NB as a Business

A reply on ACOR from fellow parent, Neil Hutchinson, who's boy Sam is in treatment with Max here in San Diego for recurred NB. This guy is really on the ball.

Well, I think we should treat NB as a business and put together a business presentation for:

1. the myth of competing trials (to overcome the thought that "we can't have too many trials open or we'll never fill our trial" - good trials accrue patients :)
2. why it makes good business sense to put drugs into trial for neuroblastoma (Bayer, the manufacturer of nifurtimox may get a 2nd indication for this drug which could translate into a worldwide market; how many kids would this be, what's the money involved)
3. trial ratings (I think parents should develop a rating system for trial design; positive aspects should be intra-patient dose escalation, allowing kids who have had the drug before to get it again since they are often precluded, finding a way for kids that have limitations w/respect to platelets to have access to drugs). I think we can also use this to change the mindsets of the current NB doctors to design their trials in a more patient friendly manner.
4. then we contact the drug companies that have drugs we would like to trial. One company is Hoffman-LaRoche which has a drug called nutlin. If you search the ANR abstracts (and it's probably on Pubmed), nutlin is something that reverses the off- switch on the p53 tumor-suppressor. John London and I found out about this when talking to some oncs the other day. The company probably is just looking at bigger cancer markets but what if we call them and persuade them to get the drug into trial since I think much of the pre-clinical work is done?

The whole tone would be one of collaboration and positivity (I live a mile from Sea World and it's the old "how we train Shamu"). I think so much of what we have to overcome is simply awareness: if a company knew that parents would support a trial, they may just me interested in accelerating things.