Tag Archives: guest

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Reddit AMA Guest Appearance

Reddit Tag-Along

reddit-logo

On Friday, November 18, 2016, I participated in a Reddit AMA as a co-guest in support of my friend, Jef Akst. Earlier this year she published a book titled Personal Trials: How Terminally Ill ALS Patients Took Medical Treatment Into Their Own Hands (available on amazon.com in both Kindle and paperback) about the Oral Sodium Chlorite Project I created along with Rob Tison and Ben Harris, and our journey through the DIY drug experience. Reddit asked her to do an AMA about the book and she asked me to tag along for the session to give the ALS patient perspective and as one of the subjects of the book.

It was my first time ever doing this and it was exhilarating. For two hours, Jef and I were furiously typing away trying to keep up with the deluge of questions. In fact, I am still going back and answering late questions right now. At first I was a little nervous about facing a bunch of trolls and kooks, as the Internet appears full of these days. But the questions were all quality and reflected a desire to actually learn something about the subject.

I am grateful to Jef for writing the book, telling the story of patients driven to find their own solutions to untreatable diseases. And I am extremely grateful to Reddit for giving us this opportunity to share a taste of the experience with others who may have never previously heard of ALS before today. And thank you again, Jef, for inviting me to help her tell the story.

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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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Guest Blog: Me!

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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Cross The Blogosphere

I would like to direct my readers to the blog of my friend Dan Munro. Dan writes, quite intelligently, about the healthcare space with a focus toward Information Technology (IT). As healthcare goes more and more digital in terms of research, treatment, and record-keeping, IT is critical to that development. As with the Internet boom, many gizmos and techniques will rise and fall away. Dan does a great job of sorting through it and his blog is worth a bookmark.

You might remember Dan from an earlier post featuring me. The earlier post was about the oral sodium chlorite project I created. Today Dan posted Part 1 of a 2-part series on “Biohacking”. This is the result of his own research and some discussion we had about a project in which I am involved. Part 2 comes out next week, authored by yours truly, so stay tuned!

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Thomas Ohlson – Guest Blog

I would like to introduce to my readers my friend Tom Ohlson. He was diagnosed the same year I was and we are both “venterloquists”. Although the circumstances suck, I am glad to have met him and am excited to present the following as a guest-blog entry. I feel exactly the same way.
Last week, on the 4th of July, scientists announced they may have discovered the Higgs boson. Frankly, I think if it were not a holiday and if there was nothing else newsworthy taking place, this announcement would have still have received little notice despite the significance of this discovery. This is unfortunate for a myriad of reasons, not the least of which is how American scientific curiosity has been replaced by a need for superficial sensationalism. Dont take my word for it–just look at the amount of major network news airtime devoted to the Higgs boson announcement vs. the Tom Cruise/Katie Holmes divorce announcement. Still, the discovery of the Higgs boson sounded a clarion call across the planet–great discoveries are still out there to be made and with them, comes great hope.

As we celebrated the birthday of the United States that day, it was evident just how shallow our country had become. In a nation founded by citizen scientists and deep thinkers such as Franklin and Jefferson, we now look at science like Dorothy and her companions viewed the Wizard of Oz–a mysterious force that sometimes grants wishes. Instead of wanting to know how our world works, we only want to know that it works. Instead of learning the issues ourselves, we allow the media, politicians, and PR types to influence our decisions. We have neither the time, nor inclination to learn even the simplest science–unless we learned in school, let others figure it out and well just go along with what they say. To tell the truth, I was just like most Americans until recently. If not for my diagnosis of ALS, I probably would have remained a Monday morning scientist Hey Bob, did you see the eclipse last night? Solar, lunar, whats the difference? Did you hear about Tom Cruise and Katie Holmes? If its not worth watching on the Discovery Channel, its not worth knowing. Sad to say, our interest in science has been reduced to how entertaining Hollywood can make it.
Maybe, thats not a total loss. As a grad student, I once taught a college course in American Government. The course professor insisted each student have a daily subscription to the New York Times. Granted, many in academia would argue that the Times is the gold standard for daily news. However, these were 18- and 19-yr. old undergrads, more interested in the next keg party than the minutia of daily politics. Knowing this, I argued that the students would be much more likely to read the paper if it had simple articles like those found in USA Today–simple was better than nothing. Nevertheless, my argument fell on deaf ears and I can only imagine all those New York Times subscriptions that found the recycling bin before a single article was read. That said, I suppose a glossy, but hollow science segment on TV is better than nothing.
In 2005, I was told I had a terminal illness for which there was no known cause, no viable treatment, and no cure. That was it. I was only in my early 40s, but I was a dead man walking. Most Americans believe the mantra that if we eat right, exercise, and live within moderation, we will live to a ripe old age. Despite following this instruction manual, I was broken and nobody had replacement parts. I stopped seeing my neurologist, because all he would do is shake his head and mutter how devastating ALS was, and I buried my head in the sand hoping it would all just go away. Didnt work. As my body slowly rotted away, I desperately looked for answers. Unfortunately, what I discovered was disheartening. No diseases were being cured. Big Pharma had everything geared toward marketing medical conditions. There were billions to be made in drugs and treatments to manage disease. Curing them would eliminate all that profit. ALS only effects 30,000 Americans, so developing drugs to treat it offered little financial incentive. I was screwed. So, I started to look outside the pharmaceutical industry for answers. In order to understand what I was looking for, I had to rewire my heretofore keg-seeking brain for science. Not an easy task, but these days I am devoid of a job and a life in general, so I have had the opportunity to do something I never actively did before–study.
The good news–I have found scientific disciplines of which I excel at studying. The bad news–none of them relate to ALS. However, (and this is really the point of this writing) like those undergrads I wanted to have read USA Today, I am now at least able to discern trends and discoveries of significance and I will say this–we are on the verge of a medical revolution, the likes of which have never been witnessed by the human race. That said, we are only at the beginning of this revolution and many of us may never live to reap its benefits. Nevertheless, I have no doubt that a child born today will never have to fear ALS, MS, Alzheimers, and most other diseases. Thankfully, Big Pharma has met its match in an even more powerful industry–insurance. The insurance industry has been getting killed by Big Pharmas string em along policy. The insurance industry needs us to either die quickly, or live healthy. Since western society wont tolerate early death, healthy lifestyles are the only option. So, we see the sudden advent of regenerative medicine, stem cells, gene therapy, etc.
Stem cell therapy alone, is developing at a phenomenal pace. Seven years ago, the best one could hope for was an offshore procedure, which simply transferred cells from one body part to another. Huge controversy surrounded the use of embryos, which were considered the most viable source of stem cells. Today, a patient can use their own cells, which can then be manipulated into the needed cell type, thus avoiding any ethical or rejection issues. Countries free from regulatory and Big Pharma constraints such as Israel and China, are developing stem cell technologies to rival our own. Entire organs are now being created in the lab, paralyzed mice are walking again, and some patients are seeing complete reversals of their medical conditions–all from stem cells. One can only imagine what we will see from stem cells seven years from now!
Seven years ago, there was only one drug available for ALS patients; a drug which prolonged life expectancy a whopping 2-3 months! These days, there are a number of promising drugs currently in clinical trials, with dozens more being looked at. Diaphragm pacers are now prolonging the need for invasive breathing procedures. Reports of ALS patients improving after receiving stem cell treatments both here and in Israel are generating a buzz in the ALS community. Did I contribute to any of these breakthroughs? Of course not. Did my newfound interest in science make a difference? Yes, it saved my life. I cannot overstate that enough. By understanding on at least a very basic level where the science was taking us, I found hopeand hope is what sustains me.
So, what does the Higgs boson have to do with all of this and whats its significance? Stephen Hawkings bestseller, A Brief History of Time was supposed to be a laymans guide to understanding these types of theories, but every attempt of mine to read his book resulted in me giving up after two chapters. I am obviously the last person who should attempt to explain the Higgs boson to anyone. In any event, heres my attempt. The Higgs boson, also known as the God particle, was first predicted almost 50 years ago. The boson and its corresponding field are the final pieces of one of the most successful physical theories in history–the Standard Model, which encompasses all of nature’s fundamental particles, and every fundamental force apart from gravity. The Higgs boson is believed to be a catalyst for the Big Bang and the creation of the universe. That said, the real significance for us mortals who cant fully grasp all of this is that science persevered. Despite all the crap we now occupy our lives with, despite the dwindling interest in science, despite monetary disincentives, scientific curiosity still exists. Those noble souls who toil away far from the spotlight, let us know that the secrets of the universe are still attainable and through their deeds, hope springs eternal.

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Guest: Persevering On NP001

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

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

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

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

FRS for all 37 reporting

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

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

With side effects:

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

FRS for those reporting side effects

Without side effects:

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

FRS with no side effects

Comparison: 100.4% mean improvement

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

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

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