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New sickle cell treatment ready for testing

2009-12-03 www.scidev.net A +

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.

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