Source: AP
A controversial gene-editing technology trial with only one participant ended in tragedy, and scientists were baffled and seeking to determine what killed the man, AP reported.
Terry Horgan of Montour Falls, New York, 27, enrolled in the trial in late August in hopes of finding a treatment for Duchenne muscular dystrophy (DMD).
Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin that helps keep muscle cells intact.
Terry Horgan passed away in October according to Cure Rare Disease, a Connecticut-based foundation that Terry Horgan’s brother, Rich, founded to try to save him from the rare disease.
The organization has already issued a statement indicating that researchers from all around the country are currently investigating the research and that the results will be made public.
“It will probably be 3-4 months to come up with a full conclusion,” said spokesman Scott Bauman. “At this stage of the game, saying anything is pure speculation.”
Horgan was the sole volunteer in the country to be treated using CRISPR, an acronym for Clustered Regularly Interspaced Short Palindromic Repeats.
It is a technique that edits genes by precisely cutting DNA and then letting natural DNA repair processes to take over.
Source: CRISPR Therapeutics
The exact cause of Horgan’s death remains unclear.
AP reported:
Although little is known about how he died, his death occurred during one of the first studies to test a gene editing treatment built for one person. It’s raising questions about the overall prospect of such therapies, which have buoyed hopes among many families facing rare and devastating diseases.
“This whole notion that we can do designer genetic therapies is, I would say, uncertain,” said Arthur Caplan, a medical ethicist at New York University who is not involved in the study. “We are out on the far edge of experimentation.”
The early-stage safety study was sponsored by the nonprofit, led by Dr. Brenda Wong at the University of Massachusetts Chan Medical School and approved by the Food and Drug Administration. The hope was to use a gene-editing tool called CRISPR to treat Horgan’s particular form of Duchenne muscular dystrophy. The rare, genetic muscle-wasting disease is caused by a mutation in the gene needed to produce a protein called dystrophin. Most people with Duchenne die from lung or heart issues caused by it.
At this point, it’s unclear whether Horgan received the treatment and whether CRISPR, other aspects of the study or the disease itself contributed to his death. Deaths are not unheard of in clinical trials, which test experimental treatments and sometimes involve very sick people.
But trials involving CRISPR are relatively new. And Fyodor Urnov, a CRISPR expert at the Innovative Genomics Institute at University of California, Berkeley, said any death during a gene therapy trial is an opportunity for the field to have a reckoning.
“Step one is to grieve for the passing of a brave human soul who agreed to be basically a participant in an experiment on a human being,” Urnov said. “But then, to the extent that we can, we must learn as much as we can to carve out a path forward.”