Hope for Thalassaemics

In the NST,

Hope for thalassaemic patients Ranjeetha Pakiam and Farish Shahrir KUALA LUMPUR, May 12: The painful injection that thalassaemic patient Mohd Zanel Ibrahim, 18, endures daily will become a thing of the past soon. Advances in the medical field have brought substitute medication in the form of pills and syrup which can be taken orally. A new drug called ICL670 removes excess iron from the thalassaemic patient's body caused by frequent blood transfusions. The transfusions are needed since those suffering from the genetic disease experience rapid break-up of red blood cells, leading to anaemia (low haemoglobin levels). This drug, so far, is said to have no side-effects. ................

This is a heartening advance. Thalassaemics especially those of the beta-type, usually require repeated blood transfusions. This will increase their iron level in their body over time leading to a condition termed haemasiderosis. This can affect the heart, nerves and endocrine tissues. Most thalassaemics had a short life span due to this unwanted accumulation of iron.

Then iron chelation with desferoxamine was found. Iron chelation means binding to excess iron for excretion from the body. However, the patients were inconvenienced by the fact that this drug could only be given by a intramuscular route. Thus the quest for an oral drug began.

There is currently another oral agent, desferiprone. It however is far less effective than desferoxamine and had potential suppressive effects on white blood cells. This latest oral drug of ICL 670 is so far been promising. In actual fact at a dose of 20mg/kg/day, it surpasses the chelating effects of desferoxamine. It is also a daily dosing drug thus it can be taken with ease. There has so far been no reported adverse side effects.

Despite still in Phase III trials, such promise augurs well for the thalassaamic community. Malaysia has a high number of thalassaemia patients and those with traits. One way to fight thalassaemia is effective family counselling. Partners who both have thalassaemia traits should be advised to not get married. Thus we can hopefully decrease the prevalence of this faulty gene.

We are still waiting for the day that gene therapy can cure all genetic diseases. We are still a long way from it but current research is on-going. Due to a high number of thalassaemic patients here, it would be wise for the government to invest more money in research. Pharmaceutical companies can also play a role in establishing a research center here in Malaysia!

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