Numerous new therapies for multiple sclerosis are considered based on theories as to the causes and based on animal research. Those that appear promising are typically tested in a series of tests. These are: phase I, which looks primarily at safety and, at least a hint of efficacy. If the phase I is successful, a somewhat larger phase II trial is carried out. Phase II is to get enough safety and efficacy data to plane one or more phase III trials to demonstrate the effectiveness of the drug. The phase II trial does not necessarily have to show a statistically significant effect but will provide the evidence to plan definitive trials. These are typically quite large and the FDA generally requires two successful phase III trials to approve the drug. If significant risks are seen or suggested by the trials, the FDA may mandate a “risk evaluation and mitigation strategy” or REMS program which mandates careful monitoring, evaluation and reporting of all significant side effects. Most, though not all, of the most effective drugs had REMS programs.
In the past, the FDA demanded placebo controlled trials, that is, all trials had to have a sham treatment arm for comparison with the drug being tested. Now, most new drugs are tested against another established drug, most commonly one of the interferons or glatiramer acetate. These comparison trials are of two types, Non-inferiority trials which are used to show that the drug is as good as an existing drug. This was done to gain approval of a generic glatiramer acetate. Superiority trials are used to show that a new drug is better than one of the existing treatments as was done with the testing of alemtuzumab (Lemtrada).
Throughout the history of MS, new treatments have been reported based on very limited evidence and with no good rationale. These are a significant problem as they cause diversion of a great deal of effort and resources to disproving the false claims of efficacy. One of the most recent was the claim that venous obstruction (CCSVI) was causing the disease and venous stenting was an effective treatment. A number of surgeons were doing the procedure and many patients paid as much as $8,000.00 to get the stenting. It has been shown not to be effective and a great deal of expense and effort that could have been better spent on other research was diverted to disprove the false claims.
Over the years claims have been made for a host of supposed treatments including such things as cows colostrum, royal jelly, bee stings, snake venom, amalgam fillings etc. At one time a list of all treatments suggested to be effective for MS was made and came to 7 pages single spaced just to list them. Many of these will appear from time to time on the internet. You should be wary of claims for efficacy based on limited, uncontrolled data.