March 2, 2015 1:13 PM MST
Interestingly, the new drug coming down the pike to “treat” OPMD is a derivative of sugar!
The following information was gathered and assembled for this post by yours truly, and is not in any way associated with an “official” description of the drug itself. Obviously, there is a lot of research and a chemical process that goes with its creation and production; its not as simple as a teaspoon of sugar, lol.
After a little digging around online, however, I found the following information about the basic chemical platform for Cabaletta on Wikipedia. While a few sentences in the following description of trehalose are a bit complex, it is easy to understand how trehalose is produced in nature and has not caused any side effects in the clinical trials.
I was fascinated to read that the drug is based on trehalose, which is a disaccharide. A disaccaride is a sugar (a carbohydrate) composed of two monosaccharides. It is formed when two sugars are joined together and a molecule of water is removed. For example, milk sugar (lactose) is made from glucose and galactose whereas cane sugar (sucrose) is made from glucose and fructose.
Trehalose, also known as mycose or tremalose, is a natural alpha-linked disaccharide formed by an α,α-1,1-glucoside bond between two α-glucose units. In 1832, H.A.L. Wiggers discovered trehalose in an ergot of rye, and in 1859 Marcellin Berthelot isolated it from trehala manna, a substance made by weevils, and named it trehalose. It can be synthesised by bacteria, fungi, plants, and invertebrate animals. It is implicated in anhydrobiosis — the ability of plants and animals to withstand prolonged periods of desiccation. It has high water retention capabilities, and is used in food and cosmetics. The sugar is thought to form a gel phase as cells dehydrate, which prevents disruption of internal cell organelles, by effectively splinting them in position. Rehydration then allows normal cellular activity to be resumed without the major, lethal damage that would normally follow a dehydration/rehydration cycle. Trehalose has the added advantage of being an antioxidant.
Extracting trehalose was once a difficult and costly process, but circa the year 2000, the Hayashibara company (Okayama, Japan) confirmed an inexpensive extraction technology from starch for mass production.
Trehalose is currently being used for a broad spectrum of applications.
March 6, 2015 1:27 PM MST
Here is a question for you; would you be willing to try this new med when it becomes available?
September 17, 2015 1:10 PM MDT
On May 2014 I had an email from Bioblast and I am now on their files. Going to Clinical Trials you can see that the next step will end next year.
May 2, 2016 3:33 PM MDT
I recently did a webcam interview with a research group that is working with BioBlast. I did tell them that I would be willing to consider the medication if it slowed the progression of leg muscle weakness symptoms and, of course, improved these symptoms. I can’t remember if they mentioned the symptoms of Ptosis and swallowing problems. They were referring to product “X” as a drug that would be administered via IV for an hour once per week. This could be done by a visiting nurse, according to them. It will be interesting to see what really comes of this pharmacological therapy.