Drépanocytose et thalassémie : un traitement révolutionnaire vient d’être autorisé!
L’Agence de réglementation des médicaments et des produits de santé (MHRA) au Royaume-Uni, vient d’approuver un traitement révolutionnaire contre la drépanocytose et la β-thalassémie : Casgevy.
Ce médicament innovant, le premier à utiliser la technologie CRISPR, récompensé par un prix Nobel en 2020, ouvre une nouvelle ère dans le traitement de ces maladies génétiques. Avec des résultats prometteurs dans les essais cliniques et une approche unique de modification génétique, Casgevy représente un espoir considérable pour améliorer la qualité de vie des patients.
La drépanocytose et la β-thalassémie sont causées par des erreurs génétiques dans la production de l’hémoglobine. Ces maladies sont particulièrement répandues chez les individus d’origine africaine, caribéenne, méditerranéenne, sud-asiatique, sud-est asiatique et moyen-orientale. La drépanocytose se manifeste par des crises de douleur intense, des infections graves et une anémie, tandis que la β-thalassémie entraîne une anémie sévère nécessitant des transfusions régulières.
Casgevy agit en modifiant le gène défectueux dans les cellules souches de la moelle osseuse du patient. Celles-ci sont prélevées, génétiquement modifiées, puis réinjectées au patient. Jusqu’à présent, la greffe de moelle osseuse était la seule solution pour ces patients, malgré son risque de rejet. Durant les essais, Casgevy a démontré sa capacité à restaurer la production d’hémoglobine normale et saine.
Dans l’essai pour la drépanocytose, 97 % des patients traités n’ont pas eu de crise de douleur sévère pendant au moins 12 mois. Pour la β-thalassémie, 93 % des patients n’ont pas eu besoin de transfusion sanguine pendant au moins un an après le traitement. Les effets secondaires étaient similaires à ceux des greffes de cellules souches autologues, incluant nausée et fatigue.
Source: Futurascience
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