A small study of Stargardt’s disease patients found the vast majority of individuals with the condition had improved or stabilized vision after autologous bone marrow stem cell treatment.

Out of 34 treated eyes, about 62% improved and approximately 24% remained stable, while roughly 15% showed continued progression of the disease. The participants received treatment as part of the Stem Cell Ophthalmology Treatment Study (SCOTS and SCOTS2) and were followed up at one year.

 The average central vision improvement following treatment was about 18%, and ranged up to approximately 81%. In the 17 patients that were treated, 13 showed VA improvement in one or both eyes, three individuals showed no net loss, and only one subject’s condition worsened as a consequence of disease progression. Of note, about 94% of patients had improved vision or remained stable, and the researchers reported no adverse events following treatment.

Stargardt’s disease patients with preoperative 20/400 or worse vision didn’t generally improve beyond 20/100. While such degrees of visual recovery are meaningful and beneficial from a functional perspective, this suggests that recovery of full macular function with a single treatment may be difficult, researchers noted. Still, one patient went from counting fingers to 20/50+2.

Patients with a VA better than 20/200 generally maintained macular function, and in some cases, experienced significant improvement. This may suggest that earlier treatment could provide greater opportunity for the mechanisms of action of the bone marrow stem cells to reverse damage and offer neuro-protection, investigators said.

While further investigation is needed to determine which specific mutations and degrees of maculopathy will most likely benefit, the study’s bone marrow-derived stem cell approach may be considered a treatment option for patients with this condition, they added.

Weiss JN, Levy S. Stem cell ophthalmology treatment study (SCOTS): bone marrow-derived stem cells in the treatment of Stargardt disease. Medicines (Basel). 2021 Feb 3;8(2):10.