When the first bone barrow transplants were performed decades ago, they were a groundbreaking way of using living cells to treat diseases.
Now it’s hoped that treatments known as "living therapies" could offer revolutionary cures for rare illnesses.
You’ve probably heard of CRISPR – the gene-editing technology developed here in the Bay Area.
"Being able to leverage that technology to make single repairs within a patient’s calls is going to be very important. It will allow us to treat the more rare diseases that otherwise have been very difficult to tackle," said Dr. Michelle Hermiston, clinical director of the UCSF Pediatric Immunotherapy program.
She told KCBS Radio’s "As Prescribed" on Thursday that CRISPR is one example of the kind of treatments that will be researched at UCSF’s new Living Therapeutics Initiative.
Some of the technology is very new.
The initiative is aiming to speed up their development and use.
"My hope it that we’re going to have more to offer for families," Dr. Hermiston explained. "Where we used to have - or now have - the conversation that this is a chronic lifelong genetic illness, that we’re going to be able to say we have a way to fix this."
The outcome for a child who had multiply-relapsed leukemia was less than 10%. CAR-T has taken the response rate to almost 90% in those kids with long-term remissions in the 60% range, she added.
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