A type of cell drawn from certain body tissues shows enormous potential to treat a range of diseases in the United States and Europe, but it must first receive federal approval as a regulated pharmaceutical.

That is the message of a recent report by a UW-Madison researcher. Jacques Galipeau, MD, director of the Program for Advanced Cell Therapy at the UW School of Medicine and Public Health, says mesenchymal stromal cells are the most-studied cell therapy that is not yet FDA-approved.

Mesenchymal stromal cells, which have regenerative and anti-inflammatory properties, are harvested from bone marrow or adipose tissue, and then grown in a lab to produce large quantities. These manufactured cell products are one of the most researched type of cells worldwide in human clinical trials for advanced cell therapies.

“The use of manufactured living cells to treat disease is entering mainstream medical practice in Europe,” he said. “Among these platforms, mesenchymal stromal cells have been under study for nearly 20 years and are now poised to impact patients with Crohn’s disease and graft-versus-host disease.”

To date, research has focused on both anti-inflammatory and regenerative therapeutic properties of this cell type, and the most advanced clinical studies examine their use for Crohn’s disease, heart disease and graft-versus-host disease.

The paper, co-authored by Luc Sensebe, of STROMALab in Toulouse, France, was recently published in the journal Cell Stem Cell.
The research analysis explains the potential of these cells, as well as the scientific, regulatory and business challenges to approval in the U.S.

“We outline the important scientific, translational and distributive justice issues, which can inform optimal use of these living-cell drugs, particularly mesenchymal stromal cells, for patient benefit,” Galipeau said.

His program is working to secure federal Food and Drug Administration approval for the use of these cells and other novel cell types to treat patients at UW Health’s University Hospital in Madison, Wis.

The Program for Advanced Cell Therapy offers an additional benefit to traditional pharmaceutical implementation of cellular immunotherapies. It is housed in the hospital it serves, Galipeau said, reducing the friction for seamless and sustainable deployment within a large health care enterprise.

A key to providing effective living-cell drugs is their fitness, the research analysis explained.

When cells are extracted, manipulated, frozen and flown across the country or transported by vehicle for hours to the location where they will be thawed and used, the cells become less effective.

By placing the lab that treats the cells in the health care setting, the transportation problem is eliminated, and better treatments are possible, Galipeau said.

“Our program is an engine to achieve this goal,” he said, adding it could be a model for health care institutions in other states.
For more information, visit the Program for Advanced Cell Therapy.