When Gary Landsman prays, he imagines he is in Israel and his sons Benny and Josh are running toward him. They are wearing yarmulkes, and the cotton fringes called tzizit fly out from their waistbands. He opens his arms ready for a tackle.

The reality is Benny and Josh both have Canavan disease, a fatal inherited brain disorder. They are buckled into wheelchairs, don’t speak, and can’t control their limbs.

On Thursday, April 8, in Dayton, Ohio, Landsman and his family rolled the older boy, Benny, into a hospital where over several hours, neurosurgeons drilled bore holes into his skull and injected trillions of viral particles carrying the correct version of a gene his body is missing.

The procedure marked the climax of a four-year quest by the Landsman family, who live in Brooklyn, New York, to obtain a gene therapy they believe is the only hope to save their kids.

MIT Technology Review first profiled the Landsmans’ odyssey in the cover story of our 2018 special issue on precision medicine. Advances in gene therapy technology are making it possible to treat genetic diseases like hemophilia. But because Canavan is an ultra-rare disease, few companies are working on a cure. So the family financed the daring gene treatment on their own, using funds they raised online.

Impressive advances in genome sequencing, gene replacement, and gene editing mean, in theory, thousands of rare genetic diseases could be treated. But because companies aren’t leading the way, parents say, they are being forced to embark on multimillion-dollar quests to finance the needed experiments. Adding to the ethical dilemma: in some cases, parents are designating their own children as the first recipients.

The trial in Dayton, for instance, is prioritizing children whose families have been able to raise funds to underwrite the experiment, whose costs so far are close to $6 million. “It raises the eternal equity question of who gets access to trials and who doesn’t,” says Alison Bateman-House, a bioethicist at New York University who is studying ethical issues in pediatric gene-therapy trials.

The Landsman family has raised more than $2 million, and families from Russia, Poland, Slovakia, and Italy have also used cash donations to secure spots in the trial. A Russian family even posted a copy of an invoice for “gene-therapy treatment” in the amount of $1,140,000, which included $800,000 to offset costs of manufacturing the

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By: Antonio Regalado
Title: This family raised millions to get experimental gene therapy for their children
Sourced From: www.technologyreview.com/2021/04/13/1022210/gene-therapy-expensive-treatment-genetic-diseases/
Published Date: Tue, 13 Apr 2021 10:00:00 +0000

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