If you're not improving the symptoms of the disease, the fact that there are detectable MRI changes is irrelevant. The effects of a drug are only useful if they improve the disease. The fact that it might produce detectable changes in the size of brain areas without improving the outcome for the patient is actually kinda scarry. It might suggest that unwanted side effects might be occurring.
Even if you suspect that the reason for a non-significant result may be due to a large placebo effect, that doesn't change the fact that AIMM failed to demonstrate efficacy and the FDA is not going to approve anything, no matter how safe, without an unequivocal demonstration of efficacy.
The placebo effect can be minimized by altering the instructions and thereby the expectancies of the subjects. If you imply that it is likely that they will be taking an inert placebo and that they should not expect any major effects from the drug, then there are usually less robust placebo effects.
A friend of mine, Jerry Woodward, is very knwoledgeble in this area and he believes that the only viable route for AIMM to take is to scrap MYLORAL as constituted, go back into the lab and develop a second generation formulation. He suggests that this should include an ajuvant and be paired with the withdrawal of all immunosuppressive drugs. This sounds like an eminently sensible solution to me. Continuing on and spending millions on more trials for a drug which, at best, has only very small effects IMO would be foolish.
I may have lost faith in MYLORAL. But, I have not changed my overall faith in the reasonableness and sound scientific approach that AIMM has followed all along. This company has fine scientists and sound management. The combination of the two does not always lead to success, and it did not here. However, I believe that they are simply too smart and capable not to pull back and try again with an updated and improved version of MYLORAL. They have the money to carry on for quite a while if they are not funding expensive clinical trials. If they can demonstrate that the second generation MYLORAL is far more potent than the present version, then, they can take it back into the clinic.
John de C |
