New tools and technologies fueled biomedicine to great heights in 2017. Here are just a few of our top picks. All are great examples of research informing better care for children (and adults).
1. Gene therapy arrives
In 2017, gene therapy solidly shed the stigma of Jesse Gelsinger’s 1999 death with the development of safer protocols and delivery vectors. Though each disease must navigate its own technical and regulatory path to gene therapy, the number of clinical trials is mounting worldwide, with seven gene therapy trials now recruiting at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. In August, the first gene therapy won FDA approval: CAR T-cell therapy for pediatric acute lymphoblastic leukemia. …
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. …
Vector has been deliberating about its predictions for 2013, consulting its many informants. Here’s where we’re putting our money this year; if you have other ideas, scroll to the bottom and let us know.
Genome sequencing scaling up at health care institutions
Last year we predicted genome sequencing’s entry into the clinic; this could be the year it goes viral. Technology companies with ever-faster sequencers and academic medical centers are teaming up at a brisk pace to offer genomic tests to patients. Just in the past two weeks, a deal was announced between The Children’s Hospital of Philadelphia and BGI-Shenzhen to sequence pediatric brain tumors; Partners HealthCare and Illumina Inc. announced a network of genomic testing laboratories; …
Here once again is Vector’s take on some exciting trends we’ve been watching in the pediatric health arena and what we expect to see more of this year. If you’ve got others to propose, scroll to the bottom and let us know!
Whole-genome sequencing enters the clinic
In 2000, with our genome deciphered, the Human Genome Project promised to transform medicine, predicting and preventing all that ails us. The project spawned next-generation technologies, accelerated the development of bioinformatics and shaped new perspectives on research. But if, say, a stroke patient was asked the question, “Is your life any better than 10 years ago thanks to advent of genomics?” the answer would have to be “no.” Hence the New York Times’s assertion in 2010 that the project yielded few new cures.
Now that paradigm seems to be shifting. Whole-genome sequencing has begun moving into the clinic, sleuthing out problems, offering hope for a medicine that’s more effective and more personal. 2011 saw genomic information provide biochemical insights timely and actionable enough to improve the treatment of individuals with cancer and dystonia, and, in a case at Children’s, failure to thrive and severe kidney calcification. …