L-glutamineplumpsupsickle-shapedbloodcellsandpreventsbloodvesselblockage,saytheresearchers
USNationalHumanGenomeResearchInstitute
AtreatmentthatreducesthenumberofbloodcellsdeformedbysicklecellanaemiahaspassedearlytestsinpeopleandwillsoonbeginphaseIIIclinicaltrials.
Thetrials,whichwilllookatsafetyandefficacyinalargenumberofpeople,wereapprovedinAprilbytheUSFoodandDrugAdministration(FDA)andshouldbegininSeptember.
Sicklecellanaemiaisahereditaryblooddisorderforwhichthereisnoknowncure.Theexperimentaltreatmentisapillformofthenaturally-occurringaminoacidL-glutamine,whichplumpsupredbloodcellsdeformedbythediseasemakingthemlesslikelytoblocksmallbloodvesselsandcausedebilitatingpain,saysYutakaNiihara,principalinvestigatorattheLosAngelesBiomedicalResearchInstitute(LABioMed),wherethetreatmentisbeingdeveloped.
"InourphaseIIclinicaltrialweobservedanexcellentsafetyprofileandpositivetrendsinreducingthenumberofcrisesandhospitalisationsinsicklecelldiseasepatients,"saidNiiharainapressrelease.
FewothertreatmentsforthisdisorderhaveprogressedtoPhaseIII.
Butthepriceofthedrugislikelytobeanissueindevelopingcountrieswheremedicationismostneeded.ThecostofusingL-glutaminetotreatanotherdisease—shortbowelsyndrome—isaroundUS$600permonthperpatientandNiiharaexpectsinitialcoststobesimilarforsicklecellanaemia.
ButheiskeenforL-glutaminetobemadeaffordableindevelopingcountries—particularlyinAfricawherethediseaseismostprevalent.
"ForAfricawewouldliketoplansomethingviaanon-profitorganisationsoitwillbemoreeasilyavailabletopeoplewithoutresources,"NiiharatoldSciDev.Net.L-glutamineisalreadysoldcommerciallyinmanypharmaciesinthedevelopedworldanditisoftensoldasasportssupplement.IntheLABioMedtrials,however,patientsarebeinggivenamoreconcentrated,pharmaceutical–leveldoseoftheaminoacid.
JosephOjobi,aconsultantattheJosUniversityTeachingHospitalinNigeria,saystheresearchsuggeststhatsicklecellpatientswhoswitchtoaprotein-richdietmightalsobeabletoalleviatesomeoftheirsymptomsasproteinisagoodsourceofL-glutamine.
Buthewarnsthatdietarychangescannotalterthemutatedgenethatcausessicklecellanaemiaandmightnotalleviateallthesymptoms.