IS MALAYSIA READY FOR GENE THERAPY?
It is making progress in the field, but there are many questions that must be considered, including balancing the potential benefits with unintended risks
ACCORDING to the seminal report, “Human Genome Editing”, jointly released by the US National Academy of Science (NAS) and the National Academy of Medicine (NAM) in early 2017, genome editing is a powerful new tool for making precise alterations to an organism’s genetic material.
Recent scientific advances have made it more efficient, precise, and flexible than ever before. These advances have spurred an explosion of interest from around the globe on the possible ways in which genome editing can improve human health. The speed at which these technologies are being developed and applied has led many policymakers and stakeholders to express concern about whether appropriate systems are in place to govern these technologies and how and when the public should be engaged in these decisions.
Last week, the Malaysian Industry Government Group for High Technology (MIGHT) played host to a public lecture by Dr Ismail Serageldin, a world renowed scientist, who shared his reflections on the impact of the new genetics on public health. “Are we ready for gene therapy?” he asked. Gene therapy is essentially the use of deoxyribonucleic acid (DNA) to cure disease, where DNA is the chemical substance of heredity that makes up the genes in humans and almost all other organisms.
Gene therapy involves a treatment through which copies of a normal gene (also called a therapeutic gene) are inserted into a person’s somatic cells to fight the disease caused by faulty gene present since birth. The normal copies will be expressed, thus overriding the faulty ones to fight the disease.
First encouraging results came in about two decades ago, when children with “bubble boy disease”, caused by a lack of functional immune system were cured using gene therapy. Without treatment, they would be forced to live in sterile environments forever to avoid infections.
Up to 2016, over 2,200 gene therapy clinical trials were reported.
They have demonstrated that the treatment could be used for common genetic defects such as congenital blindness, cystic fibrosis, anaemia, sickle-cell disease, Bthalassemia and Duchenne Muscular Dystrophy. Although gene therapy holds promise for the future, it is still considered as experimental. Many technical problems need to be resolved for it to become a standard medical practice.
For many years, researchers tried to develop efficient and reliable ways to make targeted and precise changes to the genome of living cells. Their efforts have paid off with the discovery of CRISPR technology — Clustered Regularly Interspaced Short Palindromic Repeats found in bacteria, which they use to defend themselves against viruses.