Roadmap for developing pluripotent stem cell-derived cellular therapies

The International Society for Stem Cell Research (ISSCR) has published a comprehensive new resource for accelerating the translation of pluripotent stem cell-derived. reNEW Associate Professor Ana Hidalgo-Simon was one of the international experts that contributed to this valuable resource during a two-year process.

Pluripotent stem cell–derived therapies hold immense promise for patients with chronic conditions such as type 1 diabetes, Parkinson’s disease, chronic kidney disease, and heart failure. Yet, until recently, there were no global standards to guide the efficient translation of these innovations through the entire pipeline – from early research to clinical testing along the regulatory pathway. To address this gap, the ISSCR launched an international initiative to establish such a framework.

reNEW’s Ana Hidalgo-Simon was on the steering committee for the resource called ‘Best Practices for the Development of Pluripotent Stem Cell-Derived  Cellular Therapies’.

“As the development and the clinical need for these therapies are both global, it makes a lot of sense to have a global set of standards that complies with regulatory requirements across constituencies. This is an excellent guide for developers both from academia and commercial entities. It can help to make the ‘I wish I have done things differently’ an unnecessary expression,” said Hidalgo-Simon.

The resource is organized into seven sections, ranging from raw material use to preclinical studies and regulatory consideration. Hidalgo-Simon was actively involved in the latter as a working group member. The outcomes from this regulatory considerations working group have been reviewed by regulators in nine regions including Australia, Europe and the United States.

The guide should ultimately lead to the acceleration of pluripotent stem cell-derived therapy development. Kapil Bharti, co-chair of the steering committee, said: “Through the Best Practices and their anticipated effect on the speed and rigor of the translational process, we hope to increase the number and quality of stem cell-based therapies reaching patients around the world.”