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Hi,
I am thinking of writing a letter to Berlin Cures, Dr Bettina Hohburger and Professor Carmen Scheibenbogen to assist them with experimental design. I am hoping to gather more ideas from people on PR who have a longer memory about Ampligen.
I am not a scientist, but it occurred to me that Berlin Cures is likely to be unaware of Ampligen and could learn lessons from the failure of Ampligen to get approved.
Ampligen was a drug that anecdotally cured or significantly improved the lives of many ME/CFS patients, but which ultimately failed to convince the FDA of its efficacy and safety.
We dont have access to the FDA rejection letter, but it is described in a press release from Ampligen's creator, HemisphereX:
It seems the FDA's calculation shows they did not show statistical significance for efficacy, but HemispherX did. The question of safety seems to be related to some patients who became worse while on Ampligen. In later presentations, HemispherX insists the number of adverse events in the drug and placebo cohorts were identical.
The experimental design of the Ampligen trial is described here: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4917909/#!po=23.6842
What lessons can we gather here? I was going to suggest the following lessons:
So any tips on experimental design for Berlin Cures? You guys read a lot of ME/CFS papers. What is the best way to measure improvement in performance? Ampligens phase 2 trial used Karnovsky scores which are a subjective measure. Step counts seem to be a flawed measure as well.
I am thinking of writing a letter to Berlin Cures, Dr Bettina Hohburger and Professor Carmen Scheibenbogen to assist them with experimental design. I am hoping to gather more ideas from people on PR who have a longer memory about Ampligen.
I am not a scientist, but it occurred to me that Berlin Cures is likely to be unaware of Ampligen and could learn lessons from the failure of Ampligen to get approved.
Ampligen was a drug that anecdotally cured or significantly improved the lives of many ME/CFS patients, but which ultimately failed to convince the FDA of its efficacy and safety.
We dont have access to the FDA rejection letter, but it is described in a press release from Ampligen's creator, HemisphereX:
The Agency stated that the submitted data do not provide substantial evidence of efficacy of Ampligen® for the treatment of CFS and that the data do not provide sufficient information to determine whether the product is safe for use in CFS due to the limited size of the safety database and multiple discrepancies within the submitted data.
It seems the FDA's calculation shows they did not show statistical significance for efficacy, but HemispherX did. The question of safety seems to be related to some patients who became worse while on Ampligen. In later presentations, HemispherX insists the number of adverse events in the drug and placebo cohorts were identical.
The experimental design of the Ampligen trial is described here: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4917909/#!po=23.6842
What lessons can we gather here? I was going to suggest the following lessons:
- Suggest that they speak with Dr Peterson and Dr Lapp about their proposed experimental design. The doctors might be able to share lessons or provide tips without disclosing confidential information about the Ampligen trial.
- The phase 3 Ampligen trial measured improvement based on exercise tolerance using a treadmill. Both the placebo and Ampligen arms improved their exercise tolerance, but the improvement in the Ampligen arms was statistically significantly larger than the placebo arm based on the company's calculations.
- The safety profile featured severe adverse events in both arms. That is likely because ME/CFS patients experience severe crashes. Berlin cures will need to design their experiment design with this fact in mind.
- Suggest they use an objective primary endpoint, such as improvement in microcirculation in the eyes rather than attempting to use
So any tips on experimental design for Berlin Cures? You guys read a lot of ME/CFS papers. What is the best way to measure improvement in performance? Ampligens phase 2 trial used Karnovsky scores which are a subjective measure. Step counts seem to be a flawed measure as well.