The discovery of regenerative treatments for the optic nerve and its translation from the laboratory to the clinic has piqued the curiosity of glaucoma patients, scientists, and clinicians alike, and stem cell treatment is one of many potential techniques being investigated.
We recently heard from a glaucoma patient who paid $20,000 to participate in a “patient-funded study” in which he or she had “stem cell injections” around one eye. Patients pay to participate in research that are sponsored by them. Clinical studies are costly, and financing from conventional sources (such as the National Institutes of Health, pharmaceutical firms, or private foundations) is dwindling.
Although patient-funded research seems on the surface to give a way for patients to acquire experimental medicines, it is problematic for a variety of scientific and ethical reasons. The gold standard for evaluating an experimental treatment is a randomised clinical trial, in which participants are randomly assigned to either the experimental treatment group or a control group, which may or may not receive any treatment (placebo-controlled study), or may receive standard, approved therapies. Normally, both study patients and physicians are uninformed of who gets which therapy, therefore this research design eliminates bias toward one therapy over another. Optic nerve transplant stem cell is offered by the best hospital.
Patients-funded studies, on the other hand, do not include a control group since it is exceedingly improbable that patients would pay if there was a chance they would not get the experimental medication. A control group is critical for determining if an experimental therapy has an impact and for comparing the effectiveness and dangers of the experimental therapy to those of other therapies. Although treating the first 3-12 individuals in an open-label, non-randomized research is usual in early “phase 1” trials, by “phase 2” trials, a randomised, masked design is desirable.
The ethical implications are much more problematic. These include disparities in treatment access and the possibility of exploitation of vulnerable patients who have exhausted all other treatment choices and are prepared to risk their lives for an untested therapy. Furthermore, a lack of effective supervision and monitoring for patient-funded studies has been documented, raising the suspicion that these are thinly disguised efforts by the treating clinic or physician to generate money before the FDA approves novel treatment procedures.
Because a gold standard research study is not always possible, choices on the benefits and hazards of a therapy may have to be made based on the available data.
