| Thanks for your questions! Yes we'll be testing combinations, as this brain cancer has not responded historically to single agent therapy for decades. With combinations of existing drugs it is possible to affect multiple driver pathways in the tumor at once. Similar to the dramatic improvement of AIDS therapy and infectious disease treatment with cocktails, tumors could respond much differently to combinations instead of a "one at a time approach" that has resulted in very small survival gains in this disease. Please see this paper for more detail on the scientific approach to mutli-agent combinations to target many pathways at once.
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4226667/ Patients have been taking the CUSP-9 protocol without any serious adverse affects on a compassionate use basis. This has now moved into a prospective clinical trial for recurrent glioblastoma patients in Germany
http://www.anticancerfund.org/projects/cusp9-a-combination-o... Personalized medicine services like this could allow clinicians to move beyond traditional randomized controlled trials. Commercial services such as Foundation Medicine provide test results to oncologists who then use their discretion to treat the patient outside of a clinical trial with scientific rationale based on the test itself as opposed to a standard of care. In a severe disease like Glioblastoma where average survival is 15 months, if the majority of patients using a system like Notable Labs live substantially longer, is a control arm necessary? The HAART cocktail for AIDS treatment never went through a phase 3 trial, so there is precedent in high need diseases. I also would like provide a link to a recent documentary that highlights the combination approach in brain cancer by telling the story of a 20 year survivor of Glioblastoma, Ben Williams. Other patients who safely used combinations of existing drugs are featured as well.
http://www.survivingterminalcancer.com/ |
If you do that then the community can start banding together to test formulations and then send the promising ones to you for further analysis.
Good luck. I love the idea and I personally have a few ideas of my own in the field that I want to start in 2016/2017
Edit * of course everyday people won't necessarily have access to approved therapies - but perhaps they can test other compounds and environmental conditions (or get the FDA to somehow approve tiny little test pellets for different drugs that would be legal for hobbyists to use in testing)