Yusuf Isah An-Nuphawi
Healthy cells and genes are fundamental to wellbeing of every living thing. Genetic dysfunctions, unlike other diseases especially in humans, requires a therapy that could go into the basic level of a living system, that is, molecular level, to correct such abnormalities in the gene-a biological code that determines physical and functional features of a living.
Gene therapy, curing genetic disorders like Sickle Cell Anaemia by changing a person’s genes, has been evolving for decades. However, despite the best efforts of researchers around the world, gene therapy has seen only limited success, the conditions are rather managed not cured. Then, the question is, why genetic diseases are difficult to cure? According to medical experts, this question borders on biological and economic factors.
On the one hand, it is very difficult to introduce new genes, for therapeutic objectives, into cells of the body and keep them working because of the body’s natural surveillance system called Immune system that can unwelcome any foreign substance. On the other hand, can any capitalist or government profit from developing a gene therapy to treat a rare disorder? Developing a new therapy is very expensive with a limited number of patients to recover those expenses from. In reality, developers may never earn money from treating such rare genetic disorders and some patients may never be able to afford them.
In the recent time, alternative management strategies are evolving and designed to improve particular symptoms associated with the disorders. For example, a genetic disorder associated with a heart defect might be treated with a heart transplant. Conditions that are characterized by defective blood cell formation, such as sickle cell disease, can now be treated with a bone marrow transplant. Bone marrow transplantation can allow the formation of normal blood cells and, if done early in life, may help prevent episodes of pain and other future complications.
Sickle Cell Anaemia patients, all over the world, are recently reassured of permanent cure as doctors at the University of Illinois, Chicago, USA, University of Loyola, Chicago in partnership with the University of Ibadan claimed to have discovered a permanent cure for Sickle Cell Anaemia. The researchers, Professor of Medicine, Victor Gordeuk, who is the Director, Sickle Cell Centre, University of Illinois, Chicago, USA and his colleagues, Prof. Damiano Rondelli and Prof. Bamidele Tayo, University of Loyola, Chicago claimed the cure is bone marrow transplant.
Corroborating the claim, the Chief Medical Director, UCH, Prof. Temitope Alonge, explained that the stem cell transplant is a standard procedure for the treatment of not only sickle cell anemia but many blood cancers in both adult and children.
His words: “With this chemotherapy-free transplant, we are curing adults with sickle cell disease, and we see that their quality of life improves fast within just one month of the transplant”.
“About 90 per cent of the approximately 450 patients who have received stem cell transplants for sickle cell disease have been children. Chemotherapy has been considered too risky for adult patients, who are often more weakened than children by the disease” he said.
He added that “Adults with sickle cell disease can now be cured without chemotherapy — the main barrier that has stood in the way for them for so long. Our data provide more support that this therapy is safe and effective and prevents patients from living shortened lives, condemned to pain and progressive complications.”
On risks that are involved in the procedure, Professor Alonge explained that: “In the new procedure, patients receive immuno-suppressive drugs just before the transplant, along with a very low dose of total body irradiation, a treatment much less harsh and with fewer potentially serious side effects than chemotherapy”.
“Donor cells from a healthy and tissue-matched sibling are transfused into the patient. Stem cells from the donor produce healthy new blood cells in the patient, eventually in sufficient quantity to eliminate symptoms. In many cases, sickle cells can no longer be detected. Patients must continue to take immunosuppressant drugs for at least a year” he explained.
According to a medical statistic, over five million Nigerians suffer from Sickle Cell Anaemia, a severe hereditary form of anaemia in which a mutated form of haemoglobin distorts the red blood cells into a crescent shape at low oxygen levels.
