CD40L and the MAB

The ALS Therapy Development Institute is a non-profit biotech fully devoted to finding a treatment for ALS. A few years ago they undertook a genome-wide association study (GWAS) on the animal model of ALS (the G93A mouse).The results implicated the costimulatory pathway which links the innate and adaptive immune systems. In an attempt to address that pathway, TDI used a monoclonal antibody against CD40L. This decreased a specific T-Cell interaction which led to the body attacking the axons of the motor neurons. A very good description of the study can be found at the Alzheimer Research Forum (Alzheimers, Parkinsons, and ALS are selective neurodegenerative diseases so research in one can provide clues into the pathways of others). This finding is interesting for a few reasons. For the first, it marks a shift in looking at ALS as a motor neuron disease to seeing it as some other dysfunction in which the neurons are injured. Second, while the treatment described isn't a "cure", it does point towards a significant pathway of disease management. Another important point is the success reported by TDI. Since the development of the murine model of ALS numerous researchers have claimed varying degrees of success in preclinical work only to have the candidate substance fail in human trials. TDI undertook an internal study to figure out why and discovered numerous variabilities for which researchers must account when designing trials. TDI's mouse program is now recognized as the "gold standard" for that model. The fact that they can report measurable and repeatable results is significant. Of course mice aren't men and this isn't a cure, but it does give quite a bit of hope that a truly effective treatment may be within reach. [UPDATE 04-02-2010: TDI discusses this via MP3 podcast.] [UPDATE 04-05-2010: The study is now available free of charge in PDF format. Very good of Nature Publishing Group to make this available to PALS and CALS!

Stem Cells for Dummies

Stem Cells for Dummies is authored by Lawrence S.B. Goldstein, PhD. Dr. Goldstein also co-authored the legislation which created CIRM. The book does, IMHO, a rather good job of presenting complex science in terms which laypeople can easily digest. It starts with a primer on biology as it applies to stem cells and a brief history of their discovery and initial uses. The differences between adult and embryonic cells is explored with frank discussion of the attributes and limitations. The role of these cells in the research and treatment of various diseases is discussed along with a review of the current state of the art (my previous post on stem cells deals with some cutting-edge techniques). There is a detailed section on the ethical and moral controversies surrounding this sensitive topic, and a section on common myths and misconceptions. Stem Cells for Dummies is written from the scientist's viewpoint but takes effort to present in a neutral tone. It is written for anyone who wants to understand stem cell technology without requiring a PhD. Rather than spend weeks cuddling with Google and sifting through the inevitable dreck, drop $10 and receive a great resource from a recognized expert in either dead-tree or electronic form. [disclaimer: I linked to Amazon for convenience and because I use their Kindle for PC application. If you can, support your local bookstores.]

Sausage-fest!

Interesting things happen to one's body due to ALS. One of the most trivial effects I have noticed involves my fingers. I am quadriplegic so my hands spend all their time on a (relatively) flat surface. Grasping or any voluntary flexing of my fingers has been nothing but a memory for years. This allows the skin to tighten and smooth a bit. You know those little wrinkles over your knuckles on each finger? I don't have any anymore! I noticed this during physical therapy a few weeks ago and wondered what these little keratin-tipped bony alien hotdogs were doing at the ends of my hands. Truly a sausage-fest.

Letter from Lou

I recently received a handwritten letter from Lou Gehrig. Obviously it was not addressed to me as the people responsible for creating me had not yet themselves been created. But it does give an intimate glimpse into the life of a remarkable man (even without the disease his accomplishments are legendary). I was struck by the proof of his equally legendary kindness and generosity, and hope to approach that in my own life as I struggle with fellow PALS to achieve the goal that eluded Lou.

Kindle for PC

Back in November of 2009, Amazon released its Kindle for PC software. The program is free and available for Windows 7, XP, and Vista. A future version for Mac is promised. While I have never used a physical Kindle (holding anything but my breath is no longer an option for me), the software appears to recapitulate most of the function of the device. The most important part for me (and for all advanced-stage PALS) is that it behaves very well with my optically-controlled assisted communication system. I use the ERICA from ERT. ERT was recently purchased by Dynavox but the K4PC should still work on that platform as well as the Tobii. Downloading and installing is very simple. Once installed you need to register the "device" to your Amazon account (like the physical Kindle each registered software counts as one of the 5 devices on which you are allowed to share your purchases). If you don't have an Amazon account you will need to set one up (I recommend letting an assistant do the data entry here although I was able to do this myself). For those who already have a Kindle, you should see your previous purchases sync to the software. If this is your only device, new purchases from Amazon will appear within 30 seconds of purchase. Each page has large, easy to read type and the size and spacing can be adjusted for comfort. To either side of the page are white arrows to turn the page. They only appear on mouse-over, and if you click even near them you will hit the target. They are spaced far enough away from the text that accidental page turn is almost impossible. If you close the application with a book open your place is saved automagically. Other bookmarks are easily set. The software is considered beta at the time of writing this post but works very well for me. Updates are automatic but can be set to manual. For software not expressly written for handicapped users it satisfies some important requirements. I am very happy to be reading books again!