AsianScientist (May 28, 2015) – A stem cell research team at the Li Ka Shing Faculty of Medicine, The University of Hong Kong (HKU), has led the first genetically modified hematopoietic stem cell transplantation in Asia for an 18 year old Taiwanese with the fatal inherited disorder metachromatic leukodystrophy (MLD).
The work was done in close collaboration with researchers from The Second People’s Hospital of Shenzhen (The First Affiliated Hospital of Shenzhen University) and the National Taiwan University Hospital (NTUH).
MLD is a devastating lysosomal storage disease caused by a deficiency of arylsulfatase A (ARSA). Without this enzyme, sulfatides are not broken down and accumulate in the white matter of the brain and nervous system, causing destruction of the myelin sheath, or demyelination.
Current approaches to MLD treatment involve enzyme replacement therapy or hematopoietic stem cell transplantation. Unfortunately, the high molecular weight ARSA protein is unable to penetrate the blood-brain barrier, while autologous stem cell transplants risk immune rejection.
Instead, a team led Dr. Lian Qizhou, assistant professor of the Department of Ophthalmology and Department of Medicine of Li Ka Shing Faculty of Medicine, HKU, conducted a hematopoietic stem cell transplant using the patient’s own cells that had been genetically modified to express ARSA.
Since the transplant was performed in September 2014, early results indicate stabilisation of the disease. The patient’s mobility and balance, capacity of hygiene management and ability to write have improved, constipation and urinary problems have resolved, and no seizures have been recorded.
Commenting on the project, Professor Zhuo Jiacai, clinical professor and head of the Division of Hematology at the Second People’s Hospital of Shenzhen (The First Affiliated Hospital of Shenzhen University), added on how it helped the patient.
“Before stem cell transplantation, the patient required chemotherapy, termed Busulfan-based conditioning, to remove original bone marrow cells and generate space for gene corrected-stem cell growth,” said Zhuo.
“This procedure potentially introduced the risk of multiple lesions including the neural system. By careful titration of therapy, the conditioning treatment was well tolerated and the patient made a rapid recovery following transplantation without obvious side-effects.”
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Source: The University of Hong Kong.
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