Getting approval for stem cell clinical trials: a step by step guide

Ellen Feigal, M.D. Senior VP for
Research & Development at CIRM

Trying to move a promising therapy from the lab into clinical trials in patients is one of the most challenging parts of any drug discovery process. But when that new therapy involves a stem cell or cell-tissue it can be doubly so. You are not only dealing with questions of whether it’s safe (always good to know) and whether it works (rather important too) but you are also dealing with a whole new area of science that adds extra questions and complications along the way.

That’s why CIRM teamed up with the Alliance for Regenerative Medicine (ARM) to come up with a new step-by-step guide on how to navigate the tough regulatory waters and get approval from the Food and Drug Administration to move a therapy into clinical trials. The result is a white paper published in the journal Stem Cells Translational Medicine.

It lays out precisely what a company needs to do, when, why and how if it wants to get FDA approval.

As Ellen Feigal, MD, CIRM’s Senior VP for Research and Development and the lead author of the article, points out in the paper:

“The field of regenerative medicine is still relatively young and involves new and novel science. The products involved might be classified as a biologic - such as human cells and tissues or gene therapy- a device, a drug or combination of those. They also span a range of areas from biology to chemistry and physics and so could come under multiple regulatory agencies. All that can create uncertainty and confusion. The goal of this paper is to help companies understand the complexity of the process and how to most effectively navigate through it.”

In our press release we quote Michael Werner, Executive Director of the Alliance for Regenerative Medicine, and a co-author of the study:

“Everyone involved in this process, whether it’s a company or the FDA, has a shared goal of bringing safe and effective therapies to the public as quickly as possible.”

The article is comprehensive but even with this information companies will face lots of challenges along the way But by helping take some of the uncertainty out of the process we hope to make it easier for companies to get their therapies where they are needed the most, in patients.

K.M.