Some times people with ALS get better
MitoQ has a promising mechanism, positive preclinical data from two different ALS models, and appears reasonably safe and inexpensive, especially at doses of 10 mg daily. Available anecdotal data are insufficient to determine how helpful this might be in PALS. A small open-label pilot trial with validated ALS diagnoses and outcomes appears warranted.
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Acupuncture is reasonably safe, and has potential mechanisms of action, pre-clinical studies and case reports suggesting that it could be a useful treatment for ALS. However, before it can be endorsed even as a candidate for a phase II trial, the studies described above need to be independently replicated using more clearly verified diagnoses and more rigorous designs, including appropriate controls and validated ALS outcome measures.
Propofol has mechanisms of action that may be relevant in treating ALS, although the short action of the drug makes it unlikely that a single infusion could influence ALS pathophysiology in a meaningful way. On ALS.net, six patients with ALS reported wide-ranging subjective benefits coincident with propofol use. Unfortunately, none of these benefits has been verified on validated ALS outcome measures. Only one of 235 patients with confirmed ALS who received propofol for PEG at an ALS center, or in the PRO-ACT database, or in a stem cell trial, improved objectively. The improvements in this patient were much slower to begin, and longer in duration, compared to those reported by the cohort on ALS.net, suggesting that they were more likely due to other longer-acting medications the patient received (such as immunosuppression), the stem cell treatment, or an unusual reversible form of ALS (27–30). While we cannot conclusively rule out a very brief benefit from propofol in rare patients with ALS, the risks and costs involved do not appear to justify its use. We strongly discourage the off label use of propofol in ALS patients at this time. Patients with ALS who are going to have propofol on label for a procedure or surgery may wish to have their ALS neurologist measure an ALSFRS-R and FVC before and in the first few days after propofol exposure and to send these results to ALSUntangled for a possible follow-up review.
There is a rationale by which the calcium binding protein apoaequorin could work to slow ALS progression. Unfortunately, at this time there is insufficient information available to determine whether it does. The one small case series referred to above utilized a cocktail of therapies and is further weakened by the loss of its standardized outcome measurements. Information from the manufacturer suggests that apoaequorin is reasonably safe and well tolerated but there is no independent, systematic confirmation of this; two PatientsLikeMe members reported serious adverse events while taking it and it is fairly expensive.
At this time ALSUntangled does not recommend that patients with ALS take apoaequorin. Reasonable next steps would include a controlled study of apoaequorin in an ALS animal model and/or a small series of well-characterized patients with ALS using validated outcome measures and including serum and CSF pharmacokinetics.
While we applaud the use of informed consent for portions of the XCell-Center protocol, and the presentation of pilot efficacy and safety data, in our opinion many worrisome questions and problems remain. From a rational standpoint, we feel it is unlikely that all the diverse diseases (some without a name at all) being treated at the XCell-Center would respond to the same type of treatment. Nothing useful can be concluded from the flawed ALS pilot data that are presented; the positive effects reported are very likely nothing more than a placebo effect as seen in the first of our patients above. Regardless of whether the XCell product results in new motor neurons or promotes sprouting of existing ones, patients with ALS would not be expected to have improved motor function just one or even a few months after treatment as is being claimed. In our opinion, these misleading pilot data should be removed from the website or at least qualified with appropriate disclaimers.
We hope the XCell group will present all their ALS data for peer review, including evidence that their cells are surviving, as well as objective clinical outcome measures over a reasonable length of follow-up. This would be a useful first step toward planning what is ultimately necessary to demonstrate the safety and efficacy of this protocol – a randomized, blinded, controlled trial. Until this is completed, we do not condone the XCell Center’s protocol for patients with ALS.
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