Skip Navigation LinksHome > March-April 2013 - Volume 19 - Issue 4 > Safety and Tolerability of Human Placenta-Derived Cells (PDA...
Inflammatory Bowel Diseases:
doi: 10.1097/MIB.0b013e31827f27df
Original Clinical Articles

Safety and Tolerability of Human Placenta-Derived Cells (PDA001) in Treatment-Resistant Crohn’s Disease: A Phase 1 Study

Mayer, Lloyd MD*; Pandak, William M. MD; Melmed, Gil Y. MD; Hanauer, Stephen B. MD§; Johnson, Kristine BS; Payne, Denise RN, MPA; Faleck, Herbert MD; Hariri, Robert J. MD, PhD; Fischkoff, Steven A. MD

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Abstract

Background: The clinical utility of cellular therapies is being investigated in a broad range of therapeutic areas. This phase 1 study represents the first exploration of PDA001, a preparation of cells cultured from human placental tissue, in subjects with Crohn’s disease.

Methods: Twelve subjects with active, moderate-to-severe Crohn’s disease unresponsive to previous therapy were given 2 intravenous infusions of PDA001 1 week apart, monitored weekly for 5 weeks, and assessed at 6 months, 1 year, and 2 years after infusion. Six subjects received 2 infusions of 2 × 108 cells (low dose), and 6 subjects received 2 infusions of 8 × 108 cells (high dose).

Results: Mean baseline Crohn’s Disease Activity Index in the low-dose and high-dose groups was 305 and 364, respectively, and mean C-reactive protein was 8 mg/L and 49 mg/L, respectively. All subjects in the low-dose group achieved a clinical response (a Crohn’s Disease Activity Index decrease of ≥70 points versus baseline), and 3 achieved remission (a Crohn’s Disease Activity Index decrease of ≥100 to <150 points). Two subjects in the high-dose group achieved response, and none met remission criteria. Most adverse events were mild to moderate in severity and included headache, nausea, fever, and infusion site reactions.

Conclusions: PDA001 infusions appear safe and well-tolerated in subjects with treatment-resistant Crohn’s disease. A response was seen in all subjects in the low-dose group. The high-dose group, with a higher baseline disease activity, had only 2 responders, suggesting a more treatment-resistant population. A phase 2 study in this patient population is ongoing.

© Crohn's & Colitis Foundation of America, Inc.

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