This brings up an interesting ethical question: Is it OK to buy your way directly into a clinical trial? At first glance, I would say no, but then upon further consideration, the trial wouldn't be able to start for anyone if those first patients didn't pay the $2 million... so it is helping others. In the past, wealthy people have approached me and have asked for help in getting unapproved treatments. My response was always that it had to be done within a standard clinical trial. They would donate money to the Musella Foundation, and I would then find a researcher willing to design and run a clinical trial of the treatment we wanted, with liberal enough entry criteria that the donor would qualify. However, it had to be open to other patients as well. This has worked out pretty good and I do not think it has the moral issue of a patient paying directly to get into a trial. There is more oversight - our medical advisory board has to approve of the project, and we are able to watch and make sure the trial really opens for others, and the money is well spent and accounted for. This has resulted in new treatments that are now being used for many other patients.
