Neurodevelopmental Disorders - Acting early can prevent much longterm healthcare costs of care for families.
Within the world of epilepsy, there are the most devastating childhood epileptic syndromes - the epileptic encephalopathies. Dravet syndrome, Rett syndrome, Infantile Spasms - West Syndrome; Tuberous Sclerosis, Lennox-Gastaut Syndrome, Otahara syndrome - etc; Landau-Kleffner syndrome (Acquired epileptic Aphasia).
Many children suffering from neurodevelopmental disorders/epileptic encephalopathies are burded by high seizure load; developmental delay/intellectual disability; physical disabilities; behavioral control problems - etc. Sleep dysfunction, autonomic dysfunction, etc. all contribute to the difficulty in keeping these patients in a somewhat decent quality of life.
Many of the problems, however, are not well controlled with the current medications available. Regarding Infantile Spasms - where the theory is that some sort of insult to the brain (i.e. genetic predisposition from tuberous sclerosis, congenital structural brain lesions, trauma, infection/etc) may lead to a common seizure type - the epileptic spasm - manifesting as hypsarrhythmia pattern on EEG with the spasm showing generalized paroxysmal fast activity correlating with the spasm. This may clinically seem rather benign BUT; if epileptic spasms are not treated ASAP and the etiology of the spasms is not discovered early - it may be too late to prevent a dramatic developmental regression - that can lead to other problems as one ages.
This is where I think 2-73 will come into play. At present, we only have a few medications. cheap medications like prednisone - may have efficacy to stop infantile spasms however it is not as efficacious as the extremely expensive Acthar-gel (synthesized ACTH). ACTH, while efficacious, is 1) terribly cost prohibitive and 2) extremely high risk for untoward side effects including death. Strong anti-inflammatory medications like ACTH/steroids and some antiepileptic medications (Sabril (vigabatrin) in the case of Infantile Spasms caused by tuberous sclerosis - also extremely expensive) are the current standards of care.
Now, lets just say a newer medication with strong anti-inflammatory activity and minimal side effects is introduced and proven to be as efficacious at controlling infantile spasms as ACTH is, without the high cost and risk for extremely untoward adverse reactions..........GAME CHANGER!
As an epileptologist, I am quite interested to see what Missling has to present this afternoon.
Good luck to all long investors!
