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Stem cells might beat drugs in delivering relief faster, more effectively to rheumatoid arthritis sufferers

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People who have RA overproduce a protein called tumor necrosis factor (TNF), which causes the inflammation and damage to the bones, cartilage and tissue. Anti-TNF drugs can block the action of the protein and reduce inflammation. Etanercept® (marketed under the trade name Enbrel) is a type of anti-TNF drug called a biologic that for years has been prescribed to treat RA. However, it can’t be targeted specifically to the site of the arthritis and, thus, requires higher doses that can cause serious side effects including fatal infections, multiple sclerosis, seizures, heart failure, cancer and more.

“Moreover, biologics in general require intense development and manufacturing processes that are challenging for reproducibility, even within the same company. So we wanted to see how delivering treatment through a very targeted system such as that which can be done using stem cells compared to a biologic drug such as Etanercept®,” said Joseph Mosca, Ph.D. He led the team of researchers from Osiris Therapeutics, Inc. Baltimore, Md., and the Novartis Research, Basel, Switzerland, in conducting the study.

The researchers began by genetically altering human mesenchymal stem cells (MSCs) in the lab to become vehicles for the cell-based anti-TNF delivery. They then injected the cells into mice that had been induced with RA and monitored them over a seven-day period, then compared the results to a group of animals treated with Etanercept®. The results showed that the anti-TNF therapy delivered by stem cells reversed or attenuated the arthritis inflammation on par with the Etanercept® — except that it did it faster.

“If this translates into fewer side-effects and/or lower compliance remains to be seen,” Dr. Mosca said. “In either case, these results illustrate the ability of stem cells to deliver proteins of therapeutic value and demonstrate their potential clinical utility in rheumatoid/osteoarthritis and other TNF-related diseases where anti-TNF biologic drugs have already shown promise.”

“The authors have shown the feasibility of a targeted approach to treatment using cells that are known to ‘home’ to damaged tissue,” said Anthony Atala, M.D., Editor of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine. “The manuscript supports and demonstrates the potential of mesenchymal stem cells as a vehicle for cell-based gene delivery.”