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Jeffrey N. Weiss, M.D.

1. Weiss JN, Levy S, Benes SC.  Stem Cell Ophthalmology Treatment Study: bone marrow derived stem cells in the treatment of non-arteritic ischemic optic neuropathy (NAION). Stem Cell Investig 2017; 4:94.

https://sci.amegroups.com/article/view/17421/17703

Abstract:

Background: Ten patients with bilateral visual loss due to sequential non-arteritic ischemic optic neuropathy (NAION) underwent autologous Bone Marrow Derived Stem Cell (BMSC) therapy within the Stem Cell Ophthalmology Treatment Study (SCOTS). SCOTS is an Institutional Review Board approved clinical study utilizing autologous BMSC in the treatment of optic nerve and retinal diseases that meet inclusion criteria.

Methods: The average age of the patients treated was 69.8 years. The average duration of visual loss in eyes treated was 9.8 years and ranged from 1 to 35 years. Affected eyes were treated with either retrobulbar, subtenons and intravenous BMSC or, following vitrectomy, intra-optic nerve, subtenons and intravenous BMSC. The primary outcome was visual acuity as measured by Snellen or converted to LogMAR.

Results: Following therapy in SCOTS, 80% of patients experienced improvement in Snellen binocular vision (P=0.029) with 20% remaining stable; 73.6% of eyes treated gained vision (P=0.019) and 15.9% remained stable in the post-operative period. There was an average of 3.53 Snellen lines of vision improvement per eye with an average 22.74% and maximum 83.3% improvement in LogMAR acuity per eye. The average LogMAR change in treated eyes was a gain of 0.364 (P=0.0089). Improvements typically manifested no later than 6 months post procedure.

Conclusions: The use of BMSC in the Stem Cell Ophthalmology Treatment Study achieved meaningful visual improvements in a significant percentage of the NAION patients reported. Improvements typically manifested no later than 6 months post-procedure. Duration of visual loss did not appear to affect the ability of the eyes to respond to treatment. Possible mechanisms by which visual improvement occurred may include BMSC paracrine secretion of proteins and hormones, transfer of mitochondria, release of messenger RNA or other compounds via exosomes or microvesicles and neuronal transdifferentiation of the stem cells.

Keywords: Non-arteritic Ischemic Optic Neuropathy (NAION); optic neuropathy; stem cells; bone marrow derived stem cells (BMSC); blindness; visual loss

 

  1. Weiss JN, Levy S, Malkin A. Stem Cell Ophthalmology Treatment Study (SCOTS) for retinal and optic nerve diseases: a preliminary report. Neural Regen Res. 2015 Jun; 10(6): 982–988.

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4498363/

Abstract:

In this report, we present the results of a single patient with optic neuropathy treated within the Stem Cell Ophthalmology Treatment Study (SCOTS). SCOTS is an Institutional Review Board approved clinical trial and is the largest ophthalmology stem cell study registered at the National Institutes of Health to date- www.clinicaltrials.gov Identifier NCT 01920867. SCOTS utilizes autologous bone marrow-derived stem cells in the treatment of optic nerve and retinal diseases. Pre- and post-treatment comprehensive eye exams were independently performed at the Wilmer Eye Institute at the Johns Hopkins Hospital, USA. A 27 year old female patient had lost vision approximately 5 years prior to enrollment in SCOTS. Pre-treatment best-corrected visual acuity at the Wilmer Eye Institute was 20/800 Right Eye (OD) and 20/4,000 Left Eye (OS). Four months following treatment in SCOTS, the central visual acuity had improved to 20/100 OD and 20/40 OS.

 

  1. Weiss JN, Levy S, Benes SC. Stem Cell Ophthalmology Treatment Study (SCOTS) for retinal and optic nerve diseases: a case report of improvement in relapsing auto-immune optic neuropathy. Neural Regen Res. 2015 Sep; 10(9): 1507–1515.

http://www.nrronline.org/article.asp?issn=1673-5374;year=2015;volume=10;issue=9;spage=1507;epage=1515;aulast=Weiss

Abstract:

We present the results from a patient with relapsing optic neuropathy treated within the Stem Cell Ophthalmology Treatment Study (SCOTS). SCOTS is an Institutional Review Board approved clinical trial and has become the largest ophthalmology stem cell study registered at the National Institutes of Health to date (www.clinicaltrials.gov Identifier NCT 01920867). SCOTS utilizes autologous bone marrow-derived stem cells (BMSCs) for treatment of retinal and optic nerve diseases. Pre-treatment and post-treatment comprehensive eye exams of a 54 year old female patient were performed both at the Florida Study Center, USA and at The Eye Center of Columbus, USA. As a consequence of a relapsing optic neuritis, the patient’s previously normal visual acuity decreased to between 20/350 and 20/400 in the right eye and to 20/70 in the left eye. Significant visual field loss developed bilaterally. The patient underwent a right eye vitrectomy with injection of BMSCs into the optic nerve of the right eyeand retrobulbar, subtenon and intravitreal injection of BMSCs in the left eye. At 15 months after SCOTS treatment, the patient’s visual acuity had improved to 20/150 in the right eye and 20/20 in the left eye. Bilateral visual fields improved markedly. Both macular thickness and fast retinal nerve fiber layer thickness were maximally improved at 3 and 6 months after SCOTS treatment. The patient also reduced her mycophenylate dose from 1,500 mg per day to 500 mg per day and required no steroid pulse therapy during the 15-month follow up.

 

  1. Weiss JN, Benes SC, Levy S. Stem Cell Ophthalmology Treatment Study (SCOTS): improvement in serpiginous choroidopathy following autologous bone marrow derived stem cell treatment. Neural Regen Res. 2016 [cited 2017 Nov 16];11:1512-6

http://www.nrronline.org/text.asp?2016/11/9/1512/191229

Abstract:

We report results in a 77-year-old male patient with visual loss from long-standing serpiginous choroidopathy treated with bone marrow derived stem cells (BMSC) within the Stem Cell Ophthalmology Treatment Study (SCOTS). SCOTS is an Institutional Review Board approved clinical trial and the largest ophthalmology stem cell study registered at the National Institutes of Health to date (ClinicalTrials.gov Identifier: NCT01920867). Eight months after treatment by a combination of retrobulbar, subtenon, intravitreal and intravenous injection of BMSC, the patient’s best corrected Snellen acuity improved from 20/80 to 20/60 +1 in the right eye and from 20/50 to 20/20 -3 in the left eye. The Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity continued to improve over the succeeding 8 months and the optical coherence tomography macular volume increased. The increases in visual acuity and macular volume are encouraging and suggest that the use of BMSC as provided in SCOTS may be a viable approach to treating serpiginous choroidopathy.

 

  1. Weiss JN, Levy S, Benes SC. Stem Cell Ophthalmology Treatment Study (SCOTS): bone marrow-derived stem cells in the treatment of Leber’s hereditary optic neuropathy. Neural Regen Res. 2016 Oct;11(10):1685-1694.

http://www.nrronline.org/article.asp?issn=1673-5374;year=2016;volume=11;issue=10;spage=1685;epage=1694;aulast=Weiss

Abstract:

The Stem Cell Ophthalmology Treatment Study (SCOTS) is currently the largest-scale stem cell ophthalmology trial registered at ClinicalTrials.gov (identifier: NCT01920867). SCOTS utilizes autologous bone marrow-derived stem cells (BMSCs) to treat optic nerve and retinal diseases. Treatment approaches include a combination of retrobulbar, subtenon, intravitreal, intra-optic nerve, subretinal, and intravenous injection of autologous BMSCs according to the nature of the disease, the degree of visual loss, and any risk factors related to the treatments. Patients with Leber’s hereditary optic neuropathy had visual acuity gains on the Early Treatment Diabetic Retinopathy Study (ETDRS) of up to 35 letters and Snellen acuity improvements from hand motion to 20/200 and from counting fingers to 20/100. Visual field improvements were noted. Macular and optic nerve head nerve fiber layer typically thickened. No serious complications were seen. The increases in visual acuity obtained in our study were encouraging and suggest that the use of autologous BMSCs as provided in SCOTS for ophthalmologic mitochondrial diseases including Leber’s hereditary optic neuropathy may be a viable treatment option.

 

  1. Weiss JN, Levy S. Autologous Bone-Marrow Derived Stem Cells in the Treatment of “Untreatable” Optic Nerve and Retinal Conditions. EC Ophthalmology. 9(5): 332-336.

https://www.ecronicon.com/ecop/pdf/ECOP-09-00307.pdf

See PDF

 

  1. Weiss JN, Levy S. Stem Cell Ophthalmology Treatment Study: bone marrow derived stem cells in the treatment of Retinitis Pigmentosa.  Stem Cell Investigation June 2018

http://sci.amegroups.com/article/view/19760

Abstract:

Background: Seventeen patients with bilateral visual loss due to Retinitis Pigmentosa (RP) underwent autologous Bone Marrow Derived Stem Cell (BMSC) treatment within the Stem Cell Ophthalmology Treatment Study (SCOTS and SCOTS 2).  Both are National Institutes of Health (NIH) and Office of Human Research Protection (OHRP) compliant Institutional Review Board (IRB) approved clinical studies utilizing using autologous BMSC in the treatment of retinal and optic nerve diseases that meet inclusion criteria.

Methods: The average age of the patients treated was 48.8 years. The average duration of disease prior to treatment was 27.6 years and ranged from 4 to approximately 60 years.  Affected eyes were treated with either retrobulbar, subtenons and intravenous BMSC or retrobulbar, subtenons, intravitreal and intravenous. Follow up was provided a minimum of 6 months. The primary outcome was visual acuity as measured by Snellen or converted to LogMAR.

Results: Following therapy in SCOTS or SCOTS 2, 11 patients (64.7%) showed improved binocular vision averaging 10.23 lines of Snellen acuity per eye over pre-treatment acuity; 8 patients (35.3%) remaining stable over the follow up period; no patients experiencing loss of overall acuity.  In 33 treated eyes, 15 eyes (45.5%) improved an average of 7.9 lines of Snellen acuity, 15 eyes (45.5%) remained stable, and 3 eyes (9%) worsened by an average of 1.7 lines of Snellen acuity. Improvements ranged from 1 to 27 lines of vision. Using the Logmar Scale and calculating delta as a ratio to pre-treatment vision in improved eyes, acuity improvement ranged from 23% to 90% with an average of 40.9% visual acuity improvement over baseline vision. Evaluation of all patients and eyes capable of LogMAR vision showed an average of 31% improvement in vision over baseline. Findings were of statistical significance (p= 0.016). There were no surgical complications.

Conclusions:The BMSC protocols of the Stem Cell Ophthalmology Treatment Study achieved meaningful visual acuity improvements or stability in Retinitis Pigmentosa that were of statistical significance. Duration of disease did not appear to affect the ability of eyes to respond. Safety was confirmed. Possible mechanisms by which improvement occurred may include transdifferentiation of BMSC into NeuN positive cells, BMSC paracrine secretions or neurotrophic factors and  hormones, transfer of mitochondria, release of messenger RNA or other compounds via exosomes or microvesicles. Given the successful outcome in this otherwise progressive condition, consideration should be given to providing this treatment option.

 

  1. Weiss JN, Levy S. Dynamic light scattering spectroscopy of the retina—a non-invasive quantitative technique to objectively document visual improvement following ocular stem cell treatment. Stem Cell Investig 2019; 6:8.

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6509422/pdf/sci-06-2019.03.01.pdf

 

  1. Interview article with MedicalResearch.com

MedicalResearch.com –  Optic Nerve Stroke: Bone Marrow Stem Cells Provide Statistically Significant Vision Improvement
https://medicalresearch.com/stem-cells/optic-nerve-stroke-bone-marrow-stem-cells-provide-statistically-significant-vision-improvement/40837/

 

  1. Weiss JN, Levy S. Neurologic Stem Cell Treatment Study (NEST) using bone marrow derived stem cells for the treatment of neurological disorders and injuries: study protocol for a nonrandomized efficacy trial. Clin Trials Degener Dis. 2016 [cited 2019 Jun 18];1:176-80.

http://www.clinicaltdd.com/text.asp?2016/1/4/176/196984

 

  1. 11. Weiss JN, Levy S. Stem Cell Ophthalmology Treatment Study (SCOTS): Autologous bone-marrow derived stem cells in the treatment of hereditary macular degeneration. EC Ophthalmology. 10(7): July 01, 2019

https://www.ecronicon.com/ecop/volume10-issue7.php

Abstract

Purpose: We report a case of hereditary macular degeneration showing drusen and macular atrophy, clinically considered consistent with Malattia Leventinese, which obtained a significant improvement in vision following treatment with autologous Bone Marrow Derived Stem Cells (BMSC) in the Stem Cell Ophthalmology Treatment Study (SCOTS) National Clinical Trial Number 01920867.

Methods: A 53 year old male presented with a history of hereditary macular degeneration showing geographic atrophy and drusen,  thought consistent with Malattia Leventinese. Screening examination showed best-corrected vision of 20/400 in the right eye (OD)  and 20/2000 in the left eye (OS). Patient met inclusion criteria of SCOTS and underwent Arm 2 OD consisting of BMSC provided retro- bulbar, subtenons and intravitreal; Arm 3 OS consisting of vitrectomy followed by BMSC provided subretinal and subtenons; followed  by BMSC intravenous. Procedures were performed without complications.

Results: Pre-treatment and post-treatment comprehensive eye examinations were performed by the patient’s eye physicians at a  major university unassociated with the SCOTS study. Following treatment in SCOTS there was prompt improvement in vision which  persisted. The patient’s corrected Snellen visual acuity improved from 20/400 right eye (OD) and 20/2000 left eye (OS) pre-treat- ment to a final 20/25 OD and 20/40+2 OS at 14 months post-treatment.

Conclusions: A case of hereditary macular degeneration, thought clinically consistent with Malattia Leventinese (ML), was treated  in the SCOTS clinical trial with autologous BMSC. The major improvement in vision that resulted suggests maculopathies showing  drusen and atrophy may respond in a strong fashion to treatment with BMSC.

 

  1. Weiss JN, Levy S. Stem Cell Ophthalmology Treatment Study (SCOTS): Bone marrow derived stem cells in the treatment of Usher syndrome. Stem Cell Investig. 2019;6:31

http://sci.amegroups.com/article/view/29013/html

Abstract:

Background: Usher syndrome is the most common form of syndromic retinitis pigmentosa and includes types I, II, and III with varying degrees of hearing loss. We present results of 10 eyes with Usher syndrome treated with autologous bone marrow derived stem cells (BMSC) within the Stem Cell Ophthalmology Treatment Study (SCOTS).

Methods: Preoperative Snellen visual acuities ranged from 20/30−1 to 20/400 with the average pre-operative Snellen acuity approximately 20/85 and the average logarithm of the minimum angle of resolution (LogMAR) acuity 0.635. All eyes had significantly impaired visual fields and patients reported hearing loss as part of this syndromic retinitis pigmentosa. Treatment using the protocols of the SCOTS study using BMSC provided by retrobulbar, subtenons, intravitreal and intravenous injections.

Results: Following treatment, 80% of the Usher eyes showed an improvement in visual acuity. Of the eyes that improved the average increase in visual acuity was 36.4% on LogMAR with improvements ranging from 23% to 94%. The average post-operative change in all treated eyes was a gain of 0.18 LogMAR and an increase in visual acuity of 28.3% on LogMAR. The results showed high statistical significance with P<0.001. Visual fields generally improved. No patient experienced a loss of vision. One patient underwent preoperative and 4-month post-operative audiometry testing which demonstrated improvement. The procedures were performed safely and without complications.

Conclusions: Findings confirm meaningful improvement in visual acuity is possible in Usher syndrome using BMSC protocols developed in the SCOTS study. Statistical significance and safety were established.

 

  1. Weiss JN, Levy S. Stem Cell Ophthalmology Treatment Study (SCOTS): bone marrow derived stem cells in the treatment of Dominant Optic Atrophy. Stem Cell Investig 2019; 6:41.

http://sci.amegroups.com/article/view/32664

Abstract:

Background: We report the results of 6 patients with Dominant Optic Atrophy (DOA) who met inclusion criteria and were treated in the Stem Cell Ophthalmology Treatment Study (SCOTS). SCOTS/SCOTS 2 is an Institutional Review Board approved and NIH registered (NCT 03011541) clinical study that uses autologous bone marrow derived stem cells (BMSC) in the treatment of optic nerve and retinal disease.
Methods: This is an open label, non-randomized clinical study using natural history of the disease as the comparator. BMSC were separated from aspirated autologous bone marrow with minimal manipulation using an FDA cleared Class II medical device. Patients were treated with combinations of retrobulbar, subtenons, intravitreal or subretinal placement of BMSC followed by intravenous injection of BMSC depending on the arm of the study chosen. There were no surgical complications.
Results: Of the patients treated, 83.3% (5 of 6 patients) experienced visual improvements and in all of these cases both eyes improved. Ten eyes or 83.3% experienced gains in visual acuity with a median improvement of 2.125 Snellen lines, or approximately 10.63 letters. Two eyes were considered unchanged compared to longstanding measurements. Using LogMAR, the average improvement in vision for all eyes was 29.5%. The average visual acuity increase in eyes that improved was 33.3%. Findings were statistically significant with P<0.001.
Conclusions: Using autologous BMSC per 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 occurred in 83.3% of eyes and averaged 29.5%. Mitochondrial transfer and neuroprotective exosome secretions from the BMSC may have been key to the improvements observed in this mitochondrial disease.

 

  1. Weiss JN, Levy S. Stem Cell Ophthalmology Treatment Study (SCOTS): bone marrow derived stem cells in the treatment of Age-Related Macular Degeneration. Medicines 2020, 7(4) 16. https://www.mdpi.com/2305-6320/7/4/16

Abstract:

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.

 

  1. Weiss JN, Levy S. Stem Cell Ophthalmology Treatment Study (SCOTS): Bone Marrow-Derived Stem Cells in the Treatment of Stargardt Disease. Medicines 2021, 8(2), 10

https://doi.org/10.3390/medicines8020010

Abstract

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