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Will the Vaxx Upend All Gene Therapy Trials?

I am a gene carrier for a heritable form of Dementia and have made myself available for clinical trials of new drugs that may someday provide a cure – probably not for me, but hopefully for my children. I want to relate an interesting story that just happened when I interviewed for a new drug trial.

As a veteran of drug trials, I know how to ask the right questions, though I had never applied for a gene therapy drug trial before: How many PET scans of the brain, how many lumbar punctures, how many overnights in the hospital, etc. Once I had qualified for the trial by proving that I am a non-symptomatic gene carrier, they started showing aggressive interest in getting me to participate. At this point, I did a deeper dive into the trial and discovered that it was an escalating dose of the novel drug that would be injected directly into the brain stem, which really got my attention. In my view, this was aggressive therapy on several fronts: gene therapy – be careful; injection into the brain – time to reconsider; escalating dose with no prior data – run for the hills!!

I didn’t need to hear anymore. Now I knew why the trial had been open for two years and was having trouble getting people to enroll. But here’s the most interesting takeaway: in the process of asking questions about the trial, I learned from the nurse researcher that if I enrolled in any gene therapy trial, I would never be able to participate in another gene therapy trial, which I can understand the rationale for, as any additional trial might have confusing results because of the prior trial. That’s when I couldn’t help myself (as an ardent anti-vaxxer), so I put the question to her: “We all know that the mRNA vaccines are gene therapies, so does that mean that your universe of potential enrollees has shrunken dramatically because you can’t accept people that have been vaccinated?” After a long silence, she said they hadn’t considered that issue before and just wanted to end the call.

All of this raises the question: will an entire class of drug therapy research be stymied by this issue going forward? Even though no one in the clinical trials world wants to consider the mRNA vaxx a gene therapy, the fact is that it is. They can’t just reclassify the vaccinated as gene therapy virgins because they will cloud the results. At the level of biology, the classification wordplay doesn’t matter. The vaccinated have been genetically manipulated. Pharma has to win the battle over the vaxx being classified as a gene therapy; otherwise, their huge bet on gene therapy meds will be truly spoiled.