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    • #17997
      Dagmar Munn
      Keymaster

      This week I wrote my column With Clinical Trial Criteria, We’re Stuck on the Outside, Looking In, about how the criteria set for most ALS clinical trials are limited to those in their first three years of the disease. Those of us who are 4+ years out are excluded.

      Why does that matter? Because doctors and medical insurers base their decision to prescribe the new treatments on “who” made up the clinical study group. Thus, patients 4+ years out are excluded from receiving the medication.

      Has anyone else noticed this issue or felt its effect?

      What do you think about this situation?

      Two good references are:

    • #18002
      Anonymous
      Inactive

      A couple of different articles I read said that around 5k people/year are diagnosed with ALS in the US. Seems that about 10% live more than 10 years, but the average 3-5 is assumed for most statistics. Researchers probably do not want to try to inject more into the numbers than they need so if the majority of ALS patients are gone between 3-5 years, they are likely concentrating on those of us who fit into the “majority” picture (the balance is those who live past 5 years which is more than 10%). Far beyond that, they have to shuffle a lot more statistics which would change the pictures they create substantially.

      The truth is that every year, they ignore another percentage of the ALS population that may give them the secret to longevity…instead of figuring out they do not know the cause of ALS…they should be looking at why some of us live much longer than the majority. In that, they could trip over some real answers to help us.

    • #18010
      Delbert Mitchell
      Participant

      I have been excluded from many possible trials due to the fact I was diagnosed in 2014. Another factor is that most of these trials are on the east coast or midwest which limits ALS participants from participating. I live in Nevada, and the closest trial centers are in Los Angeles, San Diego, or San Francisco, and yet we have a Cleveland Clinic here in Las Vegas, but they never consider people living out of these large metropolitan areas. My ALS has been very slow progressing, and my neurologist is amazed at how slow that progression is. The trials are definitely skewed to those ALS patients who are under three years, and it’s very discriminating to the rest of the ALS population.

    • #18011
      Chuck Kroeger
      Participant

      I have been excluded from trials because my vital lung capacity is less than 60%. I missed one trial that we thought might work here in the mid West because of a few percentage points.   Very frustrating

    • #18012
      Suzanne Gallo
      Participant

      Ragnar, oh my god, this post hit the nail on the head for my husband, my Pals- and I. He was diagnosed on January 11, 2021. Our first clinical ALS appt was on March 4, 2021, at U of Penn. One of my first questions for the neurologist at the ALS clinic was- what clinical trials can my PALS participate in. This is U of Penn, where HEALY platform trials are going on. The neuro doc said, he is not eligible. The notes from another neurologist at Penn, the one who gave us the ALS diagnosis, said that my PALS ALS started 3 years ago. This was because we guessed that he had been experiencing hand cramps for about 3 years. We had no idea that these hand cramps were at all related to ALS. Three years ago, our primary care doc told us that the hand cramps were arthritis.
      So we were shocked, and saddened that revealing the “guessed” timing of 3 years ago for the hand cramps- would be the record timeline for the start of ALS. We thought that the trials used the date of diagnosis, not the “estimated” time of when the PALS began to notice hand cramps. A lot of people get hand cramps for many reasons.
      So my PALS was shut out of ALL clinical trials, because the neurologist who made the diagnosis posed the start time as 3 years ago- effectively eliminating his chance of participating in clinical trials. Especially disturbing because this was our first ALS clinic. My PALS walked in. He had no breathing or swallowing issues. He had a slowly progressing disease. We are crushed.
      My husband is a very engaging, super friendly 67 year old guy. My dad passed away from ALS 4 years ago. My dad didn’t have a chance to get into any trials: he lived in the Florida Keys, and wasn’t keen on traveling- and he was 78 at the time of diagnosis.
      For my PALS and my first ALS clinic, we were prepared. When we were told that he could not participate in any clinical trials- it was a major set back for us.

    • #18013
      Elizabeth Pacheco
      Participant

      I was very disappointed when I was turned down as a participant in clinical trials. And like all of you I felt frustrated but moreover “Struck” because I was old in the disease. I wasn’t basically Sick and disabled enough to participate. I’ve been told participation can accelerate progression and count your lucky stars that your progression is slow. Was glad to see my frustrations and thoughts expressed. We need to keep pushing to be represented in clinical trials

    • #18014

      Clinical trials are excluding pALS, but clinical trials are absolutely inefficient anyway and it is not specific to ALS:

      * Actually there is a shortage of ALS patients in clinical trials, as a “normal” phase II clinical trial should involve nearly 500 patients, but we can see that there are phase II clinical trials with only a few dozen participants. So to get meaningful data the recruitment must make sure all patients have a similar profile. Yet the proposal of John of having a trial with only long time survivors is interesting … if there were enough patients.

      * Clinical trials are a very inefficient way to verify the efficacy of newly proposed drugs. This is not specific to ALS and without mentioning the two larger mortality causes that are still unsolved (heart and cancer), for example in Parkinson there were three times more clinical trials than in ALS, without any indication of progress. For Alzheimer disease, its the same or worse.

      * Drugs that are tried in clinical trials are not selected rationally: Some clinical trials tries repeatedly the same drug, failing each time, some other are just trying in another disease, a drug which failed in one disease, as the cost of abandoning a research pathway is higher that having a last random shot in the dark at another disease.

      * Drugs that are tried in clinical trials are not selected rationally: Most of the time they do not result from academic research, but from a hunch of the principal investigator. Principal investigators have to cajole hospitals in conducting trials, they won’t if they think there is a high risk of side effects that could endanger their reputation. In neurodegenerative diseases the risk of dangerous side effects  is always present.

      * Most academic research do not result in clinical trials. Academic research is the result of small teams with small projects lasting one or two months, as their main goal is to publish a paper during the semester course. Next month they will jump on a totally unrelated topic. That’s the fault the way research is funded since the 80′.

      * Most academic research do not result in drugs. There is a 1500 to 1 ratio between drugs proposed by academics and drugs that make it to the market. Look at Lipinsky rule of five to understand why (it’s also named Pfizer’s rule).

      * The goal of academic research is just to get rich quick: In the last two years there were 10 patents with “ALS treatment” in their title!

      * Most academic research on ALS is futile as it is either in-vitro or on rodents. Rodents do not have the exactly the same neural tracks as primates and its those neural tracks that are specific to ALS that are missing in rodents…

      * One third of academic research is not reproducible by the pharmaceutic industry, so there is a large distrust of academics by the industry.

      * There are many phenotypes in neurodegenerative diseases, there is nothing as simple as Alzheimer or Parkinson or ALS. Some scientists tell there are up to 5 Alzheimer or Parkinson types and a dozen ALS types. Scientists do not know exactly what is wrong in ALS or at least where the dysfunction  is located, is it in brain’s motor area, or in neuro-muscular junction or in the spine? They do not even agree on broad classification of the disease, is it a metabolic or an immune disease? Some aspects are entirely neglected by ALS research such as the cause of cachexia in ALS.

      So eliminating confounding influences is mandatory in a disease which is heterogeneous to an extreme point.

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