Barriers remain for this level of revolutionized medicine.
 | By Alexandra Sifferlin Senior Staff Editor, Opinion |
As scientists have learned more about the effects specific gene mutations have on our health, the promise of gene editing has taken shape. And in the last few years, significant progress has been made that brings a new kind of medicine into view. |
Gene therapies already exist for some conditions. A recently approved one, for a bleeding disorder, costs a record $3.5 million for a single-use vial. Gene editing with CRISPR, which builds on gene therapy, is now also being used in studies to treat diseases like sickle cell and heart disease. But barriers remain for widespread use. |
Fyodor Urnov, a highly regarded CRISPR scientist at the University of California, Berkeley, who with colleagues coined the term "genome editing," discusses the state of the science — and his view of what's holding it back — in a guest essay for Times Opinion. "So much substantive progress has been made in the field of genetic medicine that it's clear scientists have now delivered on a remarkable dream: word-processor-like control over DNA," he writes. |
The obstacles, he argues, are "not technical but legal, financial and organizational." The $3 million medication is case in point, he says. Currently, scientists who want to develop treatments for genetic diseases undergo a long and expensive clinical trial and regulatory process for a drug that will benefit a relatively small group of patients. Drug companies then price the treatments exorbitantly to make up for it. Urnov argues that the Food and Drug Administration needs a new pathway for CRISPR cures, one that's faster and more financially sustainable. |
The possibilities he puts forward are nuanced. He believes CRISPR drugs that are used to treat diseases that affect huge numbers of people, like heart disease, merit the lengthy process. But he and others envision a future where doctors and scientists can treat one-of-a-kind mutations, which isn't possible currently. From Urnov's perspective, science can already deliver. |
"Scientists can rewrite a person's DNA on demand. But now what?" Urnov writes. "Unless things change dramatically, the millions of people whom CRISPR could save will never benefit from it." |
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