The charity who provides RCH4 has never made claims about the efficacy of the drug. However, they possess considerable data collected from PALS over several years to prove efficacy.
From patient data collected through 2017, we do know:
- RCH4 is effective in 84% of PALS treated, with a noticeable slowing in the patients rate of decline.
- 52% of patients treated with RCH4 stabilize for more than four months.
- 26% of patients treated with RCH4 stabilize for more than one year.
- 4% of patients treated with RCH4 have ALSFRS-R scores that actually improve.
There are no inclusion/exclusion criteria for PALS who desire to take this new drug. The charity provides it on a humanitarian basis, which distinguishes their efforts from clinical trials. In clinical trials, there are inclusion/exclusion requirements that also include a percentage of participants to use a placebo.
PALS are taken on a first come first serve basis of those who ask for help by simply filling out a form on the charities website. This also avoids skewing of the statistical analysis in favor of the efficacy of the drug, unlike clinical trials where patients are carefully selected for best outcome. The charity has no placebo control group, and all of PALS receive the actual RCH4 drug.
How do they work out just how effective the drug is? Firstly they establish the speed of the disease decline for each person from the time of their diagnosis to the time each person starts on RCH4. This is done by patients submitting their medical testing, records and other pertinent information.
Since many PALS have not had their ALSFRS-R score assessed by a neurology specialist, it is difficult to establish the average starting score. In case the information from a neurologist is not available, the data relies on the information provided by patients,
Patients are required to send reporting containing a variety of parameters ranging from physical abilities to medical testing. By comparing the decline rate before to the decline rate after, the efficacy of the drug is established. The charity maintains a database which provides detailed, ongoing analysis of every PALS, in real time.
Since the beginning, the charity has collected the data from PALS, who all combined, were given approximately 6,000 doses of RCH4. Based on the evidence submitted through their monthly monitoring reports, the drug shows extremely strong potential to slow the progression of ALS, prolonging the lives of PALS.
For some PALS, the drug halts the decline of their ALSFRS-R score over time. Fewer PALS note the improvement, or reversal, of their ALSFRS-R score over time, but these results usually take much longer.
The cases of score reversal (improvement of the condition) may raise a question about the accuracy of their diagnosis. In case of the error in diagnosis, they can observe the efficacy of the drug applied to other neurodegenerative conditions. The patients themselves admit that as long as the treatment improves their function or slows down their decline, the name of their disease is irrelevant to them.
When asked about side effects, patients report small bruising or stinging when the liquid is cold. Since RCH4 is administered via a intramuscular shot using a syringe that is most commonly used by diabetics for insulin, the bruising and pain is not permanent.