Stem cell clinical trials are driving significant progress in regenerative medicine, offering new hope for patients with challenging conditions. In ophthalmology, experimental treatments for vision loss, such as retinal pigment epithelium cell transplants for macular degeneration and photoreceptor cell therapy for retinitis pigmentosa, have shown early signs of improving visual function while maintaining good safety profiles. Approved therapies like cultured limbal stem cells for corneal repair further demonstrate the real-world potential of this approach. In neurological disorders, advances include dopaminergic neuron replacement for Parkinson’s disease, which has yielded measurable improvements in motor symptoms, alongside ongoing research into stem cell applications for ALS and multiple sclerosis.

Orthopedic medicine is also benefiting from stem cell innovation, with therapies aimed at regenerating cartilage, repairing joint damage, and restoring spine health. Clinical studies in knee osteoarthritis have reported reductions in pain, enhanced mobility, and signs of cartilage restoration, while stem cell injections for degenerative disc disease show potential to improve disc stability and relieve chronic back pain. Collectively, these developments highlight the growing role of stem cells in shaping the future of advanced, targeted, and durable treatments.

Ophthalmology

Intravitreal Autologous Mesenchymal Stem Cell Transplantation in Retinitis Pigmentosa (Phase I; NCT01531348) – Safety, feasibility, and short-term efficacy of BM-MSCs injected intravitreally in advanced RP patients.

Early-Stage Phase I Trial of CD34⁺ Stem Cell Injection for Retinitis Pigmentosa – UC Davis – Autologous CD34⁺ stem cells from bone marrow injected into RP patients’ eyes; 4 of 7 participants improved vision with no serious adverse events.OpCT-001: First Human Trial Using iPSC-Derived Photoreceptors for Advanced Retinitis Pigmentosa – FDA approved in 2024; first patient enrollment expected in 2025.

OpRegen® (RG6501) Phase 1/2a Clinical Study for Geographic Atrophy in Dry AMD – Subretinal allogeneic RPE cells from hESCs; vision gains and anatomical improvements seen in early trials.

Neurological Disorders

Bemdaneprocel (BRT-DA01) Phase I Trial for Parkinson’s Disease – ESC-derived dopaminergic neuron precursors transplanted into the putamen; safe and showed symptomatic improvement at 1 year.

5-Year Observational Follow-Up Study of BRT-DA01 Recipients (NCT05897957) – Tracks long-term safety and clinical outcomes post-transplantation.

Orthopedic Disorders

CARTISTEM® Umbilical Cord MSCs for Knee Cartilage Defects (Phase 3, Korea) – UCB-MSCs with hyaluronate showed superior cartilage repair and symptom improvement vs microfracture over 5 years.

Mesoblast MPC-06-ID for Chronic Low Back Pain Due to Disc Degeneration (Phase 2) – Allogeneic mesenchymal precursor cells injected into the disc; signaled disc stability and reduced pain.

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