Cell Therapy: A Potential New Treatment to Restore Lost Vision

Scientific and clinical research has brought about significant advances in medicine, including ongoing clinical studies investigating the potential of cell therapy to replace cells that have been lost in inherited retinal diseases (IRDs) with the goal of restoring vision. IRDs are a group of diseases characterized by the loss of function of retinal cells, which over time leads to vision loss and, in many cases, blindness.

Inherited Retinal Diseases and Cell Therapy

are a broad group of rare genetic eye conditions that affect cells in the retina called photoreceptor cells. These cells are essential for converting light into visual signals that are transmitted to the brain, allowing vision. Loss of these cells can cause progressive vision loss in individuals, with many IRDs potentially leading to blindness. IRDs include retinitis pigmentosa (RP), Usher syndrome, Stargardt disease, and cone-rod dystrophy (CRD).

To date, there have been limited treatment options for patients affected by IRDs. Cell therapy is a new approach under investigation that aims to replace photoreceptor cells lost to disease with healthy cells, with the goal to restore function and vision to these individuals.

Cell therapy is a growing field in regenerative medicine — and IRDs are just one of the areas where it’s being explored.

Explore how it works: Cell Therapy 101

Cell Therapy Clinical Trials are Underway for Inherited Retinal Diseases

Today, researchers are actively testing cell therapies in clinical trials for people living with IRDs. These trials are studying the ability of cell therapy to safely and effectively replace retinal cells lost to disease with healthy ones — potentially restoring vision.

Clinical trials evaluate the safety and efficacy of investigational therapies and are required for a therapy to receive regulatory approval, such as from the FDA in the U.S. or other regulatory agencies outside of the U.S. Eligibility to participate in a clinical trial depends on multiple factors, including your specific diagnosis, disease progression, and age.

While cell therapies for IRDs are not yet approved, they are an important step forward to potentially transform the treatment of these diseases.

FAQ: Cell Therapy for Inherited Retinal Diseases (IRDs)

Have questions about cell therapy for inherited retinal diseases? You’re not alone. Here are answers to some of the most common questions people ask when exploring this new area of research.

IRDs are a group of rare genetic conditions that affect the retina — the layer of tissue at the back of your eye that senses light and sends corresponding signals to your brain. IRDs include retinitis pigmentosa (RP), Usher syndrome (a form of RP with associated deafness), Stargardt disease, and cone-rod dystrophy (CRD). These diseases often begin in childhood or early adulthood.

IRDs are generally caused by genetic mutations that affect the health of photoreceptor cells in the retina, which are crucial for vision. Over time, as the photoreceptor cells degenerate due to progression of disease, the loss of these cells leads to visual impairment and potentially blindness.

Retinitis pigmentosa is one of the most common types of inherited retinal diseases. It causes progressive vision loss due to the breakdown of photoreceptor cells — primarily rod cells, which are responsible for night and peripheral vision. As the disease progresses, however, cone cells, which are involved with day vision, color perception, and visual clarity, can also be affected, leading to central vision loss.

The goal of cell therapy for IRDs is to replace light-sensing cells that have been lost to disease with healthy cells. The aim is for the new cells to integrate into the retinal structure and restore the function of the lost photoreceptors, thus restoring vision.

Traditional gene therapy approaches focus on correcting a faulty gene in the DNA to treat disease. In the retina, once cells are lost to disease, they cannot grow back, so gene therapy can only be used if there are still remaining cells in the retina. Cell therapy takes a different approach by administering healthy cells to replace cells that have been lost to disease. In the case of IRDs, this means introducing healthy photoreceptor cells into the retina to replace the lost retinal cells and restore function.

Cell therapies that are currently being investigated for IRDs are gene agnostic, meaning patients with many different mutations in different genes may benefit from the therapy. Cell therapy for IRDs is designed to replace photoreceptor cells that are lost due to the disease, which could mean restoring the retina’s ability to sense light.

In clinical trials, cell therapies aiming to restore cells lost to diseases are administered via a one-time subretinal injection to the back of the eye via a specialized procedure. This allows the healthy cells to be placed where they’re needed. The procedure is performed by a highly trained retinal surgeon, and patients are closely monitored afterward.

No, not at this time. Cell therapies for inherited retinal diseases are still in the investigational stage. That means they are being studied for safety and efficacy in clinical trials and haven’t been approved for use in patients.

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