The next big strides in gene therapy for rare diseases may come from CRISPR and new approaches to delivery.
The 10-year-old from Midlothian, Va., plays tag with his friends and swims in the ocean. “I think his peers would describe him as someone who is kind, sweet and funny, and supersmart,” says his mother, Sheila Ungerer. He won a countywide citizenship award and was named classroom student of the year.
Though the therapy did not show statistically meaningful improvements compared with a placebo in one randomized controlled trial and results of an ongoing clinical trial to determine efficacy aren’t yet published, Will and the 83 other boys in Sarepta’s clinical trials now make the microdystrophin protein. Based in part on that evidence, the U.S.
Since gene therapy was first proposed to treat such genetic diseases in the 1970s, it has had thrilling highs — including the first successful gene therapy, in 1990, in a 4-year-old born with severe combined immunodeficiency, or SCID — and deeply troubling lows. Clinical trial participants haveand have even died. The biggest problem appears to be the viruses used to ferry replacement genes where they need to go.
Editing cells outside the body, or ex vivo, has worked for rare diseases that affect the blood or immune system. Yet it comes with limitations, since scientists haven’t worked out ways to extract stem cells from tissues other than blood and bone marrow and replace them later. It also comes with its own dangers. It typically requires chemotherapy to kill existing bone marrow so the modified cells can take hold and replicate.
On March 22, 2018, Conner Curran of Ridgefield, Conn., then 7 years old, became the first person to get an experimental in vivo gene therapy developed by Pfizer for Duchenne muscular dystrophy. “Before gene therapy, he really struggled to go up the stairs,” says Conner’s father, Chris Curran. “Two months after gene therapy, he’s running up the stairs.”
In a common approach used today, a virus delivers the gene of interest into the DNA. The gene usually does not end up in its typical location in the genome.Some viruses instead drop the gene of interest in the nucleus. There, it can still be active, but it remains separate from the rest of a person’s DNA.Even when a virus gets its cargo to the right cells — the right zip code — that doesn’t mean it’ll find the right address.
But on July 2, 2019, Gray got three vials of her own bone marrow cells edited to make fetal hemoglobin. The worst part was the recovery from chemotherapy, she said. “It took about seven to eight months for me to physically feel and mentally accept that I was better,” she told the crowd of scientists, ethicists and other experts. Thanks to her “super cells,” she now has a full-time job, attends her children’s football games and cheerleading events and can enjoy family outings.
Extensive scarring also obscured Antonio’s vision. He had two surgeries in 2016 and 2017 on his left eye to remove the scars, but they came back two to three months later. The procedures kept failing, so “we decided not to do any additional surgeries,” ophthalmologist Alfonso Sabater of the Bascom Palmer Eye Institute at the University of Miami Health System and the Miller School of Medicine said July 24 during a news conference. There appeared to be little hope for saving Antonio’s vision.
Antonio has recovered nearly all of his vision in one eye and about 60 percent in the other, and the scars haven’t returned. But since the effects are temporary, it’s likely that he will need regular doses for years.
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