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 Whether it is a problem of vanishing results, reliance on unrepeatable or outdated information, or the fact that not all publications are created equally, there seems to be a serious problem with relying on medical publications.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.

Medical Tourism

It's been over a month since my last post. A couple of interesting things have come over the transom but nothing really compelling for me to write about. Also I have been busy in other areas. Something compelling did show up today. An article on Nature Magazine's website concerning the controversy around stem cell medical tourism and the difficulty in regulation. While stem cells offer tremendous potential, the science necessary to fully and safely exploit that potential is still in its infancy. However, because of the potential, the desperation of people afflicted with horrible disease, and the ability to conjure and proliferate medical buzzwords over the Internet, the unwary and uninformed are preyed upon by the immoral and unscrupulous. As can be demonstrated by reading my previous entries, I am a firm believer in stem cell technology. I am anxious to participate in one or more procedures in the hope of regaining function. But the science needs a little more time to develop. Anyone offering or advocating a stem cell "therapy" is either lying or deluded. Several clinical trials are underway or will soon commence (some of which I have previously discussed). But for now, I urge the reader to save your money and preserve your health by not succumbing to the siren song of an exotic "miracle cure".

Stop the Pop

Apoptosis is an important function in multicellular organisms such as people. It keeps cancer at bay and helps keep organs healthy and fresh. But when it occurs in cells that aren't replaced (like motor neurons) nasty things (like ALS) happen. A couple of clinical trials are currently ongoing to support the mitochondria to delay apoptosis (Knopp with dexpramipexole and Trophos with olesoxime). Recently, a study was published which took a rather more aggressive approach. The approach probably isn't appropriate as a therapeutic because it isn't cell-specific. For a good explanation, please read Amber Dance's article over on the ALZ Forum. Ms. Dance is one of my favorite science writers and actually has some credentials to back up her analysis.