Currently, I am taking a drug called Ursodiol. Ursodiol is used to dissolve gallstones in patients who do not need to have their gallbladders removed or in those in whom surgery should be avoided because of other medical problems. However, ursodiol works only in those patients whose gallstones are made of cholesterol and works best when these stones are small and of the “floating” type. It is also used to help prevent gallstones in patients who are on rapid weight-loss programs.
It is believed to help make your bile more fluid so that it flows easier through constricted or narrowed bile ducts. For me, all that I know is every time I have ever tried to stop taking it I get sick with Cholangitis.
Here is an example of my Lab Reports (not taking URSO vs. taking URSO). Again, this isn’t an ongoing study that I’ve been keeping track of but I do trend my lab reports monthly to see how they are changing and if anything in particular that I do seems to make it better or worse.
To me, it’s enough of a difference to make me want to keep taking the Urso. At this stage in the disease, it’s all about a quality of life. The longer I can make it between an ERCP, the better.
So are there any other treatments on the horizon? Any hope? Recently a new study has shown some hope in a possible treatment for this horrible disease.
Novel Drug Offers Hope in Rare Liver Disease
24-norursodeoxycholic acid shows promise in primary sclerosing cholangitis
A chemical cousin of an existing drug might offer hope to patients with a rare but debilitating liver disease, a researcher said here. The existing drug is ursodeoxycholic acid (UCDA) whose value in primary sclerosing cholangitis (PSC) is debated, according to Michael Trauner, MD, of the Medical University Vienna in Austria.
But a phase II clinical trial suggests that a variant of the compound — 24-norursodeoxycholic acid (norUCDA) — can reduce liver damage owing to the disease, Trauner told reporters at the International Liver Congress.
In PSC, fibrotic obstruction of the bile ducts lead to cirrhosis and liver cancer, and the illness is becoming an important indication for liver transplant, Trauner said. It’s “not a very common disease, but it’s a very serious disease that affects young adults,” he added.
Because there’s currently no effective medical treatment for PSC, “we are very excited by this data” even though more research is needed, commented Frank Tacke, MD, Ph.D., of the University Hospital Aachen in Germany and a member of the scientific committee of the European Association for the Study of the Liver (EASL), which organizes the meeting.
Tacke said the annual incidence of PSC is about 15 per 100,000 people, and patients generally are diagnosed with the disease in their 20s and 30s. Many have long-term irritable bowel syndrome (IBS) and face the prospect of liver cancer or transplant within a decade or so of diagnosis. NorUCDA has a novel mechanism of action, he said, and represents a “new hope for this type of patient.”
“This is an awful liver disease that we have,” commented Heiner Wedemeyer, MD, of Hannover Medical School in Germany, who was not involved in the study. “It’s so difficult to handle these patients,” and “this is the first time in decades that we have something that might work.”
The compound is derived from UCDA by removing a side chain, a modification that in animal studies gave norUCDA anti-cholestatic, anti-inflammatory and anti-fibrotic properties, Trauner reported. It also appears to detoxify bile acids. To test it for the first time in human, Trauner and colleagues enrolled 161 patients from 12 countries who were either UCDA-naïve or underwent an 8-week washout before beginning the trial. None of the patients took the older drug during the 12 weeks of therapy. Patients were randomly assigned to get a placebo or one of three doses of norUCDA — 500, 1,000, or 1,500 mg daily, he said. They were followed for an additional 4 weeks after the end of treatment.
The primary efficacy endpoint was the average change in serum alkaline phosphatase (ALP) — a marker of liver distress — from baseline to the end of treatment, while secondary endpoints included the proportion of patients with partial normalization of ALP (to below 1.5 times the upper limit of normal), change in liver enzymes and bilirubin, and such things as pruritis, fatigue, and disease activity scores.
Analysis showed that norUCDA reduced ALP compared with placebo in a dose-dependent fashion — 12.3%, 17.3%, and 26%, respectively, and was significantly better than placebo for all three doses. The pattern was similar for other laboratory values, he said. Based on that data, the 1,500-mg dose will be tested in a phase III trial, Trauner said.
During his late-breaker platform presentation later, Trauner said researchers measured the quality of life and found no change, possibly because of the short study period, and also were unable to see a change in IBS among affected patients.
On the other hand, the adverse event profile was not different from placebo at any dose. Indeed, some 80% of placebo patients reported at least one adverse event, compared with 59%, 73%, and 67% of those getting the 500-, 1,000-, and 1,500-mg doses, respectively.
Similarly, 28% of placebo patients reported an adverse drug reaction, compared with 23%, 32%, and 38% of patients respectively on the low, medium, and high norUCDA doses.