Glaucoma cannot be cured, which is why there is great interest among patients, physicians, and scientists in new regenerative therapies for the disease. The clinical application of stem cell therapy holds great promise. Recent reports of patient-funded trials, where the patient pays to participate in a study and receive experimental stem cell treatments for glaucoma, have raised the question whether this approach is scientific and ethical.
Stem Cell Therapy for Glaucoma
Stem cells treat glaucoma in a number of ways. In early glaucoma, stem cells are neuro-protective and prevent the degeneration of ganglion cells in the retina. In patients with advanced glaucoma and considerable loss of vision, stem cells replace ganglion cells and replace connections as well as regrow optic nerve fibers to carry signals to the brain.
Patient-Funded Trials for Glaucoma
Private foundations, pharmaceutical companies, and the NIH traditionally fund medical research into new treatments, including stem cell therapies. However, clinical trials are expensive and funding is limited. The decreasing availability of funds from traditional sources has led to the emergence of patient-funded research where patients pay thousands of dollars to participate in a study. One patient was recently reported to have paid $20,000 to receive stem cell injections in one eye.
Is Patient-Funded Research Scientific?
On the surface, it would appear that patient-funded research is a great way for people to receive investigational therapies. However, there are both scientific and ethical factors that must be taken into consideration. Scientifically, the gold standard in medical research is a randomized clinical trial with a large number of participants. The study participants are randomly divided into a treatment group and a control group. Patients in the control group receive either a placebo or standard approved therapies. In double-blind trials, to minimize bias, the patient and doctor do not know whether the patient is receiving the test drug or not.
This is the scientific method of assessing the safety and efficacy of any new treatment. In patient-funded research, the control group is absent (since it is unlikely someone will pay to not receive therapy). The fact that this critical step is overlooked in patient-funded research is deeply concerning. Without the control group, there is no way to tell whether the treatment under investigation is truly effective.
Is Patient-Funded Research Ethical?
Equally concerning are the ethical considerations of patient-funded research. The first factor is the disparity in accessibility where the patients who need the treatment most may not be able to afford it. The second consideration is the risk of exploitation where patients who have exhausted all options may opt for a treatment for which safety is unproven. Thirdly, patient-funded trials are not subject to the same regulations and monitoring as randomized clinical trials. Therefore, some physicians and clinics may use this approach to make a quick buck off unsuspecting patients by offering treatments that do not have FDA approval.