Latest news with #ShinyaYamanaka
The Mainichi
08-05-2025
- Health
- The Mainichi
Editorial: Japan must quickly commercialize iPS-based treatment by overcoming challenges
Research into treatment using induced pluripotent stem (iPS) cells has been generating positive results one after another. Amid high expectations among patients struggling with intractable illnesses and their families, it is hoped that progress converting the technology to practical use will be quick. A clinical trial conducted by a team of Kyoto University researchers targeting Parkinson's disease patients has found that the conditions of some of the subjects administered with nerve cells generated from iPS cells have improved. Another trial on Type 1 diabetes patients triggered the cells to secrete insulin, responsible for lowering blood sugar levels. In a world first, Keio University led a clinical study using iPS cells on patients with spinal cord injuries and saw some of the subjects' motor functions improve. In April, a startup launched at the University of Osaka applied for approval from the health ministry to manufacture and distribute heart muscle sheets prepared from iPS cells for treating heart disease, marking the first application of its kind for regenerative medicine products derived from iPS cells. These are epoch-making results for diseases that were previously difficult to treat. There arose no safety issues during the research phase, implying that these achievements have brought us a step closer to getting the technology into practical use. With their ability to develop into a variety of tissues, iPS cells have been under the spotlight for their potential to recover functions lost to illnesses. Kyoto University professor Shinya Yamanaka, who developed iPS cells, was awarded the Nobel Prize in physiology or medicine. There remain, however, challenges that must be overcome. Increasing the number of iPS-derived cells administered to patients to boost efficacy raises carcinogenic risks. Unlike medicinal compounds, quality may vary among living cells used in the treatment. Careful checks are indispensable. Further confirmation of the efficacy of the iPS-based treatment is also essential. So far, clinical trials and studies have turned up different effects among individual patients. Due to the high development cost, patients undergoing the treatment are expected to face hefty bills. As there are fewer patient samples compared to those given general new drugs, it won't be easy to collect data. Companies seeking to commercialize the regenerative medicine products are likely to use a system allowing them to hit the market for a set period on condition that the firms acquire additional data on their efficacy, among other requirements. The system is unique to Japan, enabling applications for marketing drugs once their efficacy can be estimated. Even though approval for such products is considered a mere "provisional permit," it can lead to treatment in the very near term. It is hoped that Japan will continue to steadily resolve challenges and make its world-leading technology flourish as a medical revolution.
Japan Forward
25-04-2025
- Health
- Japan Forward
iPS Cell Research Can Give Japan Lead in Regenerative Medicine
このページを 日本語 で読む The day when regenerative medicine using induced pluripotent stem cells (iPS cells) will be used in real-world treatments is fast approaching. For Japan to become a world leader in the field of iPS cell technologies, the government should strengthen support for practical applications and industrial development in this promising field. A research team from Kyoto University recently conducted clinical trials in which neurons made from iPS cells were transplanted into the brains of patients with Parkinson's disease. Results showed that there were no major safety issues and the procedure was effective in improving symptoms in some patients. Parkinson's disease is a serious ailment in which the number of nerve cells that secrete substances vital for transmitting information in the brain decreases. This causes tremors in the limbs and impaired motor function. Treatment mainly involves medication to alleviate symptoms. But this strategy becomes less effective as the disease progresses. Until now, no definitive treatment approach has been identified. If iPS cells could be used to replenish lost nerve cells, it could become a completely revolutionary, groundbreaking treatment. The results of these clinical trials mark a big step towards achieving this goal. There are thought to be around 250,000 Parkinson's patients in Japan. Cooperating pharmaceutical companies are looking to file applications for approval with the Japanese government within this year. However, only seven patients participated in the Kyoto clinical trial. Additionally, only some experienced improvements in motor function, with results varying from person to person. While confirming the procedure's safety and effectiveness was highly significant, further testing and refinement are needed before it can be established as a viable treatment. Professor Shinya Yamanaka of Kyoto University succeeded in creating iPS cells about 20 years ago. He was awarded the Nobel Prize in Physiology or Medicine in 2012 for that achievement. Shinya Yamanaka speaks at a press conference on January 22, 2018. Kyoto University, Sakyo Ward, Kyoto City. (©Sankei by Junpei Teraguchi) Since then, research has progressed on technologies to produce various types of cells from the body and transplant them into patients for treatment. This is considered a game changer in regenerative medicine. Early in April, a venture company spun off from Osaka University applied for government approval for a treatment that involves transplanting sheets of cells into the heart. Clinical trials and clinical research involving cell transplants are also underway for many other diseases, including spinal cord injuries and diabetes. Nonetheless, international competition to commercialize regenerative technologies is fierce. The Japanese government has invested a total of ¥110 billion JPY (about $770 million USD) in iPS cell research over the past decade. But further support will likely be required. There is no doubt that iPS cell technology will open up new vistas in the field of medicine. We hope to see new treatments become available to patients quickly, and for Japan to lead the world in delivering transformative technologies. Author: Editorial Board, The Sankei Shimbun このページを 日本語 で読む
Yomiuri Shimbun
23-04-2025
- Health
- Yomiuri Shimbun
Medicine with iPS Cells: Research Originating from Japan Finally Bearing Fruit
Regenerative medicine, which uses iPS (induced pluripotent stem) cells and other materials to cure diseases for which there have been no treatment methods, is now much closer to practical application. It is hoped that a treatment method will be established at an early stage and it will be used for patients. Kyoto University Hospital has announced the results of a clinical trial in which nerve cells made from human iPS cells were transplanted into the brains of seven patients with Parkinson's disease, who then showed improvement in their symptoms. This is a groundbreaking achievement. Parkinson's disease is an intractable disease that causes tremors in the limbs and slowness of movement due to a decrease in dopamine, a substance in the brain that is related to the coordination of movement. It is estimated that there are about 290,000 patients in Japan. Symptoms can be suppressed with drugs that increase the volume of dopamine, but there is no fundamental cure. In the recent clinical trial, the treatment method was effective for some patients and they no longer required assistance. However, there is a need to confirm the effectiveness of the method in trials with a larger number of patients before it can be put into practical use. But, unlike conventional pharmaceuticals that can be mass-produced such as in the form of pills, the development and quality control of advanced pharmaceuticals using cells and other materials is much more difficult, and large-scale clinical trials also are not easy to conduct. For this reason, pharmaceutical companies that will manufacture and sell the drug with the cells intend to aim to collect a large amount of data by utilizing the conditional and time-limited approval, or early approval, system that applies to regenerative medicine. Under this system, unlike the usual procedure in which all clinical trials are completed before full approval and marketing, a drug company first seeks to obtain early approval, which is equivalent to a provisional license, after small-scale clinical trials, and then aims for full approval by collecting data showing treatment effects. This can be said to be a useful system that encourages technological innovation in the medical field and brings new drugs to patients faster. However, of the five products that were granted early approval separately from the recent clinical trial, the applications of two products were withdrawn due to the failure to prove the efficacy of the drugs, and the remaining three products have not yet received full approval. Although it is essential to fully verify the safety and efficacy of this product in the recent trial as well, there are high expectations that a path to practical application has been opened. Prof. Shinya Yamanaka at Kyoto University discovered technologies to generate iPS cells and was awarded the Nobel Prize in 2012. The government has intensively invested funds in iPS research. It seems that the research that has spanned more than a decade is finally bearing fruit. Recently, research on the use of iPS cells for the treatment of Parkinson's disease, spinal cord injuries and diabetes has been advancing. For example, a startup from the University of Osaka has applied for approval to manufacture and market cardiomyocyte sheets made from iPS cells to transplant them into patients with heart disease. It is hoped that collaboration between industry and academia will be strengthened to realize long-awaited treatments with iPS cells originating from Japan. (From The Yomiuri Shimbun, April 23, 2025)
Yahoo
31-01-2025
- Business
- Yahoo
Gameto launches Phase III iPSC fertility trial following IND greenlight
A Phase III trial investigating a method of maturing human eggs outside of the body using induced pluripotent stem cells (iPSC) has been given the green light after the US Food and Drug Administration (FDA) granted an investigational new drug (IND) application. US-based Gameto will now launch its multi-centre US trial to establish the efficacy of its ovarian support cell (OSC) technology designed to mature eggs outside the body using iPSC technology, in a system the company argues requires 80% fewer hormonal injections than traditional in vitro fertilisation (IVF) methods. Primary endpoints in the upcoming double-blind, randomised trial will consider the safety and efficacy of viable embryos produced outside the body and improving pregnancy rate assessed at 12 weeks. Secondary endpoints will investigate embryo development alongside maternal health and live birth rates. Dubbed Fertilo, the company describes its iPSC offering as able to use young ovarian support cells to recreate the natural egg maturation process in a dish. As a result, it has the potential to reduce the duration of treatment cycles down to two- or three-days while traditional methods rely on 10-14 days of high-dose hormonal stimulation to mature eggs. Gameto's chief scientific officer Christian Kramme said: 'Gameto's technology platform opens entirely new possibilities for addressing reproductive health challenges through cell engineering-inspired innovation. 'With Fertilo, we've demonstrated the potential of iPSC-based therapies to redefine fertility care by targeting ovarian health at the cellular level. This milestone also highlights the power of iPSCs in regenerative medicine and our computational and cell engineering platform where we have broader applications including menopause and ovarian cancer.' Gameto is entering into a relatively competitive market in terms of infertility, with research by GlobalData estimating that infertility treatments across the seven major markets brought in $2.3bn last year, rising to $3.4bn by the end of 2030. This growth is down, in part, due to the number of individuals choosing to have children later in life. GlobalData is the parent company of Clinical Trials Arena. Approval for the company's US Phase III trial comes after the company announced the first-ever live birth carried out as a result of its ovarian support cell system, to parents at the Santa Isabel Clinic, in Lima, Peru, in December 2024. Gameto's Fertilo builds on that of Japanese researcher Shinya Yamanaka, who won the Nobel Prize for Physiology and Medicine in 2012 for his discovery of iPSC. Gameto co-founder Dina Radenkovic said: 'By overcoming the major challenges of conventional IVF such as long treatment cycles, significant side effects, and the emotional and physical strain, Fertilo provides a potentially faster, safer, and more accessible solution for families. This milestone marks a turning point in reproductive health and highlights the first application of iPSC technology in IVF and the immense potential of our technology.' Elsewhere in the world of IVF treatment, Spanish fertility biotech Oxolife has presented positive exploratory Phase II data for its non-hormonal fertility pill. "Gameto launches Phase III iPSC fertility trial following IND greenlight" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
