Abstract: 70
Type: Oral
Progressive multiple sclerosis (encompassing both PPMS and SPMS) is characterized by the continued and irreversible accumulation of neurologic disability. Recent findings suggest that innate immune cells in the CNS play a predominant role in disease progression during progressive MS (PMS). However, therapeutic drugs for modulating the activity of astrocytes and other CNS innate immune cells in PMS are not available. Our central hypothesis is that targeting the innate immune response in the CNS will provide efficacious therapeutic approaches for PMS. Based on this hypothesis, we developed a multi-tiered approach supported by innovative tools and methods, unique compound collections and patient samples to identify mechanisms of disease pathogenesis, therapeutic targets and drug candidates for PMS. This approach will combine targeted (Aim 1) and unbiased (Aim 2) screens of FDA approved compounds to identify therapeutic drugs that modulate the CNS innate immune response and neurodegeneration in PMS. In parallel, we will define the molecular mechanisms that control the CNS innate immune response in PMS, and evaluate the effects of candidate therapeutic drugs on these mechanisms (Aim 3). In summary, these studies use a multidisciplinary approach to define the mechanisms that control the CNS innate immune response and its dysregulation in PMS, as well as candidate therapeutic targets and drugs. These studies will also provide valuable knowledge and research tools relevant to other inflammatory and degenerative disorders affecting the CNS.
Disclosure: Francisco J. Quintana: Nothing to disclose
Abstract: 70
Type: Oral
Progressive multiple sclerosis (encompassing both PPMS and SPMS) is characterized by the continued and irreversible accumulation of neurologic disability. Recent findings suggest that innate immune cells in the CNS play a predominant role in disease progression during progressive MS (PMS). However, therapeutic drugs for modulating the activity of astrocytes and other CNS innate immune cells in PMS are not available. Our central hypothesis is that targeting the innate immune response in the CNS will provide efficacious therapeutic approaches for PMS. Based on this hypothesis, we developed a multi-tiered approach supported by innovative tools and methods, unique compound collections and patient samples to identify mechanisms of disease pathogenesis, therapeutic targets and drug candidates for PMS. This approach will combine targeted (Aim 1) and unbiased (Aim 2) screens of FDA approved compounds to identify therapeutic drugs that modulate the CNS innate immune response and neurodegeneration in PMS. In parallel, we will define the molecular mechanisms that control the CNS innate immune response in PMS, and evaluate the effects of candidate therapeutic drugs on these mechanisms (Aim 3). In summary, these studies use a multidisciplinary approach to define the mechanisms that control the CNS innate immune response and its dysregulation in PMS, as well as candidate therapeutic targets and drugs. These studies will also provide valuable knowledge and research tools relevant to other inflammatory and degenerative disorders affecting the CNS.
Disclosure: Francisco J. Quintana: Nothing to disclose