Japanese pharmaceutical company Sumitomo Pharma has applied to Japanese regulatory authorities for market approval of a Parkinson's disease treatment utilizing induced pluripotent stem cells (iPS cells). If approved, it is expected to be the world's first Parkinson's disease treatment using iPS cells.
According to the Nihon Keizai Shimbun on the 6th, Sumitomo Pharma has applied to the Ministry of Health, Labour and Welfare for manufacturing and sales approval of a treatment that involves transplanting nerve cells made from iPS cells into Parkinson's disease patients.
Parkinson's disease is a degenerative disease characterized by the deterioration of nerve cells that secrete dopamine in the brain, resulting in decreased motor function, and there is currently no fundamental treatment.
Shinya Yamanaka, a professor at Kyoto University, discovered iPS cells and received the Nobel Prize in Physiology or Medicine in 2012. Sumitomo Pharma is collaborating with him to challenge the commercialization of this treatment.
iPS cells are derived by inducing dedifferentiation into an embryonic stem cell state, where all human cells can grow, by introducing specific genes, proteins, or chemicals into fully grown cells. Unlike embryonic stem cells, which are obtained from fertilized eggs, iPS cells can avoid bioethical controversies. Another benefit is that treatments can be made from the patient's own cells, eliminating the need for immunosuppressants.
Sumitomo Pharma stated that the Kyoto University research team confirmed in clinical trials involving six Parkinson's disease patients that there were no serious side effects, and four patients showed improvements in motor function.
If Sumitomo Pharma's iPS cell treatment is formally approved, it will become the world's first iPS cell Parkinson's disease treatment. However, the Nihon Keizai Shimbun reported that the clinical trial scale is small and additional verification regarding the treatment's effectiveness is needed, making it likely that the Ministry of Health, Labour and Welfare will grant conditional approval. Conditional approval involves submitting additional efficacy and safety data within a certain period post-marketing.
Sumitomo Pharma is also conducting larger-scale clinical trials in the United States. The company's annual investment in regenerative medicine and cell therapy amounts to 10 billion yen (approximately 943 million won). In Japan, besides Sumitomo Pharma, Qulips has also applied for approval for an iPS cell-derived treatment for heart disease.