Hannah’s Hope has funded researchers at Emory University, Columbia University, the University of North Carolina and elsewhere to carry out the steps preceding the gene transfer experiment. And results have been so promising that the Food and Drug Administration (FDA) gave a tentative go-ahead this April, pending further toxicity studies in animals. Those are underway now, but cost $646,000 – plunging Hannah’s Hope Fund to the breaking point.
Lori estimates other costs:
- $430,200 for a 2-year natural history study for 12 patients to track the course of this ultra-rare disease, so that the researchers can determine exactly what to check to assess success.
- $220,000 to manufacture the viruses and their human gene cargo
- $600,000 for the phase 1, 2-year trial for 8 children
- $450,000 for a small Phase 2 trial to assess efficacy
That’s a total of $2,346,200. Then, hopefully, a biotech or pharmaceutical company will step in for the required larger phase 3 trials that precede FDA approval.
It sounds like a lot. But for comparison’s sake, $2,346,200 equals:
- approximately 1/30th of Johnny Depp’s fee for his next film
- 1/10th of what Alex Rodriguez earns in a year
- 10 speeches for Bill Clinton
- 6 of Kate Middleton’s wedding dress
- 2 years worth of nanny-time for Brad and Angelina’s brood
- Slightly more than Kim Kardashian’s useless $2 million engagement ring
- .07% of the money spent on the Obama and Romney campaigns as of March 2012.
The government isn’t funding the GAN gene therapy trial. So what the tiny GAN community needs, desperately and now, is a Michael J. Fox, Christopher Reeve, or Julia Roberts.