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FDA Stem Cell Guide

FDA Approved vs Investigational Stem Cell Therapies

"Approved" and "investigational" are legal terms, not marketing ones. This guide explains what each really means for a stem cell product.

FDA-approved

A stem cell product is FDA-approved when the agency has reviewed the manufacturer's Biologics License Application (BLA) and concluded that the product is safe and effective for a specific medical use, in a specific patient population, at a specific dose and route of administration. Approval also requires that the manufacturer can consistently produce the product to quality standards.

Approval is narrow. A product approved to treat a particular type of blood cancer is not automatically approved to treat any other condition — using it for something else would generally be an unapproved use.

Investigational

A product is investigational when it is being studied under an active Investigational New Drug (IND) application. An IND allows a sponsor to legally ship the product across state lines and administer it to human subjects for the purpose of collecting safety and efficacy data. Investigational products are typically administered:

  • Only within a registered clinical trial with a formal protocol.
  • After review by an Institutional Review Board (IRB).
  • With written informed consent describing the risks and unknowns.
  • Usually at no cost to the participant for the investigational product itself.

Investigational status is not a stamp of effectiveness. Many investigational products never receive approval because trials show they don't work, don't work well enough, or carry unacceptable risks.

What "not approved" often looks like in the wild

Some clinics market stem cell products for conditions where the FDA has not approved any stem cell therapy. These offerings are sometimes described using phrases like "regenerative", "cutting-edge", or "cellular therapy". If the product is not on the FDA's list of approved cellular therapy products and is not being administered under an IND in a registered trial, it is unapproved for that use.

How to check

  • The FDA publishes a public list of approved cellular and gene therapy products. Cross-reference the product name.
  • Legitimate clinical trials are registered on ClinicalTrials.gov with a study number (NCT ID), a listed sponsor, and a written protocol.
  • Charging patients thousands of dollars for an "investigational" therapy outside a registered trial is a common warning sign, not a norm.