ALS

Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s Disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost. As voluntary muscle action is progressively affected, patients in the later stages of the disease may become totally paralyzed. The progressive degeneration of the motor neurons in ALS eventually leads to death. Most people who develop ALS are between the ages of 40 and 70, but victims of ALS can be as young as 20 or 30.

Neuraltus is working on the development of NP001, a small molecule compound aimed at slowing or even stopping the progression of ALS. The drug functions by attacking a novel disease target and is a promising molecule for the treatment of ALS.

Dyskinesias in Parkinson’s Disease

Parkinson’s disease occurs when the neurons in the brain that produce dopamine die or become impaired. Dopamine is responsible for smooth, coordinated function of the body’s muscles and movement. Therefore, drugs that replace or simulate dopamine activity have become the mainstay of Parkinson’s disease treatment regimens. One of these drugs is levodopa. Although it is the gold standard for the treatment of motor symptoms in Parkinson’s disease, levodopa therapy often causes dyskinesias. Dyskinesias are tremulous, involuntary movements and are often as severe as the symptoms of Parkinson’s disease itself. Currently, there is no effective therapy for the dyskinesias caused by the drugs taken by most Parkinson’s patients. Working with the Parkinson’s Institute, Neuraltus is developing NP002, a small molecule compound, to reduce these dyskinesias in Parkinson’s patients. The treatment of dyskinesias will enable Parkinson’s patients to benefit from the effectiveness of Parkinson’s therapies. In October 2009, Neuraltus began a Phase 1/Phase 2 clinical trial to treat dyskinesias in Parkinson’s patients and is currently enrolling patients. Click here for more details about the study, or to read a description of the study on the National Institutes of Health website, go to clinicaltrials.gov. If you’d like to contact Neuraltus about the study, please send an email to clintrials@neuraltus.com.

Lysosomal Storage Disorders

Lysosomal storage disorders result from a specific enzyme deficiency in the body, caused by a genetic mutation received from both parents. Individuals suffering from a lysosomal storage disorder have a build-up of unwanted substances within the cells. This can lead to any of approximately 40 known lysosomal storage disorders, including Tay-Sachs, Gaucher’s, and Fabry’s diseases. Neuraltus is collaborating with the Hospital for Sick Children in Toronto, Canada to develop NP003, a small molecule therapeutic to treat lysosomal storage disorders.