AMRF Investigational Programs

Protein ubiquitination, similar to protein phosphorylation, regulates many of the intracellular processes that are critical for the survival of malignant cells. AMRF believes that a deeper understanding of protein ubiquitination will lead to new therapies for malignancies, immunological and inflammatory diseases, and degenerative diseases of the nervous system.

Basic research has shown that within specific cancer types such as breast cancer or lung cancer, many subtypes exist that require distinctive therapeutic approaches. The AMRF Ovarian Cancer Research program believes that we must learn more about the various types of genetic, epigenetic, and molecular alterations that have occurred in cancerous tissues, and translate the most promising scientific and therapeutic leads into near-term clinical trials that will improve outcomes for ovarian cancer (OvCa) patients.

Melanoma therapy has undergone a revolution during the past 10 years, consisting of breakthroughs in both targeted and immune therapies. The AMRF Melanoma investigators have contributed importantly to this revolution and are building on these successes to identify mechanisms of resistance to these treatments, elucidate strategies to overcome that resistance, discover tests that predict treatment efficacy, and understand/minimize treatment side effects in order to optimize patient quality of life and overall survival.

Our immune system has evolved to protect us from diseases; however, multiple myeloma is a cancer that starts in the immune system and can be life threatening. The AMRF Multiple Myeloma Program aims to identify early changes in the immune system that lead to myeloma and develop treatment strategies to prevent the lethality of the disease. Understanding, diagnosing and treating the disease through scientific discovery that can translate into clinical practice is the core organizing principle of the myeloma program.

The limited recovery in brain injury and disease means that stroke and traumatic brain injury, and their attendant loss of neurons, myelin and white matter, are leading causes of adult neurological disability. Mechanisms of circuit reorganization and recovery after brain injury exist, and these might serve as targets for the development of new therapies to further improve recovery. The AMRF Neural Repair in Brain Injury and Disease program focuses on identifying mechanisms of repair and cell replacement after stroke and other injuries, and to use that understanding to develop new treatment strategies for disorders of brain cell injury and loss.

There are currently no therapies that promote functional recovery after peripheral nerve injury or that maintain nerve function in peripheral neuropathies. The AMRF Peripheral Neuropathy and PNS Nerve Regeneration group is working to identify mechanisms of axon growth and survival, remyelination, and axoglial interactions. Our goal is to discover mechanisms that promote regeneration and remyelination, that protect axons from damage and degeneration, to validate these as viable therapeutic targets, and then translate these discoveries into new treatments.

The inability of the injured spinal cord or brain to repair themselves and restore function after injury has been recognized for millennia. The AMRF Spinal Cord Injury and CNS Nerve Regeneration collaboration focuses on identifying cellular interactions and mechanisms that control the re-growth and targeting of nerve fibers after injury to the spinal cord or brain. Understanding these interactions and mechanisms will allow us to develop drugs, genetic manipulations and cell therapies to improve repair, leading to a new generation of therapies for spinal cord and brain injuries and other neurological disorders.

Despite advances in treating acute multiple sclerosis (MS), we lack treatments for critical aspects of MS that contribute to clinical progression. Stopping progressive MS requires a precise understanding of how the relationship between nerve cells and the cells that make myelin is disrupted. The AMRF MS group focuses on dynamic analysis of MS and its animal models, defining pathways used for communication between nerve cells and myelin, interrogating interactions between the nervous and immune systems, and identifying therapeutic approaches to enhance repair and forestall, if not reverse, progressive MS.