BRIEF CURRICULUM VITAE
Name: Nasser Aghdami
Educational Background:
1. 1991 – 1998:MD Oroumieh University of Medical Sciences, Oroumieh, Iran
2. 2001-2007: PhD, Tarbiat Modares University, Tehran, Iran, PhD in Immunology
Current Position in the Institutions:
2008-present Head of Department of Regenerative medicine and Royan cell therapy
Abstract: Stem Cell Therapy in neurometabolic and neurodegenerative disorders
Inherited metabolic disorders (IMDs), especially when neural system involved, cause progressive
organ failure and death early in life. Although Enzyme replacement therapy can be effective for
treating a handful of enzyme deficiencies, it does not provide a cure from the endogenous disorder,
and the patient is left with lifelong dependence on pharmaceuticals. Cross-correction after donor
leukocytes engraftment in the host tissue during hematopoietic stem cell transplantation, make and
secrete the deficient enzyme, and can improve the diseases behavior. Hurler Syndrome is the first and
most common inborn error of metabolism, which HSCT is indicated and performed.
While bone marrow transplantation seem like the most likely to stop the disease progress, but when
we need regeneration for tissue or function lost, other tissue specific stem cells such as neural or
mesenchymal stem cells seems more suitable. Experimental studies, were showed intracranially
injection of NSC in symptomatic Sandhoff (Hexb(-/-)) adult mice, increased animal lifespan,
improved neurological function, and slowed disease progression. Cells migrated far from the injection
site and integrated into the host cytoarchitecture, restoring beta-hexosaminidase enzyme activity to
improvement of neuropathologic and behavioral
Accessibility is the largest obstacle that would be overcome to have enough cells for effective
treatment. Neural stem cells (NSC) are located in sub ventricular zone but extremely rare in healthy
subjects and we need injury like ischemic injury to NSC proliferation. Moreover, NSCs may impact
conditions such as neurogenetic lysosomal storage diseases when transplanted at birth, but it is
unclear whether such interventions can be effective in well-established disease, a situation often
encountered clinically.
It seems, stem cell therapy is at its early stages and we need generated more data in animal models
and clinical trials to know more about diseases mechanisms or treatment effects. The possibility of
deriving human induced pluripotent stem (iPS) cells by overexpressing just a few transcription factors
in somatic cells has opened new opportunities for in vitro disease modeling and can improve our
knowledge about disease mechanisms and their treatment method respectively. Here, I review the
current state of research on various stem cells and identify the important challenges that must be
addressed as stem cell therapy is brought to the clinic in neurodegenerative disorders.