Our ideas will come from real patients with sporadic ALS who tried something and had their ALS improve on it (as validated by an ALSUntangled review). Most traditional ALS research ideas come from cells or animals that have abnormal ALS causing genes inserted into them, and how accurately these model sporadic ALS remains unclear.
We will be looking for huge treatment effects: improvements in validated ALS measurements. Most traditional ALS research studies are looking for a very small slowing of disease progression (typically 20% slowing of the rate of decline of the ALSFRS-R).
Because we will be looking for such big effects, our studies can be open to almost all patients with ALS. Traditional ALS research studies looking for small treatment effects try to maximize their “signal to noise” by excluding patients who have had disease for more than a few years or, have BiPAP, a diaphragm pacer or other experimental therapies underway.
Because we will be looking for such a big effect, our studies will not need a placebo group and can be enrolled and completed quickly. Traditional ALS research studies looking for small treatment effects require concomitant control groups, large sample sizes and long durations.
Only a few visits will be face to face; most “visits” will be virtual. This will minimize travel burdens to patients and caregivers.
Our research results will be transparent in real time. It takes a frustratingly long time to get the results from most traditional ALS research studies, even after they are completed. We will also publish our protocol on this website so that patients who want to try exactly what we are testing can do so.