19 February 2018

Huge changes in the treatment of children with haemoglobin disorders over the next five to twenty years will help to extend life expectancy, a leading paediatric haematologist says.

Dr Rabbi Hanna, of Cleveland Clinic, USA, said clinical breakthroughs are likely, including advances in bone marrow transplant techniques, gene therapies, and improved medications. This would provide a significant boost to sickle cell patients, for example, whose average life expectancy is just 34 years, he said.

 “We really have to provide a cure, and the only cure available has been bone marrow transplantation, with the best outcome from donors who are matched family – brothers and sisters,” Dr Hanna said.

 “In the past few years there have been big steps forward in haplo-transplants, using family members that are only half-matched, particularly a mum or dad with 50% of the child’s genes. That will give more children the chance of a cure, replacing bone marrow that is defective with new bone marrow that is healthy.”

He said new treatments are particularly important in some Middle East countries, where rates of both sickle cell disease and, in particular, thalassaemia are higher than in Europe or North America.

One in every 12 people in the United Arab Emirates is a carrier for the gene that causes thalassaemia.

Dr Hanna says haploidentical transplant trials are now in phase two and three, testing for efficacy and side-effects, while gene therapy, using modified DNA to introduce a functional gene to replace the mutated gene causing the blood disorder, offers additional possibilities for a cure beyond bone marrow transplants.

 “Early phase one studies looking at this area of gene therapy for thalassaemia are very promising, although they are in the very early stages so it’s going to take time before we see them become available in a clinical setting,” he said.

 “There are also several medications that have recently been developed for the treatment of sickle cell disease and thalassemia – two have been approved by the FDA, and others are pending. These aren’t a cure, but they can make the disease less severe.”

Source: Wallis PR



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