With the possibility of Resmetirom being approved soon, as patients, we need to be aware that availability of the drug will still be limited and some months away even if approved.  Beyond that many of us will not be proper candidates for the drug as it likely will be approved for a specific patient population and the response of insurers is still an unknown.  So what might the Madrigal research teach us that could help patients who can't qualify for the treatment?

Akero releases topline results from its phase 2B trial of 96 weeks of efruxifermin (EFX) treatment in patients with pre-cirrhotic MASH to a surprised community.

First looks suggest that Akero sets a new standard for what is possible in the treatment of liver fibrosis.  While any potential drug approval is still years away, the HARMONY trial indicates that NASH/MASH fibrosis will be treatable with drugs.

Last summer I had a medical emergency.  The odds were it would kill me but I survived with few life threatening consequences.  The problems I had are commonly fatal for 80 year old men.  There is a message in my experience which I hope to share with you in this article.  It is a bit longer than most but stay with me.  Understanding consequences is critical to survival and as patients we ignore them at our peril.

A decision on resmetirom is due in a couple of weeks. While we wait anxiously for the FDA to make its decision it may be worthwhile to take a moment and look at some of the rest of the field.

Today marks a significant milestone for Madrigal's NASH/MASH program and a major step forward towards securing the first-ever FDA approved treatment for NASH (MASH).

Hepatic Encephalopathy (HE) -- A stranger lives inside me.

I've talked with many who have experienced the condition but this was my first time. It was very different from what I imagined.  This blog is particularly aimed at care givers and family members who find themselves interacting with someone that they don't recognize.

It is not an uncommon course with this disease in its advanced stages for the patient to become angry, short tempered, and even mean. We see reports from people who no longer recognize their loved one.  The warm, gentle, loving person they have known has somehow become a vengeful tyrant. This makes the job of caring for the needs of that patient very difficult. I can't help much to lighten the burden but perhaps a bit of understanding can make it easier to accept.

Many of our members have asked how I was doing so I thought it was time to provide an update.

A quick review. I was having very painful time with back muscle cramps and had a fall which resulted in 3 compression fractures.  While dealing with that I saw my PCP who identified that I was having a critical calcium event.  My calcium was very high and that can cause a lot of damage to heart and kidneys besides potentially being fatal.  A rush to the ER led to admittance to the hospital where, during the treatment of my calcium problem, they diagnosed me as having multiple myeloma stage 3 (the highest stage) and immediately put me on a chemo program. 

Because of concern about stigma and the increasing knowledge about how fat in the liver leads to disease, the AASLD has officially adopted a new naming convention for our disease.

The new definition is under the umbrella term "STEATOTIC LIVER DISEASE", and what we know as NAFLD is now MASLD.  NASH has been changed to MASH.

We are sponsoring a webinar for patients who want to understand the change this coming week.  I encourage you to attend. This will change the way your physician talks to you about this disease. If you can't attend but would like to view it, we will distribute the recording to all registrants so go ahead and sign up.

CLICK THIS LINK TO REGISTER FOR THE WEBINAR

In my last blog I talked about my fall.  When I fell I did quite a bit of damage and have 3 compression fractures to show for it. I've been surprised by the number of people who have commented about falling and most have had no problems and dismissed the event. Perhaps its time we took a closer look.

This is an MRI of my back after the fall.

In my last blog I talked about falling and how bad that was for old folks.  I mention that frequently in my communication with members of the foundation and I was disgusted that I tripped at night and had a hard fall.

My hepatologist and every other doc always emphasized don't fall. I fell.

Last week, In the middle of a midnight bathroom run, I tripped and fell.

Not a terrible fall as nothing broken and no head impact but a big setback in my recovery from the back spasm.  NASH patients tend to become riskier to falls as the disease progresses. That one of the reasons for keeping an exercise routine.

Did you ever feel defeated by liver disease? Sarah Larsen decided to fight back.  We are giving her a special blog space where she will document her training journey from patient to athlete training to compete in the World Senior Games.

People who promote something, like politicians or health advocates, and then don't practice what they preach get my goat.

Madrigal Pharmaceuticals has released news that it has completed its filing for approval by FDA of its NASH drug candidate Resmetirom.

If you would like to read the official announcement, here is a link to it

Madrigal Pharmaceuticals Completes Submission of New Drug Application Seeking Accelerated Approval of Resmetirom for the Treatment of NASH with Liver Fibrosis | Madrigal Pharmaceuticals.

There are hinge points in society which alter things in ways that fundamentally change how society works.  The domestication of fire may have been the first. The development of projectile weapons and the efforts of the first farmers are examples.  They can be large structural change like the development of the city/state or a knowledge engine like the invention of the printing press. They may be instantaneous like the explosion of the first atomic bomb or aspirational like the Magna Carta, but in all cases society changes as a result.

Join us for a webinar about AI and machine learning.

About a month ago, I shared some of my thoughts on the recent hysterics over AI. We know that panic and fear sells, so the media often plays on these emotions to garner more exposure and views. Unfortunately, these stories often fail to provide us with any meaningful information that might alleviate our concerns and instead leave us feeling more panicked and afraid than before we turned on the TV.

The makers of the first NASH drug that has met the goals set by FDA, Ocaliva by Intercept, have once again been told that if they spend another billion dollars they can apply again. Intercept is beaten and will withdraw.

My doctor divorced me. A new kind of care is being forced upon us.

It is an old story.  They think we don't know, but we know. We get the little messages, "I wonder if we should see other people". We hear the office chatter, "Did you see the big ones that just came in"? "Sure a lot of newbies here lately." "There sure are a lot of young ones."

We're here to mark a time in history when the Allied armies of science and medicine joined in battle to defeat the silent scourge of NAFLD/NASH. For decades, much of humanity has been under a terrible shadow. Countless faceless victims mourned only by their families have fallen to NASH, a disease of modern society. As the the medical world became aware of the killer stalking their patients the word went out from a few pioneers seeking answers. Even though the early researchers were largely ignored and the spotlight fell on the stars of heart and cancer they persevered.

As I reported earlier, this was the outcome of the FDA meeting on the first drug candidate to successfully meet the FDA's requirement of reducing fibrosis by at least one stage.

The FDA advisory panel voted to reject Intercept's application for OCA as a treatment for NASH

Despite meeting the goals set by FDA, of reducing fibrosis by at least one stage, the GIDAC panel today voted against approval of obeticholic acid as a treatment for NASH.