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Stem Cell Regenerative Medicine for Cerebellar Degeneration

Research Summary: Cerebellar degeneration results in the loss of speech and motor coordination. This can be caused by a variety of conditions that include inheritable genetic disorders (Friedreich's ataxia, spinocerebellar ataxia and Niemann-Pick Type C) and associated conditions like stroke, toxins (e.g. chronic alcoholism), inflammation (e.g. Multiple Sclerosis), infections (e.g. Creutzfeldt-Jacob disease) and neoplastic disorders.  There is no cure for patients with cerebellar degeneration, and treatments are largely supportive.  While there has been much research devoted towards other neurological conditions like Alzheimer's disease and Parkinson's disease, there has been little research directed towards finding treatment for cerebellar disorders.  

Despite the underlying cause, cerebellar degeneration leads to the irreversible loss of Purkinje cells - the principal cell of the cerebellum.  Thus, cell replacement with healthy Purkinje cells represent a potential strategy for treating cerebellar degeneration. Purkinje progenitor cell (PPC) is the stem cell that differentiates into terminal Purkinje cells.  PPC are differentiated from pluripotent stem cells.  However, there has been little effort to develop a PPC therapy for cerebellar degeneration.  The technical barrier to create clinical-grade PPC has been the neoplastic risk of pluripotent stem cells.  Previous reports have shown that elimination of oncogenes or cancer-causing genes from induced pluripotent stem cell (iPSC) reprogramming can maintain pluripotency without causing tumors in mouse models.  However, oncogene-free iPSC reprogramming has not been achieved without the use of viruses, which are not safe for clinical therapy.  The objective of this research study is to develop a healthy immune-tolerant PPC from iPSC that can serve as a universal cell replacement therapy for cerebellar degeneration that is characterized by Purkinje cell death.


In collaboration with Cellular Engineering Technology, a biotechnology company, the Institute has co-developed a non-viral and oncogene-free iPSC technology. By eliminating viruses and oncogenes, safer PPC replacement therapy can now be realized.  We have developed a pipeline of healthy iPSC that have the potential to serve as universal PPC therapies that exhibit immune-tolerance without regard to the recipient's HLA status.

A requirement before cell transplantation of PPC can be used in patients with cerebellar degeneration is the need to confirm that these cells are not rejected by the patient's immune system. This cannot be fully tested without mixing these stem cells with the immunological cells of patients with cerebellar degeneration.  This research will be done at our facility and will not be injected into patients.


As proof of concept, the Institute is recruiting patients with spinocerebellar ataxia as a prototype of patients with cerebellar degeneration.  Thus, we are asking patients with spinocerebellar ataxia to sign up on the Institute's patient registry.  If chosen for the research study, it is expected that the research subject will provide medical records that confirm the diagnosis of spinocerebellar ataxia and a blood sample.  For further questions about the study, send comments or questions to 

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