This small molecule is being evaluated for the treatment of advanced non-small cell lung cancer, as part of a Phase 3 global multi-centre clinical trial. To understand why, Yamauchi’s group has developed an innovative in vitro model of PMD using retroviruses and are currently exploring potential therapeutic molecules through tests on animals.Īt the TUPLS Department of Medicinal Chemistry, Yoshio Hayashi and his group have created anti-tumour agent called Plinabulin. Studies show that mutations of a particular lipid soluble protein, proteolipid protein 1 (PLP1), lead to dysfunction in cells that act as support and insulation to nerve cells in PMD patients. That’s why “one of the key goals of this project is to help us discover drugs for rare diseases that do not pay off in the private sector,” explains Masato Tanaka from TUPLS’s Laboratory of Immune Regulation.įor instance, at TUPLS’s Laboratory of Molecular Neuroscience and Neurology, researchers led by Junji Yamauchi are working on treatments that could help patients with Pelizaeus– Merzbacher disease (PMD), a rare inherited condition involving the central nervous system that affects one in 200,000–300,000 people.
The pharmaceutical industry will mostly develop drugs that have potential for profit, leaving people with rare diseases underserviced. A five-year project called Academia Drug Discovery began to focus on collaborations between life science and pharmacy researchers, wide-ranging outreach efforts, and work on overlooked diseases. In 2017, Tokyo University of Pharmacy and Life Sciences (TUPLS) started to prioritize its drug discovery programmes.
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