Tag Archives: survival

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Liquid Hope

Usually a new hole in your stomach is bad news, often being either an ulcer or the result of some sort of violence. But for some, properly done, it’s a way to keep fed if the more normal method is no longer available. The question then is what to put through the hole. Obviously it would need to be in liquid form, but one can’t live just on beer alone (and I have tried…). Thankfully, there is a much better alternative.

Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig’s Disease) is a disease outwardly characterized by loss of muscular strength. People coping with diagnosis (PALS – Person(s) with ALS) experience a progressive loss of muscular control as the nerves communicating brain commands to those muscles die. Eventually a specific muscle, the diaphragm, becomes weakened and breathing capacity is diminished. After progressive weakening of the diaphragm, breathing capacity diminishes to the point that blood carbon dioxide levels rise and the person dies of respiratory failure.

Although no two PALS experience the same progression pattern (I call us “Snowflakes From Hell”), usually another important – yet overlooked – muscle group is impacted before the diaphragm. This muscle group is commonly known as the tongue. When the back of the tongue loses strength, it can no longer efficiently create the pre-swallow bolus made up of the food being chewed and it can also no longer guard the airway against intrusion of food below the mouth before the epiglottis closes the trachea and opens the esophagus. This creates a choking situation with the increased possibility of aspiration pneumonia. Obviously both the choking and pneumonia represent substantial threats to life, especially for those with compromised respiratory function. Not only are choking and aspiration both hazards, but the lack of proper nutrition from not being able to eat is a dire handicap in the battle against ALS.

Fortunately for people in such a situation of lingual weakness, such as PALS in mid and late stages, medical science has created the PEG tube. This is a silicone rubber tube a little larger around than your typical drinking straw. It provides a direct route to the stomach and can dramatically lower one’s bar bill (because you don’t taste, you can switch from top-shelf to well brands…). PEG tubes are actually essential tools in “treatment” of ALS by keeping up optimum nutritional (including caloric) content.

Unfortunately, the “medical formulas” many patients are told to exclusively use – such as Nestle COMPLEAT – are based almost entirely on corn syrup for calories, which is the glucose base version of high fructose corn syrup (HFCS – the difference between the two is that HFCS is much sweeter, thus being attractive to processed food manufacturers). Basically, each can is a candy bar with a multivitamin in the middle. We have all heard the news about the perils of excessive sugar intake and how it, in the form of HFCS, is pervasive in processed foods. Eliminating HFCS and still eating just as much glucose sugar, especially as a sole source of calories, is equally harmful.

As I have previously blogged, using these medical formulas for any prolonged period is very risky in terms of your pancreas. I am an otherwise extremely healthy [formerly] athletic man with zero endocrine or any other confounding health issues. Nevertheless, using the traditional “medical formula” every day for two years put me in the ICU for a few days with a severe diabetic and hepatic crisis. I took control of my treatment plan and eliminated the corn syrup by switching from formula to real food (something which hospital dieticians tell patients to NOT do).

Clearly, the traditional enteral nutrition sources are not meant for long-term use. Until recently, most PALS died relatively shortly after diagnosis. This meant a few months of solely enteral nutrition weren’t going to pose a problem. But now, with better care and with adaptive technology better able to restore lost abilities, PALS are living longer post-diagnosis. I am one of those, going past 10 years post-diagnosis. Obviously better nutritional products are required. After taking personal control of my feeding, choosing fresh food blended together with a combination of healthy sources of fat, my blood glucose, liver, and kidney functions all normalized.

Not all PALS have either the ability to make their own blenderized food (is that really a word?) or have people who can make food for them which meets their nutritional and caloric needs. Just opening a can of soup is insufficient, as almost all processed food contains unacceptable levels of sodium, HFCS, etc. Further, PALS have certain requirements such as higher fat and calories. Getting those from improper sources can be hazardous. So what can we do?

Liquid Hope is here! This is a product created as a reaction to the terrible content of the traditional formula and the negative effect on health they can have. It is basically fresh food in a pouch that meets the needs of those with special dietary concerns (dairy free, gluten free, non-GMO, etc.). It’s a full meal replacement suitable for PALS as-is, but can be mixed with avocado, coconut oil, or other healthy fat source to boost calories for those PALS experiencing dramatic weight loss. My readers can learn more about the development of Liquid Hope here.

Even though I was getting mostly fresh food, I was interested in trying out Liquid Hope. The good people at Functional Formularies agreed to supply me a 7 day supply. From the very first meal I felt great! I was fully satisfied as if I had just had a good meal at our local vegetarian restaurant (I really miss their vegetarian lasagna). After 48 hours, I had more than my usual energy, I felt clear, and I was much more regular (constipation is a frequent issue for PALS). I only added a couple tablespoons of coconut oil along with some protein and vitamin additives, like I do all my meals. I was really sad to see the last pouch go down.

In my semi-expert opinion, Liquid Hope is a fantastic enteral nutrition solution and far superior to the usual cans of “medical formula”. I am greatly looking forward to switching fully to Liquid Hope for my nutritional needs. It’s now covered by Medicare*, Functional Formularies can help with the paperwork, and my first regular shipment is on its way!

I have been watching and talking about Liquid Hope on social media for a while. Frequent readers and friends know that I am extremely anti-“medical formula” and push patients to make fresh food for their enteral nutritional needs. Now that Liquid Hope is covered by Medicare* and is provided by a growing network of enteral nutrition providers, I call on all PALS to try it and use it. Let Nestle make snacks, not food staples. PALS have a serious medical condition requiring real nutrition. Take care of yourselves. Either blend fresh (not freshly-opened) food or use an organic and healthy product such as Liquid Hope.

* [So long as you aren’t in what’s known as a competitive bid area. The problem with being in one, in my opinion, is that the reimbursement to providers is based purely on lowest-price, keeping the better products from being available. I can explain the political aspects but that’s an entirely different subject not appropriate for this blog.]

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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.

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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.

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The ALSETF

As my readers may know, my posts this year have been a little farther and further between. I have been working on some projects. Hopefully one or more can make a real difference for PALS. It’s time to talk about one of these projects: The ALS Emergency Treatment Fund.

The ALSETF is about bringing treatments in late development (post-Phase 2) to ALS patients. Right now there is no hope for the majority of living patients because over 50% do not qualify for clinical trials. For a newly diagnosed patient, the odds of living to see a drug approved which is starting trials at the same time is about 10% (actually much less considering the historical approval rate of ALS treatments). However, with recent advances into the nature of ALS, certain drugs have been developed which show real promise for treating at least a subset of PALS. More are planned to enter trials in the United States very soon. We at ALSETF mean to get that hope ASAP to PALS who are currently living.

ALSETF is a 501C3 non-profit organization with the mission to partner with government and industry agencies to enable Expanded Access Programs. Expanded Access Programs (EAPs) are FDA authorized programs that permit the use of yet-unapproved drugs under medical supervision, in specific cases where those drugs are in late stages of development and have shown preliminary evidence of safety and efficacy. EAPs are only for immediately life threatening conditions for which no effective approved therapies exist. We have open communication with the FDA’s Office of Neurology products for guidance on EAPs involving investigational drugs for ALS. We also maintain open discussion with clinical leaders on the best practices for EAPs, as well as with certain pharmaceutical companies with drugs in trial and in the pipeline.

Our focus right now is to raise up to $5M to help fund certain costs of an EAP such as upgrading manufacturing to clinical-grade, production quantity necessary for fulfilling EAP demand, etc. These are all issues which can currently prevent a pharmaceutical company (especially the small start-ups likely to take a chance on ALS) from accommodating a large EAP. We believe the financial issues can be solved and that the drugs being talked about with excitement in the ALS community can be brought to those who don’t qualify for trials now, while they are still living.

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Wide Open

Tonight I found a double-helping of tasty news: “Regulatory T-lymphocytes mediate amyotrophic lateral sclerosis progression and survival” (PDF). So what’s the big deal? Let me explain in two parts – the scientific then the soapbox.

It has been known for a while that Regulatory T-Cells (Tregs) were helpful in moderating the damage noted in ALS. In fact, one of the authors on this paper, Dr. Stanley Appel, did pioneering work in determining this. What makes this paper exciting is that numbers of Tregs and levels of their associated regulator protein FOXP3 were positively linked to rate of progression. This not only points directly to powerful therapeutic targets but also can be used to separate patient populations between fast and slow progression for more accurate (and shorter) clinical trials, all from a simple blood analysis at diagnosis.

The other thing I found very encouraging about this paper was how it was published. Part of the funding for the research came from NIH. Therefore it would be assumed to be taxpayer-funded and therefore public domain. I have seen open articles published by the paywalled giant Elsevier in such circumstances. However, the chosen journal was an expressly Open Access journal published by the European Molecular Biology Organization called EMBO Molecular Medicine. This is a paper published by top researchers who deliberately chose an Open Access journal. This behavior deserves commendation and support. Research needs to be shared, especially where catastrophic disease is concerned.

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Under Press-Sure

Over the past two weeks ALS has gotten some much-needed national coverage, first from the Wall Street Journal followed shortly by ABC News. I also just completed the first round of questions for an article due to appear in July in the-scientist.com. More opportunities seem to be materializing and I will do my best to keep the momentum going to keep the message of ALS in front of the public. I would like to call upon everyone to contact their local press, mention the WSJ and ABC News articles, and tell them YOUR stories. If you think it would help to mention a relationship to me, do so and I will back you.

There were a few points in the articles that I wanted to clarify:

  • I did not design the computer I use. It is a TabletKiosk Sahara Slate PC i440T with a Point Grey Flea-2 CCD camera with an infrared light and lense. The system uses the ERICA software made by Eye Response Technologies (since purchased by DynaVox). The only part of the computer I modified was my work environment and I installed certain programs and utilities which I use. I did help design the overhead mount which slides along the overhead track I use for my lift.
  • NP001 is indeed sodium chlorite, but when ingested orally the acid in the stomach breaks down most of it. The most optimistic data I have seen (based on rats exposed to it in drinking water) is a maximum 30% reaching the bloodstream. I would expect that to be less, and to be variable, in most people.
  • Because NP001 is already in clinical trial, I arranged the project more as an “early access” model with data-keeping as secondary.
  • I have three criteria for any such projects:
    • drug must be relatively safe
    • drug must be inexpensive
    • drug must be legal to obtain

It’s very important to me that I don’t recklessly put other PALS in physical, financial, or legal jeopardy with any project. My intention is to help not harm. We all already have enough problems to deal with.

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Guest: Persevering On NP001

A few days ago my friend, who goes by the handle Persevering on the TDI Forum, posted an evaluation of the PALS who have been self-reporting their experience with the Neuraltus Phase 2 trial of NP001 (which I have discussed here in the past). While the data looks exciting, I must caution that this isn’t official data and makes some assumptions which, if wrong, could totally invalidate the evaluation. However, I have enough faith in Persevering to bring this to your attention. For those who don’t have Patients Like Me accounts, I reproduce the graphs here.

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I have analyzed the NP001 phase 2 data from PLM to date.

I am aware of 37 PLM members who have participated in the trial and 34 who have entered sufficient data to track progress. This is roughly 1/3 of the listed full trial enrollment, and offers a great sample to predict actual trial results. The trial consists of 6 infusion cycles over 21 weeks (147 days), followed by 4 follow-up visits adding 16 more weeks. 13 have completed dosing. 11 will complete within 4 weeks, and all within 8 weeks.

In my thinking, since each infusion cycle is 4 weeks total, the first non-dosing follow up at week 25 (175 days) ends the dosing phase, and I have chosen to review data to day 175 in terms of FRS change. The variable of interest is FRS slope, and is very commonly used to evalauate efficacy for ALS clinical trials. For example, it was used in the recent report of Dexpramipexole phase 2 data, and was used for the official Lithium clinical trials in the US.

FRS for all 37 reporting

In my opinion it is possible to review reports of side effects for commonality to approximate those getting drug (either dose) versus placebo. I do not believe it is 100% accurate, and I also expect those getting a higher dose to have more or greater side effects, but it is not entirely possible to segregate the drug groups (high vs. low). Some may not be prone to side effects on drug and others could experience “nocebo” effects, so again this is not a true substitute for unblinding, which should occur later and allow a timely re-analysis of PLM data.

With that assumption/disclaimer, the summary statistics are:
(Green is improved FRS score, yellow is stable FRS down to -0.49/mo, and red is loss of FRS)

With side effects:

  • n = 20
  • Mean ALSFRS-r slope: 0.00
  • Standard Deviation ALSFRS-r slope: 1.24

FRS for those reporting side effects

Without side effects:

  • n = 14
  • Mean ALSFRS-r slope: -1.01
  • Standard Deviation ALSFRS-r slope: 0.68

FRS with no side effects

Comparison: 100.4% mean improvement

2-tailed t-test p-value for difference = 0.0093

The most exciting outcome to me is that the difference in progression rate is very statistically significant (p<0.05), even with only 34 data points! This would be unprecedented for a phase 2 ALS clinical trial. For example, the recent exciting report regarding Dexpramipexole, with a 31% mean improvement versus placebo in the first 12 weeks had a p-value ~ 0.20, a value 4 times higher than acceptable to conclude drug is better than placebo, by convention (based on a sample size of 53).

There is rumor of an official NP001 trial efficacy review soon, based on all data available on January 24, 2012. Let’s hope for statistical significance there as well, and potentially FDA accelerated approval, as occurs for cancer and HIV drugs.
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Thanks to Persevering for the work and for the guest-blog!

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Nothing Vented Nothing Gained

I have long been an advocate for PALS going on a vent. Many opt out for various reasons, one of which is, surprisingly, discouragement from medical professionals. Horror stories of cost and the difficulty of home care are common.

I beg to differ with that. Cost can indeed be an issue but with creative financing and the possibility of obtaining long-term care insurance before formal diagnosis, cost can be managed. In terms of home care, with a few simple rules adhered to, the risks of complications are minimal. Up until now I had only my own experience to use as proof of my contrarian view. It turns out I was correct all along.

In a study of over 100 PALS 78 were recommended for tracheotomy. 38 underwent the procedure. Of those, the one-year survival rate was approximately 80% (the same as lung transplant) and only one died from respiratory causes. As the study noted:

“Nowadays, starting [home venting] should no longer necessarily be considered as the beginning of the end for these patients. When the appropriate medical resources are in place and the patients wish to continue living, the old mentality of focusing only on palliative care which is still common in the management of this disease must make way for high technology and compassionate care.”

Vents today are about the size of a grammar school student’s backpack and very quiet. Assisted Communication Computers are covered by insurance and Medicare and are very easily converted to general-use Internet-capable computer systems. Web-based social media platforms give unprecedented communication and social access to today’s PALS.

I have cheated The Reaper for 4 years now and others have for longer. In my view the old bastard can piss off until I am good and ready to go. My quality of life is what I make of it. Technology is my friend and has been for most of my life. As the esteemed Steve Saling says, “Until medicine arrives, technology is the cure.”

I believe this and urge all PALS to use technology to win the battle against ALS until medicine provides the final victory.

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Headlines

First off, there is some wonderful news: Diaphragm pacing system receives FDA approval for use with ALS patients. This is a device which extends time until vent by electrically stimulating the diaphragm. Note that this will not replace a vent as the diaphragm muscle needs neurons to release acetylcholine to the muscle fibers in order for them to contract. However, I urge all PALS reading this to immediately begin talking with your neurologist about whether this would be right for you. Here is some good information regarding the DPS.

Next up is the recent news regarding the Neuralstem trial involving stem cells implanted in the spines of PALS. Readers can see a poster-style synopsis of current trial data here. The initial results are encouraging, and one patient has even shown improvement. The next step in the trial is cervical implantation where the cells will have the chance to impact the phrenic nerve (the “money” nerve that serves the diaphragm and breathing). There is still much to learn about this technique before it is available to but a few. Remember that this is still a safety trial. I would urge readers to consider the words of Neuralstem’s CEO.

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The Cost of Breath

Despite having a “fatal illness”, with a little technology and someone to keep an eye on me for the inevitable adjustments, I can live until age draws the final curtain on the story of my life. My quality of life remains high regardless of my current certain physical limitations. There is nothing wrong with my mind: I am still as intellectually productive as ever.

Nothing would please me more than to earn my own keep (and I am not alone in this desire), but people classified as terminally ill don’t appear to be considered viable members of the workforce. So I volunteer my time to raise awareness and try to understand relevant research, sharing what I learn with others (a part of my previous career and the genesis of this blog). I find this work fulfilling and it keeps my opinion of my quality of life quite high, focusing on accomplishment rather than loss. Those close to me can attest that I can get cranky between projects when I have nothing to do so I try to stay busy. As I am quadriplegic I must use a computer system that tracks my eye movement to approximate the use of a mouse. With this computer I write and respond to email and online chat offering technical advice and answering questions from other PALS, scour the Web for research information, create videos to promote awareness, created a website (with coding assistance from a friend far more skilled than I), and play with my home computer network among other projects.

The decision to vent or not is a highly personal one with many factors contributing to the decision. I chose to live on a vent for a few reasons. First, I had finally recently achieved real happiness in life and didn’t want the dream-come-true to end so soon. Second, I saw some hope on the horizon with the state of research into the disease and possible ways to overcome it and I still hold this view more than ever. Last, and important to my perception of quality of life, I still had a contribution to make to the world and I have only grown more intense in that belief. I am simply not done living, with all which that experience entails.

But my every breath now comes at a price. Because I cannot move to assist myself I must have an able-bodied person within hearing distance of any alarms or signals from my equipment. The skill requirements aren’t high and can be taught in a weekend, but nevertheless someone must be alert and on duty 24/7. Family is often preoccupied by the demands of daily living, volunteers are few and far between, and government assistance is non-existent for people in my condition. The one factor which should NOT be involved in deciding whether to live on a vent is the cost of attendants. Medicare will not provide for in-home attendants. There are even very few institutions which will take people on mechanical ventilation and hospice is not an option due to the vent being a life support device. With the enormous wealth floating around our for-profit healthcare system it is astounding that no insurance (not even my high-end corporate policy) will cover the cost of attendants. So my only option, given my desire to live, is to become a beggar on an offramp of the information superhighway. This is terribly humiliating to me.