Sally Temple, co-founder and chief scientific officer of Luxa Biotechnology LLC, said in her opening keynote that stem cell research is driving "a remarkable change" in how scientists approach neurodegenerative diseases, which affect millions worldwide and have few treatments that alter their course.
Temple said cells from patients can be reprogrammed into stem cells and grown into brain organoids, which are lab-grown miniature brain models. Her lab used them to trace a genetic form of frontotemporal dementia to a new drug target, and candidate therapies are now being tested in clinical trials.
Transplanted cells are being tested to restore movement in Parkinson's disease, seizure control in epilepsy and vision in age-related macular degeneration, she said. More than 1,200 patients had received pluripotent stem cell products in 116 clinical trials as of 2025, with no generalizable safety concerns.
Dopamine-producing cells transplanted by BlueRock Therapeutics survived 18 months in Parkinson's patients and improved their motor scores, and a phase 3 trial is now under way. In an early-stage trial, an interneuron therapy from Neurona Therapeutics cut disabling seizures by a median 89 percent in nine epilepsy patients, she said.
Luxa's own therapy injects adult retinal pigment epithelium stem cells beneath the retina. One legally blind patient in its trial improved to 20/40 vision, which is good enough to drive, and the gain held for more than a year with no safety issues, Temple said.
"It really takes the team," Temple said. She added that bringing a therapy from discovery to the clinic required development work, clinical manufacturing, clinical sites and regulatory oversight, as well as patients with "the generosity and courage" to take part.
AJP Takeaways
- Sally Temple, co-founder and chief scientific officer of Luxa Biotechnology LLC, delivered the opening keynote at BIX 2026 in Seoul on Oct. 28, 2026, on stem cell treatments for neurological diseases.
- More than 1,200 patients had received pluripotent stem cell products in 116 clinical trials as of 2025, with Parkinson's disease, epilepsy and age-related macular degeneration among the targets for cell replacement.
- Luxa's adult retinal pigment epithelium stem cell therapy for dry age-related macular degeneration showed no safety issues in its first-in-human trial, with results published in Cell Stem Cell in September 2025.
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