Lead Inventor: Vincent P. Butler Jr., M.D. Problem or Unmet Need: Myelin is an insulating material coating many types of neurons which allows proper conductance of their electrical signals. Disturbance of this myelin sheath can result in a host of...

Lead Inventor: Vincent P. Butler Jr., M.D. Problem or Unmet Need: Myelin is an insulating material coating many types of neurons which allows proper conductance of their electrical signals. Disturbance of this myelin sheath can result in a host of cognitive and physical disabilities, including speech impairment and loss of dexterity. Improper myelination can be caused by both genetic factors, such as in leukodystrophies, or by physical damage to the neurons, as seen in compression injuries of the spinal cord. In order to improve the conduction of demyelinated neurons, researchers tested the effect of digoxin, also known as digitalis, a Na+/K+ ATPase pump inhibitor widely used for various heart conditions (Kaji, R. et al. (1990) Effect of digitalis on clinical symptoms and conduction variables in patients with multiple sclerosis. Ann. Neuro. 28(4): 582.). Inhibition of this pump leads to an increase in the concentration of sodium ions in the cell, lowering the resting potential of the neuron, making it easier to trigger an action potential. While several of the multiple sclerosis patients treated with digoxin did show signs of improved conductance, the effects were small, presumably due to the fact that digoxin does not cross the blood-brain barrier well and therefore could not act strongly on the demyelinated neurons. Details of the Invention: In order to gain the benefits of a Na+/K+ ATPase pump inhibitor at the site of injury, Dr. Vincent P. Butler Jr., M.D., prepared scillarenin from proscillarenin, an analog of digoxin which penetrates the blood-brain barrier. Dr. Butler then tested scillarenin in a mouse model of multiple sclerosis, where the mice had been induced to create auto-antibodies against myelin, resulting in demyelination and its classical neurological manifestations. Within two hours after dosage, administration of scillarenin was able to reverse paralysis of previously flaccid limbs. Additionally, a single dose of scillarenin continued to be effective for 6-8hrs after administration. This effect has been shown using both structural classes of these Na+/K+ ATPase inihibitors, collectively termed cardiac genins. Applications: • For treatment of demyelinating diseases including multiple sclerosis, compression damage to the spinal cord, peripheral neuropathy and congenital demyelinating disease • Management of periods of acute symptoms common in Multiple Sclerosis Advantages: • Cardiac genins cross the blood-brain barrier, exerting 20 times more neural effect without increasing the risk of cardiotoxicity associated with digoxin and its analogs Opportunities • Sponsored research support • Licensing Patent Status: Issued Patent (US 6,306,845)

Pat. Title: METHOD FOR TREATING DEMYELINATING DISEASE

Publication #:  

App. #: 09/240,935

Patent #: 6,306,845

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