Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects

Phase I Study of the Safety and Preliminary Efficacy of Intracerebral Transplantation of HuCNS-SC® Cells for Connatal Pelizaeus-Merzbacher Disease (PMD)

The purpose of this study is to determine the safety and preliminary effectiveness of human central nervous system stem cells (HuCNS-SC®) transplantation in patients with Connatal Pelizaeus-Merzbacher Disease (PMD).

Enrolled subjects will be transplanted with HuCNS-SC cells into the brain and will receive immunosuppression for nine months. The study observation period is for one year after transplant surgery. Thereafter, subjects will be enrolled in a long-term observational follow-up study for four years.

Inclusion Criteria:

• Confirmed clinical diagnosis of connatal PMD
• Molecular genetic confirmation of mutation in the proteolipid protein 1 (PLP1) gene
• MRI consistent with PMD as interpreted by a qualified neuroradiologist

Exclusion Criteria:

• Other significant congenital brain abnormality not related to PMD
• Previous participation in gene transfer or cell transplant trial
• Presence of neurological signs and symptoms not consistent with PMD
• Current or prior malignancy
• Prior organ, tissue or bone marrow transplant