Keio University

The most effective drug for Parkinsons disease hasnt changed in 50 years. But fresh research published in one of the countrys top scientific journals is helping build the case for a more cutting-edge approach that uses stem cells to restore important brain functions.The second most common neurodegenerative illness, Parkinsons, is caused by the loss of certain nerve cells. These cells produce a chemical messenger, dopamine, that plays a crucial role regulating movement. Since the 1970s, a drug called levodopa, which the body converts into dopamine, has been the mainstay treatment for combating the tremors, slowness, stiffness and balance issues that come with Parkinsons.Levodopa doesnt stop the disease, however, so scientists have spent decades trying to find a more permanent fix. The latest development in that search comes from two clinical trials testing stem cell-derived therapies transplanted into the brains of Parkinsons patients.The first of those trials, conducted at the Kyoto University Hospital in Japan, evaluated seven people who received what are essentially blank slate cells that can turn into dopamine-producing neurons. These cells were created using a technology pioneered at Kyoto University, where roughly 20 years ago, scientists discovered how to reset some adult human cells to act like stem cells.The studys main focus was safety. Results, published Wednesday in Nature, show no serious adverse events were reported, though researchers did identify 73 mild to moderate events. Importantly, there was no evidence the introduced cells started growing in an unchecked, tumor-like fashion a major concern with stem cell implants.The study also explored whether this therapy had any effect on patients disease. Two years post-treatment, the six participants who were evaluable had gotten better on a scale clinicians use to measure the severity of Parkinsons motor symptoms. Researchers looked at whats known as on time, when medications are adequately controlling these symptoms, as well as the opposite off time, and found scores improved an average of 36% and 20%, respectively.Another test indicated that dopamine-producing cell activity had increased substantially. This was determined through a technique in which researchers strap a radioactive element to a molecule of levodopa and then use a PET scan to follow its path through the nervous system, somewhat like how a submarine tracks objects on a sonar display.The second trial took place at sites throughout the U.S. and Canada, and employed a different kind of stem cell that comes from human embryos. It enrolled a total of 12 participants and followed them for a year post-transplant.Similar to the Japan study, there were no deaths, serious adverse events or tumor-like tissue growth related to the introduced cells. Trial runners did report two serious events overall. One participant was hospitalized with COVID-19, while another had a seizure that was attributed to the surgical procedure.The trial also saw improvements in off scores on that disease severity scale and positive results from the radioactive levodopa test.The cell therapy tested, called bemdaneprocel, is being developed by Bayer subsidiary BlueRock Therapeutics, which back in August 2023 announced some of the data published Tuesday. The company expects to start a Phase 3 trial before the end of June.Hideyuki Okano, a stem-cell scientist at Keio University in Tokyo, argues more research is needed to confirm these types of cell therapies are effective against Parkinsons.Still, in an accompanying Nature editorial, he called the new trial results encouraging because they suggest treating Parkinsons patients with donor cell transplants is likely safe. That both trials proved to be safe, and hinted at possible efficacy, is an important step towards the establishment of this cell therapy for Parkinsons disease in wider society, he wrote.Cell therapy for Parkinsons isnt a new idea. In 1989, a team led by neuroscientist Olle Lindvall performed the first cell transplantations for Parkinsons patients. While the landmark study didnt show a major therapeutic benefit, a few interesting signals encouraged the research community to keep exploring.One ethical concern from that trial was that it used a relatively large amount of tissue derived from aborted fetuses. In the decades since, Parkinsons and cell biology experts have made significant progress identifying other, less controversial sources of stem cells that can be scaled more easily.A milestone of that work came in 2020, when the technique invented at Kyoto University was used to turn a Parkinsons patients own skin cells into the early stages of dopamine-producing neurons, which were then implanted into his brain.According to Okano, as of December, there were 115 clinical trials testing 83 products derived from pluripotent stem cells. And among those targeting central nervous system disorders, the ones for Parkinsons disease are at a more advanced stage of development.Further behind Bayer and BlueRock, Aspen Neuroscience, a San Diego-based biotechnology company, has a Parkinsons therapy derived from patients own cells currently in early-stage human testing. '

TOKYO, Japan I February 13, 2025 I Restore Vision Inc., a clinical-stage biotechnology company advancing gene therapies for retinal disorders, today announced that the first patient in its Phase I/II clinical trial with RV-001 has been dosed at Keio University Hospital. RV-001 utilizes adeno-associated virus (AAV) vectors to deliver a unique functional gene encoding “Chimeric Rhodopsin”, a light-driven G-protein activation mechanism to re-establish light activation in retinal interneurons. The goal is to expand light sensitivity to retinal interneurons via an intravitreal injection of RV-001. This milestone marks the initiation of the world’s first clinical trial of optogenetic gene therapy, which leverages a proprietary “Chimeric Rhodopsin” for patients with retinitis pigmentosa (RP). “Over 2 million people worldwide suffer photoreceptor loss in retinitis pigmentosa, resulting in severe vision loss and eventual blindness” said, CEO and Ophthalmologist Yusaku Katada, MD, PhD. “Our “chimeric rhodopsin” approach has broad potential to alter light sensitivity across multiple subtypes of RP patients. [1] We are continuing to advance the Phase I/II trial to assess safety, light-activation and light-sensitivity in these patients, post-administration of our lead gene therapy product RV-001.” Phase I/II Safety, Tolerability, and Exploratory Efficacy Trial Highlights Connecting Innovation, Academia, and Investment Restore Vision, Inc., a spin-out startup from Keio University, bridges cutting-edge academic innovation with entrepreneurial application to drive progress in retinal therapies. Developed through collaborations with Keio University School of Medicine and Nagoya Institute of Technology, RV-001’s optogenetic technology addresses the limitations of traditional approaches by combining high light sensitivity with a unique G-protein activation mechanism. [2],[3] This groundbreaking technology has the potential to be expanded to other retinal diseases, paving the way for broader applications in vision restoration. The Japan Agency for Medical Research and Development (AMED) and other academic grants, with key contributions from Keio University and Nagoya Institute of Technology, supported RV-001 development. About Restore Vision Restore Vision Inc., a clinical-stage gene therapy company, specializes in developing transformative gene therapies for inherited retinal disorders. By integrating academic excellence with entrepreneurial vision, the company is committed to pioneering treatments that address critical unmet medical needs and improve patient outcomes worldwide. [1] Roberts et al. Arch Ophthalmol. 2010 [2] Sasaki et al. PLoS One. 2014 [3] Katada et al. iScience. 2023 SOURCE: Restore Vision

The first optogenetic "Chimeric Rhodopsin" gene therapy tested in humans. RV-001 starting dose was generally well tolerated in this open-label trial. RV-001 a gene-agnostic approach for RP patients. TOKYO, Feb. 13, 2025 /PRNewswire/ -- Restore Vision Inc., a clinical-stage biotechnology company advancing gene therapies for retinal disorders, today announced that the first patient in its Phase I/II clinical trial with RV-001 has been dosed at Keio University Hospital. RV-001 utilizes adeno-associated virus (AAV) vectors to deliver a unique functional gene encoding "Chimeric Rhodopsin", a light-driven G-protein activation mechanism to re-establish light activation in retinal interneurons. The goal is to expand light sensitivity to retinal interneurons via an intravitreal injection of RV-001. This milestone marks the initiation of the world's first clinical trial of optogenetic gene therapy, which leverages a proprietary "Chimeric Rhodopsin" for patients with retinitis pigmentosa (RP). "Over 2 million people worldwide suffer photoreceptor loss in retinitis pigmentosa, resulting in severe vision loss and eventual blindness" said, CEO and Ophthalmologist Yusaku Katada, MD, PhD. "Our "chimeric rhodopsin" approach has broad potential to alter light sensitivity across multiple subtypes of RP patients.[1] We are continuing to advance the Phase I/II trial to assess safety, light-activation and light-sensitivity in these patients, post-administration of our lead gene therapy product RV-001." Phase I/II Safety, Tolerability, and Exploratory Efficacy Trial Highlights Patient Criteria: Participants with advanced RP, agnostic to genotype, and characterized by severe vision loss, no- to limited vision. Safety and Efficacy: In addition to safety, exploratory efficacy endpoints include improvements in light sensitivity and visual function. Connecting Innovation, Academia, and Investment Restore Vision, Inc., a spin-out startup from Keio University, bridges cutting-edge academic innovation with entrepreneurial application to drive progress in retinal therapies. Developed through collaborations with Keio University School of Medicine and Nagoya Institute of Technology, RV-001's optogenetic technology addresses the limitations of traditional approaches by combining high light sensitivity with a unique G-protein activation mechanism.[2],[3] This groundbreaking technology has the potential to be expanded to other retinal diseases, paving the way for broader applications in vision restoration. The Japan Agency for Medical Research and Development (AMED) and other academic grants, with key contributions from Keio University and Nagoya Institute of Technology, supported RV-001 development. About Restore Vision Restore Vision Inc., a clinical-stage gene therapy company, specializes in developing transformative gene therapies for inherited retinal disorders. By integrating academic excellence with entrepreneurial vision, the company is committed to pioneering treatments that address critical unmet medical needs and improve patient outcomes worldwide. Media Contact Hikaru Miyazaki COO, Restore Vision Inc. Email: [email protected] SOURCE Restore Vision Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED