Working with mice, researchers have used gene therapy to restore sight to the blind, reprogram the body’s own T cells to attack cancerous tumors, prevent the formation of amyloid plaques in Alzheimer’s brains, and more. But the US Food and Drug Administration and many other international regulatory bodies have been slow to approve human trials due to the high risks associated with tinkering with a patient’s DNA.
Liz Parrish thinks that the FDA and the medical establishment have it backward. Parrish is the founder and CEO of BioViva, a Seattle-based company that is pushing for open access to potentially life-saving gene therapy technology now, not later. The real risk, Parrish argues, is that more than a hundred thousand people die each day, and millions more suffer, while we wait for regulatory agencies to approve drugs that can save lives.
BioViva isn’t a research institute and Parrish isn’t a scientist. Instead, the company positions itself as a “translational engine,” a private company bringing together international researchers and physicians to speed the delivery of experimental therapies.
The only way to truly know if these therapies work in human bodies — as opposed to mice or human cells in the lab — is to give patients the right to decide if the risk is worth it. And the more people who choose to undergo these therapies, the more data BioViva can collect and publish about their true risk and efficacy.
Read full, original post: Who Should Decide If Gene Therapy Is Too Risky, Patients or the FDA?