ALS News Today Forums Forums ALS Progress Research Topics NPt520-34 Was granted orphan drug status!

  • NPt520-34 Was granted orphan drug status!

    Posted by Amanda on November 26, 2019 at 11:55 pm

    The Orphan Drug Act was passed in 1983 with the intention of motivate pharmaceutical companies to target drugs that would benefit people with rare diseases. When you look at the government website https://rarediseases.info.nih.gov/diseases/fda-orphan-drugs they list 12-13 kinds of ALS; however, under each there are only two drugs approved for the treatment of ALS.

    • “Edaravone (Brand name: Radicava) – Manufactured by Mitsubishi Tanabe Pharma Corporation
      FDA-approved indication: May 2017, edaravone (Radicava) was approved for the treatment of amyotrophic lateral sclerosis (ALS).
    • Riluzole (tablet) (Brand name: Rilutek) – Manufactured by Sanofi
      FDA-approved indication: December 1995, riluzole (Rilutek) was approved for the treatment of patients with amyotrophic lateral sclerosis. Riluzole extends survival and/or time to tracheostomy.”

    Knowing that there are only two approved treatments for ALS, that makes research for new medications to slow the progression, or more importantly stop the onset or cure ALS even more important. Since ALS is classified as a “Rare disease” new drugs or drugs in the research phase such as NPT520-34 are, in my opinion, of great importance.

    ALS News Today recently published an article on NPT520-34. This is the drug that has success crossing the blood brain barrier and hopefully will not only benefit pALS, but people who suffer from other rare disease such as Parkinson.

    Are any of our members involved with the clinical trials for NPT520-34 or have you been keeping up with the research on this drug? What are your thoughts?

    If you are interested in reading the article on ALS News Today the link is https://alsnewstoday.com/2019/08/20/npt520-34-granted-fda-orphan-drug-designation-als/

    Also, this article was made into a flash briefing!

    Let us know your thoughts and opinions.

    john-russell replied 4 years, 3 months ago 5 Members · 7 Replies
  • 7 Replies
  • john-russell

    Member
    November 29, 2019 at 7:32 am

    Do you know of any articles descring these dozen or so types of ALS? I have long wondered if ALS is really a very specific diagnosis since function and longevity following diagnosis is so varied.

  • Dagmar

    Member
    November 29, 2019 at 12:16 pm

    What a good question John! I searched and could not find any definitions for the 14 “types” of ALS referenced in the article: https://rarediseases.info.nih.gov/diseases/fda-orphan-drugs

    If any of our members can help out with links or information about these “types” it would be appreciated!

  • bev

    Member
    December 3, 2019 at 8:07 pm

    <p style=”text-align: right;”>so many disease mimic the symptoms of ALS. I just had an emg after 10 years following symptoms and it still points to PLS. I lost my balance and fell numerous times and eventually lost my ability to speak. I had head trauma in 2 traumatic falls. Speech therapy failed and I’m not eligible for clinical trials because I don’t have an ALS diagnosis. PLS is a catch all diagnosis, a type of monally.</p>
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  • jean-pierre-le-rouzic

    Member
    December 4, 2019 at 3:57 pm

    There are many subtypes of ALS but it is very artificial as there is no biomarker for ALS.

    As long as there is no biomarker, we do not really know what is exactly the disease, except that its clinical signs in the “classical” form involve both upper and lower failing motoneurons.

    For example here is ALS type 23:

     

     

  • Dagmar

    Member
    December 4, 2019 at 4:01 pm

    Thank you Jean-Pierre for the helpful reference!

    In fact, through your link I discovered this page which lists 24+ subtypes of ALS and, most importantly – – how they received the classification. It is a detailed read… but it looks to identify the specific genetic source of each “type.”

    Here is the link: https://www.omim.org/entry/105400

  • jean-pierre-le-rouzic

    Member
    December 5, 2019 at 4:02 am

    but it looks to identify the specific genetic source of each “type.”

    Actually most (95%) of ALS cases have no genetic origin, as they are proteopathies, mainly due to TDP-43, which is also shared with several kind of dementias (FTD, 1/3 of Alzheimer, some Parkinson).

    Even for the familial cases were there is an obvious genetic origin, scientists did find proteopathies.

    But one can ask what creates the proteopathies in the first place, it can be a lot of things, including DNA or RNA damage as most proteins involved in ALS are related to DNA or RNA damage. Probably what we subsume under the umbrella term “aging”.

    But really there is no scientific consensus about ALS, scientists do not even agree on very broad hypothesis such “dying forward” or “dying backward”. In one case the disease starts in the Pons or the medulla oblongata, in the other case it starts at the neuromuscular junction or even with muscle waste. There are strong evidence for both cases.

    On a related topic, there is a recent tendency in clinical trial to divide the patients in subgroups. But those subgroups are not based on genetics, they are based on various biomarkers. Maybe that would help to better stratify ALS.

  • Dagmar

    Member
    December 5, 2019 at 10:53 am

    Good point!

  • john-russell

    Member
    December 8, 2019 at 7:59 pm

    Read your link Dagmar. Interesting. Of course if genetics were not involved everyone ex]posed to a trigger would be sick. I was surprised that the information in the link was presented several years ago.

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