Biomarkers could aid diagnosis and lead to strategies for treatment
Soon after systems biologist Juergen Hahn published a paper describing a way to predict whether a child has autism from a blood sample, the notes from parents began arriving. “I have a bunch of parents writing me now who want to test their kids,” says Hahn, of Rensselaer Polytechnic Institute in Troy, N.Y. “I can’t do that.”
That’s because despite their promise, his group’s results, reported March 16 in PLOS Computational Biology, are preliminary — nowhere close to a debut in a clinical setting. The test will need to be confirmed and repeated in different children before it can be used to help diagnose autism. Still, the work of Hahn and colleagues, along with other recent papers, illustrates how the hunt for a concrete biological signature of autism, a biomarker, is gaining speed.
CTSA Program paves way for nationwide single IRB model.
Developing new treatments for diseases often requires large numbers of clinical research participants enrolled in the same study at numerous geographical sites. These multisite clinical trials are well-positioned to discover whether a promising therapeutic is safe and effective, and may provide medical professionals with the information needed for treating their patients. However, the initiation of such studies may be delayed because each site typically relies on its own Institutional Review Boards (IRBs) to provide ethics reviews of the risks and benefits of the proposed research.
"This milestone is a giant step toward a nationwide model for greater efficiency in IRB review..."
Researchers transforming animals with the latest genome-engineering tools may be disappointed by draft rules released by the US Food and Drug Administration (FDA) on 18 January — two days before US President Barack Obama leaves office. It is not clear how the administration of incoming president Donald Trump will carry the proposals forward, however.
The most controversial of three proposed regulations declares that all animals whose genomes have been intentionally altered will be examined for safety and efficacy in a process similar to that for new drugs.
Many researchers had hoped that the FDA would be less stringent about evaluating organisms whose genomes have been edited with precise tools — such as CRISPR and a separate technique called TALENs — than it is for animals that have been given DNA from different species or created using less-sophisticated means. Alison van Eenennaam, an animal geneticist at the University of California, Davis, calls the draft FDA proposals “insane”.
“The trigger for their regulation is whether the animal was intended to be made, and what does intention have to do with risk?” she says. “The risk has to do with the attributes of the product.”
BETHESDA, Md. — At what was supposed to be the end of his tenure atop the National Institutes of Health, Dr. Francis Collins is still a very busy man.
In his seven and a half years leading the agency, Collins has been involved in the response to Ebola and Zika. He has helped secure the first funding increases for the NIH in decades. Congress just this month funneled billions of dollars into several major projects — the Precision Medicine Initiative, the cancer moonshot — that Collins helped craft.
This was supposed to be the finale for Collins. But, as he told STAT in a recent interview in his offices here, he loves the NIH and believes in public service, so if asked he would consider it a “privilege” to remain director under President-elect Donald Trump. Many researchers and members of Congress would like to see him stay. Nobody knows yet if he will.
Below is his full conversation with STAT, in which he reflects on NIH's past and future. The transcript has been lightly edited for clarity and length.
Tell us about a few programs that you are most excited about.
As far as areas of exceptional opportunity, I’d start with the Precision Medicine Initiative. This unprecedented national study of health has recently been renamed and now it is called "All Of Us."