FDA Must Do Its Due Diligence on Gene Therapies

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There’s no grand conspiracy that’s kept us using fossil fuels lo these many years. The problem of weaning people onto something cleaner is that nothing else can do the job. Solar promised too much too early, that it couldn’t possibly deliver, and nuclear scared people, not just with hiccups like Three Mile Island but genuine nightmares like Chernobyl.

It would be great if super-effective nuclear and super-clean solar could take care of all our needs. But they can’t, not yet. I’m sorry that conservatives have to be the Mean Dad and say “you can’t have that,” but at this point I’m angry that we have to keep saying it, whether it’s to insipid children like Alexandria Ocasio-Cortez and grownups who should know better like Bernie Sanders.

Unfortunately, now we have to give this bad news to some very ill people – or at least to some snake-oil salesmen who might dupe them.

President Trump promised in his recent State of the Union address that the United States was committed to fighting deadly diseases, notably childhood cancer. While some new techniques promise to be great weapons in this war, they haven’t been proven yet and might therefore cause friendly fire, to continue the allegory. Controversial gene therapy drugs could save many lives in the future, but for now the Federal Drug Administration (FDA) needs to make sure such drugs aren’t rolled out until we know they’re safe.

Gene therapy is an experimental treatment that involves introducing targeted genetic material into a person's cells to fight or prevent particularly onerous diseases, such as immuno-deficiencies, hemophilia, Parkinson's, cancer, and HIV. The therapy is an attractive proposition, because it promises to correct genetic disorders by either a) restoring a normal-functioning copy of a gene or b) reducing the toxicity arising from a mutated gene.

That certainly sounds nicer than chemotherapy or aggressive pharmaceuticals, but the reality is much more complex – and potentially risky – as a result the delivery systems used. The most common delivery is a virus that, while altered to be as safe as possible, still carries with it some risks.

But because it’s got so much potential – and there’s so much money to be made – the FDA is contemplating “expedited regulatory review pathways” to get them to market. This would make gene therapy drugs available to patients as quickly as possible, particularly for products that promise to treat or cure diseases where traditional treatment options are currently inadequate.

Normally the FDA stepping on the gas pedal would be a good thing. But with gene therapy, what we need is just enough regulation to make sure the treatments are safe – we need guard rails to keep us from driving off a cliff.

Just as with snake-oil salesmen of old, there are fraudulent drugs on the market, pushed out to help people in need. Some manufacturers are developing therapies outside usual regulatory channels, and the FDA has been good about realizing the need to clamp down on them. “In these cases, the manufacturers have failed to respond to the FDA’s request to have a dialogue with the agency about how to come into regulatory compliance,” the administration has said. One glaring example is Chinese scientist He Jiankui, who “startled and outraged the scientific world” by genetically editing two twin girls without permission, ostensibly to protect them against HIV.

Another atrocious example is Jesse Gelsinger, and 18-year-old suffering from ornithine transcarbamylase deficiency (OTC). The gene therapy used to treat him caused “a chain reaction that the testing had not predicted -- jaundice, a blood-clotting disorder, kidney failure, lung failure and brain death,” according to The New York Times. Following Gelsinger’s death, it was revealed that in the previous seven years of U.S. human gene therapy experiments had caused 691 “serious adverse events” (up to and including death).

Granted, these tragedies were all back in the 1990s, but as recently as 2016 the journal Nature warned that “Gene-therapy trials must proceed with caution – The perils of the past must not be allowed to happen again.”

Apparently not everyone has learned this lesson. Just last year, the Novartis-owned company AveXis filed for FDA approval for a gene-therapy of spinal muscular atrophy (SMA) – at a tidy price tag of $4 to $5 million for one procedure patient, this despite the fact that safer, proven treatments already exist for SMA.

Gene therapy could have unlimited potential – just like solar and nuclear power – but its efficacy and safety are unproven. Such treatments could dramatically improve quality of life for otherwise untreatable patients – or they could be another Chernobyl. Let’s make sure the FDA does its due diligence before we take such a risk.

Jared Whitley is a longtime Washington, D.C., politico who worked in the Senate, the White House, and the defense industry. He has an MBA from Hult International Business School in Dubai.

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