For decades, new innovations in stem cell biology and genetic manipulation have driven a wave of proposed treatments based on these technologies. Although there has been intense speculation and expectation about when and how these methods will finally begin to show true potential for impacting patients’ lives, there is still only one gene therapy which has been approved by the US FDA. However, the results of a recent clinical trial suggest a new type of gene therapy may soon be available.
In a very promising new study, doctors have applied both gene and stem cell therapies together to stop a childhood neurodegenerative disease known as cerebral adrenoleukodystrophy (ALD). This is a severely debilitating disease affecting nerve myelination, and patients diagnosed with ALD usually do not live more than a decade after the disease is identified. Until now, the only viable treatment for children afflicted with this genetic disorder has been a hematopoietic stem cell (HSC) transplant from a matched donor. Transplantation from a HSC donor not only requires the availability of a compatible, healthy individual, but also carries significant risks of transplantation-related complications.
In this clinical trial, published in the New England Journal of Medicine, HSC from affected patients were isolated and then treated with a lentivirus-based vector to introduce a healthy copy of the mutated ABCD1 gene. The lenti-transduced HSC were then put back into the ALD patients. As described in this study, almost all the treated patients remained free of major neurological disabilities two years after treatment and continued to express healthy ABCD1 protein. Given the normally rapid progression of ALD symptoms, this result represents a monumental success. It now seems that gene therapy may begin to live up to its early hype, and truly revolutionize the treatment of genetic disorders.
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- Eichler F, Duncan C, et al. Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med. 2017 Oct 4.