There are two big conferences in the world for people who deal with liver disease. EASL is in Europe in the spring and AASLD is in the US in the fall. This is where the latest research is released, where the cutting edge of knowledge about the disease is shared, and where the future of liver medicine can be glimpsed. It is a time for the superstars of the field to strut their stuff and be recognized by their peers and it is a window into the best of today and a glimpse of what we as patients can look to in the future.
We will follow this note with a more useful review of information we hope will be useful to you as patients, but I wanted to share a general perspective of what I see happening broadly.
Unless you are newly diagnosed, you will remember how excited we all were two years ago when we all believed Ocaliva from Intercept would be the first drug approved for the treatment of NASH. When the FDA said no it was a real setback for everyone. When that was combined with the failure of several other high profile phase 3 trials industry and the patient communities were quite depressed for a while. Investment pulled back and researchers questioned whether they had made a mistake going after a liver therapy.
But, unless you are a patient with a progressive disease, time heals. The lab work continued to advance, there were still clinical trials happening despite the pandemic, and the great engine of discovery may have wobbled a little but the journey to a better future continued.
My sense of where we are in our search for therapy for liver disease is greatly encouraged by what I saw at the meeting. The science at the cutting edge of discovery is exquisite. Therapy for us has been defeated in the past by the fact that liver is just so very complex. It is, by far, the most complicated chemistry in the body and in the past we just mashed it all together and tried to make sense of the average responses and that led to undesirable side effects, marginal therapeutic response, and clinical trial disappointment.
Science is moving incredibly fast. Today, the labs are able to study single cell chemistry. They are able to map the individual molecules and to see what they do in real time. They can analyze the cellular processes in exquisite detail and as we learn how to apply these tools it is clear that we will succeed.
As a patient, I'm dismayed that there are no treatments and frustrated by the gap between the science and the regulators but I'm enormously heartened by the fact that this tension exists. Even 5 years ago the plodding pace was just how it had always been and there wasn't really a case for doing it better. It is frustrating to be in that first wave of a new tide but that tide will lift all boats and we will be better for it.
I recently participated in testimony before the FDA. Here is a link to the recording if you are interested in how patients are speaking to the regulators today. Everyone always thinks their kids are smart and beautiful, but I think this is one of the best patient sessions we've seen and I'm proud to have been a part of it.