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.
I was recently alerted to a blog called Science-Based Medicine which had a post that was rather critical of me. The post was written by David Gorski, a managing editor of Science-Based Medicine. If you read the post, you can probably understand why I took a little umbrage to Dr. Gorski’s characterization of my efforts. Below is my response to him, shared here for my readers’ convenience.
“I find your attempts to discredit me by dissembling most curious for a chap claiming to champion honest science. First of all, the multiple conflations between NP001, my personal project, and MMS are deliberately misleading. The only relationship between the three is that they contain some amount of sodium chlorite. MMS is a rather high concentration that is broken down to chlorine dioxide by the citric acid with which it is mixed. NP001 is a very low concentration of sodium chlorite delivered intravenously. My oral delivery is the same concentration as NP001, with at best about 30% delivery of NaClO2 to the blood after gut passage (according to FDA publications on animal exposure). My project (https://sites.google.com/site/alschlorite/) was intended more as an open-label experiment for people who didn’t qualify for the Phase 2 trial and in the absence of an Expanded Access Program (which some of us are working on for during the Phase 3).
NP001 and its cousin WF10 have excellent safety and tolerability in multiple studies and the case for sodium chlorite having the desired immune modulation is extensively covered in PubMed despite your claims to the contrary (please see http://www.ericvalor.org/?p=86 for an easy guide through a few of the publications by Dr. Michael McGrath, the inventor). And despite your casting doubt as to the integrity of Neuraltus by pointing at a lack of publication in PubMed, the record on clinicaltrials.gov clearly shows that Neuraltus has satisfied thus far all FDA requirements to move forward in the regulatory process for NP001 and another candidate drug (http://www.clinicaltrials.gov/ct2/results?term=neuraltus). Further, I trust the results published on Patients Like Me done by my friend known online as Persevering (http://www.ericvalor.org/?p=42). We have good reason to believe that our correlation between side-effects and live drug is accurate. I won’t reveal that right now because NP001 still has trials to go and I don’t want to pollute the subject pool.
I didn’t just guess about NP001 and run to buy a bottle of MMS like you insinuate here. I did a careful review of literature by Dr. McGrath, including email with Dr. McGrath himself. I had other sources of information which explicitly stated that sodium chlorite indeed was the active ingredient in NP001. I then worked with a chemist with years of experience using sodium chlorite in the water purification industry to examine feasability and safety. Congratulations on your own discovery, but you could have emailed me for documentation (I am quite easy to Google).
The lithium project (in which I participated) was not a collection of anecdotes but rather a self-reported trial using the standard measuring tool of the time – the ALS Functional Rating Scale or ALSFSR. This is the same questionnaire used in neurological clinics. Karen Felzer did a fantastic job analyzing the data. Unfortunately the data showed futility, like the official trials that followed ours.
The lithium project (http://alslithium.atspace.com) was organized because all other ALS organizations expressed no interest in testing the fantastic report making headlines. We patients therefore took it upon ourselves to make an initial, admittedly unofficial, trial to test the report. We knew that the results would not be as reliable as a real clinical trial, but our results would be enough to carry on further to a real clinical trial if the findings in the study paper were real. Shamed by our effort, other organizations hastily constructed trials which proved our findings. Even years later, organizations were burning precious money doing real trials when they could have looked at our results and not wasted precious time which could have been spent on other studies.
My reports are not a figment of my imagination as you suggest. Go to my site listed above and look at the video evidence I have posted. My doctors have also physically seen the improvements, much to their surprise. Although I don’t make ANY claim of definitive evidence, I do take my effort quite seriously and soberly.
The real story here, which you ignore or dismiss, is patient empowerment. For too long have we been isolated and told to just wait to die. We can use technology now to organize and, more importantly, mobilize. We now can and will take action to promote OUR agenda, not the feeble selfish agenda of certain advocacy organizations which do very little beside provide employment for their directors.
We are trained in highly-skilled technology trades. We might not be doctors, but we are engineers trained to research and resolve highly complex problems. We are capable of learning and comprehending complicated concepts. We understand the limits of our abilities and, further, that without our efforts the pace of the fight against disease is much too slow for people living today. And we have no delusion that anything we are doing is intended to replace clinical trials. Rather we intend to augment and push forward the actual science.
I will let Jamie Heywood respond further on behalf of PLM.
– Eric N. Valor”
A few days ago, Matt White, who lives with ALS, addressed the Butler NCAA basketball team prior to their Final Four victory over Michigan St. Normally I don’t care about basketball (I am a hockey fan) but a Facebook friend posted the Youtube link above and I viewed it. The thing that struck me about Mr. White’s address was his promotion of Sir Ernest Shackelton as a role model. If you don’t know who he is or don’t know his story, please take a moment to read the previous link (most notably the portion on the Trans-Antarctic Expedition).
Sir Ernest is a perfect role model for PALS because of his dogged determination, his cool ability to make rational choices in grim situations, and his use of those characteristics to survive a lethal predicament (multiple predicaments, actually). Not only did he survive but he ensured his men would as well. Contrast the experiences of Scott and, worse, Franklin. Even though Shackelton was forced to abandon his original goal, he achieved an even greater goal by surviving.
I would like to thank Mr. White for including Sir Ernest in his address and would like to concur. Even though PALS may be forced to abandon their goals, it is my hope that many can join me in “pulling a Shackelton” and doing what is necessary to survive an impossible situation.
I recently received a handwritten letter from Lou Gehrig. Obviously it was not addressed to me as the people responsible for creating me had not yet themselves been created. But it does give an intimate glimpse into the life of a remarkable man (even without the disease his accomplishments are legendary).
I was struck by the proof of his equally legendary kindness and generosity, and hope to approach that in my own life as I struggle with fellow PALS to achieve the goal that eluded Lou.
happy birthday Internet! I am proud to have known you before the web became your most prominent feature.
The site was down last night and this morning. We increased our allowed monthly bandwidth and it was created as a new account rather than credit to our existing. Once it was cleared up it took overnight for the servers to sync. Joan did a credible job as a DNS administrator!
It’s Hocktober! I’m excited to the point of levitation. Unfortunately this will be the first season I will not attend any games in person, but this is clearly the reason Philo Farnsworth invented TV!
I wanted to thank everyone who has donated so far, and to reiterate that no amount is too small (even $5). I have been asked about the Paypal buttons. The first is a user-defined amount and the second is $25/month for 24 months. I also wanted everyone to know that I have left no stone unturned. I was diagnosed by the ALS teams at both UCSF (who follows me) and Mayo Clinic. I was even tested for an extremely rare bacteria possibly acquired during a surf trip in the South Pacific. I appreciate your concerns and your questions and am happy to share my knowledge and experience.
Thank you all again.
Busy day. First, the Sentinel published a follow-up to the story last year, and yesterday a clinical trial I have been monitoring for two years finally got FDA approval. I have read the published pre-clinical animal studies and had the pleasure to converse with the CEO via email. i am not expecting a miracle but am anxious to see what happens regardless. Note that I am geographically unable to participate…
Good news! I got picked up by the Huffington Post.
we finally got the Paypal buttons up! It is quite a bit trickier to get that going (especially for a site like this) than one might think. Thanks to all of you who have already responded. Please spread the word. We should be able to populate the Photographs page tomorrow. I will sneak a few from my Facebook page…
Hello and thank you for visiting. I will update here periodically to give an idea of what this experience is like, try to publicly answer questions thrown my way, and announce important new research in the quest for a treatment and/or cure. Older posts will be archived in the future as we change and improve the site.
Please check back often and don’t hesitate to ask me a question; I spend a lot of time on research and enjoy sharing what I learn.