FDA & Japanese Health Ministry (厚生労働省) To Develop iPS cell clinical rules

In a rare sign of stem cell international regulatory unity, the Japanese Health Ministry (厚生労働省) and the US FDA have agreed to develop a joint, unified regulatory framework for clinical studies of human iPS cells for use in treating retinal diseases. Presumably the rules would also guide clinical use of iPS cells to treat other diseases.

The two regulatory bodies aim to have the joint rules in place by 2015.

To me this seems like a positive development, but it may come a bit too late as the first clinical study use of iPS cells in human patients is poised to begin in less than a year in Japan, well before the joint regulatory framework would be in place.

In fact, it was just announced that the proposed first in human use of an iPS cell product to treat macular degeneration cleared yet another hurdle as a Japanese Health Ministry subpanel OK’d the project.

Interestingly, the subcommittee meeting that gave the OK to the human iPS cell study was apparently the first meeting of its kind open to the public, which seems like an important, positive development.

It’s unclear if attendees were allowed to ask questions or if anyone attended other than reporters. I suspect no scientific data was publicly presented, but I’m not sure.

It is notable that the same screening panel, including chair Ryozo Nagai, that oversees the iPS cell project has asked for additional data from Masayo Takahashi’s team in the form of what appears to be whole genome sequencing data on the iPS cells.

It is also encouraging that the panel also will consult with cancer experts as they further evaluate the project and consider potential safety risks.

Three months ago I publicly called for the Takahashi team to publish their pre-clinical data before doing the first human experiments, but to date that has not happened.

Some called my post making that call unrealistic on that topic as, they asked, why would anyone publish proprietary data that might give their commercial competitors an advantage? Indeed, there is no rule that pre-clinical data has to be published before clinical studies begin.

However, patient safety must come first before commercial considerations and I believe given the historic nature of the first ever human trial of iPS cells that the full stem cell community should be given the opportunity to consider the pre-clinical data, not just regulatory bodies in private.