Taze Me Bro!

Rob Goldstein of the ALS Therapy Development Institute (ALS-TDI) talks to Dr. Seward Rutkove about his invention of device using Electrical Impedance Myography, which recently won the $1,000,000.00 Prize4Life award for biomarker development for ALS. This is available as an mp3 stream. Very interesting discussion and well worth a listen.

Press Release

This is a press release I created to help us get our story of struggle out to the media. Please cut and paste the text below and put your own contact information in the Contact: line then send to your local media with a cover letter explaining your own struggle and advocacy activities. With enough synchronous distributed local coverage we can generate national interest. If you can't cut and paste then email me and I will send you a copy. YOU are the story. But together we can create the change we demand. --------(cut and paste below)------------ People with ALS Changing Their Own World Contact: Much like the people in the Middle East, people here at home who are subject to another kind of tyranny are using social media such as Facebook, Twitter and Youtube to organize and spread the word about their condition. These people are known as PALS, or Person(s) with ALS. ALS, commonly known as Lou Gehrig's Disease after the famous baseball player who died from it, is a progressive and incurable deterioration of the nerves controlling voluntary muscle movement. This leaves the person totally paralyzed and eventually unable to even breathe. Because the expected lifespan from diagnosis rarely exceeds five years, the population of living PALS is low and those living aren't able to make themselves into public figures. Until now. With the advent of social media, as well as the technology of mobile computers with eye-tracking input systems and text-to-speech synthesis, PALS are able to compete on an even footing in cyberspace with more physically capable people. Many of these PALS are in serious medical conditions such as near total paralysis and some on mechanical ventilation via tracheotomy. As the astrophysicist and PALS Stephen Hawking said, "My body may be crippled but my mind is free." Unsatisfied with the level of advocacy and awareness generated by organizations with that as their claimed mission, PALS are doing it themselves and placing pressure on those representative organizations to change old operating procedures. Other organizations representing other medical conditions are very vocal and aggressive in public awareness (the foundation of funding and national priority for research) and this new group of PALS demand commensurate action from the organizations representing them. Examples of the new paradigm are: • an emergency Facebook movement to transfer one PALS from an abusive institutional care facility to an entirely new fully automated care facility (which was designed by another PALS who was also the first resident) http://www.govostes.com/blog/?page_id=172 http://www.youtube.com/results?search_query=saling+als+residence+&aq=f • the "Often Awesome" serial documentary of a PALS' life with ALS from diagnosis to current time http://www.youtube.com/results?search_query=often+awesome+&aq=f • various Facebook groups including a direct petition to the ALS Association http://www.facebook.com/pages/Petition-Against-ALSA-National/139603146055424?ref=ts • a movement to draft a Hollywood star as a spokesman https://www.facebook.com/alsspokesman • use of the popular video creation site xtranormal.com to craft and distribute their own Public Service Announcement http://www.youtube.com/watch?v=NgNVvHbIIiI Facebook as a corporation is aware of this group of PALS and recently invited a few to its Palo Alto, CA, headquarters to take part in a documentary being aired in March, 2011, on MTV. The documentary will be about how various groups have used Facebook to effect change.

Public Service Announcement

This is a little video I put together. It was a bit of work to do with my optical tracking system. I need your help getting it distributed so please share via Facebook, Twitter, email, whatever. Some may wonder why I didn't mention the ALS Association (ALSA). ALSA to this date refuses to mount a national PSA campaign like other more successful organizations, so I did my own. The two organizations I did mention I feel are more effective. But this is really about awareness so please help me and many others get the word out. Thanks!

Be Yourself

"I'm nobody! Who are you?" The concept of "self" is critically important in the immune system. Each of our cells present a unique set of molecules on their outer walls which signal to our wandering immunity police that they belong ("your papers, please"). Any other cells not presenting the same combination are considered foreign invaders and are attacked ("your papers are not in order!"). This works great for resisting infection but is a major impediment to transplant surgery. Even "tissue matching" is inexact, requiring recipients to have their immune system forever after repressed in order to maintain the graft. Finding a way to allow for a graft of desirable foreign tissue but also maintain a robust immune system is a therefore a major goal. A stem cell research grant by theCalifornia Institute of Regenerative Medicine has a quite novel approach: regenerating the thymus, the "police academy" where the immunity cells learn to recognize the body's own cells from foreign invaders. The thymus atrophies in adulthood, so regenerating it from stem cells from an appropriate HLA line might trick the immune system into accepting the graft with the same HLA type. Quite a clever approach, and yet another reason that I am glad to have voted in favor of forming CIRM (ironically shortly before I was diagnosed with ALS).

Apollo 13

"Houston, we have a problem." Apparently you can't believe everything you read. Whether it is a problem of vanishing results, reliance on unrepeatable or outdated information, or the fact that not all publications are created equally, there seems to be a serious problem with relying on medical publications. While research papers provide valuable clues, only after those results are repeatedly verified can they reach the status of fact. And even that could change over time as the sample set grows beyond what is practical for a single study. It seems that even rigorous science can fall prey to the human failing that people see what they want to see. This results in selective reporting as well as selective publication (rarely do negative results get submitted). And in the pharmaceutical industry there is the ever-present threat of monetary corruption. Note that this should not create a panic of Nihilism, but rather instill healthy skepticism and diminish false hope.

Axon-Axoff

Why do axons go downhill? Gravity has nothing to do with it... The linked study, though not specifically aimed at ALS, does seem to tie together a few "hot topics" in the pathology: axonopathy, oxidative damage, and mitochondrial dysfunction. With many plausible theories of damage, trying to link some together may provide more clues as to cause and/or (more importantly) ways to effectively treat the disease. Part of the tie to ALS in this study is the fact that mice bred to lack the SOD1 gene exhibit a similar disease process as mice bred with a copy of the malformed SOD1 gene which is a known cause of familial ALS. Reinserting the gene in the "knockout" mice, but only in the mitochondria, rescued the mice from disease. Mitochondria are the power plants of the cells and as a consequence also produce molecules which can cause cellular damage (ROS, the "industrial waste" of cellular energy production). Anything which disrupts the ability to clear out this waste can cause damage to the cell. Damage to mitochondria impacts their ability to provide energy to the cell. Motor neurons have high energy requirements and due to their extremely long axons have mitochondria operating at large distance from the cell body. There are currently a few clinical trials going into Phase III aimed at mitochondrial support. While the study doesn't provide an answer (except that this problem is "way beyond eating blueberries"), it does provide some important clues for further investigation.

More Baby Steps

In June I posted a discussion about a prospective regenerative therapy. On December 1, I received a press release that the company developing this therapy has filed an IND with the FDA. This is a huge step forward for people suffering from motor neuron disease (especially late-stage PALS like myself). However, it is but the first baby step in the clinical trial journey. Let's hope that the FDA acts swiftly and positively on this IND.