Therapeutic Strategy
OUR FOCUS
Q Therapeutics is focused on restoring the normal activity of neurons by providing the support functions present in healthy central nervous system tissue.
Q-Cells® are adult progenitor cells, restricted in their ability to differentiate only into glia, the unsung heroes of the brain. While little was known about their function until recently, we now know that glial cells play an essential role in creating a healthy cellular environment for neurons and make up about 50% of the brain's mass. Our unique Q-Cells® approach is comprehensively protected by more than 20 issued patents with more pending.
Following our initial trials in transverse myelitis and ALS, we are hopeful that the restorative mechanism of Q-Cells® will be applicable to a wide range of central nervous system diseases, including demyelinating conditions such as multiple sclerosis, cerebral palsy and stroke. Other neurodegenerative diseases and injuries that our approach is potential applicable to include spinal cord injury, Huntington’s disease, traumatic brain injury, Parkinson’s disease and Alzheimer’s disease.
Current Targets
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Amyotrophic Lateral Sclerosis (ALS, or Lou Gehrig’s Disease)
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Transverse Myelitis
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Multiple Sclerosis
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Spinal Cord Injury
Future Targets
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Huntington’s Disease
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Parkinson’s Disease
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Alzheimer’s Disease
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Stroke
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Traumatic Brain Injury
UNMET NEED
Q Therapeutics is addressing the critical unmet need for treatments that target the underlying causes and mechanisms of currently incurable CNS diseases.
Every year, hundreds of thousands of people suffer from debilitating – and often fatal – diseases of the brain and spinal cord. Currently, the available treatments for most devastating neurodegenerative disorders target only symptoms, not the underlying causes of central nervous system (CNS) disease or injury.
By augmenting the population of healthy glia in the brain and spinal cord, Q-Cells® have the potential to modify the course of neurodegenerative and demyelinating diseases and injuries by rescuing damaged neurons or replacing diseased glial cells.
TARGET INDICATIONS
Our initial targets are orphan diseases, such as transverse myelitis and ALS, which benefit from an accelerated FDA approval process and market exclusivity.
Transverse Myelitis
Transverse myelitis is a rare disease caused by inflammation of one level of the spinal cord. Attacks of inflammation in transverse myelitis damage or destroy the myelin sheath around nerve cell fibers, likely by damaging the oligodendrocytes that produce myelin. An estimated 44,000 people in the U.S. are affected by transverse myelitis, for which no effective cure currently exists.
Amyotrophic Lateral Sclerosis (ALS, or Lou Gehrig’s Disease)
Amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease) is a neurodegenerative disease of the motor neurons in the brain and spinal cord that control muscle movement. The degeneration of motor neurons in ALS causes muscle spasticity, progressive weakness and difficulty with speaking, swallowing and breathing. ALS is the most common of the five known motor neuron diseases, affecting approximately 30,000 people in the U.S. The only approved treatment for ALS has minimal impact on patient survival.
Multiple Sclerosis
Multiple sclerosis (MS) is a currently incurable inflammatory disease of the central nervous systems that leads to the damage or destruction of myelin in the brain and spinal cord. While the cause of MS is not clear, the underlying mechanism of the disease is thought to be either autoimmune destruction or oligodendrocyte failure. In the U.S., approximately 400,000 people are living with MS and more than 10,000 new patients are diagnosed each year.
Spinal Cord Injury
Spinal cord injury is a broad term covering damage to any part of the spinal cord or the nerves at the end of the spinal canal. Spinal cord injuries often result in permanent alterations in strength, sensation and other body functions below the level of the injury. An estimated 12,000 people sustain severe spinal cord injuries each year, and there is currently no way to reverse the damage done to the spinal cord.
