Wow, my head is spinning from these recent clinical trial results. Within minutes of one another, two conflicting studies were released. The first I’ll highlight is of tocilizumab (the drug that my friend and colleague Kazu Yoshizaki developed to treat Castleman disease and tested on himself to prove that it was safe before giving it to other humans). Tocilizumab blocks the interleukin-6 receptor, which is important for propagating inflammation and the cytokine storm seen in Castleman disease and COVID19.
The investigators of a study of tocilizimab reported that it achieved the primary efficacy endpoint of the study, which means that a significantly lower proportion of the 65 moderate-to-severe COVID19 patients on tocilizumab died or needed to be on a ventilator by day 14 than the 64 moderate-to-severe COVID19 patients not on tocilizumab. Unfortunately, they provided very little additional information…
Within a few minutes of the above exciting results:
Confusingly, sarilumab, which ALSO blocks the interleukin-6 receptor (though at a different location), was reported to have no benefit in a group of severe and critically severe COVID19 patients. A big takeaway from the study: patients on sarilumab had similar outcomes to patients in a previous study that got us excited about the potential for IL6 blockade, but the control group in this study had a similar outcome too…
There was reason to be hopeful from within the sarilumab data for the most critically ill patients:
Among those on placebo: 55% of 77 patients died or were on a ventilator.
Among those on 200mg sarilumab: 46% of 94 patients died or were on a ventilator.
Among those on 400mg sarilumab: 32% of 88 patients died or were on a ventilator
There may also be some suggestion of activity from the proportion of patients discharged from the hospital: 41% of placebo patients were discharged versus 39% in the 200mg group and 53% in the 400mg group.
Nevertheless, we’ll keep pushing forward with everything that we’ve got to extract data on existing and novel drugs that may be effective for COVID19 in our CORONA (COvid19 Registry of Off-label & New Agents) project.
David Fajgenbaum, MD, MBA, MSc, is a groundbreaking physician-scientist, disease hunter, speaker, and bestselling author of the acclaimed memoir, Chasing My Cure: A Doctor’s Race to Turn Hope Into Action. Best known as the ‘doctor who cured himself’ (Doctor Cure Thyself, NY Times), Fajgenbaum went from being a beast-like college Quarterback to receiving his last rites while in medical school and nearly dying four more times battling Castleman disease. To try to save his own life, he spearheaded an innovative approach to research and discovered a possible treatment that has put him into an extended remission. Now, he is spreading this approach to other diseases (His method could save millions, CNN) and sharing lessons he learned about living from nearly dying through Chasing My Cure, which has been translated into five languages and named one of the “Best Non-Fiction Books of 2019” by Next Big Ideas Club.