Stem Cell Therapy Seen as Safe, May Improve Motor Function, Early Findings Suggest


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Parkinson's News Today
MAY 7, 2019 Jose Marques Lopes, PhD

#AANAM – Stem Cell Therapy Seen as Safe, May Improve Motor Function, Early Findings Suggest

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Treatment with mesenchymal stem cells (MSCs) — adult stem cells with regenerative effects — is safe and well-tolerated, and improves motor function in people with mild to moderate Parkinson’s disease, according to early findings from an ongoing small study.

The research, “Preliminary Report on the Safety and Tolerability of Bone marrow-derived Allogeneic Mesenchymal Stem Cells infused intravenously in Parkinson’s disease Patients,” was presented during the 2019 American Academy of Neurology (AAN) Annual Meeting, being held in Philadelphia.

MSCs — adult stem cells found in multiple tissues, such as umbilical cord, bone marrow and fat tissue — have regenerative and immunomodulatory effects, which makes them potential therapies for the chronic neuroinflammation associated with Parkinson’s.

Researchers from the University of Texas McGovern Medical School, City University of New York, and Baylor College of Medicine are assessing the safety and feasibility of allogeneic (from a donor) MSCs. The stem cells were taken from the bone marrow of a healthy adult, purified, and delivered intravenously (into the vein) in increasing doses to patients with idiopathic (of unknown cause) Parkinson’s.

The study enrolled 20 participants, including 11 men, ages 45-78. All were in off state — which refers to the resurgence of symptoms due to a gradual decline in levodopa’s efficacy — and were at stage 3 or less in the Hoehn & Yahr (H&Y) scale, correlating with mild-to-moderate disease severity. Stage three is considered mid-stage and is characterized by loss of balance and slowness of movement.

Each dose group consisted of five patients who received one of four doses of MSCs: 1, 3, 6 or 10 x106 MCS/kg of body weight. Participants are evaluated over the course of a year, at weeks 3, 12, 24 and 52.

The study’s primary outcome is safety, defined as no transfusion reactions, adverse events or organ damage. Secondary outcomes include the treatment’s impact on Parkinson’s progression, as assessed with the Unified Parkinson’s Disease Rating Scale (UPDRS), the Timed Up and Go Test of mobility and balance, the Parkinson’s Disease Questionnaire (PDQ-39) — a measure of health status and quality of life — the H&Y scale, the Columbia-Suicide Severity Rating Scale, neuroimaging, and immunologic profile.

None of the patients experienced adverse reactions within 24 hours of the single intravenous stem cell infusion. In later assessments, the most frequent side effects were hypertension, arthralgia (pain in a joint), and nausea, which were mild and temporary in all cases and did not require treatment.

To date, the first 14 treated patients showed lower (better) UPDRS-III motor scores at 12 weeks of follow-up.

“[A]llogeneic MSC infusions appear to be safe and well tolerated in subjects with mild to moderate Parkinson’s,” the researchers said.

“Our preliminary results warrant the completion of the study with the goal of identifying an ideal, well-tolerated dose that is associated with an improvement in cognition, motor function, and disability,” they added.