What side effects are associated with this type of intervention?

Common treatments for Retinitis Pigmentosa (RP) include gene therapy, vitamin A supplementation, and omega-3 fatty acids. Gene therapy, which targets specific genetic mutations, can cause immune reactions, inflammation, and potential off-target effects. Vitamin A supplementation may lead to liver toxicity and hypervitaminosis A if not monitored properly. Omega-3 fatty acids are generally well-tolerated but can cause gastrointestinal issues in some individuals. Treatments similar to CNS10-NPC cells, such as cell transplantation, have shown medium- to long-term safety in initial studies but may carry risks of immune rejection, infection, and potential tumor formation. Further studies are needed to confirm the long-term safety and efficacy of these treatments.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically involving neural progenitor cells or similar stem cell therapies for Retinitis Pigmentosa (RP). The most notable FDA-approved treatment for RP is Luxturna (voretigene neparvovec-rzyl), a gene therapy for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. This treatment works by delivering a normal copy of the RPE65 gene directly to retinal cells. Other treatments are primarily supportive, focusing on slowing disease progression and managing symptoms.

What other types of research exist?

Promising alternatives to CNS10-NPC cells for Retinitis Pigmentosa treatment in 2024 include: 1) Induced Pluripotent Stem Cells (iPSCs) which can be differentiated into retinal cells, 2) Gene therapy approaches targeting specific genetic mutations causing RP, and 3) Retinal implants or prosthetics that can restore some degree of vision. These alternatives are being actively researched and have shown potential in preclinical and early clinical studies.

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Stem Cell Therapy

Stem Cell Implantation for Retinitis Pigmentosa

Phase 1

Recruiting

Led By David Liao, MD, PhD

Research Sponsored by Cedars-Sinai Medical Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

4. Participant will be medically able to undergo ophthalmic surgery.

Participant with diagnosis of retinitis pigmentosa

Must not have

Any known allergy to any component of the delivery vehicle or diagnostic agents used during the study (e.g., dilation drops), or medications planned for use during the peri-operative period including corticosteroids, tacrolimus and mycophenolate

Contraindications to use of anesthesia

Timeline

Screening 3 weeks

Treatment Varies

Follow Up performed 5 times over 15 months

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing if introducing special cells into the eye is safe for patients with vision loss. The cells might help protect and support the existing nerve cells in the eye, potentially slowing down or stopping vision loss.

Who is the study for?

Adults diagnosed with Retinitis Pigmentosa (RP) who have visual acuity of 20/200 or worse, or a central visual field of 40 degrees diameter or less. They must be able to undergo eye surgery and sign informed consent. Excluded are those on certain medications, with allergies to specific antibiotics, recent substance abuse, ocular abnormalities that could affect the study's outcome, systemic diseases affecting ocular function, pregnant/nursing women, and unwillingness to use contraception.

What is being tested?

The trial is testing the safety of implanting CNS10-NPC cells into the subretinal space in RP patients. These neural progenitor cells are designed to become astrocytes in hopes of treating RP. This is an initial human test following animal studies.

What are the potential side effects?

Potential side effects aren't specified but may include typical risks associated with ophthalmic surgery such as infection, inflammation, vision changes or loss. There might also be immune reactions due to cell transplantation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am medically cleared for eye surgery.

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I have been diagnosed with retinitis pigmentosa.

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I have limited side vision.

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I have been diagnosed with retinitis pigmentosa.

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I have been diagnosed with retinitis pigmentosa.

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I have limited side vision.

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I have symptoms of retinitis pigmentosa.

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I am 18 years old or older.

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I have a history of night blindness.

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I have changes in the color of my retina.

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I have been diagnosed with retinitis pigmentosa.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am not allergic to any medications or substances used in this study.

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I cannot have anesthesia due to health reasons.

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I can travel to the study center and understand the consent without mental impairments.

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I have a history of HIV or hepatitis A, B, or C.

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My eyes are healthy and have no history of conditions that could complicate surgery.

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I am on or expect to be on steroids for more than a week.

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I am not currently on immunosuppressant therapies and have no contraindications to them.

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I have swelling in the central part of my retina.

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I have G6PD deficiency.

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I cannot have my pupils dilated due to a medical condition.

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I am currently pregnant.

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I do not have major eye problems that would affect surgery or study results.

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I am not nursing or planning to nurse for 6 months after treatment.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ performed 5 times over 15 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~performed 5 times over 15 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and performed 5 times over 15 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Safety, as evaluated by changes in Spectral Domain Optical Coherence Tomography (SD-OCT)

Safety, as evaluated by changes in Visual Acuity

Safety, as evaluated by changes in visual field

Secondary study objectives

Spectral domain optical coherence tomography (SD-OCT)

Visual Function Questionnaire-25 (VFQ-25)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups

Experimental Treatment

Group I: Group 2Experimental Treatment1 Intervention

Visual acuity between 20/80 and 20/200 Single, unilateral, subretinal injection of 1,000,000 CNS10-NPC (n=10)

Group II: Group 1BExperimental Treatment1 Intervention

Visual acuity of 20/200 or worse Single, unilateral, subretinal injection of 1,000,000 CNS10-NPC (n=3)

Group III: Group 1AExperimental Treatment1 Intervention

Visual acuity of 20/200 or worse Single, unilateral, subretinal injection of 300,000 CNS10-NPC (n=3)

0 / 24Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Retinitis Pigmentosa (RP) include investigational therapies like CNS10-NPC cells, which are neural progenitor cells derived to become astrocytes and transplanted into the subretinal space to support retinal health. This approach aims to replace or support damaged retinal cells, potentially slowing disease progression. Other treatments include vitamin A supplementation to slow retinal function decline and omega-3 fatty acids like DHA to support retinal cell structure. These treatments are crucial for RP patients as they offer potential ways to preserve vision and slow the progression of the disease.