Celgene Corp. says its experimental stem cell treatment showed promise in a tiny, Phase I study of patients with Crohn's disease who failed to respond to prior therapy, according to initial results.
The study tested just 12 patients with Celgene's PDA-001 stem cell treatment derived from human placental tissue. But the company was encouraged enough by the results that it will conduct multiple Phase II studies across a number of diseases.
Since PDA-001 is harvested from normal, full-term human placental tissue, Celgene said, it is likely to avoid the controversy over embryonic stem cells, which are vehemently opposed by religious and anti-abortion groups.
"We are encouraged that in these patients with Crohn's disease our unique, placenta-derived therapies show signs of clinical benefit," Robert Hariri, CEO of Celgene's Cellular Therapeutics unit, said in a statement.
"We will continue to aggressively pursue the clinical development of this and other cellular therapies derived from what we see as one of the richest sources of uniquely functional and versatile cells."
Stem cells are a hot area of medical research because they are believed to be able to transform into many other types of cells that could one day help treat a wide variety of injuries and illnesses.
The 12 patients with active moderate-to-severe Crohn's who were unresponsive to at least one prior therapy were given two infusions of PDA-001 one week apart. Six patients received a lower dose of the cells, and six received a higher concentration, or high dose, of PDA-001.
The study met its primary safety goal and demonstrated encouraging signs of clinical benefit, including clinical remission among four patients in the low dose group, Celgene said.
The company declined to speculate on why more dramatic results were seen in the lower dose patients.
Celgene discussed the stem cell study at a meeting with analysts and investors in New York at which it was showcasing its drug research pipeline.
Crohn's disease is a chronic inflammatory condition of the gastrointestinal tract that affects almost one million people in the United States.
Current, less than ideal, treatments include anti-inflammatory or immunosuppressive drugs that can lead to significant side effects and complications. Patients often require surgery or become unresponsive to therapy.
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