The discovery of regenerative treatments for the optic nerve and its translation from the laboratory to the clinic has piqued the attention of glaucoma patients, scientists, and physicians alike, and stem cell therapy is one of many potential methods 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 got "stem cell injections" around one eye. Patients pay to participate in research that are financed by them. Clinical studies are costly, and financing from conventional sources (such as the National Institutes of Health, pharmaceutical corporations, or private foundations) is dwindling.

Although patient-funded research seems on the surface to offer a way for patients to access experimental treatments, it is contentious 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 ignorant of who gets which therapy, thus this research design reduces bias toward one treatment over another. Stem cell for optic nerve damage is offered by the best hospitals.

Patients-funded studies, on the other hand, do not include a control group since it is very improbable that patients would pay if there was a chance they would not get the experimental therapy. A control group is critical for determining if an experimental therapy has an impact and for comparing the effectiveness and dangers of the experimental treatment to those of other therapies. Stem cell for optic nerve damage can be taken care of by the best hospital and doctors.

The ethical implications are much more serious. These include disparities in treatment access and the danger of exploitation of vulnerable patients who have exhausted all other treatment choices and are ready to risk their lives for an untested therapy. Furthermore, a lack of appropriate supervision and monitoring for patient-funded studies has been observed, raising the potential that these are thinly disguised efforts by the treating clinic or physician to earn money before the FDA approves novel treatment techniques.

Because a gold standard research study is not always possible, judgments on the benefits and dangers of a therapy may have to be made based on the available data.