On this episode of the podcast, Terry and Bob discuss gene therapies, which have the potential to cure and treat many genetic disorders by delivering nucleic acids into patients’ cells and replacing genes. Gene therapies can be lifesaving for many patients, but accessing them can be very difficult. They explain how the best approach to getting gene therapies more widely available is through innovation and markets.
Terry and Bob refute the idea that since gene therapies have received government funding then they should become government property once developed. By this logic, they point out, Google and Silicon Valley companies, which have also received government funding, should be government property too. In fact, anyone who has received student loans, following this argument, would owe their earnings to the government.
While the government does help facilitate good research, they often give small grants, which are gamed by establishment players who know how to navigate the grant process. Dr. Gary Michelson discusses this perverse dynamic back on Episode 27. The government picks winners and losers and rarely invests in the most innovative approaches. They note how the market process and price system are the best ways to bring prices down. Look at VCRs, for instance, which used to cost $700. This price signal encouraged innovation and democratization of the product as more players were incentivized to compete and bring costs down.
Bob interviews John Crowley, Chief Executive Officer of Amicus Therapeutics, whose story has been featured in the Wall Street Journal and even Hollywood. His children were born with Pompe disease — a severe and fatal neuromuscular disorder. Bob left his role at Bristol-Myers Squibb to try to find a cure for them, developing a new experimental treatment that he credits for saving their lives.
John argues that you can judge a society by the way it treats its children. Since rare diseases often affect children, research in this space is desperately needed. He also argues that the development of drugs to treat conditions that affect a wide range of patients often come from rare disease treatment breakthroughs. If we are going to solve Alzheimer’s and Parkinson’s disease it will be through developing treatments for rare diseases. Though his company hasn’t been profitable in 10 years, it has exciting technology. He argues that drug developers have a moral obligation to make sure medicines are priced fairly and accessible.
Patient correspondent Kate Pecora interviews Ciji Green, whose daughter Maisie has spinal muscular atrophy, one of the first conditions to respond to gene therapy. Life expectancy for Maisie was initially expected to be only two years. But gene therapy has halted and even reversed the disease. Ciji explains her fight with insurance companies — backed by “Maisie’s Army” — to get her daughter the treatment she deserves. Now Maisie is flourishing.