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

Trial Summary

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.

What data supports the idea that Stem Cell Therapy for Retinal and Optic Nerve Disorders is an effective treatment?

The available research shows that Stem Cell Therapy for Retinal and Optic Nerve Disorders can be effective. For example, in a study on patients with relapsing optic neuropathy, one patient experienced significant improvement in vision after treatment, with visual acuity improving from very poor to nearly normal in one eye. In another study on age-related macular degeneration, 63% of treated eyes showed improved vision, and the treatment was safe with no complications. These results suggest that the therapy can help improve vision in certain eye conditions. ^{1 2 3 4 5}

What safety data is available for stem cell therapy in retinal and optic nerve disorders?

The Stem Cell Ophthalmology Treatment Study (SCOTS) and SCOTS 2 have evaluated the safety of autologous bone marrow-derived stem cells (BMSC) in treating retinal and optic nerve disorders. These studies are compliant with NIH and OHRP standards and have been approved by Institutional Review Boards. Across various conditions like Retinitis Pigmentosa, non-arteritic ischemic optic neuropathy, age-related macular degeneration, and relapsing auto-immune optic neuropathy, the procedures were conducted safely with no reported complications. The studies demonstrated improvements in visual acuity and stability in most cases, indicating a favorable safety profile for the treatment. ^{1 2 3 4 6}

Is the Stem Cell Therapy for Retinal and Optic Nerve Disorders a promising treatment?

Yes, the Stem Cell Therapy using autologous bone marrow-derived stem cells (BMSC) shows promise for treating retinal and optic nerve disorders. Studies have shown improvements in vision for patients with conditions like Retinitis Pigmentosa and optic neuropathy. The therapy uses the patient's own stem cells, which can help repair damaged eye tissues and improve vision without needing donor matching or strong immune system drugs. ^{1 2 4 7 8}

What is the purpose of this trial?

This trial will use stem cells from patients' own bone marrow to treat severe vision loss caused by retinal or optic nerve damage. The stem cells will be injected into the eye to help repair the damaged tissues. SCOTS is the largest ophthalmology stem cell study registered at the National Institutes of Health, using autologous bone marrow-derived stem cells for retinal and optic nerve diseases.

Research Team

Steven Levy, MD

Principal Investigator

MD Stem Cells

Jeffrey Weiss, MD

Principal Investigator

Coral Springs Florida, Vienna Austria, Dubai UAE

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

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]

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]

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 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]

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]

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]

References

1. China 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). [2022]

2. India 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. [2022]

3. Switzerland pubmed.ncbi.nlm.nih.gov

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

4. China pubmed.ncbi.nlm.nih.gov

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

5. China pubmed.ncbi.nlm.nih.gov

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

6. India pubmed.ncbi.nlm.nih.gov

Stem Cell Ophthalmology Treatment Study (SCOTS) for retinal and optic nerve diseases: a preliminary report. [2022]

7. United Arab Emirates pubmed.ncbi.nlm.nih.gov

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

8. England pubmed.ncbi.nlm.nih.gov

Advances in bone marrow stem cell therapy for retinal dysfunction. [2020]