Sep 26, 2019

Patients issue new pleas for experimental gene editing

Photo: Sandra Baker/Getty Images

Patients and parents of children with life-threatening diseases may start asking for experimental gene editing as a form of treatment, STAT reports.

The big picture: Bioethicists, genetic engineers and lawmakers are now asking the same ethical questions that led to the 2018 law allowing "the right to try" experimental drugs prior to FDA approval: namely, do patients have a right to try gene editing technology?

What they're saying: "It's so intuitively simple: replace or correct a gene that's not functioning properly. There is so much hype, more and more people will think, I want that," bioethicist Alison Bateman-House of New York University told STAT.

  • In a potentially prescient move, lawmakers in California passed a law banning people from using unproven therapies amid concern that people would start biohacking themselves or using do-it-yourself CRISPR kits.
  • There are only 2 approved gene therapies in the U.S., one for a form of blindness and the other for spinal muscular atrophy.

The bottom line: "Parents are increasingly asking scientists for access to experimental compounds that have never even been tested in animals" to help save their sick children, per STAT, and more such pleas for genetic technologies are likely only a matter of time.

Go deeper: The cost of gene therapies could test the health care system

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Genetic technology's double-edged sword

Illustration: Sarah Grillo/Axios

As gene-altering technologies become more accessible, there's also a growing risk that they'll be dangerously misused or abused.

Why it matters: "You're talking about manipulating DNA to create a designer pathogen. That’s a terrorist threat," former FDA Commissioner Scott Gottlieb told me.

Go deeperArrowOct 1, 2019

CRISPR used to treat first U.S. patient with genetic disorder

Photo: Gregor Fischer/picture alliance/Getty Images

Doctors are using CRISPR to treat a woman with sickle cell disease, the first U.S. patient with a genetic disorder to receive such treatment, NPR reports.

The big picture: Advances in gene editing technology have given new hope to people with diseases that not long ago seemed untreatable.

Go deeperArrowOct 11, 2019

Gene therapies' accessibility problem

Adapted from PwC Health Research Institute; Graphic: Axios Visuals

Gene therapies are physically out of reach for many patients, on top of their cost, according to a new PwC Health Research Institute report.

Why it matters: This further exacerbates the rural-urban divide in access to care.

Go deeperArrowOct 2, 2019