Ch-Ch-Ch-Changes...

This is my last blog post at Friends4Eric.

I have decided to change my home domain to EricValor.org and in the process consolidate the blog directly into the website. The new website allows me to bring a much better website experience to you. Over the year, I expect to add more information content, including a calendar of events such as a calendar of select events and other information of high interest.

Thew new website address is www.ericvalor.org and the direct address to the blog is www.ericvalor.org/erics-blog. I believe I was able to bring all the subscribers over automatically but please excuse any errors and/or hiccups. Although my old domain will redirect to the new, this blog address won't, so please update your bookmarks!

Thank you all for your and I look forward to a few more years of service to you all - until we finally have a cure!

- ENV

Hope Now for ALS

There has been a lot of recent controversy surrounding the experimental treatment in clinical trials known as GM604. A lot of misinformation has been tossed around on both sides of the issue. I want to beg your attention for a little while to explain what's really going on.

First, as many of you know, I was the single late-stage PALS who received GM6 in compassionate use. The intent behind this was to get a look at differences in biomarker candidate levels between earlier-stage and late-stage PALS. Any outward physical manifestation of improved condition noticed would be a bonus and, due to my advanced condition, no improvement in condition was expected. Nevertheless, Genervon and I came up with the idea to try to chart improvements in the tongue. The rationale is that since my tongue is only moderately affected, and because the hypoglossal nerve contains one of the shortest motor neurons in the body, any possible improvement would be noticed there first.

Because of my growing cooperative relationship with Genervon dating back to my first blog post on GM6, I was granted the only Expanded Access outside of trial. They were interested in getting a look at GM6 behavior in late-stage PALS and I had proven to them my organizational skills in preparing my own medical surveillance team and in communications by preparing the mechanism for data capture. Even a single Expanded Access Program can be a burden on such a small company not optimized for such work. My knowledge and experience gained over the past few years was of considerable help in filling out and transmitting (and following up on) my own paperwork.

Thousands of single Expanded Access requests would be overly burdensome and even if Genervon enlisted the help of the ALS Emergency Treatment Fund, the maximum number of patients who would be able to participate would be measured in the few hundreds. Not only would patients have to pay for drug but would also have to pay for their own medical surveillance team and at least one hospital visit for the first infusion (this alone represents several thousand dollars). If you are required to give biological samples, the cost tops $10,000.

Genervon shared with me much of the top-level data from the Phase 2 to compare against my own data. Even though the trial population was small, the data were stronger in separation between treatment/placebo cohorts than in any legitimate trial results I had seen before. And GM6 was demonstrated safe over a much larger group spread over three different neurological diseases (including ALS) plus a healthy safety group. For these reasons I suggested to and worked with Genervon on applying for the FDA Accelerated Approval Program in order to get GM6 to all PALS paid for by insurance and Medicare.

And thus began the shitstorm...

Researchers, neurologists, and leaders of certain advocacy organizations who believe in the FDA's 60 year old regulatory formula - comprised of designing, completing, and analyzing Phase 1, 2, 3 trials over a period of 5-15 years - are failing in their proclaimed mission. They simply have to stop regarding patients as helpless victims willing to eat rat poison if someone said it cured ALS, Genervon as somehow the 19th Century snake oil salesman, and themselves as the White Knight riding to our rescue. The very process of obtaining an experimental drug requires a lot of medical oversight, which we appreciate and rely on. However, patients are intelligent adults whose only desire is to change the status quo of scientific research for the benefit of both the current and future generations of PALS.

The 1992 FDA Accelerated Approval Program (AAP) was designed to meet the needs of patient populations where there is an urgent and unmet need. In 2012, Congress passed and the President signed into law the Food and Drug Administration Safety and Innovation Act (FDASIA), strengthening the agency's ability to advance public health by equipping the FDA with tools intended to expedite the development and review of innovative new medicines that address certain unmet medical needs. Among the objectives, Title IX expanded the scope of products that qualify for accelerated approval. Specific language in this law states that the FDA is to incorporate novel approaches to the review of surrogate endpoints based on pathophysiologic and pharmacologic evidence in such guidance, especially in instances where the low prevalence of a disease renders the existence or collection of other types of data unlikely or impractical. It is obvious that Congress and the President had in mind diseases just like ALS when passing and signing FDASIA into law, yet the FDA has done very little to incorporate these guidelines.

With Congress now discussing the 21st Century Cures Act, we at Hope Now for ALS believe that we are on the right side of history by insisting that PALS are given opportunities to access new investigational treatments through the FDA's Accelerated Approval Program which, with its requirement for post-marketing Phase 4 data surveillance to confirm efficacy and safety, will continue to provide invaluable data on new treatments for ALS. As most patients are ineligible for standard clinical trials, this is our only option to contribute to research that will provide the same data at a faster rate among a larger population of patients - providing much needed data on subsets of the patient population. The Phase 4 requirements of Accelerated Approval also have the ability to save billions of dollars in research that is better spent developing more new and better investigational treatments for a myriad of neurological conditions.

I will grant that the biomarker candidates are new and not yet "proven", but FDA did allow them as endpoints in the Phase 2. They are not brand-new fabrications by Genervon and are backed by a lot of recent research by respected researchers. And they were all quite uniform in response to GM6 while the placebo group all continued in the abnormal direction. In my n=1 case report the biomarker candidates sometimes went in the reverse direction, but ALWAYS TOWARD NORMAL LEVELS. This is a great indication that GM6 promotes neuronal homeostasis - the holy grail for ALS research.

The Phase 2 was indeed also only a very small population, and in previous ALS trials of similar size it was impossible to collect reliable efficacy data in such a small cohort. However, this trial was very different from previous trials. The effect registered was much larger than in previous such trials (especially dexpramipexole) and was backed up by multiple secondary measurements not subject to any placebo effect. The combination of surprisingly-large effect size and objective biological markers sets this aside from previous trials (which also used the ALSFRS almost exclusively). There was an erroneous though well-intentioned attempt to use the released FVC information as evidence of poor trial design. However, the comparison used a very inappropriate analogy population and was built on an assumption based on incorrect data.

I do have serious issues with a point used in arguments against GM6: The lithium debacle. The media reports which came out obviously created a lot of excitement within the patient community. Our first reaction was asking and pleading the research community to quickly follow up with more trials to confirm that study and the response from the research committee was absolute disinterest. Therefore the patient community took it upon themselves to create a verification study, which we did. We did *NOT* merely go out and start using lithium off-label. In fact, it was only after our trial data was being released that the research community decided to do a confirmation study. By then we had already demonstrated that lithium had no effect in ALS and begged the research community to not waste time and millions of dollars.

But again, the research community ignored the patient community.

The Hope Now for ALS movement isn't for GM6 to skip the regulatory process. It's to get FDA to use its existing programs and Congressional mandate to provide potentially life-saving treatment to PALS. This is especially important now that truly-effective treatments are very near (including NP001, Neurown, etc.). Caution is obviously warranted but ALS is a race against a clock that doesn't care. More aggressive strategy is thus required which necessitates a little less caution and a lot more courage.

In summary, the facts are:

• Genervon asked FDA for Accelerated Approval at the post-Phase 2 meeting where they presented the complete trial data plus the case report for my Compassionate Use project. I know this to be true because I co-wrote the cover letter to the data package and it specifically asked for Accelerated Approval (and it was me who urged Genervon to pursue AAP).
• The FDA should have responded with specific instructions on how to file. They did not and thus we were all left in a state of confusion. Then FDA took the unusual step of calling on Genervon to publicly release proprietary data. Genervon has no duty to do so and FDA has no authority to make such a request.
• Genervon has perfectly complied with law and regulation. All they want is to help and they believe GM6 can do that. The data so far looks good (and I can say that, having actually seen it where all others commenting otherwise have not). It's not a slam-dunk, but it's positive and safe enough that I think all PALS should have access to it - not just those eligible for clinical trial.
• The FDA Accelerated Approval Program, in place since 1992 to deal with fatal diseases for which no other treatments exist, is the best way to save lives. It opens access WHILE CLINICAL TRIALS STILL CONTINUE. It's used for cancer and other diseases with less-severe prognosis. Why not ALS?
• GM6 has a perfect safety record in over 50 patients across 3 separate neurological conditions plus a healthy initial safety cohort.
• This is about patients deciding for themselves what risk to take in treatment. This is NOT about a company trying to avoid the clinical trial process or enrich itself on patients desperation. The AAP is an existing program which gives patients access to potentially life-saving treatment while collecting the valuable efficacy data.
• Contrast Genervon's completely legal and transparent actions to other companies marketing unproven products such as lunasin and aimspro directly to patients using email. Those companies use slick pitches with "proof" based purely on non-accepted metrics and anecdotes.
• The movement behind GM6 is entirely grassroots.

The above are facts. All of the "expert opinion" going around is just biased speculation.

Scuttle Rebuttal

I am a little peeved at a new attack on the push for FDA's Accelerated Approval Program for the ALS treatment called GM6 (known as GM604 in clinical trials). This attack basically follows the same line as the one from the ALS Association (and in fact incorporated it by reference). Both are sloppy and disingenuous in style and content. They attempt false comparison to other previously-failed trials without including the reasons for those previous failures and they also try a very clumsy smear by comparison to an act of intentional clinical fraud. I am disappointed that such comparisons are presented to a public looking for facts as well as hope.

First let me discuss the false comparison to previous trial:

1. The comparisons are false primarily because the old trials exclusively used the ALS Functional Rating Scale (ALSFRS) which is a horrible metric by even the most generous interpretation. This is the only way a call for large trials can be justified, because the numb insensitivity of the ALSFRS (even the Revised version) requires such to make any kind of meaningful conclusion in a heterogeneous disease like ALS. However, the GM6 trial used several biomarker candidates and other objective clinical measurements as surrogate end-points (keep that phrase in mind) which correlated which the subjective end-points such as the ALSFRS-R. The biomarkers used were suggested and evaluated by Dr. Robert Bowser, a 2015 winner of the Sheila Essey Award for significant research contributions to fighting ALS.
2. Brain-Derived Neurotrophic Factor (BDNF), one of the neurotrophic factors listed in Dr. Dickie's post, doesn't cross the Blood Brain Barrier (BBB). It's almost impossible to get a therapeutic dose into the patient without an intrathecal infusion (directly into spine) using a pump over time. This has numerous obvious drawbacks. It's also very unclear whether a single neurotrophic factor is useful in ALS, which encompasses a host of deficiencies.
3. Cilliary Neurotrophic Factor (CNTF) does cross the BBB and early animal model tests indicated efficacy. However, two human trials in 1996 using subcutaneous delivery and intrathecal delivery as well as a review in 2004 revealed no efficacy in lower doses and serious side-effects at high doses. It's also important to note that the animal data came well before the excellent work ALSTDI did characterizing the extreme difficulties in using that model. Any ALS mouse data released prior to 2009 (and any subsequent found to not strictly follow those guidelines) should be considered suspect. I have personal knowledge of the difficulty in using this model and the false-positive data which can result from improper use.
4. Next, Insulin-like Growth Factor 1 (IGF-1) also crosses the BBB but has a very very short biological half-life, meaning it is broken down and excreted in a matter of hours. That makes therapeutic levels almost impossible to maintain. A form was created with a buffering agent attached to IGF-1 which roughly doubled the half-life but even that was woefully inadequate. Anyone who remembers the IPLEX debacle of a few years ago knows the story.

The comparisons to such single-target neurotrophic factors as BDNF, CNTF, and IGF-1 are therefore flawed in logic and fact. It is very disingenuous for Dr. Dickie to compare GM6 to them as GM6 is a master regulator and acts in 12 relevant pathways simultaneously. This information is already in the public domain freely available for anyone to look up.

Next, Dr. Dickie compares the GM6 results with those of the initial results of NP001 (actually he links to his own blog post where he addresses the anecdotal reports which came before the official results were published). What he failed to mention was the updated post-hoc analysis which showed a halt of disease progression in 27% of patients in the trial. Further, the analysis showed statistically significant evidence of two biomarkers which identified responding sub-groups. This is a tremendous achievement in ALS clinical trial history. Unfortunately the biomarkers aren't the same in the GM6 trial so the comparison of the two is incomplete at best. The only real similarity is that both trials used biomarkers as secondary end-points. However, the GM6 trial used them in a way that didn't require post-hoc analysis.

The comparison with lithium is especially troubling. First, it was far from "recent", with patient excitement starting in 2007. You can find the data collected in the first PALS-led and created clinical trial which coincidentally also involved lithium. What both ALSA and MNDA failed to report about the study which started the excitement ("Fornai, et al., 2008") was that the study essentially "cooked the books" by assigning PALS with slow progression of disease to the treatment group while putting the more standard PALS in the placebo group. This was revealed only after the paper was published. In the GM6 trial, run by two leading and internationally well-respected ALS researchers and clinicians, all participants were randomly assigned to receive either drug or placebo. The only way the comparison to the lithium study would be accurate is if the researchers deliberately placed certain patients in each cohort. Genervon merely supplied GM6. The trial was run and data collected by the two principle investigators (fancy name for doctors who run clinical trials). The analysis was then also done by a contract research facility. So any implication that Genervon somehow fabricated the data is false and besmirches the reputations of two prominent ALS doctors.

It must be noted and repeated that the standard FDA clinical trial practice is indeed extremely important in terms of protecting the public from the unscrupulous. The collection of objective scientific data is the foundation of good medical care. Nobody calling for the Accelerated Approval of GM6 disputes this. However, because ALS is so rapidly and uniformly fatal, we are calling for FDA to utilize the discretion it was granted in the face of an earlier similar crisis (the AIDS epidemic). Further, we call on everyone to realize that the GM6 trial used much more than the ALSFRS as a metric and thus smaller trial populations are much more statistically significant than before. The other end-points used in the Phase 2 are objective and not subject to placebo effect. Therefore, the indication of efficacy observed in the trial should be considered stronger than in previous trials which relied almost solely on the ALSFRS.

The Accelerated Approval Program was created to bridge the gap between the need for data and the urgent unmet needs of patients with rapidly-fatal diseases. The GM6 trial was unique in the strength of the preliminary efficacy signal, largely due to the objective biomarker end-points used. Just like the early days AIDS crisis, PALS have no meaningful treatment options and thus no hope. We want to use the very FDA program created to deal with that situation. We admit the need for more scientific data. But we don't want to die while it's collected.

Condition Green

As many of you might already know, I was the late-stage PALS mentioned in the recent Genervon press release. I got interested in this drug some time ago, did some research on it and wrote a blog post about it. I had contacted the company, Genervon, to get information for my post. Thereafter, a dialogue was maintained regarding clinical trial status and future development plans. Being that I am a late stage PALS and still extremely active in awareness, advocacy, and science, they agreed to my request for compassionate use. It was another 9 months going through the process of authorization (mostly because my local hospital had never done anything like this before and together we created a new protocol).

During that time the Phase 2A results came out and I was given access to some of the data. Those, combined with my own experience, gave me the satisfaction that this drug was safe and quite likely effective. I share the concerns about trial size, but like all PALS am concerned for the time required to go through the usual phases of clinical trials. The clinical trial program actually has four parts:

• Phase 1 - single dose usually in healthy subjects for gauging safety
• Phase 2 - use in actual patients looking at safety and initial efficacy
• Phase 3 - larger patient population with different doses, efficacy and SAEs
• Phase 4 - market surveillance for adverse events

Not only does it take time to fully enroll and execute a large clinical trial but it takes even more time to secure the funding necessary to begin each phase. This is especially true in this current era of venture capital avoiding biotech investment.

I have helped launch other initiatives to get PALS access to experimental treatments. It is critical that patients get more than one or perhaps two chances at early access to treatment while they are newly diagnosed. Drugs that are possibly effective must be made broadly available to patients who are facing otherwise-certain death. Based on the safety and the indication of efficacy in GM6 (mainly borne of my personal experience), I got behind the effort to seek what FDA calls Accelerated Approval so that many more PALS can try it and see where it takes us. Accelerated Approval requires full data surveillance for efficacy, not just serious adverse events (SAEs). The efficacy data determines whether final approval is made. Basically, Accelerated Approval is like a Phase 3 where patients/insurance pay for participation. I believe all PALS would gladly participate in such a program.

If the wider data don't support the continued use of GM6 I will be the first to admit it. But right now I believe GM6 has the capability to effectively treat ALS in a way no previous drug ever has. And I want to get that opportunity as quickly as possible to as many PALS as possible.

After publishing the press release and posting it on social media and online forums, another PALS started a petition to the FDA to demonstrate the support in the ALS Community for this Accelerated Approval. I would like to urge all who are concerned about ALS - PALS/CALS/Friends - to sign this petition and share it among your social circles. At that link you can sign the petition and post comments to be included with your name. You can also find links to email Senators who oversee FDA and proposed text for those messages.

It is imperative that the comments left on the petition signatures be respectful. FDA isn't the enemy. They really would like nothing better than to approve a treatment for ALS but need the data to support it. I think we have the data because even though the population was small, the slope of decline as measured by the ALSFRS-R was reduced significantly during the short treatment window. Also, certain biomarker candidates were tracked and correlated with progression. Nevertheless, FDA has to be very careful with the precedent it sets so we as patients must be partners with them in these decisions.

My own experience with GM6 has been positive. The worst part of the entire project was getting the PICC line and the lumbar punctures for CSF samples to make biomarker measurements. I experienced absolutely no adverse events related to the drug. Insofar as benefits, I must admit that the small gains in function noted in the press release are most likely due to surviving neurons branching out new axon terminals to cover the neuromuscular junctions (NMJs) abandoned by the dying motor neurons affected by ALS. GM6 will NOT regrow dead motor neurons. However, it does induce healing in injured ones. In my case, I probably don't have many injured motor neurons - most of mine are gone. But people who are more recently diagnosed have a higher chance of regaining some lost function in addition to stopping progression.

Based on the information I have seen and my own positive experience, along with the considerable (at best) delay in commencing a larger Phase 2 or 3 trial, I think GM6 deserves Accelerated Approval. I also think this could set a beneficial precedent for future drugs which show similar safety and efficacy signals in early trials. Hence my hope for GM6 getting into the larger population of PALS.

IceBucket The Blue Sky

The #ALSIceBucketChallenge has been nothing short of a miracle for patients and researchers. Internet memes are rather capricious, having a nearly random hit/miss ratio. That this became so huge is a stroke of incredible luck. The awareness, and resulting increase in donations, has been a huge windfall. This surge couldn't have come at a better time as researchers now have exquisite investigational tools not available even 5 years ago.

There is then the begged question, "Why did it take a patient and a tractor trailer full of luck to bring awareness to the public?" For decades there have been organizations claiming to represent ALS patients. Yet never has there been a sustained national awareness project executed. Patients were left largely on their own to create awareness. This is a question to which we as patients should demand an answer.

Nevertheless, the windfall is upon us. I believe that this boost in funding should be used to create a critical mass of awareness and outreach. As stated, Internet memes are capricious and subject to fading from the public memory with all the speed and ferocity with which it entered. This is the perfect time to keep the message sustained in the public view. Certainly the money to do so is now available.

Another question the ALS patient community should be asking is how much of the massively increased donations are going to be actually used for research, and in what programs. Some donations are going directly to research facilities but the bulk of them are not. Is that bulk to be hoarded and doled out in tiny slices and without focus to a wide variety of basic research projects, never giving any sufficient amounts to fully complete the work? Or will a significant effort be launched which will fund focused work on high-value pathways, including helping fund clinical trials in humans (there are a few promising treatments languishing for lack of funding to pay clinics to conduct Phase 2-3 trials).

This is the time for organizations representing the ALS patient community to step up. The shelf-life of popular public awareness is notoriously short. We need to take this opportunity to create a program of sustained awareness and lobbying for research funding, along with a focused research effort encompassing basic research through to human trials. It is also the time for all research and advocacy organizations to come together as a united front in order to make ALS nothing more than an unpleasant memory.

Of Mice And Me

As many of my readers know, about two years ago I came across a study investigating a novel molecule for the treatment of Alzheimer's. The molecule, J147, is a synthetic derivative of curcumin. Curcumin and other similar molecules have long been under study for neurodegenerative diseases. Unfortunately curcuminoids have rather poor bioavailability, meaning they are quickly excreted from the body and require high amounts to have a therapeutic value. Like curcumin, J147 is "orally available" (meaning it is introduced to the body by eating it) but is more than 100X as potent. This means a much smaller quantity is necessary for therapeutic effect. So far, we haven't found a toxic dose of J147. Work on toxicity is ongoing.

In the Alzheimer's study J147 had remarkable results in that model. The pathways acted upon were quite relevant to ALS. These include potent antioxidant effects, significant reduction of microglia activation and migration, and reduction of heat-shock protein expression which indicates a shift back toward cellular homeostasis. More recent data (unpublished) indicates an effect in reducing astrocyte activation, which is sufficient to rapidly kill even healthy motor neurons.

Unfortunately, because J147 is pleiotropic, pharmaceutical companies weren't interested. The current research paradigm is to focus on single molecular targets. For diseases with a single mechanisms, that's a fine method of attack. But ALS has quite a few things going on simultaneously. All prior single-target treatments have failed and the current growing opinion is that successful treatment would require a cocktail of drugs. Better to have a single pleiotropic substance than a mixture of chemicals with uncertain interactions.

In April, 2013, I created SciOpen Research Group in order to have an entity capable of negotiating research and licensing of novel molecules with the promise of treatment of ALS. J147 is our first project. In the early summer of 2013, SRG applied to Prize4Life for access to their colony of G93A transgenic research mice at Jackson Laboratories. Our research proposal for J147 was accepted and we were given granted sufficient animal numbers to properly conduct our study. We received the mice and started the experiment at the end of January.

We are very excited to have commenced our first research program and demonstrate that guerrilla biotechs can perform quality science. To that end, we created a crowdfunding campaign on Indiegogo to obtain funding for the next step of the experiment - microscopic tissue examination. This will tell us exactly what J147 did to help the motor neurons in the mice.

Please donate if you can. All donations are tax-deductible. If you cannot donate please spread word about SRG and our need for funding this new and exciting research.

Pot Luck

An article appeared on social media about a group of parents using cannabidiol (CBD) for their children's epilepsy. Unlike the usual reports of people using marijuana and subjectively reporting "improvements", this group of patient advocates went and filed an Investigational New Drug (IND) with the FDA. Don't get me wrong - I support the medical (and recreational) use of marijuana, but heretofore the real scientific data available has been extremely thin. Rather than going on Silk Road to get a bunch of medicine then post wonderful stories on social media, this group created a real clinical trial in cooperation with FDA and a company named GW Pharmaceuticals which supplied a pure oil formulation of CBD. This is a very important development in patient-driven access to investigational drugs. Far better than the usual DIY projects (even the handful started by yours truly), this type of project can deliver real, verifiable, and scientifically-accepted results.

The body contains cannabinoid receptors both in the CNS and periphery. The most well-known cannabinoid ligand is THC (a CB1 agonist) which is responsible for the euphoric psychoactive effect in marijuana. Both natural and synthetic cannabinoids long been of interest in treating disease. What's of most interest in medicine are the anti-inflammatory effects of CB2 agonists such as cannabidiol or CBD. Endogenous CB2 receptors are upregulated in the spinal cords of SOD1 transgenic mice. CBD agonists show symptomatic improvement in several inflammatory diseases. There is evidence that CB2 receptors are upregulated in response to the inflammatory microglial activation in ALS. Several studies have shown that CB2 agonists have a beneficial effect in transgenic SOD1 mice. This data shows that more work, perhaps in in human patients, is warranted.

Alternative medicine is very popular in the ALS Community because, frankly, there is nothing currently available proven to extend the lives of PALS. Unfortunately most experiments are done without adequate objective observation and recording of data. Instead all that is reported are vague descriptions of improvement, skewing any rational perception of the particular alternative medicine. This causes more desperate patients to attempt the alternative with the same lack of adequate reporting.

This post, however, is not about calling for an IND for CBD (which would nevertheless be a good idea). The point here is to spotlight that a group of patients and/or advocates got together to do an experiment outside of an institutional clinical trial. They led the way and did it themselves while preserving the valuable objective data. They created their own hope in a seemingly hopeless situation. This is the ultimate expression of DIY Medicine, done properly and openly. Any other method is a waste of time, money, and health.

There is actually much more opportunity than just experiments with speculative alternative medicine. Hope exists for the approximately 60% of living PALS who don't qualify for clinical trials. That hope is the FDA Expanded Access Program (EAP). PALS should request EAPs for those investigational treatments which have passed the Phase 2 endpoint requirements of safety and suggested efficacy. Furthermore, they should support efforts to bring EAPs to the ALS Community. Living, even for the healthy, requires hope. We, the ALS Community, like everything else we have accomplished, must create our own hope by being pioneers and responsible citizen scientists.

Outta Tsai't!

I would like to give a little blog-love to my [Facebook] friend and a fantastic artist Francis Tsai. I came to know him through the ALS community and very soon became a fan of his art. When I learned he was selling prints online to help finance his care, I purchased a couple.

The first one I purchased was one he did prior to onset called "Trixie":

This was a gift to a friend who collects pin-ups. My next purchase was a much more recent one called "Horned", which sports Francis' motto "Adapt - Survive - Prevail":

The thing I really enjoy about "Horned" is that it completely (and very literally) illustrates Francis' motto. Not content with just fading away, he got a computer and some software and created it using only his eyes!

Francis truly exemplifies the willpower of PALS and the clever use of technology to overcome the physical limitations which come with advanced ALS. A person is the result of his/her mind, NOT the physical body. With a little willpower and some appropriate technology, PALS retain purpose and personal productivity and quality of life remains high.

Surf on over to Francis' DeviantArt and StorEnvy sites (linked in the images above) and check out his art. While you're there, purchase a couple of prints. The money goes to a worthy cause and they make great gifts!

PSAwesome

On Monday of last week, this excellent PSA was released by Team Gleason. It was shown on the big screens inside the Superdome but needs to be shown repeatedly on major network television (ie ABC/CBS/NBC). Please watch, share with friends, and send to your local network television stations. This is the kind of message that needs to get in front of the eyeballs of America. This is the celebrity action we have been asking for. Now let's do our part in getting it out there.

Rescuebook

Technology is extraordinary. The level of communication made possible today by technology is unprecedented. This ability is also available for people with extreme disability (I wrote this purely with eye movement to distribute to the world...).

Response-able

I was recently alerted to a blog called Science-Based Medicine which had a post that was rather critical of me. The post was written by David Gorski, a managing editor of Science-Based Medicine. If you read the post, you can probably understand why I took a little umbrage to Dr. Gorski's characterization of my efforts. Below is my response to him, shared here for my readers' convenience.

UnBearable

The hit movie "Ted" has stirred up some controversy in the ALS Community. I usually ignore such jokes (and sometimes even laugh along, including bits involving Stephen Hawking and a recurring "Hawking-esque" teacher character in Family Guy a few seasons ago). But this was over the line. I have lived with ALS for 7 years. In 5 years, ALS took away from me everything I had built in the previous 20. It is currently robbing me of the most productive years of my life. Man to man, I hope you DON'T get Lou Gehrig's Disease.

MacFarlane and Wahlberg owe the ALS Community some awareness.

PALS can't bear Ted.

Petition for awareness.

Pet The Petition

A few months ago, I posted about publishing companies trying to charge for access to research studies paid for by taxpayers. Today my friends at The Doctor Weighs In posted about a way for each of us to really make a difference in this fight.

The White House has an online petition system where citizens can weigh in on issues before the President. If the petition gets 25,000 "signatures" within 30 days of it being created, it will be presented to the President where it must become part of the policy discussion. This is a great way for We, The People, to address and direct our government (especially in these times of corporate-captured government).

There is a petition currently in circulation to demand that scientific research paid for with taxpayer dollars remain freely accessible to anyone wishing to read it. The current signature count is just shy of 16,400 and I strongly urge my readers to sign and share this petition by email and/or social media. Working together we can ensure our intellectual property isn't stolen and we are then charged for its return by the very thieves who stole it.

All Alone

It's the middle of ALS Awareness Month. Individuals have been doing an excellent job of pushing awareness in their local circles. This includes pet groomers holding awareness and fundraising events,

R U A WARE?

It's ALS Awareness Month. As per usual, certain advocacy agencies are content with passive website pages and extolling their constituents to do the heavy lifting of spreading the word (a practice that has dramatic fall-off beyond the tiny individual circles of family and close friends). The agencies bleed enough contributions from the constituency to pay salaries and provide token services (with no consistency between Chapters). Meanwhile, The general public is still unaware and uninformed about the deadly lottery that is ALS.

Up until very recently, ALS was a disease of silence. As Person(s) with ALS (PALS) progressed, they lost their abilities to communicate and mingle in their communities. Very quickly they were shut away in homes or hospitals to die in mute angst with a capable mind trapped in an inert body.

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 with some on mechanical ventilation via tracheotomy. As the astrophysicist and PALS Stephen Hawking said, "My body may be crippled but my mind is free." Tech-savvy PALS are able to use combinations of technology to dramatically increase their standard of living, to contribute to research about the disease, and even band together to lobby government to speed up access to promising pharmaceuticals. As wonderfully-explained in a recent forbes.com article, "Technology is the cure" (thanks to Steve Saling for coining that phrase).

On a personal note, technology is saving my life. Many years ago my father was in the US Air Force flying support missions for the Apollo moon flights. On occasion I would be in ARIA control watching technicians tending to walls of huge computers. I thought that was the neatest thing ever. Years later, I was blessed to turn a childhood hobby (began when I commandeered my dad's IBM PC in 1980) into my career, and I now have in front of me more computing power than all the Apollo vehicles combined.

When I was diagnosed, I knew that my ability to communicate was going to disappear. Knowing that hands-free computer technology existed, and already having a long-term presence in "cyberspace", I knew that I would be able to be social and productive as my physical ability declined. This was paramount in my decision to accept mechanical ventilation. Without the ability for my mind to escape my physical confinement, I would have years ago allowed myself to succumb to ALS. Instead I have remained active and have had the extraordinary privilege to meet and participate in the global ALS Community.

Unsatisfied with the level of advocacy and awareness generated by organizations with that as their claimed mission, PALS in the global ALS Community are taking it upon themselves to increase awareness and advocacy, and are 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. The new computer technologies, mobile devices with apps, and ubiquitous connection to the Internet and social media allows PALS to congregate, exchange ideas, and mobilize. No longer are they shut away and forgotten.

This month, are aware or are you just a ware for fundraising that does little?