Methods to treat neurodegenerative diseases
AssigneesThe General Hospital Corporation, Children's Medical Center Corporation, President and Fellows of Harvard College
InventorsClifford J. Woolf, Brian Wainger, Evangelos Kiskinis, Kevin Eggan
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Abstract
The present invention is based on the discovery that motor neurons derived from patients with a neurodegenerative disease have decreased delayed rectifier potassium current and increased persistent sodium current compared to motor neurons derived from control healthy individuals. The present invention is also based on the discovery that the class of compounds known as “potassium channel openers” can be used to treat neurodegenerative diseases, including ALS, Parkinson's disease, Alzheimer's disease, epilepsy, and pain.
Claims17
A method of diagnosing a subject with amyotrophic lateral sclerosis (ALS), the method comprising:
- providing a motor neuron from the subject;
- determining a level of activity of the motor neuron;
- comparing the level of activity of the motor neuron with a reference level of activity of a motor neuron;
- and
- diagnosing the subject as having ALS if the level of activity of the motor neuron is higher than the reference level of activity.
A method of treating, or reducing a risk of developing, ALS in a subject, the method comprising:
- providing a motor neuron from a subject;
- determining a level of activity of the motor neuron;
- comparing the level of activity of the motor neuron with a reference level of activity of a motor neuron;
- identifying the subject as having, or at increased risk of developing, ALS if the level of activity of the motor neuron is higher than the reference level of activity;
- and
- administering to the subject identified as having or at increased risk of developing ALS a therapeutically effective amount of a potassium channel opener, thereby treating or reducing the risk of developing ALS in the subject.
A method of identifying a candidate compound to treat ALS, the method comprising:
- providing a motor neuron from a subject with ALS or a motor neuron derived from a subject with ALS;
- contacting the motor neuron with a test compound;
- determining a level of activity of the motor neuron;
- comparing the level of activity of the motor neuron in the presence of the test compound with a level of activity of the motor neuron in the absence of the test compound;
- and
- selecting the test compound as a candidate compound if there is a lower level of activity of the motor neuron in the presence of the test compound than in its absence.
Dependencies are inferred from the claim text.