There is great interest among glaucoma patients, scientists and doctors alike, in discovering regenerative therapies for the optic nerve and translating them from the laboratory to the clinic -; and stem cell therapy is one of several promising approaches being studied.
Recently, we heard from a glaucoma patient who enrolled in a “patient-funded trial” in which the person paid $20,000 and received “stem cell injections” around one eye. Patient-funded trials are studies in which patients pay to participate. This approach was developed because clinical trials are expensive and funding from traditional sources (such as the NIH, pharmaceutical companies, or private foundations) is decreasing.
Although patient-funded research would appear superficially to provide an avenue for patients to obtain therapies under investigation, it is controversial for a number of reasons, both scientific and ethical. Scientific considerations are deeply concerning: the gold standard for evaluating an experimental treatment is a randomized clinical trial in which participants are randomly allocated to the experimental treatment group or a control group that may not receive any treatment (placebo-controlled study), or may receive standard, approved therapies. Usually, study patients and doctors are both unaware of who receives which treatment and this study design minimizes bias towards a particular treatment.
In contrast, patient-funded trials do not have a control group, since it is extremely unlikely that patients would pay when there is a possibility of not receiving the experimental treatment. A control group is very important to determine if an experimental treatment really has an effect and also to compare the efficacy and risks of the experimental treatment versus other treatments. Although it is common in an early, “phase 1” trial to treat perhaps the first 3-12 patients in an open-label, non-randomized study, by “phase 2” trials, a randomized, masked…