Over the past two years, I have been participating in Gilead’s GS-9674 PSC study, which involves taking an experimental drug now labeled as Cilofexor. Cilofexor is a “nonsteroidal FXR agonist”, and its function is supposedly to reduce bile production in the liver. As we know, it’s the backup of bile from PSC which causes damage to our livers over time. During the study, I was also allowed to continue to take Urso, as they are studying the combination of Cilofexor and Urso used together to reduce bile buildup in the liver.
The study is coming to a close, and the good news is that they are now extending the study to current participants under what they call “open label”. This means that everyone who was involved with the study, including those given the placebo, will be allowed to continue taking the experimental medication for an additional two years. The clinician I have met with over the past two years said that the fact that they are allowing open label continuation is really good news, and that, had the study not been successful, they would not have done this, and it could be a sign that they will seek FDA approval of the drug.
I would like to share my experience with the study with everyone here, in hopes that maybe it provides some hope that there may be a treatment available in the future for this terrible disease.
As part of the study, I was required to have a liver biopsy done both at the beginning of the study, as well as the end of the study, which I just had done about a month ago. The specialist who read the biopsies said in his report that “ the one from 2019 showed bridging fibrosis, while the one from 2021 showed portal fibrosis. There was a regression, which suggests perhaps improvement”.
To be honest, I feel that taking the study drug over these past two years provided significant benefit. During my time with PSC, I have had these predicable “episodes” every 3-4 months, which starts with sharp RUQ pain, followed in the next couple of days by itching, heartburn, and sometimes nausea. These symptoms always occur in tandem, so it definitely appears to be the PSC. While on the study medication, I noticed a significant reduction in both the number of these episodes, and, when they did occur, the symptoms were less severe than before taking the study medication. While it is always possible I received the placebo, I had significant itching when first starting the medication, which is a side effect of the medication, and the clinician told me it was very likely I was receiving the drug and not the placebo. Thankfully the itching resolved as my body got used to the drug. My Fibroscans also improved as well, which were taken at periodic intervals throughout the study, so all good signs.
Additionally, my liver enzymes were stable the entire time, using the combination of Urso and Cilofexor.
Hopefully we will see the full results of the study published in the next year or so, but I wanted to share my experience here. I will post again as I progress further into the open label portion of the study.
