They're back, ... NP001

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patoyeah

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September 22, 2016 ? Neuraltus Pharmaceuticals, Inc.

This stuff helped some heroic pals, a few years ago. Then the company confusingly and mysteriously stopped progressing the trial

This may be one of the most promising treatments around, but can this outfit be relied on?

May work best on upper motor neuron issues.

Take care,

Pat
 

lgelb

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Early stage biopharmas run out of $ all the time and have to pause to raise it, while negotiating with the FDA in re what to do next. As we all know, investors are dubious about ALS because the ROI to date has been dismal unless you are a day trader. And trials of IV-infused rx obviously cost more.

Also, they crunched the first IIa trial numbers and found that elevated CRP was somewhat predictive of benefit (as measured by the seriously-flawed FRS, mind you) so now they're keying on that as an inclusion criterion. Smart and necessary.

It looks like the secondary endpoints are respiratory.
 

ehd42

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Hi Patoyeah

Please don't be discouraged by the events that take place throughout the development of a clinical trial. I have been either a principal or sub- investigator in more than 150 clinical trials over the past 40 years and I have learned that early trials are frequently discontinued for a whole variety of reasons, so I wouldn't be discouraged by the events surrounding a single trial and new trials are always entering the pipeline. In any case, there are dozens of clinical trials for ALS in development or currently being conducted around the world and it is just a matter of time before some of them will be conducted in the United States.
I remain very optimistic that effective treatment for ALS will arrive within the foreseeable future based on accelerating advances in science and medicine, and in particular, in neuroscience.

Check out the following websites for more information on current clinical trials for ALS:

ALS Clinical Trials | ALS Clinical Research | The Northeast ALS Consortium (NEALS)

ALS Clinical Trials | ALS Therapy Development Institute

best wishes,

Eliot
 

patoyeah

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Eliot, u r very kind. Thank u...what condition is your body in? U have had this awhile

That trial was heart breaking. Had friends in that trial. They improved. Then the trial was stopped, no explanations given. The pals begged for the drug, under compassionate use, but got no help...they quickly got worse and died, only one is left alive.

U r very kind thank u... there were some special people in that trial, and they improved w/ NP001

Thanks,

pat
 

lgelb

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In addition to the sites mentioned, clinicaltrials.gov is the most comprehensive. I linked to the 85 open trials, some ex-US.
 

ehd42

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Hi Pat,

To answer your question, I was diagnosed in 2009 with a limb onset, within a year I was on BiPAP. I began using a power wheelchair about 2 1/2 years ago and I now need to use noninvasive ventilation 24/ 7. I have no bulbar symptoms so far and my weight is stable (no PEG). Symptoms of weakness and paralysis continued to advance and I'm slowly losing function of my hands. I now use voice recognition software for computer input. In spite of this I have tried to focus my attention to learning everything I can about ALS and its management in order to maintain the best quality of life possible as symptoms advance. Fortunately, I found that I have been able to adapt and adjust to the loss of function in ways that work for me most of the time, and this allows me to enjoy what is still possible. ALS forced me to retire as a physician and I really do miss my work, however my training and experience have really helped understand what is happening to me and it also helps me find the best options available so that I can continue to enjoy my life and the people I love. It is true that everyday is a struggle one way or another ( Hoyer transfers are pain); nevertheless, I can usually find a way to deal with problems in ways that works.
I am optimistic that progress in science and technology will continue to increase at an ever-faster pace and so there will eventually be answers and effective treatment for all of us.
I'm glad I that I joined this forum because of its sense of community and how much people care about one another.

Best wishes,

Eliot
 

frankb

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Thanks, Ed, for joining our group and for sharing your great attitude.
 

chally

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Thanks Ed, I like your sense ability in dealing with this nightmare. Keep posting! Chally
 

KimT

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Eliot,

I really appreciate your joining this group. Thank you for the time you put in to help the rest of us and share your knowledge.

I'm glad you're having slow progression and have no bulbar involvement.

Please stay with us.
 

scaredwifetx

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I also want to thank you Elliot. You have helped several of us. I am glad your progression is slow and have shared most of your post with Steve.
 

*ENV*

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This short book was written by my friend Jef Akst. She researched the issue for a couple of years after doing an article with me about DIY drug trials for The Scientist magazine (for which she is a senior editor). The story talks about the OSC (Oral Sodium Chlorite) program which I developed as a sort of "expanded access" program for people who couldn't get into the original Neuraltus trial. I developed the program along with Rob Tison and Ben Harris, both of whom were in the Neuraltus trial in the high dose arm, and both of whom benefited greatly. Rob did a guest post on my blog with his scientific analysis which jibed with the post-hoc findings of Neuraltus. Unfortunately both Rob and Ben are no longer with us.

The book was recently released in dead-tree form for those without e-readers. It's worth the very low price to read about patients empowering themselves with Internet learning and meeting. Social media is the perfect tool for us to use to band together and force the change we want in the system.

 

*ENV*

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A better resource is Antidote, formerly TrialReach. They have been excellent partners in being a resource for PALS. Just fill out a few questions and you will be presented with the relevant clinical trials within a radius you specify.

In addition to the sites mentioned, clinicaltrials.gov is the most comprehensive. I linked to the 85 open trials, some ex-US.
 

lgelb

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Since many PALS can consider travel in early stages, when they are more likely eligible for intervention trials anyway, seeing all the protocols without pre-filtering allows for more personal choice.

Best,
Laurie
 

*ENV*

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"Pre-filtering"? If you mean making sure results shown are relevant, then perhaps. People constantly complain that clinicaltrials.gov is very hard to parse. Antidote is trying to make relevant trials easier to find and enroll in. Originally they were focused on cancer but after I wrote asking to consider adding ALS to their list, they put it on the top of the development list and even came out from London to meet me and film a promo video (https://antidote.me/conditions/als/). I find the search and results much more user-friendly, but your mileage may vary...
 
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