Having spoken to someone high up in the food chain, the results from the Phase 2 were so good, researchers had trouble believing them. Basically, they know what they have and if we all started talking about it, they would have mutiny on their hands.
CF Foundation has put the kabosh on miglustat in one swoop. Miglustat, an already approved drug, increased CFTR function by 120%. Normally, that is when the CF Foundation would be jumping up and down and start doing trials immediately. However, they had already invested so much money in these vertex drugs, that they decided NOT to investigate miglustat even though miglustat could be available to us in 1.5 years or less because it is already approved for another illness.
All of this is business after all. Unless we come together like the AIDS community and demand that we be given these drugs like they did with AZT, we have many years of waiting. As an adult with CF, I am willing to sign a waiver saying I'm not going to sue or hold them liable, I just want to opportunity to get the drugs before it is too late for me.
CF Foundation has put the kabosh on miglustat in one swoop. Miglustat, an already approved drug, increased CFTR function by 120%. Normally, that is when the CF Foundation would be jumping up and down and start doing trials immediately. However, they had already invested so much money in these vertex drugs, that they decided NOT to investigate miglustat even though miglustat could be available to us in 1.5 years or less because it is already approved for another illness.
All of this is business after all. Unless we come together like the AIDS community and demand that we be given these drugs like they did with AZT, we have many years of waiting. As an adult with CF, I am willing to sign a waiver saying I'm not going to sue or hold them liable, I just want to opportunity to get the drugs before it is too late for me.