New drug for sickle cell in final stage

Manama : A new hopefor Sickle Cell Disease (SCD) patients has been revealed with the global discovery of a medicine that is believed to permanently end their suffering.

Emmaus Life Sciences, an international biopharmaceutical company, recently announced that it’s in the process of making its L-glutamine treatment for SCD official, expecting it to be finalised during the ongoing international SCD Awareness Month (September).

The company stated that it has submitted its Non-Disclosure Agreement (NDA) to the Food and Drug Administration (FDA) in the United States.

According to Rare Disease Report news agency, the company’s NDA includes data of the company’s Phase 3 clinical trial that demonstrated a reduction in the frequency of sickle cell crises and hospitalisations. Besides it showed a reduction in cumulative days hospitalised and a lower incidence of the life-threatening acute chest syndrome.

“The clinical trial enrolled 230 adult and paediatric patients as young as five years of age, across 31 experienced sickle cell disease treatment centres in the US.  There were no major adverse events attributable to the treatment,” the agency reported.

Meanwhile, Emmaus Chairman and CEO Yutaka Niihara, in a televised interview, said: “It’s an amino acid that’s required by a normal person in fairly high doses. It’s a backbone of the protein and other antitoxins. We’re sure that it will decrease the damage to the hemoglobin, where SCD patients have problems. The damage to the red blood cells will also decrease, in the case of SCD.” 

“The significance of L-glutamine that  unlike other amino acids, it’s nature’s way to provide as much amino acid that the body needs without any side effects. The element has been tested and proven to reduce painful crisis of SCD patients and their stay in the hospital by 41 per cent,” he elaborated. 

However, an announcement is expected from the company in September, confirming the endorsement of the cure by international pharmaceutical authorities.

It’s believed that Bahrain holds one of the highest SCD percentages in the Middle East. According to the latest figures released by Bahrain Society for SCD Patients Care, 20 Bahrainis have died of the disease since January. 

In 2015, 31 patients succumbed to SCD. About 20 male and 11 female patients died last year.