The regenerative eye scientist, Alan Marmorstein, Ph.D., is drawn to research by the need to find new cures for diseases. As an aspiring researcher, he never envisioned his investigative journey would take him down the path of testing ways to prevent or restore loss of eyesight. However, a post-doctoral lab assignment, a National Eye Institutes grant and inspiration from retina surgeons presented him the opportunity to research disorders of the eye. It launched him on a transformative career trajectory.

“Initially, I was disappointed, because I was interested in pursuing a different line,
 says Dr. Marmorstein. “After I started working in the lab, I fell in love with the eye as a system. The eye is a unique organ in the body that you can literally look into. You can monitor what you are doing and see the effect of your research much easier than in a lot of other places in the body. It’s very easy to figure out if something can see.”

At Mayo Clinic, Dr. Marmorstein’s research to prevent blindness includes delivery of stem cell replacement therapy to slow or stop progression of diseases that rob people of their central field of vision. Mayo Clinic Center for Regenerative Medicine supports Dr. Mamorstein’s research as part of its mission to empower discovery, advance the practice and develop new cures that address unmet patient needs.

Research focus

Dr. Marmorstein’s research focuses on age-related macular degeneration and an inherited form called Best disease, which typically afflicts people under the age of 65. Macular degeneration is a loss of central vision and a leading cause of blindness in older adults. It affects more than 10 million Americans, according to the American Macular Degeneration Foundation. While there are eye injections to ease symptoms of some forms of macular degeneration, researchers are trying to solve the puzzle of how to stop the progression and ultimately cure the disease.

“The goal of our research is to keep as many people as we can from losing their central vision. Very likely any future treatments will involve a surgical intervention. We are seeking a therapy that’s off the shelf, meaning that patients can be treated readily. Our hope is that once someone is diagnosed we could immediately offer a date for surgery,” says Dr. Marmorstein.

In dry macular degeneration, the center of the retina deteriorates. With wet macular degeneration, leaky blood vessels under the retina cause blurred vision. Little is known about the cause of age-related macular degeneration. Best disease is linked to a genetic mutation affecting retinal pigment epithelial (RPE) cells, which are the layer of cells that support the health and function of light sensing cells in the retina. RPE cells are essential to maintaining vision.

Stem cell therapy

Working with the Center for Regenerative Medicine Biotrust, Dr. Marmorstein’s team is engineering replacement RPE cells derived from induced pluripotent stem cells (IPSC). The process is like turning the clock back in time to when the stem cells were first forming in the mother’s womb. At that time, cells were dividing and differentiating and could become any type of cell or tissue within the body. The human IPSCs are manufactured from tissues in the Biotrust and are reprogrammed to become retinal pigment epithelial cells that could replace the faulty cells linked to vision loss.

“The Biotrust provides us with cells from human skin samples. The expert staff at the Biotrust trained my team how to grow induced pluripotent stem cells from human samples. In addition, they provided us support in terms of finance, personnel and knowledge to do this research,” says Dr. Marmorstien.

In a further demonstration of ingenuity, Dr. Marmorstein’s team has developed a biodegradable scaffold for the delicate process of delivering the cells to the precise location within the retina where they are needed to potentially preserve or restore vision. While a plastic scaffold that would permanently remain in the eye has been used elsewhere, Mayo’s scaffold places the cell within the eye and disintegrates within two months.

“This is a unique instrument for delivering the RPE cells. The scaffold is manufactured from human fibrin, which is a protein that is naturally involved in blood clotting and wound healing,” says Dr. Marmorstein.

Dr. Marmorstein’s research team includes Jarel Gandhi, Ph.D., who designed the scaffold and Raymond Iezzi, M.D., an ophthalmologist who helped develop the surgical procedure.

A graduate of State University of New York at Albany, Dr. Marmorstein earned his Ph.D. at the State University of New York Health Science Center at Brooklyn and completed a post-doctoral fellowship at Cornell University. He began his research career at the drug company, Merck. After holding research positions at Cleveland Clinic and the University of Arizona, Dr. Marmorstein joined Mayo Clinic in 2013.

“I came to Mayo because of its unique blend of science, research, education and practice. The sharing of expertise is unmatched. I’ve had members of the retina surgical staff attending my lab meetings, and their input has been invaluable. My team draws on all available resources, such as the Biotrust, bioinformatics, biostatistics, engineering and assistance with regulatory issues to advance research,” says Dr. Marmorstein.

The key to advancing research into human clinical trials is to gain Food and Drug Administration approval for Investigational New Drug use of the RPE cells and biodegradeable scaffold in humans. Dr. Marmorstein hopes to clear that hurdle and test his research in phase 1 safety trials within 18 to 24 months.

####

The post Delivering innovative stem cell therapy to slow vision loss appeared first on Mayo Clinic News Network.

The biggest break in that quest to date, the Rochester Post-Bulletin reports, came when Dr. Marmorstein and his lab team at Mayo Clinic developed a new process for growing retinal pigment epithelial (RPE) cells, which are often used in eye-related research. According to the newspaper, stem cells reprogrammed from a patient's own skin cells can become these new and improved cells. Researchers believe that transplanting the new RPE cells into the retina could restore vision to those affected by macular degeneration.

To Dr. Marmorstein's (and other researchers') delight, these new cells, which are created from induced pluripotent stem cells, are proving to be "of better quality than the standard RPE cells" used in research. And demand is growing, according to Twin Cities Business magazine. There's just one catch. (Isn't there always?) The process for making the new cells "is not trivial" and is "more expensive," according to the P-B/ Read the rest of the article.
______________________________________________
This story originally appeared on the In the Loop blog.

The post In the Loop: The fight against inherited eye disease appeared first on Mayo Clinic News Network.

In the past, in even the best of doctor-patient relationships, it was usually the doctor who made the final decision about treatment. Now that's changing. On this week's program, we explore "participatory medicine" with patient-engagement advocate Dave deBronkhart, Jr., better known to his blog followers as e-Patient Dave. Also on the program, Mayo Clinic ophthalmologist Dr. Raymond Iezzi, Jr., talks about the bionic eye, an implant that brings sight to people with serious vision impairment.

Myth or Matter-of-Fact: Patient engagement means more time spent in your doctor's office, leading to higher health care costs.

Mayo Clinic Radio is available on iHeart Radio.

Click here to listen to the program on Saturday, April 11, at 9:05 a.m., and follow #MayoClinicRadio.

To find and listen to archived shows, click here.

Mayo Clinic Radio is a weekly one-hour radio program highlighting health and medical information from Mayo Clinic. The show is taped for rebroadcast by some affiliates.

The post MAYO CLINIC RADIO appeared first on Mayo Clinic News Network.

Miss the program?  Here's the podast. MayoClinicRadio PODCAST April 11, 2015

Myth or Matter-of-Fact: Patient engagement means more time spent in your doctor's office, leading to higher health care costs.

To listen to the program at 9:05 a.m. Saturday, April 11, click here.

Follow #MayoClinicRadio and tweet your questions.

Mayo Clinic Radio is available on iHeartRadio.

Mayo Clinic Radio is a weekly one-hour radio program highlighting health and medical information from Mayo Clinic.

To find and listen to archived shows, click here.

The post Mayo Clinic Radio: e-Patient Dave/Bionic Eye appeared first on Mayo Clinic News Network.

The Alberta government had no intention of ending Audrey's career. But in 2009, it looked as if failing eyesight might. The crusading lawyer, who had always had perfect vision, began having trouble reading and driving.

She consulted with a local ophthalmologist, who diagnosed cataracts and recommended surgery to remove them. But unconvinced of the diagnosis, Audrey sought a second opinion at Mayo Clinic in Arizona.

This wasn't her first experience with Mayo Clinic. Years earlier, her husband had consulted physicians there after learning he had cancer. And she had been seen doctors at Mayo about her own hearing loss — the result of radiation therapy for non-Hodgkin's lymphoma. When she began having eye problems, she felt Mayo Clinic was the obvious choice. 

"I have a vacation home in Phoenix, where I usually spend Christmas, and I asked if I could possibly be seen at Mayo over the holidays," she says. "I got an appointment immediately — before even arriving in Arizona."

Audrey underwent an extensive exam that yielded both good news and bad news. Her ophthalmologist, Joanne Shen, M.D., confirmed that Audrey's intuition had been correct — she didn't have cataracts. But she did have a problem that was potentially more serious: age-related macular degeneration, a chronic condition that blurs the high-resolution central vision needed for reading, driving and recognizing faces.

Until recently, little could be done to halt the progress of the disease, which is the main cause of central vision loss in older adults. But now, antibody medications injected into the eye can delay, prevent and in some cases reverse macular deterioration. To maintain the effects, injections must be repeated every four to eight weeks.

"I would happily have flown to Arizona every six weeks for the injections, but I was able to find a retina specialist in Alberta to administer them," Audrey says. "The results have been just amazing. As a result of the treatment, there has been no further deterioration. I have my life still. I can drive, and I'm able to read, which is essential for both my legal work and for pleasure — I really love reading. So I'm very thankful to Mayo Clinic for diagnosing me correctly. Having cataract surgery would not have improved my vision and quite likely would have made the macular degeneration worse."

The experience reinforced her belief in the value of a second opinion and the expertise available at Mayo Clinic.

"I would encourage anyone needing a second opinion to go to Mayo Clinic," she says. "Their care and expertise are second to none."

 HELPFUL LINKS

• Learn more about macular degeneration.
• Explore Mayo Clinic’s Ophthalmology services.
• Join the discussion about Eye Conditions on Mayo Clinic Connect.
• Contact the International Appointments Office.
• Request an appointment.

The post Second Opinion Leads to a Second Chance at Sight appeared first on Mayo Clinic News Network.