Clinical Studies
Background
Progressive Multifocal Leukoencephalopathy (PML) is a deadly demyelinating disease of the central nervous system, which occurs in immunosuppressed individuals with AIDS, leukemia, or in organ transplant recipients. PML has also been reported in patients with inflammatory or auto-immune diseases treated with immunomodulatory medications. PML is caused by a reactivation of the polyomavirus JC (JCV), which destroys oligodendrocytes, the myelin-producing cells in the brain. JCV is a benign virus, which infects the majority of healthy individuals without causing any disease. The goal of these NIH-sponsored, IRB-approved studies is to characterize the immunological, virological and radiological prognostic factors of disease evolution. HIV-positive or HIV-negative immunosuppressed individuals will qualify for these studies if they have neurological symptoms and brain lesions suggestive of PML, or if they have a positive brain biopsy or detectable JCV DNA in their cerebrospinal fluid.
Study 1
- Title: Cellular localization and Immune Response to JCV
- Purpose: To determine precisely where in the body JCV is hiding and how the immune response prevents it from causing brain disease. One of the goals of this study is to characterize determinants of JCV latency and reactivation in the body.
Study 2
- Title: Role of Inflammation in PML
- Purpose: To determine precisely what are the host or viral factors that may predict a favorable outcome for PML patients and the role of inflammation in preventing JCV from causing brain disease. One of the goals of this study is to help establish non-invasive markers of PML evolution by studying the brain metabolism in PML lesions using Magnetic Resonance Imaging (MRI), Magnetic Spectroscopy (MRS) and other advanced imaging modalities.
Study 3
- Title: Translational Development of Dendritic cell-based immunotherapy for PML
- Purpose: To determine how to harness the patient’s own immune response to combat PML. One of the goal of this study is to boost the cellular immune response against JCV by administration of autologous dendritic cell vaccines to patients with PML Study 4. Title: PML treatment study with mefloquine Purpose: To determine the role of the anti-malaria drug mefloquine (Lariam) in the treatment of PML. The goal of this multicenter, international study is to determine whether mefloquine, which is active in the test tube in preventing JCV replication, is useful in improving the clinical outcome of patients with PML. Biogen Idec is the sponsor of this study.
If you are interested to refer a patient or if you have any questions about any of these studies, please feel free to contact Igor J. Koralnik, MD at 617-735-4460.
