Background:
- The study reports the results of 6 patients with Dominant Optic Atrophy (DOA) who were treated in the Stem Cell Ophthalmology Treatment Study (SCOTS).
- SCOTS/SCOTS 2 is an Institutional Review Board approved and NIH registered clinical study that uses autologous bone marrow derived stem cells (BMSC) to treat optic nerve and retinal diseases.
Methods:
- The study is open-label and non-randomized, using the natural history of the disease as a comparator.
- BMSC were separated from aspirated autologous bone marrow using an FDA cleared Class II medical device.
- Patients were treated with combinations of retrobulbar, subtenons, intravitreal, or subretinal placement of BMSC, followed by an intravenous injection of BMSC. The specific treatment arm was chosen based on various factors, including the degree of visual loss and the patient's medical status.
Results:
- 83.3% (5 out of 6) of the treated patients experienced visual improvements, and in all these cases, both eyes showed improvement.
- Ten eyes (or 83.3%) experienced gains in visual acuity with a median improvement of 2.125 Snellen lines, which is approximately 10.63 letters.
- Using LogMAR, the average improvement in vision for all eyes was 29.5%. For eyes that showed improvement, the average visual acuity increase was 33.3%.
- These findings were statistically significant with a p-value of less than 0.001.
Conclusions:
- Using autologous BMSC as per the protocols developed in the SCOTS/SCOTS 2 clinical studies resulted in statistically significant visual acuity improvements in patients with DOA or Kjers Optic Neuropathy.
- Improvements were observed in 83.3% of eyes, with an average improvement of 29.5%.
- The study suggests that mitochondrial transfer and neuroprotective exosome secretions from the BMSC might have been key factors in the observed improvements in this mitochondrial disease.