PBC Q&A with Dr. Gideon Hirschfield

Dr. Gideon Hirschfield is a leading voice in the field of autoimmune liver disease*. Dr. Hirschfield is the inaugural Lily and Terry Horner Chair in Autoimmune Liver Disease Research at the Toronto Centre for Liver Disease (TCLD), Toronto General Hospital and Professor of Medicine in the Division of Gastroenterology at the University of Toronto. Dr. Hirschfield, a physician scientist, is a co-director of the Autoimmune Liver Disease Program at TCLD; this program offers lifelong, comprehensive care for people with autoimmune hepatitis, primary biliary cholangitis, and primary sclerosing cholangitis. Additionally, Dr. Hirschfield manages a broad platform of translational and trial-based clinical research to advance treatment for people living with autoimmune liver disease to help them feel better, function better, live longer, and avoid liver transplant.

Ivette Williams is PBC Patient Advocate. She is the Regional Leader of the Illinois PBC Support Group. Ivette has been an active member and strong supporter of the American Liver Foundation since 1999.

Ivette recently sat down with world-renowned physician scientist Dr. Hirschfield to ask the questions on the minds of many living with Primary Biliary Cholangitis.

What is the basic physiology of Primary Biliary Cholangitis, or PBC?

I think this first question is very important. It’s so important for patients to know what kind of disease PBC is and what it means when you have it.

PBC is an autoimmune liver disease. In an autoimmune disease the body’s natural immune system incorrectly recognizes the “self” as an invader and launches an immune response; in autoimmune liver disease, the liver is recognized as something that does not belong causing the immune system to attack the liver and cause damage. We think PBC arises because of a mixture of genes and environment. Your genes predispose you to autoimmune disease and something in your environment triggers the development of PBC.

PBC damages the liver in two distinct ways. First, your immune system attacks the small bile ducts inside your liver. The bile ducts wither as the immune system essentially punches small holes in them. The bile inside those damaged ducts leaks out, causing secondary, additional, injury to the liver. The main function of bile is to break down fats and oils by acting as a soap, or detergent, in the digestive process. Bile is very inflammatory and causes a lot of scarring to the liver when it leaks from the ducts. The reduced function in these small bile ducts leads to the symptoms, signs and consequences of PBC.

Why do PBC and Autoimmune Hepatitis, or AIH, often overlap?

This is an even more complicated question because we don’t really know the cause of any autoimmune disease. It isn’t as easy to diagnose as something like Hepatitis B, which is a viral infection that you either have or you don’t. These are diseases we diagnose by exclusion, that is we look for common causes of liver disease and if there is nothing else going on, we consider autoimmune disease. PBC and AIH are both autoimmune liver diseases. In both diseases, there is an attack on the liver from the immune system.

There’s actually a fair amount of controversy in the medical community about how often these two diseases truly overlap. I’m certainly on the conservative side of the equation and I think it’s very rare to find true autoimmune hepatitis in people with PBC. It can be complicated to make an

accurate diagnosis because we don’t have a single test for either disease. With PBC, we can use the mitochondrial antibody test but even that is not perfect. It isn’t as easy as just taking a liver biopsy because a liver biopsy is only as reliable as the experience of the person who is looking at it. Someone could have a lot of inflammation if their PBC is aggressive but that does not mean they have AIH. It’s important not to not rush to make a diagnosis and to ensure you have someone who knows a lot about PBC looking at the biopsy to guide the provider. I don’t believe there are as many people with overlap as we currently believe.

That being said, it is absolutely possible for someone who has had PBC for a long time developing what looks like autoimmune hepatitis. We would call this a sequential overlap. But that may not be too surprising. If you are someone with PBC and then you develop celiac disease or thyroid disease, you wouldn’t think it was ridiculous since it’s another autoimmune disease. I believe the trouble arises when doctors rush to make a diagnosis.

What causes liver pain? Is it real?

Our patients with PBC do have a dull ache. In our program, we see about a third of them with this dull ache in their side. It isn’t the sort of pain that would take you to the ER, but it is certainly real. We don’t know exactly why this occurs. But I think what happens is when the bile ducts aren’t working perfectly and the bile is building up in the liver cells, it causes the liver to be slightly enlarged.

Doctors can usually feel this inflammation during a physical examination, but not always. In someone who has a disease, including PBC, the liver is ever so slightly bigger. This subtle increase in size, we call it hepatitis, automatically contributes to that pain. It is safe to treat that pain. Some people get some relief with Tylenol, but it’s not usually a pain that needs any specific intervention and there’s certainly no operation or surgical procedure that would help. That pain is a real pain. It adds to the chronic quality of life burden when there are low level symptoms all the time.

Should everyone see it hepatologist?

North America is an enormous place. Wherever people live, there will be people with PBC. What you want is care that fits, for you. People with PBC should at least see a gastroenterologist. Some of those people should see a hepatologist or a hepatologist with a special interest in PBC. It’s unrealistic to think everyone should see a hepatologist because not everyone lives near one.

Part of the work I do is to develop ways to help gastroenterologists, or even primary care doctors, know how to confidently make a diagnosis of PBC, how to treat a patient, and what to look for to ensure someone is properly responding. We are trying to develop a simple three-pronged tool that looks at your age, bilirubin, and alkaline phosphatase to determine if you are stable or if specialist input is required.

Right now, in 2020, patients with rare diseases can more easily connect with experts, no matter where they live. All the technology we are relying on during this pandemic, remote meetings through zoom, webinars, telephone consultations, will only improve care for people living with PBC. Rarely have the barriers, if ever, been broken down in the way they are right now. Now, people can have their blood tests done locally and, using my webcam, I can give them a lot of good advice without them needing to travel six hours to see me.

Should everyone with PBC consider a live donor?

First, it’s important to note, most people who have PBC won’t need to have a liver transplant. The goal is to diagnose people early enough and see them through the steps of treatment. We start someone on ursodeoxycholic acid (or urso) and expect a treatment response in 6 to 12 months. If they are a non-responder, we can consider obeticholic acid, the other licensed therapy in North America for patients who either don’t respond well enough to urso or are intolerant of it. But, if obeticholic acid isn’t enough, there are other drugs or trials that can be considered.

In a small proportion of people their livers will continue to be damaged and, at some point, they may need to be considered for a liver transplant. Once someone is listed, they can consider identifying a live donor to avoid the difficulty of finding deceased donors. It can be hard to find the right liver when waiting on the list—there aren’t enough donors, the system is imperfect and doesn’t necessarily account for ever disease in the same way.

Live liver transplant has been a lifesaver for many patients with autoimmune liver disease, PBC, PSC, and AIH. It has made a huge difference for these patients but it’s not something you should worry about until the conversation of transplant arises. It is not a race you want to start early. No one wants a liver transplant until they need a liver transplant. It’s a big operation and what we really want is everyone with PBC to receive good treatment and avoid transplant.

What percentage of transplant patients, would you say, has PBC return?

Broadly speaking, I’d say that PBC returns in quite a lot of patients who have had a liver transplant, but it’s only a minority where it’s troublesome. That minority tends to be the people who started their journey young, required a transplant young, and needed to live longer with their transplant. The consequences of PBC, which is normally a slow disease, even in transplant patients, rarely becomes significant. Over the years, we have done enough research to know that everyone who has a transplant for PBC should go back on ursodeoxycholic acid. Try not to worry about recurrence too much because of all the recurrent diseases after a liver transplant, PBC is the least concerning.

When it comes to over-the-counter medications, vitamins, and supplements, are they dangerous for people with PBC?

Most people with PBC should lead normal lives. We try to prevent most people with PBC from ever getting cirrhosis. What that means is that for most people with PBC it’s safe to take most medications, but you still always want to check with your provider. It’s not a good idea to take medicines that are not prescribed or if you don’t know what is in them.

Many medications are perfectly reasonable. Try to avoid Advil. People with PBC sometimes do need Advil because they have arthritis or other injuries. Broadly speaking, if you don’t have cirrhosis, occasional Advil is usually fine. Many common drugs are perfectly safe for people with PBC, including statins, which are very safe. Tylenol is fine. There is no reason you can’t take Tylenol and other similar medicines that you might need for blood pressure, diabetes, etc. Once you have cirrhosis, then, of course, we are more careful with Advil and generally more careful with any medicine.

In hepatology, our goal is look after your liver so well that you have time to develop all the other problems people typically develop. And, when you do, that you can get treated like everyone else. I often tell my patients one of the reasons I never discharge them from my care is because if something happens, I want to be there to tell your provider to treat you normally so you don’t miss out on the best care. Sometimes doctors can be really scared of people with liver disease and feel like they can’t prescribe anything; when this happens, people don’t get the right treatment for something that is quite simple.

Does my initial AMA, the anti-mitochondrial antibodies, ever need to be repeated? Can it be negative and then positive? Or positive and then negative? Why does this happen?

If an AMA is positive then, broadly speaking, you don’t ever need to repeat it. If you have a negative AMA, you can still have normal PBC. Again, I think COVID has taught us a lot—remember, a test is just a test. Every test is different. Some are better than others. Some tests don’t work because of technical reasons. Some tests have a bigger range, and some are more sensitive.

When we see someone who is AMA negative, we repeat the test because we might have a slightly better test. Sometimes it becomes positive and we have the information we want. We try not to rely on just one test. We try to keep things in context.

If you treat someone with PBC who has a positive AMA with ursodeoxycholic acid, the amount of antibody in the blood can go down over time. If someone does happen to repeat your AMA, it can be negative. We even see this in clinical trials—we may get a patient who has PBC, we have a positive AMA from 1995, and you repeat the test to get into the trial and it’s negative. But the clinical trial will still take you because they know you have PBC and that over time the level just went down.

It is a very important, useful test but it is not the only tool. And remember the number related to your AMA has no relevance. You are not going to have more severe PBC because the AMA is a high number.

When I first begin seeing a someone, if their AMA test is on the edge of being positive and the liver tests are only barely elevated, I might wait to make an official diagnosis of PBC. PBC is a lifelong disease. I will follow that patient for a bit and repeat the blood work. Because once you make the diagnosis, you want to make a confident diagnosis, because it means something to that person, and it will influence their care forever. I think getting the diagnosis right at the beginning is so important and makes all the difference. If you believe you have a disease and you understand what you have, you’ll take your medicine, you won’t forget. There’s nothing worse than trying to live with a chronic disease if there are some ifs, ands, or buts.

Does stress make PBC worse?

Well, stress makes everything worse! But yes, people with PBC will generally feel run down. The immune system is affected by external factors but not in a way we can measure. We know that people can get cold sores if they are stressed. I think where stress has the most impact in PBC is in the symptoms.

PBC is a symptomatic disease. Our goal in medicine is to treat the disease and stop the disease progression. But at the same time, we need to work to make the quality of life for our patients as good as possible. That is a very difficult task and not always perfect. People will have fatigue. They are itchy, have dry eyes, joint aches, restless legs, etc. Clearly, if you have stress as well, it’s so much

harder to cope with chronic symptoms. If you have chronic symptoms that are wearing you out, coping with life on top of that is not very helpful.

Broadly, of course, stress has non-specific effects on your health. But in medicine, we are focusing on how that might impact your symptoms. Remember that doctors are not the best at everything. As an individual, it’s important you actively work to avoid stress and secure a good support network. Support can come from more than just doctors, nurses, physician assistants, and nurse practitioners, it can come from patient foundations, your own community, your family, your friends, patient advocates, etc.

One of the things we discovered, and one of the reasons we are so keen on groups like the American Liver Foundation, the PBCers, the Canadian PBC Society, and the PBC Foundation in the UK, is that when we looked at tiredness in people with PBC, we found that one of the factors associated with the most fatigue was social isolation. Social Isolation is suddenly something we have all become aware of as a result of the COVID pandemic. It’s important to tackle social isolation and find ways to connect with people who understand your disease and patient foundations, like the ALF, provide means to do that. Talking about the symptoms and stress, talking about living with a disease, is so important.

What’s an acceptable range for my alkaline phosphatase to be?

This is another complicated question. I’ll start from the latest evidence and work backwards. Broadly, we now say that the lower your alkaline phosphatase the better. People with PBC that get their alkaline phosphatase closest to normal do the best. This doesn’t mean that everybody must have a normal alkaline phosphatase to live a full life. Some of our patients started the disease at age 45 instead of 85 and, for them, we will have a more aggressive goal to have a normal alkaline phosphatase.

Largely speaking, I think most clinicians are looking to try and get a person’s alkaline phosphatase less than 1.5 times the upper limit of normal. The normal is about 120ish. Increasingly, we want it to be less than 200 or 180. Generally, what we are looking for is to avoid you being in the “200 Club,” or 200 and above. If we can do better, say 160-150, or even normal, that’s even better!

What is Hepatic Hydrothorax?

Hepatic hydrothorax is not something that is PBC specific but rather occurs when you’ve got very advanced cirrhosis and your liver is struggling. When you have cirrhosis, pressure builds up, it may cause varices, and your body will begin to retain fluid. Your body thinks that you’re in a desert. The fluid you retain usually stays in your belly. This is called ascites. There are actually tiny holes in our diaphragms. In some people, these holes are a teeny bit bigger. Every time we take a breath in, there’s a change in pressure. In some people, the fluid moves from the belly through these holes and accumulates in the area around the lungs, the pleural space. This is hepatic hydrothorax.

The amount of space around our lungs is much less than in your abdominal area. On the right side, where your liver is, there is even less space. The fluid usually forms on the right side, not the left. One liter of fluid in your lungs could make you very breathless, whereas in the tummy, you can have tens of liters of fluid because the stomach can expand where the lung doesn’t like being squashed.

Do I need to know what stage I am, and how do I find out?

I think the more appropriate question is what is my risk of developing late stage disease? If we can get someone the best treatment, for them, we can avoid late stage disease all together. Much of your general risk can be gathered that information through blood tests to determine how well you are responding to ursodeoxycholic acid or obeticholic acid.

The stage of your liver diseases is also important. It is relevant to your care and how effective drugs are in your treatment. Most people are now staging people using blood tests, ultrasound, and a newer technology called elastography, where we measure liver stiffness. Live stiffness is a sort of marker of scarring, inflammation and bile duct damage. The best time to use elastography is after you’ve been treated with ursodeoxycholic acid or Obeticholic acid, we get a much cleaner picture.

While elastography is what I utilize most, not everyone has access to this technology. Some physicians will still use a liver biopsy to stage people with PBC. It isn’t what I do, and it isn’t what the guidelines say, but there might be reasons a biopsy needs to be done. There might be times for your individual health and your individual clinician’s practice where they look at a liver biopsy to help work out the degree of damage. If that is the case, then, so long as your clinician explains how and why it is going to help you, it can be a reasonable test, but one that warrants conversation.

Of course, if you have advanced stage disease, more fibrosis or scarring, that’s something that you and your doctor need to know. Our focus should never be on just stage as we manage your journey with PBC. Most people are diagnosed so early and, if we focus on the best treatment, you have the best chance of never becoming late stage.

Should I be concerned with high cholesterol with PBC? Should people with PBC take statins?

The current belief is that when you live with liver disease, your cholesterol goes up. This happens for people living with PBC. We know that people with PBC have a characteristic feature of lipid deposits under their eyes. The good news, for really quite interesting reasons, is the cholesterol that goes up in PBC is not usually “bad” cholesterol. It might give you skin deposits but it’s not the sort of cholesterol that causes lipid deposits in your coronary arteries. Because of this, we don’t need to treat everybody with PBC who has high cholesterol.

It’s really important for doctors to know their patient, more than just their cholesterol, and consider all of the different things your physician can measure. It’s important a primary care doctor look at you as a whole person if you do develop high cholesterol. There are risks outside of PBC; it could be related to your family history or maybe you’re a smoker who hasn’t managed to stop. If you didn’t have PBC and that doctor would treat you, then it’s perfectly safe to be treated. If you do need them, it’s perfectly safe to take statins to lower cholesterol. Remember not to worry because it’s not due to the PBC that your cholesterol is elevated, it’s just because, overall, you have risk factors like anybody else.

Now, why do PBC patients seem to generally be vitamin D deficient?

Well, that’s a good question. It’s actually slightly more complicated than just PBC patients. I think half the world is deficient in vitamin D. Vitamin D deficiency is very, very common. We don’t get enough sunlight. But, in someone with mild bile duct disease on top of that, there is a subtle, but significant, impact on most fat-soluble vitamins.

We particularly worry because bone health is so important. Most of our patients are women. Many patients will be post-menopausal. Most of our patients don’t want a bone fracture. You’re less likely to fracture your bones if they are made properly and bones require calcium and vitamin D. We give most patients with PBC vitamin D supplements because it is in their best interest, it’s safe, it’s effective, and you can buy it over the counter.

We will measure someone’s vitamin D level occasionally but not necessarily repeatedly. Nearly everyone with PBC should be on vitamin D anyway.

Do lab results tell you if your PBC is progressing? If not, how do we know that?

When you look after someone with PBC there are a number of things to consider when trying to determine what Is happening to them. First, you talk to them. Second, you look at their blood tests. Third, you might occasionally look at their ultrasound. And the fourth, if you can, you look at their fibroscan. It is by using all that information over time that we can understand whether the disease is progressing. Blood tests can be helpful in determining if your PBC is progressing, but we truly want to pick up disease progression long before it is irreversible, and blood tests can be quite insensitive to that.

Tests like alkaline phosphatase, bilirubin, and platelet count are particularly important. We want to prevent the bilirubin from going up, platelet count from going down, and we use alkaline phosphatase as our flag to see if ursodeoxycholic acid is doing enough or if we need another drug.

Techniques, like combining blood tests with a fibroscan and looking at spleen size through ultrasound can be better for looking for disease progression. When we take a deeper look, we can do something about it before it’s progressed so far that the treatment becomes too hard. We do need blood tests, but it is not enough to consider them without the wider context.

Many of us have itching and fatigue. Is there really a reason?

Itching is not specific to biliary disease. Fatigue is very common in all chronic disease but it’s equally prevalent in patients with bile duct inflammation and autoimmune liver disease. However, we don’t fully understand why people get itchy and fatigued.

The itching, we believe is something within the bile that doesn’t get excreted properly. It doesn’t have to make you jaundiced but it’s substances within the bile which are irritants to smell nerve fibers in your skin. That causes you to scratch. One of the worst symptoms that anyone can have is bad itch.

Fatigue is much more complicated. We believe it is a combination of the disease’s effect on the brain and muscles. This, in combination with other factors like bad sleep hygiene, social isolation, blood pressure medications, or sleep apnea, result in this complex fatigue.

The reason why it has been so hard to treat either of these symptoms is the lack of full understanding. I would say, broadly speaking, as we treat more people with PBC more proactively, with early diagnosis and use of first- and second-line treatments, we can make an impact on symptoms.

There are some very exciting advances coming for itching and PBC in particular. New drugs on the horizon are very much focused on improving itch and, sometimes, even improving PBC. Fatigue

remains much harder, honestly, much, much harder, to treat. We know these symptoms are very important to our patients as they can significantly affect quality of life. It’s proven much more challenging to find the solutions we want but there is ongoing work.

When should I have my family members tested for PBC?

The way I look at this is if you have 20 people with PBC, only one of them will have a family history of someone else in their family having PBC. This average is common. But this also means that if you tested 20 people, only one of them would benefit from that. Broadly speaking, we don’t recommend everyone get their family screened. We tend to take an individual approach where we listen to family history (if there is knowledge of someone else in the family with PBC), look at the severity of the person’s PBC, and the age at which they got PBC. I don’t test the majority of my patient’s families. I would considering testing a family, particularly daughters, if someone was diagnosed very young or someone who told me they had an aunt with PBC.

One of the reasons we don’t test family members is that PBC can be diagnosed at many different ages in someone’s life. We would be very scared to give non-specific testing which might give false reassurance. For example, say I tested a 30-year-old daughter of a 60-year-old woman with PBC, but that daughter wouldn’t develop PBC until they turned 40. I wouldn’t want to tell them at 30 they haven’t got PBC, as if they will never get PBC, just because I tested them once. You have got to be careful about implementing testing which is why it is usually the exception, rather than the rule, that I screen family members.

How does the production of protein correlate to the severity of your illness?

I believe in this question you are referring to what we call albumin, which is the most abundant protein that’s circulating in your blood and is made by the liver. Generally, the liver is a very, very forgiving organ. Really quite clever. I’d say it is better than the kidney. Maybe not as important as the heart but not bad. Actually, it hangs on to until the bitter end. The liver only stops making protein when you have got really bad liver disease. For the majority of our patients, protein production is normal and not something we need to worry about, except for a small minority of patients. Once your albumin levels start to drop, it is a sign that your PBC is more significant and more likely to lead to consequence, including liver failure and liver transplant.

Why do so many people with PBC have joint, foot, and bone pain?

We know that autoimmune diseases are systemic, meaning the whole body is affected. One of the symptoms that is quite common in autoimmune disease are aches. It isn’t only people that have PBC who get these sorts of aches. Additionally, some of our patients will develop very mild forms of arthritis. Sometimes it is so mild that they go to the rheumatologist who can’t find anything wrong. There is probably some mild inflammation in the joints in a lot of people with PBC that doesn’t damage the joints but does impact quality of life. Unfortunately, we don’t really know all the causes for this sort of pain.

What’s the cause of esophageal varices? And how are they treated?

Esophageal varices are the consequence of portal hypertension, which is the technical term we use.

Think about it like this:

Blood goes through the liver. It has to get back to the heart. That’s the job of the vein draining into the liver (the hepatic vein) and the vein draining out of the liver (the portal vein). As the liver scars, that route for the blood becomes impaired. It’s a bit like a river that use to have a really nice, clear flow but then starts to get blockages, tree trunks crossing the river. Scar tissue gets in the way like those tree trunks. But the water still has to flow and will find a new path; it is the same for the blood, it has to keep flowing and will find another way back to the heart. That’s when it opens up these shunt vessels, vessels that were there when you are developing in your mother’s tummy, to carry blood back to the heart by a different route. Some of those vessels are veins in the esophagus, which are thin walled and drain blood onto what’s called the azygos valve which drains back to the heart. The problem with this is that these veins were not designed to drain a lot of blood under high pressure. We worry when varices develop that they might bleed because they are superficial and don’t have a strong wall.

To treat varices, we first need to look for them, which usually means doing an endoscopy. We can either give someone medicine to reduce the pressure build up. Or, sometimes, we have to put elastic bands on the varices because they are visible and may look like as though they will pop. We don’t want that to happen, so we get rid of them by using elastic bands to sort of squeeze and scar them.

There are no symptoms of varices until you bleed. We truly want to avoid that bleeding which is why understanding your stage is relevant to your ongoing care in PBC. You don’t want an endoscopy unless you need one but at the same time, if you do need one you don’t want to miss out. We are not yet at the point where we can diagnose esophageal varices without having quick look with an endoscope.

Can you talk about PBC and COVID-19? What do we know about the number of PBC patients who have COVID?

We don’t know too much. I think what we do know so far, is that it does not look like patients with PBC are more prone to get COVID. This is good news! It doesn’t look like they are more likely to do worse with COVID unless thy are very sick already from their liver disease. We would never want anyone with cirrhosis and jaundice to get sick. We know that if they get any sort of sickness, COVID or otherwise, it’s quite a stress on their body. Where I think we have seen problems have been people who have got advanced liver disease, maybe people waiting for a transplant or patients after transplant who are not back to their full health. If those people have been unlucky enough to get COVID-19, they can run into problems.

It is important to note that most of our patients are women (not all, but most) and COVID seems to be worse in men. There are risk factors for COVID which we really need to focus on more so than a pre-existing autoimmune condition:

– Age, but you can’t change that!

– Weight

– Diabetes

– High blood pressure

Would you recommend weight loss surgery or someone with PBC with a high BMI?

So, that’s a very good question and the answer is, quite pretentiously, I might. The liver is a forgiving organ, but it’s not very happy when it’s got both PBC and fatty liver. But it can be very hard to lose weight. Some of the best treatments for weight loss and diabetes has been bariatric

surgery. I think if you do not have cirrhosis, broadly speaking, weight loss surgery is safe. Even some patients with cirrhosis can consider weight loss surgery. It’s important to make sure that the surgeon doing it is in a high-volume center where they have a lot of experience.

This comes back to why I see patients with relatively mild disease. We, as doctors, should be able to advocate for those people and say, “you know what, this is a more pressing problem than your PBC.” It’s not good being overweight or having diabetes and the most effective treatment for losing weight is bariatric surgery. People who have had bariatric surgery live longer, their diabetes improves, their weight gets better, as does their cholesterol. We don’t stop people from having it. We just like to make sure we understand what stage of disease they’re at and make sure that they’ve been well explained the side effects of the treatment.

What is the current research being done for PBC?’

There’s lots of research going on. I think there’s research going from the laboratory up to clinical trials.

The laboratory research still works on why do people get this disease; what genes do you need to work on? We are part of a big consortium from around the world looking to see if we can find some more genes that associate with PBC. Can we use animal models to map out what happens to PBC, to test new drugs in animals first?

Then you fast-forward to the research that’s going on in a clinic. This research is around drug trials: new drugs; drugs that affect the PPAR pathway; drugs that help symptoms, like ASBT inhibitors; and determining how we use existing drugs, like obeticholic acid. There’s also a lot of work around symptoms: how bad are the symptoms, what can we do about the symptoms, what do patients’ value.

We have very active research and, it’s important to note, we don’t want COVID-19 to get in the way of that. We want our patients to continue to be partners in our research because, although it may seem slow do you as an individual, ultimately it makes a big difference in the long term to a disease. The reason why we have ursodeoxycholic acid is because of research.

Thank you to Intercept Pharmaceuticals for supporting this program.

*Newly renamed to steatotic liver disease

Last updated on January 17th, 2024 at 03:33 pm