Tag Archives: cheerleader

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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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Reply All – ALS Reversals

Reply All Podcast – The Reversal

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As many of you may know, Dr. Richard Bedlack has been investigating a very rare phenomenon known as “ALS Reversal” where the normally inevitably fatal disease can stop progressing and even where the patient recovers slightly or nearly fully. Over the past few months Dr. Bedlack has been interviewed for a podcast called “Reply All” (I know the timing because I was also being interviewed for supporting material). The podcast is worth a listen, and you can get read the transcript at the Reply All website.

This is more good exposure for ALS awareness. Thanks to Dr. Bedlack and to Reply All for a great story.

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Quora Top Writer Of 2017!

WOW! – Quora Top Writer Of 2017!

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I just received an email today notifying me that I have been named a Quora Top Writer Of 2017! My contributions are tightly focused in the topic of Amyotrophic Lateral Sclerosis (which I created on Quora) with some attention in the broader topic of Neurodegenerative Diseases, along with a few answers in the topics Science, Physicists, and Stephen Hawking (to give a long-term patient’s perspective on some questions asked about the Professor, including one asking how he fathered children where my answer has 1.4 million views and over 20,000 up-votes – the Quora equivalent of a Like). I have to thank my friend Laura Copeland for introducing me to and getting me involved at Quora. Laura and I met in 2011 when she interviewed me for a story in my local newspaper. She and I remained friends ever since.

Quora is probably the best place to go for answers to questions about anything from science to global social issues and politics to personal hobby interest (maybe I should start a Surfing topic..?). It’s a highly erudite place, especially for a social media site and has astonishingly remained so for many years. Quora is a place where trolls are not tolerated and from which is almost totally free.

I am quite flattered to receive this distinction and am happy that my contributions have been deemed useful for the many people who have read my answers and those who have engaged in enlightening discussions after. It’s been a wonderful experience so far, where I have been able to definitely expand global public awareness of ALS/MND is a positive and engaging way. I am thankful for the opportunity and for the response. I look forward to many more years of engagement and enlightenment.

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Top 10 Quora Writer

Top 10 Most Viewed Writer On Quora

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Wow! I just stumbled across my full Writer Rankings on Quora (http://www.quora.com). Based on number of views in the last 30 days, I am a “Top 10 Most Viewed Writer” in the following Categories:

  1. Stephen Hawking – #1 with 96,450, #2 has 4,764;
  2. Amyotrophic Lateral Sclerosis – #1 with 86,333, #2 has 194;
  3. Scientists – #2 with 83,523, #1 has 104,262;
  4. Physicists – #2 with 83,290, #1 has 359,824;
  5. Neurological Diseases and Disorders – #3 with 444, #1 has 1,222;
  6. Science – #10 with 83,114, #1 has 234,073.

Also:

  • Neurodegenerative Diseases – #23;
  • Neurology – #31;
  • Charities – #39.

This is very humbling. It’s also quite gratifying to find out that my attempts to share what little knowledge I have gained over the years are deemed useful by others. That’s all I want to do by providing answers on Quora.

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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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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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TransFatty Lives – a film review

Last Saturday evening I watched TransFatty Lives and was stunned by the unique method of simultaneously telling two stories. The first story is his slow but inevitable descent into total quadriplegia following a diagnosis of ALS and the second is writing a time capsule letter to his son to explain his absence and inability to participate deeply in his son’s life. The film was scattered with amazing images showing POB’s delightful deliberate eccentricity and with scenes both hilarious and disturbing. Some scenes were personally disturbing as I remembered my own experience with that phase of decline. Others were colorful and outrageous in a way only Patrick could make them.

TransFatty Lives is a perfect film for seeing the effects of a fatal diagnosis on a young hedonistic man. As he faces each step of decline he becomes a little more introspective and gains more awareness of the value of the little moments that give life its value. How POB takes the viewer along reveals his genius – you don’t know you have learned something until the next scene begins.

Even more than “The Theory of Everything” or “You’re Not You”, “Transfatty Lives” is the most important film involving ALS. The faithful and honest treatment of both the horror and triumph which is ALS, and the amazingly creative style of POB, makes this a must-see for all PALS and CALS and their families. It should also be widely promoted for all people worldwide. Even for those for whom ALS is just a disease named for some baseball player, this is a wonderful film about human trial, triumph, survival, and love.

This film is amazing to experience. It is much more than a simple documentary. I easily rate this 5 stars, two thumbs up, one poop, etc. Rent or buy this film immediately and have a viewing party.

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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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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.

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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.