Tag Archives: drug development

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Disaster and Development

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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Awful Truth: What’s Old Is New – Again

ALS-AwfulTruth18

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

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

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

Please enjoy my renewed video message.

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MAGIC Mitochondria (and more)

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

Hello and welcome back to my podcast.

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

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

ENV AAN Cert

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

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

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

Edaravone Adverse Events

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

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

ALZ Forum Logo

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

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

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

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

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

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

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Cyborg Is As Cyborg Does – Reply All Interview

World’s First Fully-Functional Cyborg

Reply All Cyborg

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

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

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

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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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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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On Masitinib

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

Masitinib:

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

The press release.

The study (open access!).

And the first USA patient gets approval for Compassionate Use!

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ALS-New-Drug-OLD-Scam

So apparently this is indeed not something new… While looking up the domain information for our friendly RCH4 website (which expired yesterday, July 5, 2016) to see if it would be updated, I stumbled across an older version (2011) of the site from the same person. This time he called himself “Michael Curram” of “Rixbiotech”. The address is exactly the same as “Michael Richards” who put up the RCH4 website, and the contact email given on this older website is the same – “alsnewdrug@aol.com”.

You can see the front page of the older website which was named “als-amyotrophic-lateral-sclerosis-a-new-drug.com” (history provided by Web Archive’s Internet Wayback Machine). Unfortunately none of the other pages were picked up from the site. But the substance described by this site is different from that of RCH4, apparently being some kind of way to correct a suspected autoimmune disorder that would be applicable to a variety of conditions.

The language sounds suspiciously like that of the substance “TDI-846” which was developed by the ALS Therapy Development Institute and successfully treated the SOD1G93A mice. It’s an antibody specifically for rodents and is available on the market for testing purposes only – NOT for human consumption. ALSTDI has more recently developed a human version which they are putting into human trials, but whatever this website was promoting isn’t it. The website promoting RCH4 made it sound to me like a knock-off of GM6, the peptide manufactured by Genervon. Michael Curram/Richards might well be trying to create a treatment for ALS, but he is clearly an amateur and is not going about it the right way. This makes me extremely wary and I urge all PALS to stay far away from anything this guy is promoting.

Domain information is:
Domain Name: ALS-AMYOTROPIC-LATERAL-SCLEROSIS-A-NEW-DRUG.COM
Updated Date: 2013-11-29 12:27:20
Creation Date: 2008-12-29 17:12:46
Registrar Registration Expiration Date: 2014-12-29 17:12:46
Registry Registrant ID:
Registrant Name: Michael Curram
Registrant Organization: Rixbiotech
Registrant Street: 56 Amanda Close
Registrant City: Chigwell
Registrant State/Province: Essex
Registrant Postal Code: IG7 5JG
Registrant Country: GB