Inside a couple’s quest to pay for an experimental gene therapy to save their children

IMG
Image credit: Jennie Landsman

“We need your help, we really do,” Gary [Landsman] says, his voice breaking. The Landsmans’ two sons—Benny, then 18 months, and Josh, four months—both have a fatal genetic brain disorder called Canavan disease. Benny, limp on his mother’s lap, is already affected by nerve loss. Josh isn’t yet. But he will be if nothing is done.

Jennie hit Google and started e-mailing scientists. Here’s what she learned: there may be a way to fix the genetic error in the boys’ brains. But the family would have to pay for it themselves.

The Landsmans had discovered gene therapy, technology that uses viruses to add healthy genes to cells with defective ones.

In Florida, a single boy was treated with a Canavan gene therapy in 2017 after his parents paid for the experiment. They did it under an exemption in federal rules called “expanded access,” which can allow unapproved drugs to be offered to specific patients “whose life is immediately threatened.”

Related article:  Alzheimer’s disease targeted with gene therapy trial

That experiment fell into a gray zone, not quite research and not quite medicine. It is the same pathway the Landsman family is trying to follow.

Their video, posted to Facebook and later GoFundMe, a crowdfunding site, went viral. By now, they’ve been on TV and in People magazine. Eight thousand donors have already given more than $1.5 million.

Read full, original post: Two sick children and a $1.5 million bill: One family’s race for a gene therapy cure

Outbreak
Outbreak Daily Digest
Biotech Facts & Fallacies
Talking Biotech
Genetics Unzipped
Video: Test everyone – Slovakia goes its own way to control COVID

Video: Test everyone – Slovakia goes its own way to control COVID

As Europe sees record coronavirus cases and deaths, Slovakia is testing its entire adult population. WSJ's Drew Hinshaw explains how ...
mag insects image superjumbo v

Disaster interrupted: Which farming system better preserves insect populations: Organic or conventional?

A three-year run of fragmentary Armageddon-like studies had primed the journalism pumps and settled the media framing about the future ...
dead bee desolate city

Are we facing an ‘Insect Apocalypse’ caused by ‘intensive, industrial’ farming and agricultural chemicals? The media say yes; Science says ‘no’

The media call it the “Insect Apocalypse”. In the past three years, the phrase has become an accepted truth of ...
globalmethanebudget globalcarbonproject cropped x

Infographic: Cows cause climate change? Agriculture scientist says ‘belching bovines’ get too much blame

A recent interview by Caroline Stocks, a UK journalist who writes about food, agriculture and the environment, of air quality ...
organic hillside sweet corn x

Organic v conventional using GMOs: Which is the more sustainable farming?

Many consumers spend more for organic food to avoid genetically modified products in part because they believe that “industrial agriculture” ...
benjamin franklin x

Are most GMO safety studies funded by industry?

The assertion that biotech companies do the research and the government just signs off on it is false ...
favicon

Environmental Working Group: EWG challenges safety of GMOs, food pesticide residues

Known by some as the "Environmental Worrying Group," EWG lobbies for tighter GMO legislation and famously puts out annual "dirty dozen" list of fruits and ...
m hansen

Michael Hansen: Architect of Consumers Union ongoing anti-GMO campaign

Michael K. Hansen (born 1956) is thought by critics to be the prime mover behind the ongoing campaign against agricultural biotechnology at Consumer Reports. He is an ...
News on human & agricultural genetics and biotechnology delivered to your inbox.
Optional. Mail on special occasions.
Send this to a friend