Experimental Medical Therapy

• As scientists develop a better understanding of the abnormalities in the electrical system of the brain that causes epilepsy, especially the workings of ion channels (proteins which are an essential aspect of how brain cells communicate with each other), new potentials for treatment develop.
  - Treatments are first tried in animal models of epilepsy to see if they are effective and safe before testing them in humans  
  - Many small and large pharmaceutical companies are working with epilepsy specialists on bringing new epilepsy medication to market.  The process of conducting clinical trials and bringing a new medication to patients is very complicated, time-consuming, and costly.

• A clinical trial is a scientific study of an experiment treatment in human patients (subjects).
  - The most important type of clinical trial is called a double-blind randomized controlled trial (RCT).  The FDA requires multiple RCTs to be performed before approving a new medical treatment.
     * Randomized means that subjects are assigned to active treatment or a control.  The purpose of randomization is to avoid bias (researchers may consciously or unconsciously assign subjects who they think will do better to active treatment).
     * The control might be a lower dose of the study medication or a placebo (sugar pill).  The purpose of using a control is to ensure that any benefit from the treatment is not due to a placebo effect (anytime any therapy is given by a medical professional, even a sugar pill, there will be some improvement).    
     * Double-blind means that both the medical team and subject are not aware if the subject is getting active treatment or placebo (treatment status).  Open-label means that everyone is aware of the treatment status.
     * RCTs follow a strict timeline with distinct periods.
        o Informed consent.  The medical team thoroughly and carefully explains all of the details of the study, potential risks and benefits, and alternatives to participation.  Subjects have to give permission before continuing in the study.
        o Screening period: To determine if the subject qualifies for the trial.  The purpose is to make sure that subject has the appropriate type of epilepsy for the experimental treatment, and to also make sure that there are not any other coexisting medical conditions (like pregnancy or heart disease) which would make participation in the trial potentially dangerous.  There may also be an observation period before randomization where a seizure diary to kept.  Subjects who have too few seizures during this period may not be eligible to continue in the trial.
        o Treatment period.  A defined length of time where subjects are randomized to active medication vs. placebo.  Seizure frequency and any adverse events (side effects or new medical complications) are tracked very carefully.
        o Taper period.  Some trials include a period where subjects are carefully taken off of active therapy or placebo.  Some studies skip this step and transition into an open-label extension study.  This can be an opportunity for all subjects to have access to the active treatment
        o Withdrawal.   Subjects have the right to exit the study at any point they choose.  The medical team usually also reserves the right to withdraw a subject from a study if they feel it is in the best interest of the subject.

• Clinical trials are an opportunity for patients to have early access to new treatments.  The downside is that since the treatment is experimental, all of the potential risks have not been defined.  Participation in a clinical trial however takes place under very close observation by a medical team.  By participating, subjects may benefit themselves and epilepsy patients in general, by helping move experimental treatments for testing to the public at large.

• Websites where patients can find clinical trials
  - www.clinicaltrials.gov
  - www.epilepsygroup.com