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Small, Early Study of Placenta-Derived Stem Cells Shows Safety in People with Relapsing-Remitting and Secondary-Progressive MS: Further Study Planned

October 2, 2014

Infusions of stem cells derived from placenta (a formulation known as “PDA-001” manufactured by Celgene Cellular Therapeutics) were shown to be safe in a small, phase 1 study of 16 people with relapsing-remitting or secondary-progressive MS. Fred Lublin, MD (Mouth Sinai Medical Center, New York, NY) and colleagues in the United States and Canada report their findings in Multiple Sclerosis and Related Disorders (Published Online: August 28, 2014). This study was not designed to show effectiveness. The authors comment that the next step, a proof-of-concept clinical trial, is planned.

Background: There is exciting progress being made through innovative research related to the potential of many types of stem cells both for slowing MS disease activity and for repairing damage to the nervous system. There are many types of stem cells that are undergoing research and which are producing knowledge about their potential use in treating MS. Many of these studies involve adult mesenchymal (pronounced messENkimmul) stem cells, which are present in many tissues of the body, including bone marrow, fat (adipose tissue), and placenta obtained after birth. These cells are being tested for their ability both to treat immune disorders and promote tissue repair. Further study is necessary to determine whether and which cells might prove beneficial for treating people with MS. The current study involves PDA-001 (Celgene), a formulation of stem cells derived from full-term human placenta.

The Study: Investigators randomly assigned 16 people with relapsing-remitting or secondary-progressive MS to receive two infusions of low-dose PDA-001, high-dose PDA-001 (four times the amount of cells) or inactive placebo, one week apart. Participants underwent MRI scans monthly for six months and were followed for another six months. Thirteen participants were taking disease-modifying or symptom medications during the study.

The primary endpoint established for the trial was to rule out the possibility that treatment would worsen MS disease activity to the point of a person experiencing more than five new active MS brain lesions on two consecutive monthly MRI scans, and any impact on the frequency of relapse. Secondary endpoints included the EDSS scale which measures disability, and measures of fatigue and quality of life. As in most phase 1 trials, this study was not designed to determine the treatment’s effectiveness.

The primary endpoint was achieved, meaning that disease activity did not worsen to the point of a person experiencing more than five new lesions on two consecutive monthly scans. One participant experienced a relapse. New lesions appeared in two participants in the high-dose group and in one participant in the low-dose group. The person who had a relapse experienced an increase of more than .5 on the EDSS scale; EDSS scores remained stable or decreased in most participants. There were no trends observed in any other clinical scales.

Two participants experienced serious infusion-related adverse effects, but these resolved without affecting treatment. Other common adverse events – all mild or moderate – included headache, upper respiratory infection, fatigue, infusion site reactions, and urinary tract infection.

Conclusion: The authors comment that this first trial of placenta-derived stem cells appeared to be safe and well tolerated in a small number of people with MS. They also noted that a proof-of-concept clinical trial is planned to further explore safety, feasibility, dosing and potential benefits of this approach.

With the urgent need for more effective treatments for MS, particularly for those with more progressive forms of the disease, this study represents a potentially important step toward understanding the potential therapeutic value of adult stem cells for treating MS.

The National MS Society is currently supporting 12 research projects exploring various types of stem cells, including cells derived from bone marrow, fat and skin, and has supported 70 stem cell studies over the past 10 years.  Read more about stem cells and MS.

About Multiple Sclerosis

Multiple sclerosis is an unpredictable, often disabling disease of the central nervous system that disrupts the flow of information within the brain, and between the brain and body. Symptoms range from numbness and tingling to blindness and paralysis. The progress, severity and specific symptoms of MS in any one person cannot yet be predicted, but advances in research and treatment are leading to better understanding and moving us closer to a world free of MS. Most people with MS are diagnosed between the ages of 20 and 50, with at least two to three times more women than men being diagnosed with the disease. MS affects more than 2.3 million people worldwide.

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