Neurodegenerative therapy: Vector(s) containing an inducible gene encoding a cell cycle regulator useful for treating neurodegenerative disorders.
The present invention provides a vector containing a gene useful for treating neurodegenerative disorders, especially Alzheimer’s disease. These vectors can be used in therapeutic applications to prevent neurodegenerative disorders or to slow down their progression with therapeutic efficacy.
The Medical Problem
In our aging society, a still growing number of patients suffer from age-associated CNS-disorders. Neurodegenerative disorders, namely Alzheimer’s disease (AD) and stroke giving rise to vascular dementia (VD) are most prominent. By 2025 about one-third of the European population will be older than 65 years and, thus, be at high risk for AD, stroke and VD. Stroke is the most common neurologic disease and the leading cause of adult disability. Both morbidity and mortality of stroke sharply rise with increasing age. Next to stroke, AD is the second leading cause of adult disability, and represents the fastest growing area of unmet medical need.
AD and stroke share a cell death pathway that is activated by unscheduled neuronal cell cycle re-entry and subsequent partial replication of DNA. Moreover, the selective cell death of neurones which have replicated their DNA could be shown in AD recently. This directly proofs the link between cell cycle activation and cell death in neurons of the adult human central nervous system (CNS) and identifies the molecular control of cell cycle activation as therapeutic target.
- Therapeutic concept of neuroprotection by neuron-specific targeting of cell cycle
- Neuroprotective effects had been proven on in vivo models of ischemic cell death and excitotoxicity mimicking the effects ofAß.
- High therapeutic efficacy and minimal or no side-effects
- Anticipation to bring these therapeutic tools in a joined effort to a first clinical application within a framework of five years
- AD or stroke will be in the direct focus of the first therapeutic application
- Approach will also be applicable to other neurodegenerative disorders and non-neuronal disorders with unscheduled cellcycle activation, such as cancer or atherosclerosis
patent pending: EP2218784A1 and US200120004277A1