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Desarrollo de Fármacos

Kim G Johnson1, Michael Kaplitt2, Stephen Kaminsky2

  • 1Duke University School of Medicine, Durham, NC, USA.

Alzheimer's & dementia : the journal of the Alzheimer's Association
|December 25, 2025
PubMed
Resumen
Este resumen es generado por máquina.

La terapia génica LX1001 muestra resultados prometedores para homocigotos APOE4 con enfermedad de Alzheimer. El tratamiento fue seguro y bien tolerado, reduciendo biomarcadores clave de Alzheimer.

Palabras clave:
terapia génicaAPOE4enfermedad de Alzheimerbiomarcadorestau

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Área de la Ciencia:

  • Neurociencia
  • Genética
  • Farmacología

Sus antecedentes:

  • Los homocigotos APOE4 experimentan un declive cognitivo acelerado en la enfermedad de Alzheimer (EA).
  • APOE2 es una variante protectora asociada con un menor riesgo de EA y un declive más lento.
  • LX1001 es una terapia génica experimental que utiliza AAVrh.10hAPOE2 para administrar el gen APOE2.

Objetivo del estudio:

  • Evaluar la seguridad y tolerabilidad de LX1001 en homocigotos APOE4 con EA.
  • Evaluar la capacidad de la terapia génica para convertir el genotipo APOE4/4 a APOE2/4 en el sistema nervioso central.

Principales métodos:

  • Estudio de escalada de dosis de Fase 1/2 (NCT03634007) en cuatro cohortes de dosis única ascendente.
  • LX1001 administrado en el líquido cefalorraquídeo (LCR) en la unión craneocervical.
  • Participantes: homocigotos APOE4 (≥50 años) con deterioro cognitivo leve a demencia moderada, PET amiloide positivo y biomarcadores de LCR de EA consistentes.

Principales resultados:

  • Quince participantes recibieron la dosis; 50% con deterioro cognitivo leve, 14% con demencia leve, 36% con demencia moderada.
  • LX1001 fue generalmente seguro y bien tolerado; no se observaron anomalías de imagen relacionadas con amiloide.
  • Expresión de APOE2 dependiente de la dosis en el LCR; estabilización en el LCR Aβ42/40 y PET amiloide; reducción en el LCR t-tau, p-tau y PET tau.

Conclusiones:

  • LX1001 es la primera terapia génica investigada para homocigotos APOE4 con EA.
  • Los datos sugieren que LX1001 es seguro y bien tolerado.
  • LX1001 demostró una reducción en los biomarcadores de tau en LCR y PET tau, correlacionada con el declive cognitivo.