Descripción del proyecto
Lucha contra la enfermedad de Alzheimer utilizando el potencial inmunoterapéutico natural de las células encefálicas
La enfermedad de Alzheimer la causan múltiples factores perjudiciales que provocan el deterioro cognitivo. En la actualidad, no existe ningún tratamiento efectivo para esta enfermedad, ya que tratar un único factor no pararía o revertiría la enfermedad, como han evidenciado los muchos fracasos experimentados en ensayos clínicos recientes. El proyecto ADAMtx, financiado con fondos europeos, pretende desarrollar un enfoque novedoso para un tratamiento integral. Anteriormente, los miembros del proyecto habían descubierto unas células microgliales del encéfalo (de microglia asociada con la enfermedad), que combaten específicamente enfermedades neurodegenerativas. En el proyecto actual, los investigadores desarrollarán varias dianas diferentes que simularán la actividad en esas células microgliales. El objetivo final del equipo es establecer la propiedad intelectual y asumir el desarrollo de las dianas gracias a la participación de capitalistas de riesgo y la creación de una empresa naciente.
Objetivo
Alzheimer's disease (AD) is a heterogeneous disease in which multiple detrimental factors contribute to cognitive loss and disease escalation. Currently there are no effective therapies for AD. Targeting any single symptom of disease-escalating factor (e.g. amyloid beta, tau, neuroinflammation etc.), even if successful, is not sufficient to modify the disease, as seen in the multiple failures of recent phase-III clinical trials. Thus, there is a desperate need for new approaches for development of AD therapeutics, which will be more comprehensive and not etiology-specific. Using single cell genomic analysis of the immune system in AD mouse models, we discovered a novel microglia type, disease associated microglia (DAM), intrinsic immune cells of the brain that fight AD and neurodegenerative disease. There are several revolutionary aspects to our approach to modify AD course. Fundamentally, based on our unique DAM pathways and target discovery platform we will develop novel AD-immunotherapy for boosting the brain’s innate neuroprotective mechanisms that fight neurodegeneration in AD. Development of targets that boost DAM cells is a major activity of this PoC plan, and we are in different phases of development of several targets that increase DAM activity including advanced stages of the targets Trem2 and P2ry12. The first goal of this PoC grant is to develop and strengthen our IP around AD immunotherapy targets. The second goal is to design a viable and scalable business model with venture capital and establish a startup (ADAMtheraputics) that will translate our novel technology for effective AD-immunotherapy for Alzheimer patients. We believe that our unique approach of targeting the brain’s intrinsic protective immune cells, to boost their activity and numbers, will dramatically impact AD therapy.
Ámbito científico
Programa(s)
Régimen de financiación
ERC-POC-LS - ERC Proof of Concept Lump Sum PilotInstitución de acogida
7610001 Rehovot
Israel