Affecting about 11 million people in the U.S., advanced “dry” age-related macular degeneration is a leading cause of blindness among people older than 50 — with no known cure.
An upcoming Phase 2 of a clinical trial for human subjects seeks to change that — through stem cell therapy.
A team from the National Institutes of Health is planning the first U.S. clinical trial of induced pluripotent stem cells.
An Evolving Science
Known as iPSCs for short, these cells were discovered 12 years ago and won researchers a 2012 Nobel Prize. They are created by sending plain old adult cells biologically back in time — essentially creating embryonic stem cells without the ethical baggage.
“When iPSCs were discovered in 2007, there was a lot of hype that we could easily turn them into therapies,” Kapil Bharti of the NIH’s National Eye Institute told STAT. “But there were many unanswered questions. I hope this reignites the field.”
In addition to this US study, a handful of other iPSC-based clinical studies of various kinds are ongoing in Japan and China.
The NIH team “used retinal cells created from human iPSCs to treat a form of macular degeneration in rats and pigs, with results promising enough that they hope to start recruiting macular degeneration patients for a clinical trial in the next few weeks,” STAT reported.
The results proved promising enough the research team hopes to soon conduct human trials.
“In the NEI study, scientists led by Bharti started with blood-making cells, called CD34+ cells, isolated from the blood of three AMD patients, and then turned them into iPSCs. They added growth factors and other biochemicals that shape the cells’ destiny: Although pluripotent cells can become any type of cell in the body, the specific combination of biochemicals produces one and only one. Bharti and his team created retinal pigment epithelial cells, the kind that die early in macular degeneration. Each batch took about 11 weeks.”
A Cure in Sight?
Researchers hope that replacing the dead and dying RPE cells will halt the degeneration of photoreceptors — preventing the disease from getting worse. But saving RPE cells won’t save the photoreceptors to restore vision. But the research team is developing an AMD therapy that combines photoreceptors with an RPE patch that may actually cure AMD blindness.
Stem Cell Face-Off
The study essentially puts two different forms of stem cells in a face to face competition to test their effectiveness. A trial at the University of Southern California is starting with stem cells derived from human embryos.
“You know where we are putting our bets,” said Dr. Mark Humayun of USC’s Keck School of Medicine. “But in general, cell replacement therapies are a promising approach in the treatment of AMD. So both [kinds of stem cells] should be explored.”
A Brighter Day
It’s been a long, slow road, but the momentum of stem cell science is steadily building steam. And that’s not only good news for researchers but also the patients who stand to benefit. At Blatman Health and Wellness, we already use stem cell therapy injection to help people with a host of ailments — back pain, joint pain, knee difficulties, and shoulder therapy. Many of our Cincinnati patients have benefited from such approaches.
But it’s just the beginning. Over the next several years, we are sure to witness exciting developments that would be considered “miracles of science” just 10, 20, 30 years ago. Stem cell therapy offers hope to not just knee and shoulder pain, but could some day cure degenerative diseases such as muscular dystrophy.
It’s an exciting time to be a doctor and a researcher — and that means a brighter future for humanity. There may soon come a day when these diseases fade into the memory of history — like widespread outbreaks of polio, measles, or the Black Plague.