Gene Therapy Is Where The FDA Needs To Provide Excellent Oversight

Written by Michael J. Daugherty on March 7, 2019

Conservatives want two things – a less intrusive federal government and one that does good work. The U.S. Food and Drug Administration (FDA) has an important job to make sure that all drugs rolled out for public consumption are safe. Right now, there is a controversy over something called “Gene Therapy” that both provides hope for those suffering from illnesses like SMA (spinal muscular atrophy) and some dangers.

Checking to make sure drugs are safe is the primary responsibility of the FDA. The FDA’s responsibilities, according to their own website, include regulating “prescription drugs (both brand-name and generic)” and “non-prescription (over-the-counter) drugs.” The FDA explains that human gene therapyseeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use.” The concern is that the FDA has announced an expedited review of some gene therapies that might lead to short cuts.

President Donald J. Trump promised to use the power of the federal government to fight cancer and rare diseases. Gene therapies provide some promise, but pose some dangers. The president declared in his 2019 State of the Union speech to Congress, “no force in history has done more to advance the human condition than American freedom. In recent years we have made remarkable progress in the fight against HIV and AIDS. Scientific breakthroughs have brought a once-distant dream within reach. My budget will ask Democrats and Republicans to make the needed commitment to eliminate the HIV epidemic in the United States within 10 years. Together, we will defeat AIDS in America. Tonight, I am also asking you to join me in another fight that all Americans can get behind: the fight against childhood cancer.” The president needs to use the resources of the FDA to take care that gene therapy treatments are properly tested before being used to fight these illnesses.

Gene therapy can be used to fight HIV and cancer, yet it is a relatively new and experimental concept that has proven dangerous in certain circumstances. The Digital Journal published a press release that described gene therapy as “an experimental treatment that involves introducing genetic material into a person’s cells to fight or prevent disease. Researchers are studying gene therapy for a number of diseases, such as severe combined immuno-deficiencies, hemophilia, Parkinson’s disease, cancer, and even HIV, through a number of different approaches.” The FDA is right to explore experimental treatments like gene therapy, but pushing through approvals may lead to unintended consequences.

There are two big hurdles that need to be addressed before the FDA rolls out a number of new gene therapies – the first being safety. Expedited regulatory review pathways may allow fraudulent drugs to hit the market. PM Live reported on January 16, 2019, “faced with the upsurge in activity, the regulator has set out its plans to make sure it is able to handle the coming workload, and make them available to patients as quickly as possible. It intends to make ‘maximum use’ of expedited regulatory pathways, including the regenerative medicine advanced therapy (RMAT) designation and accelerated approval, particularly for products that offer potential cures for diseases with inadequate treatment options at the moment.” Expedited review might not be enough review. The report indicates that “robust post-market tools” will be used to monitor issues that may not reveal themselves in clinical trials. The history of gene therapy includes catastrophic cases that argue against short cuts.

There are some cases of fraud and some that show inherent dangers. The PM Live report cited a fraud case from last year that caused a furor “by Chinese scientist He Jiankui whose gene-editing of twin girls to protect them against HIV.” The New York Times reported in 1999 on a case that shows the danger. Jesse Gelsinger who died as a result of gene therapy treatments because of unforeseen complications. Gelsinger “suffered a chain reaction that the testing had not predicted — jaundice, a blood-clotting disorder, kidney failure, lung failure, and brain death.” The report indicated that “Jesse’s therapy consisted of an infusion of corrective genes, encased in a dose of weakened cold virus, adenovirus, which functioned as what scientists call a vector.” There was testing done on animals and one human patient, but Gelsinger “suffered a chain reaction that the testing had not predicted.” The FDA has to make sure that new therapies do not have the same problems because it is difficult to test these relatively new procedures.

A second issue is the high cost of treatment. Reuters reported on December 15, 2018, that a new Novartis gene therapy “could be cost-effective to healthcare systems at $4 million to $5 million per patient.” That cost is considered “cost-effective” by a big drug company only because the cost will be borne by health insurance companies and government payers. Companies will try to shift a massive cost onto third-party payers, because then they can charge far more for a therapy than they would in a true free market with consumers negotiating prices.

The approach of using gene therapy has a flaw; the clinical trial data is extremely limited and the long-term implications of it are unknown. The FDA and regulators should use common sense before fast-tracking experimental and risky technology because this new therapy poses a real safety concern. Government regulators should not bow to well-funded special interests motivated by making money by rushing a new gene therapy drug to market too early.

Mike Daugherty is CEO and Founder of LabMD, a cancer detection laboratory based in Atlanta, Georgia. He is author of The Devil Inside the Beltway: The Shocking Expose of the US Government’s Surveillance and Overreach Into Cyber-security, Medicine and Small Business.