Monday, August 12, 2013

Brigades of Oily Minions

Today's post will be a collection of miscellaneous stuff related to amyloidosis that I have run across in the past couple of months. The first item is important. The others, not so much.


CPHPC Clinical Trial Now Recruiting (in the UK)


A new clinical trial to study the effectiveness of CPHPC is officially recruiting participants in the UK as of June 28, 2013. Here is the link on the ClinicalTrials.gov web site:
http://clinicaltrials.gov/ct2/show/NCT01777243?term=NCT01777243


The title of this clinical trial is "A Study to Evaluate the Safety of GSK2398852 When Co-administered With GSK2315698 in Patients With Systemic Amyloidosis." Although the acronym "CPHPC" is not listed in the title, it is in the keywords at the bottom of that link. The sponsor of the trial is GlaxoSmithKline, hence the GSK numbers in the title of the study.

This trial is good news for fibrinogen amyloidosis patients, since it is for all types of systemic amyloidosis, not just AL amyloidosis. Previous testing in patients with fibrinogen amyloidosis has been promising. If you are interested and live in the UK (or if you want to move there), check out the details at the link above and see if you meet the inclusion criteria.


Grow a Spare Liver?

A July 3, 2013 article from MedlinePlus has the title "Scientists create human liver from stem cells." First I need to state that the stem cells used in this research were not embryonic stem cells, whose use is controversial (at least in the US.) There are two main forms of stem cells, embryonic (harvested from embryos) and induced pluripotent stem cells, which are taken from a person's skin or blood. Induced pluripotent stem cells (iPS cells) are the type that are harvested from AL amyloidosis patients for use in stem cell transplants.

The very short version of the article is that a team of scientists in Japan used some iPS cells to grow liver-like structures called liver buds, which were then transplanted into mice where they matured, connected to the blood vessels of the mice, and began performing many functions of a mature liver.

This is of course a long way from growing an entire human liver, but it's a start. The first practical use of this technique will likely be to study the effect of potential new drugs on the human liver. Then they might be able to grow mini-livers from the stem cells of people with liver disease, and transplant those mini-livers into those patients to take over some liver functions of the failing liver. Growing an entire liver to replace an existing liver would likely be more challenging if it is even possible, but who knows?



More Animal Amyloidosis

In the book "XIth International Symposium on Amyloidosis," which was published in 2008 and reported on the November 2006 amyloidosis symposium, I ran across this article in the section on familial amyloidosis: "Systemic amyloidosis of fibrinogen origin in the wild living stone marten (martes foina)." A marten is a small mammal in the same subfamily as weasels, ferrets and minks. Those of you with good memories may recall a previous post in which I mentioned an article titled "Familial Renal Amyloidosis in Abyssinian Cats." This article on the stone marten went into the details of how they determined that the origin of the amyloidosis in these animals was the fibrinogen alpha chain, but they did not determine whether or not it was hereditary. In any case, those of us who have tested positive for the fibrinogen amyloidosis mutation now have some more trivia to amaze and impress our friends.


Surprising Search Results

Last month someone found this blog while searching for the phrase "lean forward shampoo." I assume this person was looking for articles about the benefits of leaning forward instead of backward over a sink while getting a shampoo in a salon, when one of the hits was my July 10 post about our week on the cruise ship where I had to take Mom from the salon to the ship's medical facility because her lungs were filling up with fluid. The Internet is just full of surprises, isn't it?



A Rag Man

In case you have not figured it out yet, the title of today's post is another anagram of "fibrinogen amyloidosis." The Internet is just full of surprises, isn't it?

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