Tag Archives: survival

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Disaster and Development

Hello and welcome back to the Cripple Command Center podcast. My name is Eric Valor and I will be your host for this episode. It’s my podcast so I get to hog up all of the miniscule glory from an obscure blog about a very niche subject. I promise I won’t let it go to the wig. On tap for this episode are: An update on the latest Neuraltus NP001 confirmatory Phase 2 trial, the new Brainstorm Phase 3, and a note about two decades’ worth of data on Oregon’s Death With Dignity Act.

But first, I would like to urge my readers and viewers to go to my friend Patrick O’Brien’s GoFundMe page and donate to his new independent film project. Patrick is a PALS like me, totally quadriplegic and on mechanical ventilation to live. Nevertheless, he continues on with his passion for independent filmmaking. His first project after he was diagnosed and became quadriplegic and vented, “Transfatty Lives”, won the TriBeCa Film Festival Audience Award and is available on Netflix and i-Tunes, among others. It’s amazing and I urge you all to watch it.

Please go, click, and donate. Patrick deserves the support to keep his work going.
http://www.gofundme.com/WheresTheBeef

Another urgent matter is regarding the ALS community in Puerto Rico. As you know, a severe hurricane devastated the island, effectively removing the communications infrastructure. This is especially dangerous for PALS by making it even more difficult for them to obtain the assistance they need. You have no doubt already heard of some patients dying in care facilities because the generators which powered ventilators ran out of fuel.

There are over 200 PALS in Puerto Rico who need assistance. Team Gleason, of which I am a board member, has committed at least $10,000.00 to this specific relief effort. I urge everybody watching, hearing, or reading this to donate. Your contributions will help us reach as many PALS as possible with supplies and equipment. With assistance from groups like Baton Rouge Emergency Aid Coalition, Cajun Airlift and networks of physicians in Puerto Rico and on the mainland, we will coordinate getting relief directly to those with ALS. Please consider donating to help PALS with little resources and little help on the horizon.
#NoWhiteFlags
https://app.etapestry.com/onlineforms/TeamGleason/gleasondonate.html

OK, to business. Neuraltus began their second Phase 2 trial of NP001 in February on 2017. The trial was to have 120 participants in 18 sites across North America and the 120th patient will have the final dose in December! Results are expected mid-2018. The previous trial “failed” but a post-hoc analysis found a responder group based on a set of inflammatory markers found in the blood and restricted this trial to patients fitting the responder profile. The results this time are expected to be much better.

Next item is the new Brainstorm Phase 3. This trial is the follow-up to the impressive Phase 2B which concluded in July of 2016. In the July 18, 2016 Update from Hope Now For ALS, HNFA issued its review of the Brainstorm data showed remarkable results in a group of patients classified as “Responders” (ALSFRS-R slope improved 100% after treatment compared to a 3-month lead-in observed slope of decline). “Responders” were almost half of the treated group. Their ALSFRS-R scores were significantly better than the placebo group and the surveilled biomarker candidates showed significant improvement. The Phase 3 is a multiple-dose trial where the Phase 2 was a single-dose. The results are expected to be even better, though ALS has shown itself to be quite elusive to hit when previous “slam dunk” treatments were thrown at it. Stay tuned here and to the HNFA website for news as soon as it’s released.

https://clinicaltrials.gov/ct2/show/NCT03280056?term=brainstorm+cell+therapeutics&rank=1
http://www.brainstorm-cell.com/patients-caregivers/clinical-trials/
http://www.hopenowforals.org/2016/09/brainstorm-phase-2-results/
http://www.businessinsider.com/r-brainstorm-enrolls-first-patients-in-advanced-als-stem-cell-trial-2017-10

Now for a subject that is intensely personal and not a recommend topic for dinner conversation . It’s definitely a dating taboo. This subject is the Oregon Death With Dignity Act (DWDA) which was passed in 1997. The law, which went into effect in 1998, has strict restrictions on terminally-ill patients who would then request a prescription to end his/her own life on their own terms rather than the much more unpleasant terms dictated by terminal illnesses.

According to the Medscape article which prompted this segment of the podcast:

“To obtain a DWDA prescription, patients must be adults of sound mind, have Oregon residency status, and have a terminal illness diagnosis. In addition, two physicians must confirm the patient’s diagnosis and prognosis, the patient must be offered hospice care, and the patient must make one witnessed written request and two oral requests at least 15 days apart.”

https://www.medscape.com/viewarticle/885868

It’s a very common-sense approach to ensure only those truly with a terminal illness have access to this program and that people requesting it are not in a state of undue stress or confusion. According to the 20 years of data compiled, the program is a total success. This is where we get into danger of ruining Thanksgiving dinner – especially when Drunk Uncle gets his inevitable too many beers and bourbons before the turkey is even out of the oven and resting.

The issue is being discussed in every individual state and on the federal level. There are many points of resistance, including religious groups and people who have had the cultural stigma of suicide ingrained in their lives. But is it really suicide if one ONLY has a medically-verified shortened life of suffering and loss of personal dignity? Isn’t it better to go out while you still have your human dignity, where family and friends can remember you whole and relatively hearty? These are questions which only the individual patients can answer for themselves.

I made my decision because I knew that I could suck the marrow from he bones of technology and life a full life full of triumph and tragedy, love and loss, creation and oblivion. I have done all that rather more intensely in my shortly over a decade of living with ALS than the previous nearly 4 decades of healthy living. But I feel that every citizen of the United States should have the right to make this decision for themselves. Adults of sound mind should be allowed to be the masters of their own lives. We do not have the luxury of any opinion about when, where, how, and by whom we are brought into this world. It’s the ultimate indignity to be denied the ability to choose our own terms of exit after tragic illness sets the date. However, this is an individual’s choice. I invite discussion on the Comments Section following each text transcript on my website.

I think 2018 is going to be a landmark year in the otherwise long and somber history of ALS. The previous clinical trials of Brainstorm’s Nurown and Neuraltus’ NP001 have had results that were much more remarkable than any in ALS history and, although history has been brutal with Phase 3 failures, there’s no reason to believe that these Phase 3s will be any different from their previous results. I believe this even more since sub-groups of PALS have been identified as very likely to respond to treatment, something not done in past clinical trials for other ALS treatments. We now have two “Real Deal” prospective treatment options going for registration (known to people not in the clinical trial business as approval) which should soon provide effective treatment for many PALS, finally extending lives in meaningful quantities. And for the unlucky among us, the Death With Dignity issue has become a national debate. With excellent data such as those coming from Oregon, hopefully it will become a nationally-recognized right for those of us unfortunate to contract a fatal disease to control our own destinies.

But like everything, PALS will need to go out and get these things for themselves. If Neuraltus and Brainstorm do decide to apply for registration, they will need our help by showing our support for approval and demanding FDA follow through. Likewise, we must also forcefully demand that lawmakers recognize our natural-born rights to control our own lives. Again, like how every gain PALS have made came from one or a few of us starting a small movement, with more joining in and staying active until the goal was achieved, we together can always achieve our goals. Not bad for a bunch of people confined to wheelchairs and beds who cannot physically speak. With a little clever use of technology we are still very potent advocates for ourselves.

A parting point is a political one. It has to do with the new Republican tax bill recently introduced in Congress. One particularly problematic issue with the bill, among multiple others, is the elimination of the Orphan Drug Tax Credit (ODTC). According to NORD, the National Organization for Rare Disorders:

“The ODTC allows drug manufacturers to claim a tax credit of 50 percent of the costs of clinical research and testing of orphan drugs. The ODTC is part of a package of provisions enacted in 1983 in the Orphan Drug Act (ODA) that provide incentives for drug companies to develop products for rare diseases. This legislation has been extremely successful, resulting in over 600 therapies for rare diseases coming to market in the last 35 years.

Without the Orphan Drug Tax Credit, approximately 33 percent fewer orphan therapies would have been developed, and 33 percent fewer orphan therapies will be developed going forward if the tax credit is repealed. This means that at least 10 fewer therapies would come to market for rare diseases each year, only exacerbating the stark need for rare disease treatments even further.”

This is a serious issue for developing treatments not only ALS but all other rare diseases. This is simply unacceptable for not just the patient community but all of America. Giving companies incentives to develop treatments for rare diseases creates jobs and innovation. And, when effective treatments are found, they create economic boosts from the sustained new jobs associated with the new treatment’s production and distribution. But primarily the patients could remain healthy and continue fully participating in the local and national economies. The ODTC is not a “loophole” and eliminating it is penny-wise but pound foolish, paying for tax cuts by eliminating excellent investments in the future of America.

Please help stop this by telling Congress that this is unacceptable. The ODTC is one of the only tax credits that actually saves lives. Tell your Senators and Representative to oppose repealing the Orphan Drug Tax Credit, and stand up for the 95 percent of rare disease patients still searching for a treatment.

https://salsa3.salsalabs.com/o/51076/p/dia/action4/common/public/

I would be remiss if I did not remind my viewers, readers, and listeners that open enrollment for health insurance plans administered under the Affordable Care Act (ACA, also colloquially known as Obamacare) runs from November 1st to December 15th. Certain states are open longer – check your state exchange website for the dates you need to know. This is half the length of open enrollment in previous years because the Trump Administration cut it from 3 months to 6 weeks (a month and a half). Trump also decimated the advertising budget, so people needing coverage under the ACA might not be made sufficiently aware of the new shorter schedule. So, remember this year to get your enrollment completed before December 15th. Don’t wait until the last minute – take some time to take care of yourself today. December 15th is the deadline so make sure your enrollment is complete with plenty of time left. Log on to either your state exchange or the national website and make sure you’re covered by the plan of your choice before December 15th.

That’s this episode of the C-to-the-3 podcast on ericvalor.org. I have been and ever will be Eric N. Valor. Until next time, keep breathing easy.

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Awful Truth: What’s Old Is New – Again

ALS-AwfulTruth18

I am re-releasing an older video I did with a new somewhat tighter look and some upgraded language (words I never liked when I originally released it). Essentially, nothing has changed in 6 years since 2011 to 2017. Radicava was approved for use in the USA, but it only works in very recently diagnosed patients and only for about a year. I classify it with Rilutek as a medication with a rather modest effectiveness. No truly effective and approved treatment exists yet for ALS.

And PALS are still being forced to make the terrifying and grisly choice between bankrupting their families merely for the privilege of living or just accepting a rapidly accelerated death where one morning their children or spouses would find them cold, pale, and never to wake again. Everything is covered by Medicare except the people required for being close by for the inevitable situation of something happening with the ventilator – normal moisture or mucus buildup, the air hose popping off, or something drastic going wrong with the machine – any of which mean death in minutes. Immediate family CANNOT be caregivers because the stress is just too great already. The health consequences would splinter a family at a time when being close together is the most important.

It is despicable that even when new treatment options which promise significant extension of life for PALS that they may be unable to enjoy that gift with their families.

Please enjoy my renewed video message.

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BCRA: The Anti-Healthcare Bill

Audio Podcast (192kbps MP3 download)
(video at bottom of text)

Hello and welcome back to the ericvalor.org Blogcast Podcast.

My name is Eric Valor and I will be your host for this episode. Please make yourselves comfortable and feel free to order something from the bar.

As my longtime readers know, this is not a political blogcast. The scope of this blogcast is limited to the subject of ALS, living with the disease, research into treatments for ALS, experiences of Person(s) with ALS (PALS), and my personal reflections. I routinely deny requests to “guest blog” general health subjects (also because they are just attempts to spread spam links). But I have to address a subject which affects 17% of our entire economy and the daily lives of 99% of our citizens, and has devastating implications for PALS and others similarly affected by severe conditions. That subject is the long-awaited Republican response to the Patient Affordable Healthcare Act, also known as the Affordable Care Act or more colloquially Obamacare.

On Thursday, June 23rd, 2017, the Republican Senate Majority Leader released a “discussion draft” of their supposedly “better” healthcare plan which was promised to lower medical costs and improve medical care for American citizens, and “rescue” us all from the “disaster” of Obamacare. Just like the ridiculous House bill (which Paul Ryan apparently hurriedly cobbled together over a long weekend), the Senate Better Care Reconciliation Act snatches access to healthcare from, and makes it much more expensive for, tens of millions of Americans. We waited 7 years for this? I call it the anti-healthcare bill.

Republicans incessantly whined for 7 years about how terrible Obamacare was, how they had “a much better plan”, how the ACA was “passed in secret with no hearings, input from Republicans or the public, and was written behind closed doors. Actually it was available online for a year for public comment, had over 100 hearings, and included over 100 Republican amendments. After all that time this embarrassment on paper is the best they can come up with? This naked frontal assault on the poor and middle-class WILL LITERALLY KILL ME and others with ALS and other deadly conditions – all to give around $1 TRILLION to the already-hyper-wealthy. Moreover, it was literally written in complete secrecy behind closed doors (in such secrecy that one of the supposed authors of the bill never saw it until today) and will have no hearings with less than 10 hours of debate and amendments before a vote is called about a week from now. Undoubtedly Senator Tortoise McGee wants to rush this vote before senators go on recess and get an earful from constituents.

Article on the BCRA from The Guardian

Obamacare is NOT “failing” (as Republicans ludicrously tried to proclaim even before ACA went into effect). The reality of the situation is that tens of millions more Americans have access to affordable healthcare. Medical bills are the number 1 cause of bankruptcy in America. And that comes from a lack of insurance.

The ACA is only “failing” in those states which intentionally refused to cooperate with the Medicaid expansion where the federal government paid 100% of the costs for 3 years and thereafter covered 90%. These same states also refused to set up state exchanges, forcing residents onto the federal one. No wonder they have problems – and all just so Republican governors and legislatures wanted to score political points at the expense of their citizens. People may try to say this is just partisan finger-pointing but unfortunately for their view it’s also true. In the states which cooperated and implemented the provisions of the ACA it’s working out wonderfully.

The ACA in its final form was not designed to lower medical costs because that was negotiated out of the bill by Republicans and Pharma lobbyists. But it did, in fact, reduce the rate at which America’s healthcare expenditures increased, and it created significant affordable relief for tens of millions who would otherwise continue without care until forced to show up in the ER with a catastrophic condition. An ounce of prevention is worth a pound of cure.


All major medical organizations have released statements in complete opposition to BRCA. I would like to quote from the statement from NORD (National Organization for Rare Disorders):

“First, the BCRA will cut hundreds of billions of dollars of Federal funding from the Medicaid program by instituting per capita caps and optional block grants. Medicaid is a critical lifeline to millions of individuals with rare diseases across the United States. … State programs for Medicaid home and community-based services (HCBS) waivers (1915 waivers) may also be jeopardized due to financial constraints.

Second, the BCRA … would phase out Medicaid expansion starting in 2020 and concluding in 2024, likely leaving many individuals with rare diseases without health insurance.

Third, the BCRA does not adhere to several of our principles relating to prohibiting discrimination against individuals with pre-existing conditions. … [The BCRA] would still bring back annual and lifetime limits and limitless out-of-pocket costs by allowing states to amend the Essential Health Benefits (EHB) through section 1332 waivers. These vital protections … would therefore be removed if a state opts out via a 1332 waiver.

Finally, the BCRA does nothing to incentivize healthy individuals to enter the individual market and help stabilize premiums by offsetting the cost of more expensive individuals.”

NORD Statement

I would also like to quote Judith Stein, the Executive Director for the Center for Medicare Advocacy (CMA):

“Never in 40 years of Medicare & Health care advocacy have I witnessed the kind of secrecy, and determination to take away health coverage we are witnessing today. A health care bill would strengthen coverage and delivery programs. This bill gratuitously weakens Medicare, decimates Medicaid, and guts insurance for over 20 million people.”

According to CMA, the BCRA includes:

  • The end of Medicaid expansion: Millions will lose coverage.
  • Medicaid per capita caps: Cuts would actually deepen over time.
  • Repeal of Medicare tax increase: Undermines Medicare’s finances.

CMA Statement

This is a statement I added to a change.org petition calling for a “Medicare for All” program:

“I am currently living only because of Medicare and Medicaid. I have Lou Gehrig’s Disease, and used to be a top-10% wage earner. The disease forced me into bankruptcy slightly before I was even middle-aged. The United States is the wealthiest country in global history, and we have much more than enough taxation right now to pay for guaranteed healthcare. Medicare functions at a much higher efficiency than any other private for-profit insurance, because it doesn’t have a powerful incentive to maximize profit by denying me the services I paid for. Even with “Medicare for all” as a basic level of healthcare, there is still plenty of market left-over for private insurance for things like elective procedures.

It’s time for our public tax dollars to be applied toward services for the public, not for the enrichment of some private corporation. The last year has seen a massive awakening in public attention toward healthcare. If you are not aware of this by now, it’s because you are not paying attention to the voices of your constituents. But we are, and are very much aware of your actions (or lack thereof).”

Please consider signing the petition. It’s not likely to be successful by itself but it will show Congress that there is significant resistance to the GOP plan and that the right move is to actually expand Medicare in order to ensure the right of healthcare for all citizens.

Change.org Petition Link

The BCRA is a hideous piece of legislation that severely jeopardizes the poor, the elderly, and the handicapped like me. It’s basically a tax cut for the hyper-wealthy that is paid for by the suffering and death, yes death, of people coping with ALS and other deadly conditions that were stricken through no fault of their own. It’s a serious threat to my life and the lives of many of my friends. That required me to make this political statement.

Thank you for watching and please vote carefully and diligently in 2018. It can change lives in a major way. In the meantime, please contact your senators immediately and urge them to vote “No” on the BCRA. Until next time, keep breathing easy.

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MAGIC Mitochondria (and more)

Audio Podcast (192kbps MP3 download)
(video at bottom of text)

Hello and welcome back to my podcast.

My name is Eric Valor and today I have a few different subjects to cover. I will cover the new ALS treatment recently approved by the FDA, the latest message from Hope Now For ALS, MAGIC in yeast cells, and trouble for stem cell therapies.

But first, I would like to make a personal announcement. Some of you may already know this, but I was recently accepted to the Academy of Neurology as a researcher. It’s not a huge deal but it’s nevertheless something I am proud to have on my CV.

ENV AAN Cert

Now, to business. My first item on the board is the first drug to be approved for ALS in 22 years.

In May of 2017, the FDA approved edaravone, also called Radicut or Radicava, for use in the United States. Edaravone was developed and originally approved for use in Japan in 2001 for protection from the effects of a type of stroke. Its MOA, or method of action, is as a scavenger of free radicals. These molecules have an unpaired electron in one of their atoms, making them extremely reactive with other molecules. The radicals at subject are called reactive oxygen species or ROS, produced as a byproduct of the mitochondria creating energy for the motor neurons. These molecules, when not properly controlled, cause significant damage to cellular structures. There have been many attempts to eliminate these ROSs as a treatment for ALS, but all previous attempts have failed.

There are some side effects resembling allergic reactions, from redness and itching up to anaphylaxis, which requires immediate emergency medical assistance or the person can perish). The incidence of serious adverse effects (SAEs) was low, with the most common, dysphagia or difficulty swallowing, occurring in 12% of patients. Milder adverse events occurred at the same rate as placebo.

Edaravone Adverse Events

The dosing regimen is 14 days of one infusion per day of 100 milliliters administered over one hour followed by 14 days with no infusions. Subsequent cycles are 10 days of infusions followed by 14 days without. Edaravone showed up to 33% slower progression in patients who were fewer than 2 years post-diagnosis, were still ambulatory, and could still feed, dress, and bathe themselves. Three out of four clinical trials of edaravone for ALS failed to meet clinical endpoints, but the fourth, when restricted to the PALS described previously, met its endpoints. What that means is that it seems effective only in people very early on in progression.

The second item on the agenda is the recent update which Hope NOW for ALS posted about its activity. On May 10, 2017, HNFA released a statement describing their May 1, 2017 meeting with key officials at FDA CDER. The statement also mentioned the approval of Radicava and how it is the first drug approved to treat ALS in 22 years. The main point of the HNFA statement was to indicate willingness by the FDA to consider updated clinical trial methods to make clinical trials more accurate and humane. It’s a hopeful message and indicates, along with the new approval of a treatment for ALS, that the FDA may be really changing how it sees and deals with life-threatening or fatal conditions.

ALZ Forum Logo

Third, the ALZ Forum has a nice article on mitochondria making MAGIC. In a study published in the March 1st edition of Nature, a team from Johns Hopkins University describe mitochondria in yeast cells untangling misfolded cellular proteins before tearing them apart for recycling the components. The process was termed “mitochondria as guardian in cytosol” or MAGIC. Aggregated or misfolded proteins which become tangled in each other are known to be torn apart in cellular machinery called proteasomes. Without mechanisms for breaking down these aggregated proteins they would clog the entire cell like the white of a boiled egg. You can see the same process happen as you fry your breakfast in the morning. That would be very bad for the cell and ultimately us.

In MAGIC, these same aggregated proteins are imported into the intermembrane area, a small space between the outer and inner membranes of the mitochondria. There the proteins are untangled from each other, then passed into the inner mitochondria where the individual proteins are chopped up. When heat shock proteins in the cytosol of the cell aren’t working properly this puts more stress on the mitochondria which are already very hard at work creating energy for the neuron. Think of it like hauling a heavy trailer up a mountain road in your car. Your engine strains under the load, getting hotter and pumping more smoke out of the tailpipe. The “smoke” from the mitochondria is the ROSs. The authors further reported that this process also happens in human cells. If those holds true then it would tie together two critical factors of neurodegenerative disease: protein aggregation and mitochondrial dysfunction. That’s would be an important finding as it would further elucidate the mystery of ALS, Alzheimer’s, and Parkinson’s.

In another story, again from the ALZ Forum, it appears that significant efficacy differences exist between clinical-grade stem cell lines and their research-grade counterparts. The differences may explain why some clinical trials fail. Two studies in the February 14 edition of Stem Cell Reports (study 1 and study 2) suggest that the outcomes could have been anticipated if the production lines were animal-tested the same way as in preclinical studies. The two subject studies looked at the unsuccessful trials by StemCells Inc. of spinal injury treatment using neural precursor cells. The company reported that the cells remyelination and motor recovery in mice with spinal injury.

But in two different trials with the same cells expanded using the Good Manufacturing Process (GMP) standard, required for production for use in humans, the cells failed to demonstrate efficacy. When the same lines were later tested in mice for the subject studies, they matured at about half the rate as the research-grade cells and largely remained as undifferentiated clumps. In one study about 4 percent of the grafted cells continued to divide and in some cases extended neurites into the surrounding tissue. Obviously injecting undifferentiated stem cells is a very bad idea and no two stem cell lines are identical. Together these studies provide strong evidence for preclinical testing of clinical-grade cells prior to use in humans.

Finally, another announcement: Beginning with this podcast (and retroactively back to the prior podcast) the video portion will be included at the bottom of the transcript. This will make viewing easier for my blog readers.

Thank you for reading and/or viewing. Leave a comment with your thoughts or any questions, and subscribe to get a notice in your email whenever a new episode is published. Until then, keep breathing easy!

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Cyborg Is As Cyborg Does – Reply All Interview

World’s First Fully-Functional Cyborg

Reply All Cyborg

I am the world’s first fully-functional cyborg! Need proof? My part in this Reply All podcast starts at 16:35.

This interview took place over about 3 weeks including one live telephone call and approximately 40 questions over email to which I replied both with text and individual MP3 files of the audio of my computer speaking each answer. It was a rather interesting experience and one that would certainly come in handy for any future interviews. Sruthi Pinnamaneni and Rick Kwan did a great job of stitching all of the questions and answers together to make a single coherent interview.

My desire was to demonstrate that life goes on after diagnosis and that there is still PLENTY that someone can still do despite full paralysis and being dependent on a ventilator. Hopefully other more newly-diagnosed PALS listening to the podcast can take a little inspiration to keep living and contributing your individual wonderful gifts to the world. Together, our voices are amplified and we can create the change we want to happen in the world.

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Neuraltus News!

Phase 2B Enrollment Open Now!

On Thursday, September 22, 2016, Neuraltus Pharmaceuticals announced the commencement of their long-anticipated Phase 2B for their lead candidate NP001. NP001 is a molecule that reverts macrophages (white blood cells) from an activated state where they hunt down and destroy pathogens and injured tissue to a calmer state where they nurture and protect other cells. I have blogged about NP001 extensively in the past. This trial follows up their Phase 2A trial which completed a few years ago. Unfortunately many of the participants in that trial are no longer with us, including my friends Rob Tison and Ben Harris with whom I launched the concurrent Oral Sodium Chlorite Project.

What It Is

This Phase 2B trial is to confirm the results of the post-hoc analysis of the responder class found in the Phase 2A. In that analysis, Neuraltus discovered that patients who were given the highest dose (2mg/kg body weight) and had elevated levels of pro-inflammatory proteins called IL-18 and C-reactive protein responded quite favorably to the drug. If this Phase 2B returns the expected results, NP001 would have a strong case for the same accelerated approval that FDA just granted for the Sarepta DMD drug eteplirsen. We could have the first new treatment since riluzole and the first truly effective one.

Sign Up Now!

I encourage all PALS to use the Clinical Trials tool on my website, provided by our friends at Antidote. It is very important that this trial is fully enrolled as soon as possible so that it is quickly completed and NP001 gets a shot at getting on the market. That is the best chance for it to get to ALL the PALS whose lives could be extended. We did it for the Phase 2A and can do it again for the Phase 2B.

This is a very exciting moment in the history of ALS.

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Guest Blog: Me!

SRG Research News – First A Little Background

As most of you know, I started SciOpen Research Group as a way for me to be able to fire actual bullets in the battle against ALS (well, actually metaphorical, but you get the idea). Our first project failed to extend life in the classic ALS mouse model so we retained the money raised to conduct the planned second part of that experiment. We had another project already in the research pipeline waiting to take the next step in development. For two years SRG was working on creating a novel molecule which would treat the desired pathway without becoming toxic like the reference molecule does at therapeutic doses. Suddenly we had the opportunity to collaborate with researchers already investigating the same pathway, albeit in different conditions (watch the video announcement), with their own library of candidate molecules.

Our collaboration’s first phase is to create a novel transgenic mouse species which represents a 100% drug efficacy in order to be a proof of concept. The project should run through the last half of 2016. As you will see below, a study was recently published which shows that SRG is definitely onto something. Our target protein is significantly elevated in human patients, and that targeting it brings positive results. The study is great indirect support of our project’s goal.

And now, the guest blog featuring myself!

Good News For Our Latest Project!

A recent report published in Science magazine strongly suggests that SciOpen Research Group is onto something with its currently ongoing study of necroptosis in ALS. Necroptosis is a “cousin” of apoptosis. In contrast to apoptosis, which happens regularly in the body, necroptosis is a form of programmed cell death which happens under inflammatory conditions and in which the components of the dead cell spill into the extracellular space. The spilling of the cellular components trigger a response in which immune cells are recruited to the area. Necroptosis is known to be a driver of both genetic ALS and sporadic ALS.

The subject study is not a direct support, in that it was looking at how the optineurin protein contributes to ALS. However, the results showed significant increase of the MLKL protein in human patients and that elimination of the RIPK3 protein or inhibition of RIPK1 had modest but nevertheless positive effects on survival of the SOD1 mice (along with positive biological evidence). This suggests that SRG is on the right track with its MLKL study. We believe that acting on MLKL will have a stronger effect without disrupting other cellular functions which depend on RIPK3 and/or RIPK31 (MLKL is involved only in necroptosis).

This study is YOUR study. It would not be position without your support. SciOpen Research Group is the world’s first fully functional “guerilla biotech”. We function only with your support and study pathways other research organizations either miss or ignore. And we can do it for much less because we are purely volunteer and have no overhead. 100% of your donations go directly to research. To support us you can make a tax-deductible donation (USA residents only) by going to our Donations page, purchase some SRG Gear, and/or go shopping on Amazon Smile and name SciOpen Research Group as your charity of choice (we are a registered and approved nonprofit under IRS 501c3). We work on ALS for you, the ALS Community, because we are part of the ALS Community. Help us continue our novel research into eradicating ALS.

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On Masitinib

I may have to revise my opinion of masitinib (which would not upset me in the slightest). Some of my readers may know that I have not been very optimistic about the probability of the drug being an effective treatment option for ALS. It’s been around for some time as a veterinary drug. But the company AB Science is developing it for ALS and other conditions.

Masitinib:

Preclinical information appears encouraging, although the study has a few issues. The rat model is not like the mouse model and is not very suitable for a survival study. The survival data are also very difficult to interpret due to the curious use of different numbers of animals in each cohort. I will defer to the opinions of my more statistics-inclined members (please feel free to comment!). The cellular data have a similar issue because they were taken in vitro rather than vivo. Nevertheless, it’s encouraging and we can hope for quick human trials.

The press release.

The study (open access!).

And the first USA patient gets approval for Compassionate Use!

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A Boost For Joost

“There are two things people take for granted every day: Time and health. When you lose those, then you wake up.”
— Joost van der Westhuizen

In 1994, Nelson Mandela became the first black President of South Africa. That same year, South Africa also hosted the Rugby World Cup. In that tournament, the home team, the Springboks, overcame considerably unfavorable odds and decades of international isolation (due to the government’s policy of apartheid) to win the Rugby World Cup. This is widely considered one of the greatest moments of South African sporting history and was the basis for the 2009 film Invictus. On that team was a young scrum-half named Joost van der Westhuizen.

In 2003 Joost retired from rugby. By then he was a superstar of South African rugby, having more caps than any other South African player. In 2011 the rugby world suffered a blow with the news that Joost had been diagnosed with ALS. Rather than retreat from the world, Joost decided to make a difference in the lives of people also coping with this dread diagnosis. He formed the J9 Foundation to educate the general public and medical practitioners about ALS, grow ALS research in South Africa, and to aid other South African PALS.

Joost’s story has been made into a documentary called “Glory Game“. In addition to the trailer, you can read about the movie here. The film has done well in South Africa and is now going to be shown first in Vancouver, British Columbia, on April 10, 2016 and in Los Angeles, California, on April 15, 2016. I urge all my friends in those areas to go see it. I have seen it and it’s simultaneously hilarious, upsetting, and uplifting. It shows the courage and determination which made Joost van der Westhuizen such a force on the rugby pitch. I am proud to call him and the Director of the film, Odette Schwegler, my friends.

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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.]