When Cortene contacted BHC with an idea, and an early draft protocol designed to test CT38 as a treatment for ME/CFS, Dr. Suzanne Vernon and I agreed to help them refine the protocol and conduct the trial. The Cortene-funded trial was an FDA and IRB-approved protocol, and BHC carried out the protocol with detailed precision. We had a routine contract with Cortene to carry out the trial. It was challenging, because CT38 is a novel substance that has previously been tested only in healthy controls, as there is no animal model for ME/CFS.
Cortene has a vision that CT38 will be curative for people with ME/CFS based on the known mechanisms and their hypotheses. Before CT38 was tested in ME/CFS, I had no clinical data to form an opinion about efficacy, but I was willing to put the drug to the test. The phase 1/2 trial was meticulously carried out at BHC, with courageous volunteers. We learned that even the lowest starting dose, an estimate based on responses previously in healthy controls, resulted in a strong physiologic response in the first two ME/CFS subjects. Revisions were made to the protocol to revise the dosing regimen, with full disclosure to and oversight by the IRB and FDA. Once the study was complete, the contracted role of BHC ended; it then became Cortene’s responsibility to analyze the data. I have stayed on as a medical advisor to the company as the data were analyzed, the paper published, and next steps determined. Cortene is optimistic about the results, and I am pleasantly surprised by the results and hope the next phases of research can move forward.
It is hard for all of us to remain dispassionate and allow the scientific process to take its course when the needs are great and so much is at stake. However, science, and especially clinical trials, must be carried out with an intellectual willingness to accept whatever the data teach us, regardless of our emotional or financial investment. Research protocols are usually carried out under a business contract at a separate institution than the pharmaceutical company, to reduce the risk or perception of bias. Research participants, so understandably eager for a positive response, are subjected to placebo assignment or other study designs to reduce subject bias in larger clinical trials.
We should be careful how we use or react to the word “cure” at this stage of the scientific process. None of us know what CT38 can really do for a larger pool of people with ME/CFS or anyone with Long COVID until larger, controlled trials are complete. Cortene asserts that if the disease mechanism reversed by CT38 is a primary cause of ME/CFS, then the drug has the potential to be curative rather than supportive. That is exciting but remains to be proven. The preliminary results from the first, small, proof-of-concept CT38 trial in ME/CFS provide a strong enough signal to move forward with the next steps. Given the known heterogeneity of ME/CFS, it is unlikely any treatment breakthrough will have the same response in everyone who meets the diagnostic criteria. We must be prepared for that outcome. But for all with ME/CFS, I hope that CT38 proves to be an effective treatment for as many as possible.
Now it is Cortene’s responsibility, like all pharmaceutical companies, to raise money for these larger, placebo-controlled trials of CT38. BHC’s role with the company was to help them understand ME/CFS, develop an illness-specific protocol, oversee implementation of the protocol and interpret the data.
My resolve, and BHC’s mission, is to broadly encourage and develop research, education, and clinical expertise to improve the lives of people with ME/CFS and related conditions, not to support or promote any specific product or ideology. I do not own stock in Cortene, and Cortene has not donated funds to BHC. BHC has purposely maintained a collaborative approach with all working toward solutions for ME/CFS. We remain steadfastly invested in our mission.
Read Dr. Vernon’s September 3rd post, Cortene Study Published.