RE:RE:GBM
I don't work in an area of medicine that deals with GBM but my understanding of the next steps would be
- investigational new drug approval.
- phase 1a/b trial. 1a is where you give a small dose and see. Then work up to what you think is the full dose needed. 1b is where you test a few more with the full dose.
- For GBM they would be treating patients who are just about to go palliative and there are no other standard options. They would do the treatment and then see if it meet RECIST criteria. This is a way of assessing a response to a cancer drug. In essence, if it shrinks 30% in longest diameter and no new cancers pop up then that is a response. Hypothetically, if the 1b patient's are salvaged from palliation and the tumour shrink dramatically then you would probably have access to the fastest FDA supports available as you are not treating something for which existing treatments work. If there was another drug that worked then you would have to do a comparison trial but no such thing exists in end stage GBM. So if it works as well as it does in animals it could be a faster process then ever seen before. Wishful thinking...
By way of comparison, the most common drugs that I'm aware of trying to work on GBM are small molecule agents that you take as a pill and they are often targetted to slow down growth and then, given enough time, cause some shrinkage. Those trials take a while as many don't respond and those that do, can take 3-6 months to see any changes on CNS imaging. Ruvidar would potentially have visible changes within weeks or a month so that may help.
All hopeful but who knows. I'm intrigued by the fact that there are no similar drugs out there so it is attacking GBM by a new mechanism.
IMO