Age-related macular degeneration (AMD) is a progressive condition which results in loss of central vision over time. Early symptoms of AMD include dry AMD, which develops deposits called drusen under Bruch’s membrane and geographic atrophy; advanced stages, known as wet AMD, feature choroidal neovascularization as one characteristic feature.

Macular degeneration treatment can help patients maintain their vision. Eye injections with anti-VEGF medication are used to block leaky blood vessels while laser therapy may also save some vision by dismantling abnormal vessels.

1. CIRM Grant Supports USC Regenerative Medicine Project

As people age, their chances of dry macular degeneration increase significantly. This condition impairs vision and disrupts daily activities such as driving, reading or recognizing loved ones’ faces – but fortunately it is preventable and treatment options exist to slow its progress.

The team’s work could pave the way to a therapy that restores vision by replacing damaged retinal cells with healthy ones from transplanted ones. Scientists have discovered that implanted cells produce chemical messengers which communicate with nearby retinal cells via paracrine signaling, stimulating them to behave more like those transplanted. With support from CIRM grants, their research will expand further and they hope to produce a therapeutic injection to be administered directly into humans.

Neovascular age-related macular degeneration (nAMD) and diabetic macular edema are the two leading causes of adult blindness in America,1 with current treatment being focused on inhibiting formation of vascular endothelial growth factor and angiopoietin 2 with drugs known as anti-VEGF agents. Though these treatments have proven highly successful at preventing vision loss, more innovative strategies may enhance their efficacy further.

USC team receives CIRM grant to accelerate development of retinal implant and prepare it for clinical trials; their research was also supported by National Institutes of Health grant.

USC stem cell researchers are making breakthroughs in other areas of medicine as well. Gage Crump, PhD, an associate professor of cell biology and regenerative medicine; and Rob Maxson, PhD, professor of biochemistry and molecular medicine are using zebrafish and mice to understand why skull bones prematurely fuse during craniosynostosis, with assistance from a grant from the National Institutes of Dental and Craniofacial Research of $3 Million.

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2. USC Regenerative Medicine Team Develops Retinal Implant

USC team’s new retinal implant converts visual information from the world into electrical impulses that stimulate ganglion cells in blind retinas, sending signals directly to patients’ brains so they can perceive shapes and edges despite being completely blind. Argus II has already been utilized by hundreds of individuals globally but at present only provides black-and-white vision; researchers are working toward expanding its capabilities.

The implant’s cellular component is comprised of retinal stem cells, which have already proven effective against other eye conditions like diabetic macular edema and age-related cataracts. Transplanted into the eye via microinjections, electrodes then convert electrical impulses into appropriate stimulation to restore vision.

This project could be an important step toward developing treatments for wet macular degeneration (WMD), an eye condition which affects more people than dry AMD and can result in permanent blindness. With help from CIRM grants, this project could quickly move towards clinical trial stage where its safety and efficacy will be tested against human subjects.

Other treatments to address wet macular degeneration include injectable medications like ranibizumab, bevacizumab and aflibercept. These injectable medications work by blocking abnormal blood vessels that leak fluid into the macula, leading to macular edema (fluid build-up in central retina).

An extensive eye examination can detect macular degeneration and its severity. Doctors can perform various tests, such as using graph paper grids to gauge vision loss caused by disease progression. Also available is optical coherence tomography technology which creates digital images of cross-sections of retina using scanning laser technology.

Gene therapy, stem cell injections and dietary supplements such as vitamin C and omega-3 fatty acids have all shown promise as experimental treatments to slow or restore vision loss due to macular degeneration. Animal studies have also demonstrated their efficacy.

3. USC Regenerative Medicine Team Develops Injectable Solution

7 million Americans currently experiencing early-stage macular degeneration could benefit from an injectable solution for macular degeneration, known as macular degeneration. This disease affects the macula of the retina – responsible for sharp vision at the center of visual field – when retinal pigment epithelium (RPE) degenerates due to degenerative changes, leading to loss of photoreceptor cells which results in blurred or distorted vision. It is estimated that injecting soluble growth factors could prevent further RPE cells from degenerating.

Although research into macular degeneration has made advances, current treatment options are limited. Eye injections containing medications that inhibit vascular endothelial growth factor (VEGF) remain the gold standard for treating neovascular age-related macular degeneration (nAMD) and diabetic macular edema (DME); however, patients often require multiple injections over time due to suboptimal results or gradual vision loss.

Wet macular degeneration occurs when a choroidal neovascular membrane forms under areas weakened by drusen and atrophy, where new blood vessels leak fluid and bleed into the macula, causing distortion and permanent damage. Lucentis and Avastin, medications which block VEGF, may help slow or stop its progress by stopping new blood vessel formation and blocking their formation.

USC researchers supported by a CIRM Disease Team Therapy Development III Award have created an implant that features one layer of healthy RPE cells to replace damaged ones in the macula. After surgically placing these implants in 15 patients, some saw significant improvements to their vision attributed to paracrine signaling – whereby implanted cells secrete substances which communicate with nearby native retinal cells encouraging them to behave similarly to healthy implanted cells.

Scientists involved with this project hope their efforts will provide a less invasive way of treating macular degeneration and other chronic, previously incurable diseases. Next year they plan to conduct a clinical trial on an implant that releases sustained release VEGF inhibitors that should improve treatment’s efficacy and safety.

4. USC Regenerative Medicine Team Develops a Personalized Treatment

Macular degeneration affects the central part of the retina (macular pigment epithelium or macula) and leads to vision loss in many older individuals as they age. It may either be dry or wet; wet being distinguished by abnormal blood vessels which leak under the retina causing hemorrhages that result in blurred vision, distorted images, or an inexplicable central blind spot symptomatically exhibited through blurry vision, distortion images or central blind spots; symptoms usually include gradual vision loss that progress over time as untreated; further complications such as driving or reading may prevent individuals from participating in activities which could otherwise benefit from treatment such as driving or reading become necessary activities of daily living.

Although macular degeneration cannot be reversed, USC researchers have recently developed an innovative treatment known as Dreamland that could offer relief to dry AMD sufferers. This therapy uses nanoparticle delivery systems to administer RNA therapeutics directly into the eye and target retinal pigment epithelium cells directly – potentially improving vision in those suffering dry AMD by decreasing abnormal blood vessels and improving retinal circulation. Based on years of research at ISCReM (Institute for Stem Cell and Regenerative Medicine), its discovery was first featured in Science Advances.

The team is currently conducting a clinical trial to evaluate their approach in patients suffering from geographic atrophy due to dry AMD. The outcomes will help them ascertain if scaffold-based stem cell-derived bioengineered RPE implants are safe and effective solutions for this indication.

Current market estimates project that the global macular degeneration market will reach $21.9 billion by 2025 due to increasing awareness, recent approval of several biosimilar drugs, and an aging population in developed nations.

USC researchers are developing innovative technologies that may alter the course of disease. Tina Guanting Qiu is an ophthalmology physician, trained retinal surgeon and stem cell biologist with over two decades of international experience. She has worked for multiple companies developing drug discovery, delivery and cell/gene therapies for neuro-retinal disorders like glaucoma and macular degeneration; her work has been featured on multiple TV and newspaper programs including The Doctors; she serves as medical and scientific advisors to small companies including BetaStem Inc. and Inotek; also serving on numerous scientific and medical advisory boards and scientific advisory boards