I recently wrote about my view that for the first time a cirrhosis patient could look forward to real medical therapies. A few folks felt that I was planting false hopes and that such miracles weren't going to happen. In light of that, I thought I might provide a broader view of the situation today.
Some of you are old enough to remember that not so long ago Hep C was unknown. We called the illness non A non B hepatitis. Today we have a cure. A miracle perhaps but also a lesson.
I spent my life as an engineer and I guess that gives me a somewhat different perspective than those in the health sciences. That relates to this discussion if we step back and consider what the difference is between science and engineering.
We live is a magical time for the scientists. We have powers undreamed of a generation ago. For example, you might watch a few videos about some of the many new tools recently available to researchers and consider what it means to have tools like this.
SEEING THE UN-SEEABLE FROM THE DISCOVERY CHANNEL
From living cells to the atoms themselves
These are certainly interesting you say but what does that have to do with a treatment for my NASH? That is the question. Was it fair for me to say that actual therapies are on the horizon?
Discovery isn't made in a vacuum. You don't just find truth one day in your laundry. Progress is built brick by brick upon the efforts of those who have gone before, and most importantly it depends upon the tools available to do the work. This is where the line between science and engineering exists.
Science is the discovery of things that are unknown. It is the probing at the boundaries of knowledge, imagining what might explain some unanswered question and proposing how to prove or disprove that idea. Usually the quest began because of some mystery or anomaly exposed by the current generation of tools. Answering this new question becomes the realm of the engineer because new machines or ways of solving problems must be developed in order to probe the workings that produced the question.
OK, a bit theoretical here, but what does that have to do with therapies for liver disease? I'm glad you asked. The solutions to great problems go step by step. In the beginning there are so many unknowns you are just searching for any kind of unifying idea. As you learn about the details the future possibilities gradually become more clear. At some point you learn enough that you can say with confidence "This is a solvable problem". You may know for certain that you don't have the answer yet, but you do know that there is one and that you can find it.
That is where we are with diseases like NASH and cirrhosis. Our tools have advanced to the point that we can watch live cells operate in real time. we can see to the very fundamental core of the processes that they do. Even though it is mind bendingly complex we know that we can understand and manipulate the chemistry of those cells.
We are now doing the engineering, the construction of a solution and we are quickly kicking the scientists to get out of the way and find new frontiers to explore because our tools and knowledge are advanced enough to build solutions, not to just look for relationships. That is why I am not speaking of miracles when I say that we can see therapies on the horizon. The timeline is murky but not impossible to glimpse anymore. When a problem becomes that of exactly how do we do this? Instead of can it be done at all? We have crossed an important threshold and in medicine that battle has changed in just the last few years. The message then is this
If you can force your heart and nerve and sinew
to do their turn long after they are gone,
and so hold on when there is nothing in you
except the will that says to them hold on.
Yours is the liver that can endure
and what is more, survive to a cure.
Apologies to Kipling, our best to you and yours. Remember diet is critical to your liver. In case you forgot, here is a place to start again.
This is an excellent series of videos from NASH day. The Foundation was a co-sponsor of this effort . If you would like to know more about it from world experts this series of 7 videos is an excellent place to start.
The picture was taken June 2018. My hubby and I are about to be 55. It’s hard to believe that in the past 11 months I was diagnosed with cirrhosis, diagnosed with HCC, had 3 surgeries and about to have a 4th, been admitted 4 times to hospital for non surgical reasons and in the ER 17 times. I’ve lost count of the number of CT scans, MRIs, ultrasounds, and other diagnostic Radiology tools. I don’t remember all my doctors names or even what their specialty is. I’ve gone from not being able to walk to the toilet from my bed to hiking to see a waterfall. I’m alive.
When I was first diagnosed, my doctor told me his goal was to get me to die of an old age other than Cirrhosis complications. I was severely decompensated, leaking 2-3 liters a day of ascites from a surgical incision, and didn’t have a clue. I was scared to death because of all the things I had found on Dr Google. I was ready to give up.
One of the things he really emphasized was to not pay attention to any statistics. There were things I needed to do to help my body heal...but he couldn’t do it without me really jumping in and owning what needed to happen. I had to cut my sodium intake to less than 2000mgs per day. I needed to cut out processed foods, focus on lean protein and plant based protein. Even though I don’t drink, no celebration drinks for anything. I needed to increase my activity in whatever way I could...even if it was lifting a soup can 10 times with each hand. He also asked me to listen to what he said and not be afraid to ask questions.
It’s been a year since diagnosis. Yes, there are still things I have to deal with every day...some days more than others. And this disease is never far from my mind. Every thing I do or don’t do impacts it. I do constantly have a running calendar in my head of my doctors appointments and test dates. Due to being diagnosed with HCC in May and had an Ablation last week, everything is slightly increased in speed. I see my Hepatologist every three months and have blood work and CT scans every three months. Every day my breakfast appetizer is a handful of pills that keep the symptoms at bay.
It doesn’t matter what the cause of your Cirrhosis is. Mine is from NASH...but it could also be left over damage from being a hardheaded college kid who liked to party too much 35 years ago...or from being a teenager who liked “speed” a bit too much to get through what life had dealt me. We all have a past. Sometimes it’s yesterday. Sometimes it’s 50 years ago. Our past doesn’t define us.
You matter. Your story matters...even if you’re not ready to share it...it’s still important.
My story isn’t done yet. It’s being written everyday. Someday, not soon, there will be a finishing period to my story. But not yet. I’m going to live! How about you?
The National Fatty Liver Registry (NFLR) is in its design stage. As outlined in our Screening Project introduction, there is a great need for identifying patients at risk and with liver disease. Our goal is to build the largest database of tests for liver disease in the world. At scale and full deployment, our target is one million tests per year, capturing valuable data for epidemiological and interventional research.
The fundamental goal of the Foundation is to alert the individual patient to the risks they may have of developing liver disease as a result of lifestyle issues. The Foundation is also commited to building a research resource which will deliver lasting benefit to society at large and to the research community.
The Foundation is taking a proactive, leading sponsorship role in the development of the NFLR. We are working with leading organizations and partners to design and implement this strategic project. If you have interest in being part of the effort, contact us for more information and how you can help.
I just returned from a conference called the NASH Summit. It is a gathering of about 200 of the top liver researchers and scientists in the world. Small but very much cutting edge. I must say that as a cirrhosis patient I am so encouraged, perhaps verging on rapture, at the progress being made to develop treatments for liver disease. (I'll get some guff for that kind of language but understand that as patients we know there is no medical help for us today)
I go to these meetings and I am always so encouraged by what I see there. This was the first conference where we have presented a poster of our progress which was fun. We usually are audience not part of the show. Here is a link if you would like to see it.
This was our first real opportunity to announce that Intercept has awarded us a grant to begin the first phase of our screening program aimed at finding people with liver disease before they have symptoms. Here is a link to information about that plan.
All good. Indeed, but coming back to the "real" world is kind of like a hangover. The problem is that the drug trials that are required for treatments to be proven can't happen in the world as it exists today. Even though liver disease is epidemic and threatens the lives of millions of people no one knows who they are. The number of active patients who can be considered for participation in a clinical trial is tiny compared to the need. So as someone who appreciates research I am so pleased, but as a patient I know that any treatment will be delayed by years because of the clinical trial problem. I try not to be angry about it though I fail at times.
The problem is that even though we know that there are millions of people who are slowly killing their livers, because it rarely has symptoms, we wait until it becomes a life threatening issue to act. It is standard practice not to screen for liver disease even when we know statistically that someone is in danger. When we didn't have the ability to screen that made sense, but now we do and that gets my goat. It is what led to our championing of our screening program but we will need a lot of help to make a difference in any reasonable time frame. Maybe we'll get it so stay tuned.
One quick example from the conference to show you just how bad it is. Research has shown that up to 70% of people with type 2 diabetes have undiagnosed liver disease. That is a shocking number since there are around 30 million of those folks. A company that does clinical trials reported that they had 35,000 patients in their database. They asked how many had diabetes and it was 18,000. So how many also had a diagnosis of NASH. What would you guess that number would be? Thousands surely given what we know of the disease. The answer 219. TWO HUNDRED NINETEEN!!!!! I knew it was a bad situation but that really depressed me. That is the real world today. A very large amount of disease cooking along and no effective outreach so no timely clinical trials and death by end stage liver disease. Interesting statistically unless you happen to get on the wrong side of the line.
Enough for now. Split brain doesn't help. I'm headed to Houston this week which is where we plan to put our pilot screening center so baby steps for now. I'll let you know how it goes.
An ironic curse with the clear implication that 'uninteresting times', of peace and tranquillity, are more life-enhancing than interesting ones. Another cautionary message is 'be careful what you wish for', and yet another 'fools rush in where wise men never go'.
Cliche man is here apparently, but the old warnings aren't necessarily wrong. I want to let you know that the foundation has entered an 'interesting time'
We have been greatly honored with support and now it is time for us to stop talking and start dancing. Intercept Pharmaceuticals has agreed to provide the first funding for our screening program and we plan to open our first pilot facility in Houston in the summer. For those who have joined us recently, we advocate building 400 screening centers across the US and to screen 1 million people a year who are at risk for liver disease from the large co-morbid (people with multiple diseases) population.
We have also been invited to present a poster at the 2018 NASH-Summit later this month. This is a gathering of world leaders in liver disease research. At conferences like this industry leaders present their latest results and give talks about where things are headed but they also have an area where promising results can be presented in poster form. Sort of like being the warm up act for the Beatles. This will be our first experience presenting a poster so that will be 'interesting" but it is an honor. Space is always limited and there are thousands of people working in the field so we were pleased to be included.
We have formed a partnership with HealthUnlocked to provide the digital platform for our online community called
Check it out. The thing that sets this apart as a patient tool compared to platforms like FaceBook is that it is ad free. We do not flood you with constant junk that just wastes your time. If you are in a forum you are likely ill and don't need to have your life energy sucked at by marketing pukes. Some day I'll tell you how I feel about that.
In association with Intercept we are also producing some patient videos and a media campaign to help educate people about liver disease and things they need to know about the system. A major problem in society is that people just don't understand liver disease, its causes, consequences, or management. A central mission of the foundation is to try to improve that.
We hope you are well. If you think others might be interested in any of this please feel free to forward this, and if you like what we are doing any financial support you can send along will be greatly appreciate.
When I think about screening for liver disease I often find that tune from My Fair Lady, "Why Can't A Woman Be More Like A Man" running through my mind.
It is an odd mental tick I suppose. One of my favorite musicals connecting to a potentially terminal illness, but the challenge we face as liver patients would largely vanish if only a liver was more like a breast.
OK, I stretch the analogy a bit here but consider how cancer is managed. We search diligently for cancer and while there are significant differences between cirrhosis and breast cancer the statistics are interesting. There are around 40,000 deaths annually from each disease, but we search out the tiniest incidence of breast cancer that we can find and manage it aggressively but we ignore liver disease until it presents serious symptoms. Think about that for just a moment. Why would we test breasts regularly but intentionally ignore early liver disease?
The answer is that we don't have any pills and the treatment is dietary and lifestyle counseling which we suck at and have largely failed to do well. We see that from the obesity challenges our society is facing. Beyond that, historically we didn't have any useful and inexpensive tests to really measure the disease. Plenty of ways to explain the past, but today we do understand the bio-chemisty better and we do have ways to test for disease that hasn't caused symptoms yet.
One might think that we would be rushing forward with screening but we aren't. Among the reasons are that insurance doesn't really want to pay for wellness testing and it isn't the business model for medicine. There are efforts to change to a wellness model but what we have is a very entrenched system. We also still are faced with the fact that the best we have to offer is advice about how to eat better and the diet industry is a great example of a failure as people routinely yoyo up and down with their weight.
The Foundation believes that we should be screening the at risk liver population now and working on education about food because in the long run that will still be the best therapy even after they develop expensive pills. To that end we are sponsoring a screening project designed to make low cost testing available and to provide early warning to people before they become ill. If the subject is of interest here is a link to it.
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