“This spring, a group of doctors used a personalized gene therapy to save an infant with a deadly genetic disorder. Their success has since cleared the way for a new trial to test similar therapies on more severely ill children. This research was made possible in part by greater regulatory leniency: the FDA will allow the team to reuse safety data from the first case, even as they tailor the treatments to each child’s unique mutation,” reports The Doomslayer.
The Guardian notes that
Doctors in the US have become the first to treat a baby with a customised gene-editing therapy after diagnosing the child with a severe genetic disorder that kills about half of those affected in early infancy.
International researchers have hailed the feat as a medical milestone, saying it demonstrates the potential for treating an array of devastating genetic diseases by rewriting faulty DNA soon after affected children are born.
Specialists at the Children’s Hospital of Philadelphia and the University of Pennsylvania started work as soon as the boy was diagnosed and completed the complex design, manufacture and safety testing of the personalised therapy within six months.
The baby, known as KJ, had the first dose of the bespoke treatment via an infusion in February and two more doses in March and April. Doctors said he was thriving, but would need careful monitoring for life.
As Nature notes, deregulation made this treatment possible: “The US Food and Drug Administration (FDA) would normally require each new formulation to undergo a separate clinical trial, with safety tests to ensure that the gene-editing components are not toxic. But in this case, the FDA has indicated that it will accept some of the safety data from KJ’s treatment.”
More deregulation is needed. Overregulation at the FDA delays the approval of lifesaving drugs, and thus kills many people. Many people die waiting for the FDA to approve life-saving drugs and tests. For example, at least a hundred thousand people died waiting years for the FDA to approve beta blockers. One of the FDA officials involved in delaying their approval was John Nestor. Nestor was notorious for following rules in ways designed to deliberately delay other people, such as his habit of deliberately driving slowly in the fast lane on highways in order to slow down other motorists.
The FDA didn’t approve a home test for HIV until 24 years after it first received an application. According to an FDA advisory committee, the test held “the potential to prevent the transmission of more than 4,000 new HIV infections in its first year of use alone.” That means thousands of people likely got infected with AIDS as a result of the delay in approving it. As Roger Parloff noted in Fortune, the FDA’s delay in approving the home HIV test was a “scandal.” It caused the deaths of thousands of people.
Gene therapy is restoring vision to some people with inherited blindness. Another gene therapy blocks the painful hereditary condition angiodema.
