Stem Cell Ophthalmology Treatment Study: bone marrow derived stem cells in the treatment of Retinitis Pigmentosa

Weiss, Jeffrey N.; Levy, Steven

doi:10.21037/sci.2018.04.02

Cited by 45 publications

(35 citation statements)

References 22 publications

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“…Of eyes treated, 45.5% showed improvement averaging a 40.9% increase on the LogMAR vision scale with 45.5% remaining secure during the period of follow up. This is the first time any treatment has proven beneficial to acuity or stabilized vision in a hereditary retinopathy [21].…”

Section: Discussionmentioning

confidence: 81%

Stem Cell Ophthalmology Treatment Study (SCOTS): Bone Marrow-Derived Stem Cells in the Treatment of Age-Related Macular Degeneration

Weiss

Levy²

2020

Medicines

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Background: Dry age-related macular degeneration (AMD) is one of the leading causes of vision loss in older patients. The macula accumulates drusen with loss of retinal pigment epithelial cells and photoreceptors. Abnormal subretinal neovascularization is absent. There is no effective drug therapy for dry AMD and a large proportion of patients progress to legal blindness from macular atrophy. The Stem Cell Ophthalmology Treatment Study (SCOTS) was conducted to assess the effect of bone marrow-derived stem cells (BMSCs) on dry AMD and other retinal and optic nerve diseases. Methods: Thirty-two eyes were treated with BMSC per the protocols in SCOTS. Provision of BMSCs in Arm 1 was via retrobulbar (RB), sub-tenons (ST) and intravenous (IV); Arm 2 via intravitreal, RB, ST and IV; Arm 3 via subretinal and IV. Patient age averaged 78 years old and ranged from 69 to 90. Visual acuity preoperatively ranged from counting fingers to 20/50-2 with an average preoperative LogMAR of 1.125. Results: Following treatment, 20 of 32 (63%) of eyes experienced improvement in visual acuity averaging 27.6% on LogMAR and ranging from 2.5% to 44.6%. The mean improvement in LogMAR was 0.963 with a standard deviation (SD) of 0.42. The visual acuity remained stable in 34% of treated eyes. One eye continued to worsen as a consequence of disease progression. The results showed high statistical significance with p ≤ 0.001. The procedures were conducted safely, and no complications were observed. Conclusion: Treatment of dry AMD with BMSC using the protocols developed in the SCOTS clinical trial has shown statistically significant clinical benefit improving visual acuity and potentially delaying visual loss in the disease.

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Section: Discussionmentioning

confidence: 81%

Stem Cell Ophthalmology Treatment Study (SCOTS): Bone Marrow-Derived Stem Cells in the Treatment of Age-Related Macular Degeneration

Weiss

Levy²

2020

Medicines

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“…Mesenchymal stem cells (MSCs), a multipotent stromal cell, are currently being tested in clinical trials due to having demonstrated efficacy in a multitude of retinal disease models (reviewed in Ref. 3) 4–7. Their mechanism of action was shown to be paracrine mediated through the secretion of neurotrophic factors such as nerve growth factor, brain-derived neurotrophic factor,8 and platelet-derived growth factor,9 and more recently, exosomes 10,11…”

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confidence: 99%

Mesenchymal Stem Cell–Derived Small Extracellular Vesicles Promote Neuroprotection in a Genetic DBA/2J Mouse Model of Glaucoma

Mead

Ahmed

Tomarev

2018

Invest. Ophthalmol. Vis. Sci.

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PurposeTo determine if bone marrow-derived stem cell (BMSC) small extracellular vesicles (sEV) promote retinal ganglion cell (RGC) neuroprotection in the genetic DBA/2J mouse model of glaucoma for 12 months.MethodsBMSC sEV and control fibroblast-derived sEV were intravitreally injected into 3-month-old DBA/2J mice once a month for 9 months. IOP and positive scotopic threshold responses were measured from 3 months: IOP was measured monthly and positive scotopic threshold responses were measured every 3 months. RGC neuroprotection was determined in wholemounts stained with RNA binding protein with multiple splicing (RBPMS), whereas axonal damage was assessed using paraphenylenediamine staining.ResultsAs expected, DBA/2J mice developed chronic ocular hypertension beginning at 6 months. The delivery of BMSC sEV, but not fibroblast sEV, provided significant neuroprotective effects for RBPMS+ RGC while significantly reducing the number of degenerating axons seen in the optic nerve. BMSC sEV significantly preserved RGC function in 6-month-old mice, but provided no benefit at 9 and 12 months.ConclusionsBMSC sEV are an effective neuroprotective treatment in a chronic model of ocular hypertension for 1 year, preserving RGC numbers and protecting against axonal degeneration.

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“…Those results have been determined to be statistically significant [17]. Additional reports on other hereditary conditions including Retinitis Pigmentosa [18], Ushers Syndrome [19], and Autosomal Dominant Optic Atrophy [20] have also shown statistically significant visual acuity improvements.…”

Section: Discussionmentioning

confidence: 99%

Stem Cell Ophthalmology Treatment Study (SCOTS): Bone Marrow-Derived Stem Cells in the Treatment of Stargardt Disease

Weiss¹,

Levy²

2021

Medicines

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Background: Stargardt Disease is the most common inherited macular degeneration, typically resulting in progressive central vision loss and legal blindness at an early age. We report regarding 34 eyes with Stargardt Disease treated in the Stem Cell Ophthalmology Treatment Study (SCOTS and SCOTS2). Methods: Autologous bone marrow was processed, separating the stem cell fraction which was provided Arms using retrobulbar, subtenons, intravitreal or subretinal and intravenous. The follow-up period was one year. Results: Of the 34 treated eyes, 21 (61.8%) improved, 8 (23.5%) remained stable, and 5 (14.7%) showed continued progression of their disease. Results were statistically significant with p = 0.0004. The average central vision improvement following treatment was 17.96% (95%CI, 16.39–19.53%) and ranged up to 80.5%. Of 17 patients treated, 13 (76.5%) showed visual acuity improvement in one or both eyes, 3 patients (17.6%) showed no net loss, and 1 worsened as a consequence of disease progression; 94.1% of patients had improved vision or remained stable. There were no adverse events. Conclusions: Patients with Stargardt Disease may potentially benefit from autologous bone marrow-derived stem cells (BMSC) as provided in SCOTS. Improvement or stabilization of vision was found to occur for the vast majority of reported patients and findings were highly statistically significant.

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Stem Cell Ophthalmology Treatment Study: bone marrow derived stem cells in the treatment of Retinitis Pigmentosa

Cited by 45 publications

References 22 publications

Stem Cell Ophthalmology Treatment Study (SCOTS): Bone Marrow-Derived Stem Cells in the Treatment of Age-Related Macular Degeneration

Stem Cell Ophthalmology Treatment Study (SCOTS): Bone Marrow-Derived Stem Cells in the Treatment of Age-Related Macular Degeneration

Mesenchymal Stem Cell–Derived Small Extracellular Vesicles Promote Neuroprotection in a Genetic DBA/2J Mouse Model of Glaucoma

Stem Cell Ophthalmology Treatment Study (SCOTS): Bone Marrow-Derived Stem Cells in the Treatment of Stargardt Disease

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