This article was authored by Yuzo Toda, Forum for Innovative Regenerative Medicine and Fujifilm Corporation, for EY’s annual biotechnology report, Beyond borders – staying the course.
In March 2017, Japanese scientists carried out the world's first eye transplant using donor stem cells. The procedure involved transplanting retina cells created from donor-induced pluripotent stem cells (iPSC).
It marked an important milestone in the country’s already thriving regenerative medicine sector. By using a stockpile of iPSCs rather than relying on the patient's own cells, the team significantly cut the cost of the operation and the time required to prepare the patient, taking it closer to becoming a more viable option for those suffering from age-related macular degeneration (AMD).
Japan's large and growing elderly population, plus its long-term outlook, has led to regenerative medicine becoming a key part of government strategy. The field is about not just revolutionary treatment modalities, gene- and cell-based therapies (including the burgeoning cancer immunotherapy field). It's also about using human living cells — iPSCs in particular — as tools for broader drug development, for instance in drug screening, where previously only animal cells could be used.
Regenerative medicine and cell therapy techniques will be game changers with regard to both therapies and drug development. Demographic and economic realities across the world demand such game changers.
A collaborative approach among academia, government and industry is central to Japan's regenerative medicine ecosystem. The Forum for Regenerative Medicine (FIRM) embodies this approach, bringing together more than 200 members, including industry, nonprofits, academic institutes and clinics to encourage and accelerate the development of regenerative medicines. Its mission: to create treatments of value to society, but also to be globally competitive.
Industry has a crucial role to play in turning regenerative medicine research into solutions for patients. Systematic collection and use of medical data is also key, along with cutting-edge technological strength and sustainable costs.
In 2014, Japan introduced two laws to create a robust, yet accommodating regulatory framework for regenerative medicine. One covers safety in research, clinical trials and medical practice involving cell and gene therapies, the other provides a conditional approval pathway for such medicines, similar to the European Medicines Agency's adaptive licensing program.
As of 2017, Japan had approved two regenerative medicine treatments under the new laws. One is JCR Pharmaceuticals Co. Ltd.'s Temcell for steroid refractory graft-vs.-host disease, an allogeneic stem cell therapy based on mesenchymal stem cells, whose technology came from Osiris Therapeutics Inc. and partner Mesoblast Ltd. (An equivalent product, Prochymal, is approved in Canada and New Zealand.) The second is HeartSheet, an autologous skeletal myoblast therapy for heart failure due to IHD, manufactured by Terumo Corp.
Plenty of challenges remain, not least around production, distribution, quality assurance and international standards for regenerative medicines. They can only be resolved through close stakeholder collaboration — collaboration that FIRM is committed to building and strengthening.