This article provides a bit of a timeline and
here is the 2021 ms. They censored cases pretty aggressively to improve the reportable results. As Nikki says, when you try to show a statistically significant difference between the treatment and control groups, it doesn't help that control group outcomes in ALS are a moving target, as the quality of care varies even among "centers of excellence," it's not as predictable a disease as many people think, etc. and the trial Ns are small in the scheme of things.
Moreover, anyone trying to trial ALS tx has a hurdle with the primitive, categorical-used-as-continuous ALSFRS-R scale.
One key issue will be the magnitude and range of expected benefit in a readily definable population for an expensive, invasive treatment. There is not enough data as yet to shed much light on this, increasing the likelihood that payors will have very narrow reimbursement criteria to begin with. If you can say confidently that a certain % of patients with attributes X, Y, Z and not A,B,C will benefit at least this much, that's more likely to win regulatory approval/reimbursement than a wider range of benefit and/or a less easily defined population.
If/as approved, this treatment, like the others, will be most effective when used early in less severe cases, again highlighting the importance of early diagnosis, as well as wait times for neuro/clinic visits, payor approvals, stem cell processing, and the procedure itself. We do not have an ALS system of care in this country, nor in most others.
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