CIRM-Funded UC-Irvine Team Set to Launch Stem Cell Trial for Retinitis Pigmentosa in 2015

Rosalinda Barrero has often been mistaken for a rude snob. She has the habit of not saying hello or even acknowledging the presence of acquaintances that she passes around town. But in fact this kind, loving mom of three has been steadily losing her vision over a lifetime. And she doesn’t seem blind because people are still vaguely visible as shadowy ghosts but their faces are unrecognizable.

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Rosalinda Barrero is legally blind due to retinitis pigmentosa. She eagerly awaits the launch of a CIRM-funded trial that will test a candidate stem cell-based treatment.

Barrero is stricken with retinitis pigmentosa (RP) an incurable genetic disease that gradually destroys the light sensing nerve cells, called photoreceptors, located in the retina at the back of the eye. In October, Rosalinda and her husband German spoke to the CIRM governing Board about the devastating impact of RP on their lives and their excitement about a soon to begin CIRM-funded stem cell-based clinical trial for the treatment of RP. The project is headed by UC-Irvine associate professor Henry Klassen, MD, PhD, who also spoke to the Board. Videos of their presentations are now available on our website and below:

Over 3000 known genetic mutations can give rise to RP. These mutations lead to the gradual deterioration of the so-called rod photoreceptors. These rod cells specifically provide our night vision — like on a moonless night. Rosalinda clearly remembers her childhood struggles with night blindness on Halloween:

“I didn’t like trick-or-treating because I couldn’t see in the dark. I ‘d say ‘this is not fun! I’m tripping! I’m losing all my candy!’ I wanted to stay home and hand out candy”

Unfortunately the disease doesn’t stop there. As the rods continue to die off another type of photoreceptor, the cone cells, become innocent bystanders and also gradually deteriorate later in life. As Dr. Klassen explained, it’s the cone cells that are critical for our sight:

“The cones are what humans use for almost all of their vision. Even at night when you’re driving a car with headlights you’re using mainly your cones. So if we could preserve the cones we can really help the patient.”

With the support of a $17 million CIRM Disease Team grant, Klassen and his team anticipates starting a stem-call based clinical trial in early 2015 with the ultimate aim of healing those cone cells in RP patients. The therapy uses a type of immature stem cell of the retina called retinal progenitor cells. The proposed approach relies on the injection of the cells into the jelly of the eye near the retina to promote indirect healing. Klassen explained the project rationale to the Board:

“So we’re talking about little clusters of cells that could fit on the head of a pin in the jelly of the eye and they’re just floating there. And what are they going to do? Well they just sit there and secrete all the factors they normally secrete during retinal development and diffuse into the retina. Once in the retina we believe [based on animal studies] those factors are going to reprogram the photoreceptors into becoming functional again instead of going down that road where they’re going to commit suicide.”

Rosalinda is beyond thrilled with the prospect of being a recipient of this candidate therapy. Her husband German echoed her hopefulness to the Board:

“Even though it’s not a deadly disease, [the therapy] would be life-changing not only for Rosie it would be for everyone around her. “

To learn more about CIRM-funded research related to blindness, visit our fact sheet.

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8 thoughts on “CIRM-Funded UC-Irvine Team Set to Launch Stem Cell Trial for Retinitis Pigmentosa in 2015

  1. I have RP also and this trial gives me comfort knowing that we are taking the right step towards finding a treatment or cure! God bless to all!

  2. I am working with a humble Mexican family here in the Yucatan who were just informed that the father (age 28) has RP but he has not had the genetic testing to isolate the type. His son is having the same night issues so he will be checked soon. I would love to be kept abreast of new research as I plan to be alongside this family during this difficult journey.

    • Dear Kathleen, Thanks for reaching out to us on behalf of this family. The RP clinical trial mentioned in this blog post is currently recruiting participants. jCyte is the company running the trial and you can get more information on their website: http://jcyte.com/clinical-trials/ Best wishes, Todd

  3. The treatment applied in Cuba for Retinitis Pigmentosa (RP), has the objective of controlling the development of this disease as another alternative among the efforts carried out throughout the, whole world to improve the quality of life of those people who suffer from it. The investigations carried out in our institution and in other international institutions have shown the necessity to develop a strategy to treat retinal degeneration diseases with a new conception.
    Our treatment consists of the following procedures the performance of unique surgical technique created by Dr. Orfilio Peláez Molina after years of research and experimental surgery on animal models is the most important part of the treatment. This unique surgical procedure called Revitalizing Surgery (R VS) is an ocular microsurgery in which an implant of the own orbital fatty tissue from the back of the patient’s eye is placed ondiveloping suprachoroidal bed, developing an angiogenesis mechanism that increases the choroidal and retinal blood circulation, the blood flow, the presence of different growth factor to favoring the control of the neural and vascular remodeling processes that occur in retinas with Retinitis Pigmentosa and other retinal degenerations. The increase of the cell neuroactivity due to the stimulation of different neurotrophic elements is another positive result after surgery.

    http://www.retinosispigmentaria.cu

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