Field of the Invention
The present invention relates to methods of providing therapy for nervous system conditions including, but not limited to: degenerative brain diseases, ganglioside storage disorders and inflammatory disorders.
Background
There are more than 600 known disorders that afflict the nervous system. Nervous system disorders have varied etiologies, but ultimately all have devastating effects on the individuals suffering from them. Nervous system disorders include degenerative conditions such as Parkinson's and Alzheimer's disease; inflammatory diseases such as Multiple sclerosis, spinal cord injury ischemia and stroke, psychiatric disorders such as schizophrenia, and lipid storage disorders such as Neimann-Pick-C, Tay Sachs, Batten, Sandhoff and Gaucher disease.
Neurological disorders strike an estimated 50 million Americans each year, exacting an incalculable personal toll and an annual economic cost of hundreds of billions of dollars in medical expenses and lost productivity.
The burden of neurological disease is a burden borne by every segment of society, and people everywhere. Indeed, individuals suffering from nervous system disorders often a require care 24 hours a day, seven days a week. In such cases family members from several households may need to partake in the care, and these caregivers usually have jobs, as well as their own families. Thus, the consequences of neurological disease have far reaching consequences.
Neurological disorders may be manifest at any stage of life. Some neurological diseases typically exhibit adult onset. These include Alzheimer's Disease, Amyotrophic Lateral Sclerosis, Huntington's Disease, Multiple Sclerosis, Parkinson's Disease, Stroke, and traumatic brain injury. Adult onset neurological disease accounts for between 13.3 and 16.1 million cases of neurological disorders in the United States. Unfortunately, many of these diseases and disorders are progressive, so that the burden of care for the individual increases with time.
Other neurological diseases affect infants and children. Indeed, glycosphingolipid storage diseases are a common cause of pediatric neurodegenerative diseases. Although the frequency of individual disorders is not high, together they are a significant group of disorders with a collective frequency of 1 in 18,000 live births.
Unfortunately, neurologic disease is seldom curable. This is true irrespective of the age at which the neurological disease is manifest. Thus, strategies for the treatment of these debilitating and often fatal diseases often focus primarily on pallative measures. Attempts at curing neurological disease have also been proposed. These treatments have included enzyme replacement therapy, gene therapy, and allogenic bone marrow transplantation. Unfortunately however, the treatments typically do not improve the condition nor alter the ultimate outcome of the disease. Thus, before this invention, symptomatic management was often the only approach available for treating most of these disorders.
The present inventors have determined for the first time that the use of neuroactive steroids is effective for the treatment of degenerative brain diseases and central nervous system disorders.
Managing the neuropathologic processes that contribute to the morbidity of neurologic diseases has proven to be a challenging task, since as noted above, there are many factors that can cause, perpetuate, or exacerbate neurological conditions involving the central nervous system. There has been no evidence prior to this invention that neuroactive steroids would provide effective therapy for the treatment of a wide range of neurological diseases, including congenital lipid storage diseases, inflammatory diseases, and chronic degenerative brain disease. The present invention therefore fulfills the need for an effective method of treating neurological disorders by providing methods of administering neuroactive steroids to a subject.