[Current treatment for Gaucher's disease and new prospects].
Two new useful enzymes have recently undergone clinical trials for the treatment of Gaucher's disease (GD): velaglucerase alpha and taliglucerase alpha were both approved for early access programs as of the June 2009 shortage in Imiglucerase supply. Velaglucerase has been approved by both, Food and Drug Administration and European Medicines Agency. The Phase I/II trial of velaglucerase is in its 8th year: There were no drug-related serious adverse events or withdrawals, and no antibodies. Statistically significant improvements (p<0.004) were noted in mean percent change from baseline to nine months and baseline to 48 months for hemoglobin (19.2% and 21.7%, respectively), platelet counts (67.6% and 157.8%, respectively), normalized liver volume (<18.2% and <42.8%, respectively), and normalized spleen volume (<49.5% and <79.3%, respectively). Within 2 years of initiation of therapy, all patients achieved normalization of hemoglobin level, all but one patient achieved platelet counts of greater than 100×10(9)/L, all patients achieved near normalization in liver volumes, and all patients but one exhibited a reduction of more than 50% in spleen volume. At present, velaglucerase alpha is indicated in type 1 GD symptomatic patients (children or adults) and is accepted as an orphan drug by the EMA with similar cost to imiglucerase. Taliglucerasa alpha, obtained from transfected plant cell cultures, is pending to approval.
Grupo de Estudio de Enfermedad de Gaucher y Neoplasias Hematológicas, Servicio de Hematología, Hospital Universitario Miguel Servet, Centro de Investigación Biomédica en Red de Enfermedades Raras, Instituto Aragonés de Ciencias de la Salud, Zaragoza, Spain. giraldo.p@gmail.com
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Giraldo
Paz
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Latre
D056947:Enzyme Replacement Therapy / Q000379:methods*; D005776:Gaucher Disease / Q000188:drug therapy*; D005962:Glucosylceramidase / Q000627:therapeutic use*; D006801:Humans; D011994:Recombinant Proteins / Q000627:therapeutic use*
D011994:Recombinant Proteins; D005962:Glucosylceramidase; C550184:Velaglucerase alfa, human