Inhibition of erythrocyte sickling by...

A - Human Necessities – 61 – K

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A61K 38/05 (2006.01) A61P 7/00 (2006.01)

Patent

CA 2345243

It has now been found that N-L-alpha-aspartyl-L-phenylalanine 1-methyl ester (APM) exhibits antisickling properties. In vitro testing verified that APM significantly lowered the frequency of sickling of red blood cells from each of twelve pediatric aged patients being treated for sickle-cell anemia by exchange transfusion. Sickling was also inhibited in an "index" patient after oral administration of APM. These in vitro and in vivo results identify APM as a therapeutic agent for the family of sickle cell molecular diseases.

On a découvert que le N-L-alpha-aspartyl-L-phénylalanine 1-méthyl ester (APM) présente des propriétés d'inhibition de la falciformation. Les épreuves in vitro ont permis de confirmer que APM abaissait de façon considérable la fréquence de la falciformation des globules rouges prélevés sur chacun des douze patients en âge pédiatrique suivant un traitement contre la drépanocytose par transfusion d'échange. La falciformation est également inhibée chez un patient témoin après administration orale de APM. Ces résultats in vitro et in vivo ont permis d'identifier APM comme un agent thérapeutique pour la famille des maladies moléculaires à hématies falciformes.

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