Stem Cell Therapy for Retinal and Optic Nerve Disorders

(SCOTS2 Trial)

Recruiting at 5 trial locations

Overseen By Steven Levy, MD

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: MD Stem Cells

Disqualifiers: Inadequate exam, No follow-up, No consent, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 2

Duration: 1 day

500 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores how stem cells from a participant's own bone marrow can treat damage to the retina or optic nerve, both crucial for vision. Researchers aim to determine if this treatment can improve vision or prevent further deterioration. Participants might receive the stem cells through an injection near the eye or into the bloodstream. This trial may suit individuals with documented vision issues from retinal or optic nerve damage that are worsening or unlikely to improve independently. As an unphased trial, it offers a unique opportunity to contribute to groundbreaking research that could lead to new treatments for vision loss.

Do I have to stop taking my current medications for the trial?

The trial protocol does not specify if you need to stop taking your current medications. However, you must be stable on your current treatment for retinal or optic nerve disease and unlikely to have visual improvement from it.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that using a person's own bone marrow stem cells to treat eye and vision problems is generally safe. In previous studies, these stem cells treated various eye conditions, and patients usually tolerated the treatments well. The studies reported only minor side effects and no serious issues. Since these stem cells come from the patient's own body, the risk of rejection or complications is lower. Overall, the evidence suggests that these stem cells offer a safe option for those considering this treatment. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about using stem cell therapy for retinal and optic nerve disorders because it offers a novel approach that could potentially repair and regenerate damaged eye tissues. Unlike traditional treatments, such as medications and surgery that primarily manage symptoms or slow disease progression, this therapy uses bone marrow-derived stem cells (BMSCs) administered directly to the eye and intravenously. This method aims to restore vision by promoting healing and regeneration at a cellular level, offering hope for a more lasting and comprehensive solution to vision loss.

What evidence suggests that this trial's treatments could be effective for retinal and optic nerve disorders?

Research shows that using a person's own bone marrow stem cells might help treat damage to the retina and optic nerve. In this trial, participants will receive autologous bone marrow-derived stem cells (BMSC) as part of the treatment. Studies like the Stem Cell Ophthalmology Treatment Study (SCOTS) showed that patients with conditions such as dry age-related macular degeneration (AMD) experienced improvements after receiving these stem cells. Animal studies have used these cells to repair damaged photoreceptors, the eye cells that detect light. Some patients with optic nerve damage also saw positive results after treatment. While more research is needed, these early findings suggest that these stem cells could be a promising option for treating eye disorders. ^{1 2 3 4 6}

Who Is on the Research Team?

Steven Levy, MD

Principal Investigator

MD Stem Cells

Jeffrey Weiss, MD

Principal Investigator

Coral Springs Florida, Vienna Austria, Dubai UAE

Are You a Good Fit for This Trial?

Adults over 18 with progressive or stable damage to the retina or optic nerve, and vision no better than 20/30. They must be medically stable for the procedure, have potential to improve with BMSC treatment, and not at high risk from it. Excluded are those unable to consent, at significant health risk if treated, unable to undergo proper eye exams, or unwilling to follow up as required.

Inclusion Criteria

I could benefit from BMSC treatment with minimal risk.

I had eye surgery over 3 months ago and my condition is stable.

I am over 18 years old.

Exclusion Criteria

I am unable to understand and give consent for treatment.

I am at high risk for health or vision problems if I undergo the procedure.

I cannot undergo a thorough eye exam to document any eye conditions.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive injections of autologous bone marrow derived stem cells for retinal and optic nerve conditions

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment with comprehensive eye examinations and diagnostic testing

12 months

Visits at 1 day, 1 month, 3 months, 6 months, and 12 months post-procedure

What Are the Treatments Tested in This Trial?

Interventions

• Arm 1
• Arm 2
• Arm 3

Trial Overview The trial is testing autologous bone marrow derived stem cells (BMSC) therapy on individuals with various retinal and optic nerve diseases. The goal is to assess whether this treatment can repair damaged tissues in conditions that lead to blindness or low vision.

How Is the Trial Designed?

Group I: Arm 1 Experimental Treatment 1 Intervention

BMSC provided retrobulbar, subtenon and intravenous for one or both eyes

Arm 1 is already approved in United States for the following indications:

Approved in United States as Autologous Bone Marrow-Derived Stem Cells for:

• Retinal damage
• Optic nerve damage

Who Is Running the Clinical Trial?

MD Stem Cells

Lead Sponsor

Trials

6

Recruited

1,500+

Published Research Related to This Trial

In a study involving 32 eyes of patients aged 69 to 90, treatment with bone marrow-derived stem cells (BMSCs) resulted in a significant improvement in visual acuity for 63% of the eyes, with an average improvement of 27.6% on the LogMAR scale.

The treatment was conducted safely with no observed complications, indicating that BMSC therapy could be a promising option for improving vision and potentially delaying progression in patients with dry age-related macular degeneration.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stem Cell Ophthalmology Treatment Study (SCOTS): Bone Marrow-Derived Stem Cells in the Treatment of Age-Related Macular Degeneration. Weiss, JN., Levy, S.[2020]

Bone marrow mesenchymal stem cells (BMSCs) are promising candidates for treating eye diseases due to their ability to differentiate into various cell types, low immunogenicity, and ease of use.

The review highlights BMSCs' cellular activity, migration, immuno-modulatory, and anti-inflammatory effects, which together enhance their potential therapeutic applications in ophthalmology.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Mini-Review: The Therapeutic Potential of Bone Marrow Mesenchymal Stem Cells and Relevant Signaling Cascades. Yang, W., Ma, B.[2019]

In a clinical study involving 6 patients with Dominant Optic Atrophy, 83.3% experienced significant visual improvements after treatment with autologous bone marrow-derived stem cells, with an average increase in visual acuity of 29.5%.

The treatment was safe, with no surgical complications reported, and the observed improvements may be linked to mitochondrial transfer and neuroprotective effects from the stem cells.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stem Cell Ophthalmology Treatment Study (SCOTS): bone marrow derived stem cells in the treatment of Dominant Optic Atrophy. Weiss, JN., Levy, S.[2022]

A 54-year-old female patient with relapsing optic neuropathy showed significant improvement in visual acuity and visual fields after receiving autologous bone marrow-derived stem cells (BMSCs) as part of the Stem Cell Ophthalmology Treatment Study (SCOTS), with her right eye improving from 20/400 to 20/150 and her left eye achieving 20/20 vision over 15 months.

The treatment not only enhanced her vision but also allowed her to reduce her medication dosage, indicating a potential for improved quality of life and reduced reliance on other therapies following stem cell treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stem Cell Ophthalmology Treatment Study (SCOTS) for retinal and optic nerve diseases: a case report of improvement in relapsing auto-immune optic neuropathy. Weiss, JN., Levy, S., Benes, SC.[2022]

Bone marrow-derived stem cells, particularly mesenchymal stem cells and CD34+ cells, show promise in treating retinal dysfunction due to their ability to provide paracrine trophic effects that can benefit various retinal conditions, as supported by preclinical evidence.

Early clinical trials indicate that autologous bone marrow stem cell therapy is safe and does not require immunosuppression, although the safety of intravitreal delivery of mesenchymal stem cells needs further investigation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Advances in bone marrow stem cell therapy for retinal dysfunction. Park, SS., Moisseiev, E., Bauer, G., et al.[2020]

In a study involving 10 patients with long-term visual loss from non-arteritic ischemic optic neuropathy, 80% experienced improvement in vision after receiving autologous bone marrow-derived stem cell therapy, with significant gains in visual acuity measured by Snellen and LogMAR scales.

The improvements in vision were observed within 6 months post-treatment and were not influenced by the duration of visual loss, suggesting that the therapy may effectively restore vision through various mechanisms such as protein secretion and neuronal transdifferentiation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stem Cell Ophthalmology Treatment Study: bone marrow derived stem cells in the treatment of non-arteritic ischemic optic neuropathy (NAION). Weiss, JN., Levy, S., Benes, SC.[2022]

In a study involving 17 patients with Retinitis Pigmentosa, 64.7% experienced significant improvements in visual acuity after receiving autologous bone marrow-derived stem cell treatment, with an average improvement of 10.23 lines on the Snellen scale.

The treatment was found to be safe, with no surgical complications reported, and improvements in vision were statistically significant, suggesting that this approach could be a viable option for patients with this progressive condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stem Cell Ophthalmology Treatment Study: bone marrow derived stem cells in the treatment of Retinitis Pigmentosa. Weiss, JN., Levy, S.[2022]

A 27-year-old female patient with optic neuropathy experienced significant improvement in vision after receiving autologous bone marrow-derived stem cell treatment as part of the Stem Cell Ophthalmology Treatment Study (SCOTS).

Her visual acuity improved from 20/800 in the right eye and 20/4,000 in the left eye before treatment to 20/100 and 20/40, respectively, just four months post-treatment, indicating the potential efficacy of stem cell therapy for optic nerve damage.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stem Cell Ophthalmology Treatment Study (SCOTS) for retinal and optic nerve diseases: a preliminary report. Weiss, JN., Levy, S., Malkin, A.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT01920867

NCT01920867 | Stem Cell Ophthalmology Treatment Study This study will evaluate the use of autologous bone marrow derived stem cells (BMSC) for the treatment of retinal and optic nerve damage or disease.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7235782/

Stem Cell Ophthalmology Treatment Study (SCOTS) The Stem Cell Ophthalmology Treatment Study (SCOTS) was conducted to assess the effect of bone marrow-derived stem cells (BMSCs) on dry AMD and ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT03011541

NCT03011541 | Stem Cell Ophthalmology Treatment Study II This study will evaluate the use of autologous bone marrow derived stem cells (BMSC) for the treatment of retinal and optic nerve damage or disease. Detailed ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4498363/

Stem Cell Ophthalmology Treatment Study (SCOTS) for retinal ... In this report, we present the results of a single patient with optic neuropathy treated within the Stem Cell Ophthalmology Treatment Study (SCOTS).

5. journals.lww.com journals.lww.com/10.4103/1673-5374.193251

Stem Cell Ophthalmology Treatment Study (SCOTS) Bone marrow-derived stem cells (BMSCs) have been used to rescue photoreceptors in rhodopsin knockout mice with retinal degeneration (Arnhold et al., 2007).

6. ophthalmologyscience.org ophthalmologyscience.org/article/S2666-9145(24)00125-8/fulltext

Phase I Study of Intravitreal Injection of Autologous CD34+ ... To evaluate the feasibility and safety of intravitreal injection of autologous CD34+ stem cells from bone marrow (BMSCs) in eyes with vision ...