Ray of hope for gene therapy in Sri Lanka | Daily News

Ray of hope for gene therapy in Sri Lanka

Unlike the West, the negligence of inherited diseases in Sri Lanka has resulted in patients with genetic diseases dying for want of proper diagnosis. This is partly due to limited facilities, inaccessibility and non-affordability.

However, due to free genetic testing in the state sector established by Prof. Ranil de Silva, Director, Interdisciplinary Centre for Innovation in Biotechnology and Neuroscience (ICIBN), Faculty of Medical Sciences, University of Sri Jayewardenepura (USJ), the provision of over 500 free molecular diagnostic reports to neurogenetic disease patients has been made possible.

This includes the following diseases: Duchenne Muscular Dystrophy (DMD), Spinocerebellar Ataxias (SCA), Huntington’s Disease (HD), Spinal Muscular Atrophy (SMA), Limb Girdle Muscular Dystrophy (LGMD) and Myotonic Dystrophy.

This was only possible due to the untiring efforts of PhD candidates NalakaWijekoon and Lakmal Gonawala, and a team of clinical doctors including Dr. Pulasthi Dissanayake. The success story and the roadmap of establishing a cost-effective molecular diagnostic facility in a developing country by Prof. Ranil de Silva and his team has been accepted for publication as a benchmark article in the reputed journal, Lancet Neurology which ranks first among clinical neurology journals.

Further expanding the molecular diagnostic facility, clinics have been conducted by ICIBN around the country for better availability and the convenience of the inherited disease community, bestowing a unique barefoot service to the nation in collaboration with the consultant neurologists / paediatric neurologists Dr. Sunethra Senanayake (National Hospital Sri Lanka), Dr. Darshana Sirisena (Colombo North Teaching Hospital, Ragama), Dr. Pyara Ratnayake (Lady Ridgeway Hospital for Children), Dr. Harsha Gunasekara (Sri Jayewardenepura General Hospital), Dr. Janaka Peries, Dr. Sanjaya Fernando (Teaching Hospital, Kurunegala, Dr. Athula Dissanayake (Karapitiya Teaching Hospital) and Dr. Ajantha Keshavaraj (Jaffna Teaching Hospital).

Molecular genetic testing is the ‘gold standard’ in the diagnosis of genetic diseases. Though most genetic neurological disorders are currently minimally responsive to existing treatments, they are potential candidates for Gene Therapy - an approach that can correct genetic abnormalities.

Gene therapy may revolutionize the treatment of neurological disorders in the coming decades, but there are still great challenges ahead to overcome. The treatment of genetic disorders - Gene-Based Therapy ­– is an ongoing battle, with clinical trials having been completed, some others ongoing, or those that have been approved worldwide.

Prof. de Silva has already established a rapport with scientists in Europe, USA and Japan who work on personalized drug development for neurogenetic diseases, where Sri Lanka has been successfully identified as a potential study site for Phase- 03 clinical trials providing a silver lining for the patients in the developing world. Moreover, the proteomics and metabolomics analysis results of the study will help in identifying prospective serum biomarkers to further collaboratively validate as surrogate end-points. Such surrogate endpoints will facilitate investigation of Sri Lankan natural products (cinnamon) based potential synergistic drug targets and disease modifying pathways.

Genetic testing and research has been funded solely by grants of Prof. de Silva through national (USJ, WHO) and international organizations. “Despite the national significance in genetic testing for the management of neurogenetic diseases, there has been little or no patronage from the state. We are looking forward to both, the state involvement and donations from philanthropists to support this worthy cause,” Prof. De Silva said.