We are at the forefront of research focusing on development of therapies for diseases of the central nervous system (CNS).
Our research on therapies is currently focused on developing treatment options for:
Neurofibromatosis Type 1
NF1 is part of a group of conditions known as neurocutaneous disorders that affect the skin and the nervous system. NF1 causes tumor growth along nerves in the skin, brain, near the spinal cord and other parts of the body. These tumors are usually benign (non-cancerous), however they cause a range of symptoms with varying severity among affected people. People with NF1 typically have problems with their bones, eyes and nervous system, as well as other complications, including high blood pressure, learning disabilities, attention deficit hyperactivity disorder (ADHD), seizures and speech problems. Further, some people with NF1 develop cancerous tumors that grow along nerves and are at a higher risk of developing other forms of cancer. NF1 accounts for approximately 90% of all neurofibromatosis cases and occurs in about 1 in 3,000 births. There is no known cure for NF1 and treatment options vary. Selumetinib (KoselugoTM), a MEK inhibitor marketed by Alexion Pharmaceuticals, Inc., an AstraZeneca group of companies, is the only FDA-approved prescription medicine used to treat children 2 years of age and older with NF1 who have plexiform neurofibromas that cannot be completely removed by surgery.
Amyotrophic Lateral Sclerosis (ALS)
ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, causing loss of muscle control. It most commonly affects people between the ages of 40 and 70, with an average age of 55 at the time of diagnosis. The average life expectancy after diagnosis is two to five years, but some patients may live for years or even decades. Currently there is no known cure or treatment that halts or reverses the progression of ALS, and FDA only approved 2 medications so far for the treatment of this disorder, both shown to modestly slow the progression of ALS.
Multiple Sclerosis (MS)
Multiple sclerosis is a chronic and potentially disabling autoimmune disease, and the most common neurodegenerative disease of the central nervous system in young adults. The pathological hallmark of MS is the formation of demyelinating lesions in the brain and spinal cord, with the immune system attacking the myelin sheath that normally protects nerve fibers in the brain. There are now 2.8 million people worldwide who have MS, and every five minutes, someone, somewhere in the world is diagnosed with this disorder.
Schizophrenia affects 1 in 100 people and is a chronic and disabling psychiatric illness. It is amongst the top ten causes of disease burden in working adults, and patients typically die 20 years prematurely. Antipsychotic drugs have been the cornerstone and first-line treatment for schizophrenia over the last 60 years. However, they are ineffective or not tolerated in three-quarters of patients and, even where they are effective they have limited efficacy for the negative and cognitive symptoms, and are not disease modifying.