Stem cell ophthalmology treatment study (SCOTS) for retinal and optic nerve diseases: A preliminary report

Jeffrey N. Weiss, Steven Levy, Alexis Malkin

Research output: Contribution to journalArticlepeer-review

Abstract

In this report, we present the results of a single patient with optic neuroprotective and immunomodulatory effects and havean important role in the treatment of nervous tissue diseaseincluding retinal and optic nerve diseases through brain-derivedneurotrophic factor as reported by Wilkins et al. (2009).Methodology of SCOTSSCOTS, the Stem Cell Ophthalmology Treatment Study, is thelargest ophthalmology stem cell study registered at the NationalInstitutes of Health: Identifier NCT Number 01920867 SCOTS is an open label, non-randomized, efficacy study.There is no placebo or sham arm. All patients enrolled in thestudy meet eligibility criteria and receive active treatment.Bone marrow aspirated from the posterior iliac crest is separatedto provide BMSCs within the stem cell concentrate.Inclusion criteria for SCOTS include that patients should• Have objective, documented damage to the retina or opticnerve unlikely to improve OR.• Have objective, documented damage to the retina or opticnerve that is progressive.• AND have less than or equal to 20/40 best corrected centralvisual acuity in one or both eyes AND/OR an abnormalAbstractIn this report, we present the results of a single patient with optic neuropathy treated within theStem Cell Ophthalmology Treatment Study (SCOTS). SCOTS is an Institutional Review Boardapproved clinical trial and is the largest ophthalmology stem cell study registered at the NationalInstitutes of Health to date- www.clinicaltrials.gov Identifier NCT 01920867. SCOTS utilizesautologous 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 WilmerEye Institute at the Johns Hopkins Hospital, USA. A 27 year old female patient had lost visionapproximately 5 years prior to enrollment in SCOTS. Pre-treatment best-corrected visual acuityat the Wilmer Eye Institute was 20/800 Right Eye (OD) and 20/4,000 Left Eye (OS). Four monthsfollowing treatment in SCOTS, the central visual acuity had improved to 20/100 OD and 20/40OS.

Original languageEnglish (US)
Pages (from-to)982-988
Number of pages7
JournalNeural Regeneration Research
Volume10
Issue number6
DOIs
StatePublished - Jun 1 2015

Keywords

  • Blindness
  • Bone marrow-derived stem cells
  • Ophthalmology
  • Optic nerve
  • Optic neuropathy
  • Stem cells
  • Visual loss

ASJC Scopus subject areas

  • Developmental Neuroscience

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