Tag Archives: soapbox

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Guest Blog – ALS Finding a Cure

Watch These Straight-Talk Videos from ALS Finding a Cure

I’ve invited my friend Dr. Merit Cudkowicz at ALS Finding a Cure to share some important news about what they are doing to help ALS patients and their families learn all they can about the disease. Please read and share.

Every 90 minutes or so, someone new is diagnosed with ALS.

A new patient and his or her loved ones are overwhelmed by a whirlwind of emotions. They are shocked, confused and desperate for information.

So many questions … What exactly is happening to me? What’s next? How long will I live? Will I be able to drive? Or shower? Or go to the bathroom by myself? What happens when I can’t do these things anymore?

At this critical time, patients and caregivers need plain, simple, factual information from experts who care.

That’s why ALS Finding a Cure has created a series of videos to help ALS patients and their loved ones better understand the disease and the resources and support that will be needed as it progresses.

You get facts and insights from the people who know best — individuals living with ALS, their spouses, healthcare providers and professionals. Each of the eight videos brings an honest and straightforward perspective on the impact this disease has on the lives of those touched by it.

We have strived to cover the topics that a patient, caregiver or loved is most concerned or curious about:

  • Overview describes, in layman’s terms, exactly what ALS is and what it will do
  • Resources explains the information, equipment and support networks available to help manage the disease
  • Lean In encourages individuals to realistically prepare for, embrace and take ownership of ALS
  • Relationships provides guidance on maneuvering through the disease and the importance of a journey-long support system
  • And four other videos – Nutrition, Mobility, Hygiene and Breathing & Communication – offer straight talk and on each of those vitally important topics

The video series is an extension of the primary purpose of ALS Finding a Cure, which is to find and fill in the critical gaps in ALS science so that researchers can – one day soon, we hope — develop treatments for and a cure to this disease.

But we are also committed to empowering patients and their loved ones, right now, to be proactive about understanding and managing ALS. These videos are intended to be a resource so that patients and others can make informed decisions throughout their journey.

“Lean in” is more than just the topic of one of the videos; it is the overarching theme for this series. As Eric Valor’s inspiring life makes evident, being proactive is central to understanding, coping with and owning ALS.

We hope you will find these videos to be an effective resource for anyone impacted by ALS.

Dr. Merit Cudkowicz
Chief of the Neurology Department at Mass General
Chief Medical Officer, ALS Finding a Cure

www.alsfindingacure.org

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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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ALS-New-Drug-New-Scam? – Redux

So it looks like the entity behind “ALS New Drug” is back, this time with a new website host. The site has been changed so that every page begins with erroneous whining about how ALSUntangled supposedly ended some kind of “charitable funding”. First, that person, persons, or organization has no status as a charity in any country. Second, ALSUntangled has taken no stance on the subject whatsoever because the entity refuses to cooperate whatsoever by revealing any information about itself or the product it promotes.

Let me explain the facts of the situation:

Back in July of 2015 the “als-new-drug.com” domain was purchased by a man in Great Britain named Michael Richards. Apparently around September 2015, the website was put up. A [non-exhaustive] search of the Internet and PubMed reveals no Michael Richards from Essex, Great Britain, involved in neurology or ALS.

In April, 2016, the site was brought to my attention by another PALS. I read through the site and read a lot of claims backed up by absolutely no objective information in the form of links to studies involving the drug in question, no objective or clear information about what the drug actually is or how it works, and no identification of the inventor(s) or the entity promoting the drug. A deep Internet and PubMed search for RCH4 or the “scientific” name given revealed absolutely no hits (very unusual and highly improbable for a real drug that has supposedly been in development for many years). In fact, absolutely no objective information exists about this drug except for the claims made on the website.

After failing to find any corroborating information, my Internet domain information lookup results, and my decades of professional experience identifying Internet scams, I made the initial assessment published on my blog in the post titled “ALS New Drug New Scam?”. Because this had been brought to my attention by another PALS who was considering taking this “treatment” and because other patients were apparently already using it, I felt it was urgent to publish a warning that something was not right about this. I have been publishing this blog for exactly this reason since 2009 and I am known for my understanding of the neuroscience and pharmacology of ALS. That’s one of the reasons I was invited to join the ALSUntangled Review Group.

After I published my initial assessment – based on all the available objective information – I contacted Dr. Bedlack to ask if he knew anything about the subject. He informed me that it was on the list of Open Reviews (I don’t keep the list updated in my memory). Because it’s quite a long list and Dr. Bedlack is busy running a major ALS clinic, he asked if I would be interested in taking the lead in gathering information for this project and writing an initial draft report (something I have previously done multiple times for ALSUntangled). Of course, I agreed to assist. There is no title of “Lead Investigator” for ALSUntangled but I used that in email and forum postings to communicate with others because it’s a more succinct and convenient identification of my association with ALSUntangled. I then sent a request for information to the entity promoting RCH4 at the AOL email address given as contact on the website and began asking for patient experience and information on various forums dedicated to ALS.

The questions I sent to the contact email was the standard set sent to every promoter of an alternative treatment option, plus a few of my own customized to this case which were relevant to the investigation. The questions are:

  1. What exactly is this drug and how did you discover it?
  2. How does it work?
  3. What is published on the mechanism?
  4. What pre-clinical ALS data are there?
  5. Are these pre-clinical ALS data published?
  6. How many patients with ALS have taken this?
  7. What are you measuring in patients with ALS that take this?
  8. What happened to those measurements?
  9. Over what period of time and how often are measurements made?
  10. Has anyone had any side effects from this drug?
  11. What percentage of people who take it have any side effects?
  12. What are the most common side effects?
  13. What are the most serious side effects and how often did these happen?
  14. How much do you charge patients for this drug?

Additionally:

  1. If not why not and how are you capitalized?
  2. Will you identify the members of your group so that their qualifications can be examined?

These are standard questions that ALSUntangled asks of EVERY promoter of an alternative treatment option. They are intended to gather relevant data so that a scientific evaluation of the substance can be made, and I included the financial question so patients would have some information about the possibility of long-term access. The promoter is always free to not answer any particular question. The entity behind RCH4 reacted instead with hostility – as if the questions were attacks on their very character. Moreover, apparently they have patients sign nondisclosure agreements before any distribution of the drug begins so that automatically increases the difficulty of discovering the truth of the subject. These two facts, along with the lack of any objective information made available on their site or to prospective clients inquiring about it, only reinforces my personal initial assessment that something is very wrong with this entire program.

The entity says that ALSUntangled and/or I made an allegation of some criminality on their part. In fact, ALSUntangled has made no statement of any kind about RCH4 and I merely opined based on all the [still paucity of] currently-available information and my many years of professional training and experience. The entity says I have no medical credentials. This is true, but neither does it. I do have years of dedicated learning and am recognized as an expert on the subject of ALS and treatment options for it. The entity says I have no experience with drug development. This is untrue, as I have experience both in aiding others’ programs and in developing my own via my research organization, SciOpen Research Group. I also have quite a bit of knowledge of the development process from my experience with and founding of WideTrial, my experience with and founding of Hope NOW for ALS (both organizations deal with improving clinical trials and involve dealing with regulatory authorities and pharmaceutical companies). I also have nearly a decade of experience in advocacy and awareness in the ALS space. My record is impeccable and very publicly transparent. I invite the entity promoting RCH4 to exhibit the same public transparency.

The entity says that my blog post warning patients away from whatever RCH4 is somehow cost them their “charitable funding”. I was never contacted by anyone representing themselves as being affiliated with the RCH4 entity. While I realize that I have a reputation in the ALS Community of being knowledgeable, I highly doubt any funding organization would base its decisions on my personal opinion alone. But if for some reason it did, there was obviously very little faith in the RCH4 entity to begin with.

To recap:

  • In July 2015, a domain called “als-new-drug.com” was created and shortly thereafter the website promoting RCH4 was put up on the same URL;
  • In April of 2016, I was informed about it and did a personal search on RCH4 and the entity behind it;
  • After failing to find any objective information verifying any of the claims on the website or the identity of the entity and/or supporting scientific staff (a situation that persists to this moment), I posted my findings on my personal blog;
  • I then communicated with Dr. Bedlack about RCH4 where he asked me to gather information for an ALSUntangled review, including sending the standard questions to the entity promoting RCH4 and asking PALS claiming to be taking RCH4 about their experiences, an activity I began immediately;
  • I very quickly learned that PALS were required to execute nondisclosure agreements with the prior to being provided RCH4;
  • I received a response from the entity via comment to my blog post full of overly-dramatic wounded pride and a pledge to not cooperate with the ALSUntangled investigation;
  • Patients currently using RCH4 were warned by the entity to not cooperate with the ALSUntangled investigation;
  • In an effort to smooth any hurt feelings, I recused myself from the investigation – to no avail;
  • Shortly thereafter, the website disappeared and the entity apparently began informing patients that continued supply was in jeopardy;
  • I received hateful comments from a few patients – including death threats – demanding that I take down my post (as if that would suddenly change anything?);
  • The website returned, blaming ALSUntangled and/or me for ruining a “charitable treatment program”.

I made my initial personal assessment based on my many years of professional experience and more recent scientific knowledge, and upon previous public lectures by Dr. Bedlack on how to spot treatment scams. I was not acting on behalf of ALSUntangled but entirely on my own. Afterward, I was asked to gather information for their own review – information which would have been reviewed and discussed before a report is published by the entire group which includes many well-known MDs and PhDs involved in ALS research and treatment. The amount of available objective information has not increased one bit since my initial assessment. I would love to be proven wrong but that would require objective and verifiable information. The RCH4 entity is not only not helping, they are actively resisting all efforts at learning any facts about RCH4. Facts are not just unsubstantiated claims on a website. Facts are independently verifiable objective information. All scientists and doctors, retired or not, understand that they have a duty to first provide scientific rationale and preclinical data about their drug along with a clear description of its chemical makeup before providing it to patients. That is a basic fact about drug development which apparently I know and the RCH4 entity does not.

If the RCH4 entity wants my personal assessment and warning to PALS taken down, they can very easily provide me and/or ALSUntangled with the answers to the questions sent, and allow patients to communicate about their experiences. Until then, my personal blog post will stay up as a warning to PALS to not inject into their bodies an anonymous substance sent by an anonymous source. As stated earlier, I would love to be proven wrong, and indeed welcome it. However, everything so far has proven me right.

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ALS-New-Drug: New-Scam?

UPDATE – June 29, 2016: Apparently the site is back and whining that ALS Untangled is somehow responsible for them losing “charitable funding”. ALS Untangled had nothing to do with RCH4 except having asked me to take the lead in gathering information about it from the provider and from patients. My initial assessment was indeed made quickly but was based on all the information currently available, and was made based on my decades of expert professional experience in identifying Internet scams. There is currently zero scientific evidence for any of the claims made of that website and the provider has been given multiple ongoing opportunities to back up the claims with objective evidence. I never made any claim of “criminality” – rather I feel that something is not right and that PALS should avoid injecting themselves with a completely anonymous substance.

UPDATE – May 12, 2016: Apparently the domain owner, Michael Richards, pulled the site and folded up his tent. No idea what he told “his patients”. I have absolutely no guilt over this. If all it took was one person questioning the veracity of that RCH4 whateveritwas to make him pull up stakes, then there was nothing worth putting hope into in the first place.

UPDATE: If anyone has attempted to obtain this drug and have retained emails or postal letters, please contact me so I can investigate further.

BREAKING NEWS! (April 14, 2016)

I was just alerted to a website advertising a new treatment for ALS (http://als-new-drug.com – text provided for reference but no link for reader safety). The site purports to represent a “group of retired scientists and doctors” in Europe who “discovered a previously unknown protein … which promotes ALS” and “designed a drug which safely stops production of the problem protein”. The site provides no references for the protein and a web search of the name given provides no relevant returns. The same goes for the given name of the drug. Neither is any information given about exactly who comprises this group so that their qualifications may be examined.

Domain information is:
Domain Name: ALS-NEW-DRUG.COM
Registrar WHOIS Server: whois.publicdomainregistry.com
Registrar URL: Updated> Date: 2015-09-04T02:32:35Z
Creation Date: 2015-07-05T10:28:14Z
Registrar Registration Expiration Date: 2016-07-05T10:28:14Z
Registrar: PDR Ltd. d/b/a PublicDomainRegistry.com
Registrant Name: Michael Richards
Registrant Organization: Not Applicable
Registrant Street: 56, Amanda Close
Registrant City: Chigwell
Registrant State/Province: Essex
Registrant Postal Code: IG7 5JG
Registrant Country: GB
Registrant Phone: +7.981150350
Registrant Email: privacy@wzukltd.com

This raises a number of red flags and identifies it as a likely scam:

  1. There’s no identification of the “scientists” behind this
  2. There are no links to publications about the protein
  3. There are no links to publications about this new drug.
  4. The website is cheap, poorly-designed, and unprofessional.
  5. The website is registered to an individual in Great Britain with an obscured contact email address.
  6. The website is clearly designed to create anxiety in the reader about “missing out”, thereby making the reader immediately more amenable to the presumably eventual sales pitch for the “immensely expensive” drug.

Without any evidence of efficacy, safety, or even the ingredients of this substance, I would very very strongly urge everyone to ignore this website completely.

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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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Icebreaker

My readers know that I have serious differences with the ALS Association (ALSA). However, my promise to deliver the truth (though bathed in hope in the delivery) cuts both ways. When something good happens, no matter who is behind it, I must give kudos to the deserving.

The 2014 social media phenomenon known as the Ice Bucket Challenge marked a seminal moment in the history of public awareness of ALS and in funding for research. Since then, PALS have been demanding that ALSA actually use that money rather than sitting on it. It now appears that ALSA is finally indeed mobilizing a little of that money (about $3M or 2.5%) on two wise and popular targets. This is good news, although there is a slight catch…

ALSA is helping fund a Phase 3 of the Cytokinetics drug tirasemtiv and a Phase 2B of Neuraltus’ drug NP001. Tirasemtiv is a muscular activator, meaning it causes the muscles to react more strongly than normal to a neural input. Tirasemtiv does nothing to halt the death of the motor neurons, but it can let PALS have more independence for longer than without it. NP001 is a highly purified and pH-balanced form of sodium chlorite that reverts the chronic inflammatory attack on the neurons back to a pro-growth state. Some of you might remember our dear departed friends Rob Tison and Ben Harris who experienced remarkable results during the Phase 2A. Now we know why: Based on inflammatory biomarkers discovered in post-hoc analysis, Neuraltus believes it has found a responder subgroup and is restricting the Phase 2B to those patients. I expect very good news from the 2B.

[UPDATE (07-13-2015) From my friend Jenica Lancy at ALSA GoldenWest: Today, The ALS Association announced its support of 58 new research grants totaling $11,621,638 to find treatments and a cure for ALS. The research awards announced today include investigator-initiated grants, drug development contracts, Milton Safenowitz Postdoctoral Fellowships and support of the NEALS/TREAT ALS™ Clinical Trials Network. You can see a full list of the grants here.]

Now for the catch: What ALSA is really doing is funding operations at one of the clinics which promote and direct funding toward ALSA. Both trials will be conducted by that clinic (the excellent Forbes-Norris ALS Clinic in San Francisco).

However, the fact remains that ALSA is supporting two very promising clinical trials. Some of us might wish they would do more, sooner, but they are moving in the right direction. I believe the proper response should be “Thanks! Keep it up!”. Let’s all applaud ALSA and encourage further progress along this path.

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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.