Neuraltus - NP001

[icingoncake]

thank you for explaining that to me, matt. I am a bit wary of these kinds of things. dont fully understand them myself so wait to be told of any progress but its getting to the stage where there doesnt seem to be progress and so i need to start being a tad more informed about these kinds of ideas. thanks again.

Jenny

[persevering]

Hi Matt J.

Thanks for starting this thread, and for the great summaries of highlights.

To clarify a bit of your post above, there are actually greater connections between WF10 and NP001:

http://www.bizjournals.com/sanfranci...u-gehrigs.html
For example, in this linked news article Neuraltus Pharmaceutical's CEO stated:

"But there may be reason for hope. Three ALS patients a few years ago bought a similar drug from a drug maker in Thailand and one of them saw a spontaneous remission in ALS symptoms, Gengos said, over a roughly eight-month period." [They only took drug for 3 months]

"NP-001, which Gengos said has the same "active moiety" as the Thai drug, works along the same cellular pathway."

"McGrath previously worked with Oxo Chemie AG, a Swiss company whose Thai subsidiary sold the IV version of the drug to the three patients. Oxo initially developed the drug in Germany as a topical wound healer."

There are two patent applications by Neuraltus Pharmaceuticals related to NP001, and both make extensive comparisons between their drug and WF10. The quickest confirmation comes from looking at drawings from each patent:

http://worldwide.espacenet.com/publi...C&locale=en_EP (Note that Neuraltus Pharmaceuticals was previously called Taiji Biomedical, and TJ001 is the same as NP001, the evolution with the name change)

http://worldwide.espacenet.com/publi...C&locale=en_EP

In a October 5, 2010 investor presentation, Neuraltus stated, again, that their drug has the same active moiety (active ingredient) as another drug, and presented WF10 human efficacy data with a title of "NP001 Active Moiety", before their phase I trial began. Among the data, they presented a graph with identical data to one of the two patients shown on page 9 of 28, here in McGrath's patent (which equally covers WF10 or NP001):

http://worldwide.espacenet.com/publi...C&locale=en_EP

In that same presentation, they boldly stated NP001 had a safe track record before even beginning their phase 1 trial, obviously concluding based on WF10, with the same active ingredient.

And, I could go on an on, but I expect you get the point. They are both aqueous solutions of (sodium) chlorite which are administered intravenously. And, curiously, the high dose arm of the NP001 phase 2 trial provides 2 mg/kg chlorite which equates to 0.5 ml/kg of WF10, the common dose of WF10. NP001, as per a patent application, is buffered to reduce its pH from about 12 to about 7 and reduces some low level contaminants relative to WF10.

I anxiously await something of interest in the response from MNDA, who has been talking about WF10 for some time.

[Jeannie]

I wasn't going to reply as I cannot be bothered to read 58 pages. However, assuming these patients are claiming to see improvements within a few hours or days after treatment I find impossible to believe. You would not see improvements the same day or day after receiving treatment as its not possible for nerves to repair that fast...it's the same as when folks claim they haven't walked or been able to shave for many months or years then after two days of receiving stem cells they miraculously get up and take a few steps or can shave. Ask any physiotherapist or dr you need intense physio to learn how to walk etc... it just doesn't happen over night,

Just my two cents worth.

[persevering]

Jeannie,

With all due respect, I am one of those you are essentially calling dishonest or delusional. While I agree with you on the implausibility of instantaneous nerve repair, it seems that reducing neuroinflammation results in some immediate relief. And, since the improvements only continue with the span of time, it seems that repair may be occurring as the environment becomes more favorable. We KNOW that reinnervation occurs, even without treatment, but is eventually outpaced by dennervation. By personal experience, I believe NP001 (& WF10) may make the environment more favorable, such that reinnervation wins the battle, or at least holds its own to prevent further progression.

Please ask yourself:

What do PALS have to gain by sharing their excitement of the tremendous efficacy of NP001?

Why would Dr. Michael S. McGrath found a company with one drug in mind at the time, NP001, if the experiences shared in the examples of his patent were not true?

There have been so many failures with ALS/MND treatments, I think we are all very pessimistic, and never really expect an effective treatment. I am sure the same was true for metastatic melanoma, that can now be treated with a pill as of a couple days ago!

http://www.foxnews.com/health/2011/0...oval-from-fda/

The result "takes your breath away if you're a melanoma doc," said Anna Pavlick, director of the melanoma program at the New York University Cancer Institute who participated in vemurafenib's clinical trials. For four decades, she said, skin cancer patients whose tumors had spread to their lymph nodes and other organs were forced to rely on treatments such as chemotherapy that often failed.

http://www.zelboraf.com/?cid=zel_we_...FQat7QodUmny6g

No need to read 58 pages (by the way, it is now 62), just read evaluations of NP001 phase 2 participants here: http://www.patientslikeme.com/treatm...eviews?brand=f (a couple are "public", but signing up shows 9 evaluations currently.)

[persevering]

Here is a now dated article about the premise of NP001, from June 2010. It is now in a phase 2 trial with enrollment 2/3 full, and the earliest participants having already completed all 20 drug doses.

http://alsn.mda.org/news/flip-switch

A growing body of evidence points to malfunction of the immune system as at least part of the complex ALS disease process. As noted in previous study reports, abnormal immune system activity has been observed in animal models of the disease; it also has been found in blood samples of people with ALS.***

'There's a switch'**

NP001 is administered by intravenous injection.**

According to Andrew Gengos, president and CEO of Neuraltus, the company's experimental drug is designed to flip a molecular switch in cells known as macrophages in the blood and microglia in the central nervous system.

"There's a switch that it hits," Gengos said, "that regulates these cells from an activated, inflammatory mode back to a more normal, wound-healing mode." (For more about this phenomenon, see ALS: Not Just About Motor Neurons Anymore, in the May-June 2010 ALS Newsmagazine.)**

Mice with an SOD1 mutation called G93A are a common research model of the human disease and were used in the Neuraltus experiments.**

Meaning for people with ALS***

Neuraltus is currently completing mouse studies that it hopes will show its compound changes the immune system and affects progression of ALS. If the results show the anticipated effect, they will add to the increasingly large body of evidence that shows that parts of the immune system go awry in ALS.

[icingoncake]

we will all have to wait and see on this one. for myself i am a little wary of trying to get hold of something just because it is 'similar' to something else (if im understanding right, matt) anything on the official trial will obviously only come out once the whole trial has been finished, no? wf10 is not of any interest to me just yet - however tempting it may be - and i know it is tempting. but to me i would not give my husband hope for anything that was not tested and trialed for MND. I wouldn't even know how to get him it and i dont know if i would dare even if i could. It just fels like were all waiting and waiting but i dont expect anything of note except clarifications of what we all probably know about the trial and what is already known about the other drug, wf10.

I just dont want to get hopes, but please don't listen to me if this does give you hope. We all get by as we can i guess.

[peepyh]

I don't know what to feel. I can't even allow myself to hope this is for real. Just the tiniest glimmer of a hope that maybe I could walk again, or play with my dog, rather than a drawn-out death, is almost painful.

Fingers crossed (if I still could!) that this time something works, and that we can get it in time!!!!!!!!!!!

[MattJ]

Hi Jeannie, Jenny and Paul,

Thank you for adding your thoughts to this thread. It's great hear your opinions! Bring on Monday morning. Can't wait to hear what the MNDA has to say. Hopefully things will be a little clearer then.

[Graham]

Hi Matt,

Thanks for inviting "Persevering" here and a big thanks for "Persevering" for all the info.

Paul-lfc on Build-UK forum reported the same switch being trigger by a boil infection on his back. He experienced a rapid but brief improvement that persisted. MND research should really investigate.

Meanwhile my painful remission continues. We will persevere and find the answers.

Looking forward to the MNDA statement on Monday!

Best wishes

Graham

[Prosons]

Hi All,
I wanted to chime in on this thread because it is so very important. I have ALS, diagnosed I was diagnosed April 2009. I just finished my very first week of infusions of NP001. Approximately three hours after my first infusion while eating lunch I noticed dramatically improved swallowing, swallowing was effortless and no choking. At first I was skeptical, however by the third day of infusions my swallowing improvements continued to strengthen and I had more range of motion with my tongue. My speech has improved in terms of rate of flow and articulation. The dexterity in my fingers has improved allowing me to make better use of my hands. I have increased strength in my knees. I have more range of motion in my neck. The my upper lip is stronger and my eyes do not protrude as they did prior to NP001. Please keep in mind that these changes I have described are subtle with the exception of my swallowing, ie however they are very real and tangible.

It is my opinion that the changes I have experienced are due to a reduction in neuro inflammation and subsequent reduction in spasticity. This is purly speculative on my part.

Will NP001 work for everyone? We don't know at this point.

Will NP001 work for late stage ALS? We don't know that either. What we do know at this point is that the improvements we're experiencing by some in the trial are unprecedented. The I can assure you this isn't "snake oil." I would urge you to be cautiously optimistic about this trial and the company Neuraltus. I would also urge you to spread the word and create much buzz about this trial and hopefully to the hype will lead to all of us getting the drug quicker.