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Istem-01

A groundbreaking clinical trial is underway to evaluate the safety and potential effectiveness of ISTEM-01, a novel treatment for retinitis pigmentosa caused by genetic mutations. This study aims to assess the implantation of human embryonic stem cell-derived retinal pigment epithelium (RPE) cells in patients with this degenerative eye condition. The trial offers hope for those affected by this rare disease and may pave the way for innovative tissue replacement therapies in ophthalmology.

Table of Contents

What is ISTEM-01?

ISTEM-01 is an innovative medical treatment currently being studied for patients with a specific type of eye disease called retinitis pigmentosa. It is classified as a cell therapy product, which means it uses living cells as a treatment[1]. Specifically, ISTEM-01 is made up of special cells called Retinal Pigment Epithelium (RPE) cells that are derived from human embryonic stem cells[1].

The treatment comes in the form of a medicated patch that is placed under the retina (the light-sensitive tissue at the back of the eye) through a surgical procedure[1]. This method of application is known as sub-retinal administration.

Target Condition: Retinitis Pigmentosa

Retinitis pigmentosa is a group of rare genetic disorders that involve a breakdown and loss of cells in the retina[1]. This condition can lead to vision loss and, in some cases, blindness. ISTEM-01 is specifically being studied for patients with retinitis pigmentosa caused by a monogenic mutation, which means the condition is caused by a change in a single gene[1].

How ISTEM-01 Works

ISTEM-01 aims to replace damaged or lost retinal pigment epithelium cells in patients with retinitis pigmentosa. These cells play a crucial role in supporting and nourishing the light-sensitive cells of the retina (photoreceptors)[1]. By implanting healthy RPE cells derived from stem cells, researchers hope to slow down or potentially reverse some of the vision loss associated with retinitis pigmentosa.

Clinical Trial Details

ISTEM-01 is currently being studied in a Phase 1/2 clinical trial. This means it’s in the early stages of testing in humans to evaluate its safety and get initial data on its effectiveness[1]. Here are some key details about the trial:

  • The trial is open-label, which means both the researchers and participants know which treatment is being used.
  • It aims to assess the safety and tolerability of ISTEM-01 implantation over a period of 56 weeks (about 1 year) after the treatment[1].
  • The study will also look at the placement and position of the patch in the eye and evaluate any preliminary signs of effectiveness[1].
  • Participants will be followed for up to 5 years after treatment to assess long-term safety and effectiveness[1].

Eligibility Criteria

Not everyone with retinitis pigmentosa can participate in this clinical trial. Some key eligibility criteria include:

  • Confirmed diagnosis of retinitis pigmentosa based on a genetic test showing a mutation in specific genes (RPE65, LRAT, or MerTK)[1].
  • Age between 18 and 65 years old[1].
  • Specific levels of visual acuity (a measure of how clearly a person can see), which differ depending on which group (cohort) of the study a patient might join[1].

There are also several factors that would exclude someone from participating, such as allergies to products used in the study, prior gene or cell therapy treatments, or certain other medical conditions[1].

Safety and Efficacy Evaluation

The researchers will be closely monitoring participants for any side effects or adverse events related to the treatment. They will also be looking at several measures to evaluate if the treatment is working, including:

  • Changes in visual acuity (how clearly a person can see)[1].
  • Changes in visual field (how wide an area a person can see)[1].
  • The function of the retina and RPE cells using special tests like electroretinography (ERG)[1].
  • The survival of photoreceptors (light-sensitive cells in the retina) over the implanted RPE cells[1].

Potential Benefits

While it’s important to remember that ISTEM-01 is still in the early stages of research, the hope is that this treatment could potentially slow down or even partially reverse vision loss in people with retinitis pigmentosa. However, more research is needed to determine its effectiveness and safety.

If you have retinitis pigmentosa and are interested in this or other clinical trials, it’s important to discuss your options with your eye doctor or a specialist in retinal diseases.

Aspect Details
Study Type Phase 1/2, open-label safety, tolerability, and preliminary efficacy study
Intervention ISTEM-01 (hESC-derived RPE cells) implantation into one eye
Target Condition Retinitis pigmentosa due to monogenic mutation
Primary Objective Assess safety and tolerability over 56 weeks post-implantation
Secondary Objectives Evaluate patch placement, assess preliminary efficacy, long-term safety and efficacy up to 5 years
Key Eligibility Criteria Age 18-65, confirmed genetic diagnosis, specific visual acuity requirements
Primary Endpoint Incidence of adverse events or serious adverse events
Secondary Endpoints Changes in visual function, retinal imaging, ERG, microperimetry
Follow-up Duration 56 weeks for primary endpoint, up to 5 years for long-term follow-up

FAQ

  • What is ISTEM-01 and how is it being used in this clinical trial? ISTEM-01 is a medicated patch containing human embryonic stem cell-derived retinal pigment epithelium (RPE) cells. In this trial, it is being implanted into one eye of patients with retinitis pigmentosa to assess its safety, tolerability, and potential effectiveness in treating the condition.
  • Who is eligible to participate in this clinical trial? Eligible participants are individuals aged 18-65 with a documented diagnosis of retinitis pigmentosa based on a genetic test confirming a monogenic mutation affecting RPE65, LRAT, or MerTK genes. Visual acuity requirements vary depending on the study cohort, and participants must not have certain pre-existing conditions or prior treatments that could interfere with the study.
  • What are the main objectives of this clinical trial? The primary objective is to assess the safety and tolerability of ISTEM-01 implantation over 56 weeks. Secondary objectives include evaluating the placement and position of the patch, assessing preliminary efficacy in visual function and photoreceptor survival, and monitoring long-term safety and efficacy for up to 5 years after implantation.
  • How will the effectiveness of ISTEM-01 be measured? The effectiveness will be measured through various tests, including changes in visual acuity, kinetic perimetry, retinal and RPE functionality through electroretinography (ERG), and functional survival of retinal photoreceptors using microperimetry. Additionally, imaging techniques like OCT scans and fundus fluorescein angiography will be used to assess the placement and condition of the implanted patch.
  • What are the potential risks or side effects of participating in this trial? While specific risks are not detailed in the provided information, the trial will closely monitor for adverse events or serious adverse events. This includes regular ophthalmologic exams, laboratory tests, vital sign checks, and physical examinations. Patients with allergies to study products or certain pre-existing conditions are excluded to minimize potential risks.

Ongoing Clinical Trials on Istem-01

  • Study on the Safety and Effects of ISTEM-01 and Mycophenolate Mofetil for Patients with Retinitis Pigmentosa Due to a Genetic Mutation

    Recruiting

    2 1 1 1
    Investigated diseases:
    Investigated drugs:
    France

Glossary

  • Retinitis Pigmentosa: A group of rare genetic disorders that involve a breakdown and loss of cells in the retina, which can lead to vision loss.
  • Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (hESC-derived RPE): Retinal pigment epithelium cells that are created from human embryonic stem cells in a laboratory. These cells are being tested as a potential treatment for retinal diseases.
  • Monogenic Mutation: A change in a single gene that causes a genetic disorder. In this trial, it refers to mutations in genes like RPE65, LRAT, or MerTK that are associated with retinitis pigmentosa.
  • Visual Acuity: A measure of the sharpness or clarity of vision, often tested using an eye chart.
  • Spectral Domain Optical Coherence Tomography (OCT): An imaging technique that uses light waves to take cross-section pictures of the retina, allowing doctors to see each of the retina's distinctive layers.
  • Fundus Fluorescein Angiography: A test that uses a special dye and camera to look at blood flow in the retina and choroid, the two layers in the back of the eye.
  • Electroretinography (ERG): A test that measures the electrical responses of various cell types in the retina, including the light-sensitive cells (rods and cones).
  • Microperimetry: A technique that allows for precise testing of visual function at specific points on the retina while simultaneously imaging the fundus.
  • Intraocular Pressure (IOP): The fluid pressure inside the eye, which is important in the diagnosis and management of glaucoma and other eye conditions.
  • Kinetic Perimetry: A method of visual field testing where a moving target is used to map the boundaries of a person's visual field.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-istem-01-and-mycophenolate-mofetil-for-patients-with-retinitis-pigmentosa-due-to-a-genetic-mutation/