New thalassaemia treatment gives hope

PAIN and inconvenience are often associated with treatment for thalassaemia, a genetic disorder that affects the body’s production of haemoglobin – the oxygen-carrying protein in red blood cells. This inherited disorder can cause severe anaemia and can be fatal without ongoing treatment.
With no cure, the only options for thalassaemia patients are regular blood transfusions and nightly injections through a slow infusion pump. These infusions, lasting up to 12 hours a day, five to seven days a week, limit social activities and interfere with patients’ intimate relationships.
Desferal infusions therapy, as it’s known, is also difficult for children who dislike needles and fear pain, says Professor Dr Chan Lee Lee, paediatric haematologist/oncologist at the University Malaya Medical Centre. Even older teens and young adults find these infusions extremely burdensome, he adds, and often will not comply with therapy even though they see first-hand the risks of not doing so.
But this painful procedure could soon be a thing of the past as Malaysian thalassaemia patients and their caregivers are looking forward to a clinical trial which offers an oral treatment for this condition.
Dr Chan is among three Malaysian investigators who will spearhead the trials to determine the safety and tolerability of the world’s first and only once-a-day iron chelator, ICL 670 (deferasirox).
The others include Dr Hishamshah Ibrahim, senior consultant in the paediatric department of the Kuala Lumpur Hospital and Professor Rahman Jamal, director of molecular biology at Universiti Kebangsaan Malaysia.
An easy-to-administer oral iron chelator in tablet form, ICL 670 is taken once daily after dissolving the tablet in a glass of water.
The drug was developed by pharmaceutical giant Novartis to extend the benefits of iron chelation to a greater number of patients receiving blood transfusions, and to address the needs of thousands of adult and paediatric patients who have been using Desferal (deferoxamine).
The three-centre, one-year study will cover 100 Malaysian patients. Besides Malaysia, trials are also being carried out in 12 countries across five continents ranging from the USA, Germany, Greece and Italy, to China, Hong Kong, Thailand and Taiwan.
The preliminary results of studies on paediatric patients treated with the oral chelator presented at the latest annual meeting of the American Society of Haematology, have been very promising, especially in terms of safety and tolerability, says Dr Chan.
“Oral therapy offers welcome relief and the Malaysian thalassaemic community and their professional caregivers are looking forward to the trial,” says Dr Chan.
ICL 670 would also redefine the clinical management of patients frustrated by years of inconvenience, bruising and scarring as a result of daily insertion of the deferoxamine infusion needle, says Dr Hishamshah.
“Their quality of life will drastically improve and this should impact positively on the overall treatment regime,” he says.
The Government, has also announced its intention to invest approximately RM40 million annually in the Thalassaemia Control and Prevention Programme, he adds, a move will which directly benefit the few thousand thalassaemia patients nationwide and the estimated 600,000 to one million Malaysians who are carriers of the thalassaemia gene.
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