keeping academic researchers honest and on top of their game, and, in many cases, helping to fund them.
The growing inclination among clinicians to say, “the way things are isn’t good enough,” and then push the boundaries of what’s possible to improve sick children’s lives.
People who question authority — like Martine Rothblatt, who didn’t take one drug company “no” for an answer but turned around and formed her own company.
Amazing new biomedical tools and approaches: Tinkering with epigenetic tags to push the boundaries in stem cell science. Applying CRISPR to edit disease-related genes, with whole-genome association studies steering the scissors to their proper targets. Deep sequencing to find needle-in-a-haystack mutations that happen on the fly and could explain more of our biology than we realize.
Engineers who are reimagining surgery.
Healthcare hackers, people who join up to tinker, collaborate and punch through boundaries, tackling problems that can’t be solved by any one person.
Hollywood artists who apply their craft to creating ultra-realistic medical trainers, a great improvement over cadavers or plastic mannequins. They’re helping our surgeons and proceduralists keep their heads in the game and practice tricky maneuvers in advance, making interventions safer.
The incredible drug-finding properties of zebrafish.
“Shark Tank” style pitch events. They’re popping up everywhere, and threaten to become cliché, but in medicine they’ve had the great virtue of getting scientists and clinicians to think big and appeal to new audiences. It’s been especially great to witness this in the academic environment.
Crowdsourcing challenges around tough problems in medicine, like the recent CLARITY Undiagnosed Challenge that brought experts around the world to patients’ bedsides. As CLARITY organizer Zak Kohane, MD, PhD, put it, “Isn’t that the way it should be?”
Of course, there’s much, much more to be thankful for, including all the people who work behind the scenes to make dreams a reality. All these projects matter and are helping put medicine on the right path. From ours to yours, Happy Thanksgiving.