- Pharmacological management of epilepsy: recent advances and future prospects.
- Antiepileptic Drug Development for “Therapeutic Orphans”
- Dravet Syndrome Treatment Granted Orphan Drug Designation by FDA
The incidence of Epilepsy is more predominant in the extremes of age i. Approximately 15 percent of people who have epilepsy have Status Epilepticus. Combined together approximately two to three million Americans have epilepsy but the majority of affected individuals are seizure free due to effective medications. Symptoms of the following disorders can be similar to those of Status Epilepticus. Comparisons may be useful for a differential diagnosis:. Symptoms develop due to the excessive accumulation of copper in body tissues, particularly the liver, brain and eyes.
These symptoms may include drooling, joint pain dysarthria , impaired speech dysphasia , lack of muscle coordination, tremors, involuntary jerky muscle movements, muscle rigidity and double vision. Kok Disease Hyperexplexia is a very rare inherited disorder of the neurological system. When the individual with Kok Disease is startled, the head may arch back and there may be jerking muscle movements myoclonic jerks. When startled the individual may also fall to the ground in a rigid position.
Some people with Kok Disease also experience seizures. Myoclonus is a neurological movement disorder in which a skeletal muscle undergoes sudden, involuntary contractions resulting in jerky movements. There are 3 types of Myoclonus: Intention, rhythmical, and arrhythmic. Intention myoclonus is characterized by episodes of involuntary muscle contractions that are triggered by voluntary movements, such as a purposeful action. In arrhythmic myoclonus, muscle jerks are arrhythmic and sudden.
The muscle jerking may be confined to a single muscle or involve the all of the skeletal muscles on one or both sides of the body. An extreme startle response may also be present. Rhythmical segmental myoclonus is characterized by very rapid and frequent muscle jerks. In contrast to Arrhythmic Myoclonus, this type of Myoclonus is not relieved by sleep and is not triggered by sudden stimuli or voluntary movements. Myoclonic muscle jerks may sometimes be confused with the muscle jerks and rigidity that occur in some forms of Epilepsy. Symptoms usually begin between the ages of 10 and 20 years.
The development and severity of symptoms vary greatly among patients. Exaggerated daytime drowsiness is usually the first symptom. People with Narcolepsy who have cataplexy can fall asleep so suddenly that they appear to drop to the floor unconscious. In sleep paralysis, the person with Narcolepsy want to move but cannot do so.
SE is treated with anticonvulsant drugs that attempt to prevent and control seizures. The drugs that are currently used include phenytoin, valproic acid, carbamazepine, phenobarbital, clonazepam, ethosuximide Zarontin , primidone, acteazolamide, paraldehyde, trimethadione, corticotropin and corticosteroids.
Brain surgery for epilepsy that is caused by a brain tumor or drug-resistant temporal lobe epilepsy may be tried after medications have failed to stop seizures. Surgery is generally not performed until other treatment methods have failed. The success rate for such surgeries is approximately 55 to 70 percent. It is very important to protect the epilepsy patient from self-injury during a seizure.
Protective measures should include clearing the area of any object that is hard or sharp, loosening tight clothing and placing a flat, soft object under the head. The patient should be turned on the side and if possible something soft and flat such as a pad or wallet may be placed between the teeth. Restraint is not advised. The administration of artificial respiration should be attempted only if breathing does not start after the seizure has stopped.
When the seizure is over, the patient should be allowed to sleep or be helped home if he or she seems confused.
Pharmacological management of epilepsy: recent advances and future prospects.
If the patient wants to sleep, the head and shoulders should be raised. It is possible that some people with epilepsy who have had no seizures during an extended period of time several years may reduce or discontinue anticonvulsant medications under close supervision by a doctor. One of the main advantages of this drug is that it is not a sedative, so it will not make the user feel sleepy or sluggish.
Due to occurrences of rare but serious side effects from this drug, physicians should contact the manufacturer or FDA before prescribing. The Food and Drug Administration has approved the drug Lamotrigine Lamictal for the treatment of hard-to-control partial seizures. The drug is manufactured by Burroughs-Wellcome Company. The drug fosphenytoin sodium cerebyx has been approved by the FDA for the treatment of epilepsy.
Fosphenytoin sodium is manufactured by Warner-Lambert. The drug tipiramate Topamax has been approved by the FDA for the treatment of epilepsy in addition to other medications. Topiramate is manufactured by Ortho-McNeil. In addition, Neurontin gabapentin has also been approved for use in the treatment of epilepsy. The drug Diastat diazepam suppository rectal gel has been approved by the FDA for the treatment of epilepsy. Diastat is manufactured by Athena Neuosciences Inc.
The drug carbamazepine extended release capsules Carbatrol has been approved by the FDA for the treatment of epilepsy.
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Carbatrol is manufactured by Shire Pharmaceuticals. Information on current clinical trials is posted on the Internet at www. All studies receiving U. Studies are underway on many experimental anti-convulsant drugs for the treatment of epilepsy. The website, www. For additional information on treatment research, visit the websites of the organizations listed as resources in this report.
Bennett JC, Plum F. Cecil Textbook of Medicine. Philadelphia, PA: W. Saunders Co; The financial disincentives for pharmaceutical companies to fund RCTs of AEDs among children early in drug develop should be obvious to even the casual observer. FDA policies that have traditionally worked to protect children enrolled in clinical trials, while maintaining the quality of efficacy trial results, have focused on pediatric studies of drugs marketed primarily for adults.
However helpful FDA policies may be in encouraging pediatric studies of marketed drugs, the FDA policies cannot solve the problems suffered by children with uniquely pediatric epilepsy syndromes. Until such a comprehensive plan is developed and appropriate resources allocated, children will continue to be the orphans of AED development.
According to a FDA Report to Congress, only 11 of 71 postmarketing pediatric studies promised by industry were completed The ILAE commission outlined a need for early clinical trials in children. However, because of concerns regarding the ethics of children being subject to clinical trials of AEDs too early in the drug development process, the commission recommended clinical trials in children only after Phase II and Phase III studies were completed in adults.
The additional patent protection is based on the company's compliance with the FDA's request for a pediatric study, not the eventual FDA approval of the drug for pediatric use.
Antiepileptic Drug Development for “Therapeutic Orphans”
The FDA enacted the Pediatric Rule in that required pharmaceutical companies to conduct basic studies in children for drugs approved for adults for indications that also occur among children. Many advocates for children's health heralded the rule as a major advance for pediatrics.
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Combined with the exclusivity rules that give pharmaceutical companies an additional 6 months of patent life if additional pediatric studies are performed, the Pediatric Rule offered hope that children would no longer be denied proper testing of drugs prior to routine use In October the U. District Court of the District of Columbia struck down the Pediatric Rule requiring pediatric studies, stating that Congress did not give the FDA authority to require pharmaceutical companies to perform pediatric trials.
At the time of this writing, corrective legislation is being discussed among various members of Congress to give the FDA authority to require pediatric trials. Even when the Pediatric Rule was in force, pharmaceutical companies had few incentives to develop and market AEDs for unique pediatric indications.
For example, at the Eilat VI Conference, a recent international conference on AEDs in development, not one pharmaceutical company expressed interest in developing an AED specifically for a uniquely pediatric epilepsy syndrome; no company reported plans for any clinical trials in children before approval of their AED for partial seizures in adults There is no effective program under serious consideration by governmental agencies or by the pharmaceutical industry that will facilitate drug development for pediatric epilepsy syndromes that are unique to childhood and that are relevant to the clinical treatment decisions faced daily by practicing pediatric neurologists.
At present, children with serious, chronic epilepsy syndromes that do not occur among adults can only hope that through luck their disorders will benefit from therapeutic developments intended to treat adult partial epilepsy. Volume 44 , Issue s7. If you do not receive an email within 10 minutes, your email address may not be registered, and you may need to create a new Wiley Online Library account.
If the address matches an existing account you will receive an email with instructions to retrieve your username. Epilepsia Volume 44, Issue s7. Free Access. Search for more papers by this author. Address correspondence and reprint requests to Dr. Tools Request permission Export citation Add to favorites Track citation.
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Please review our Terms and Conditions of Use and check box below to share full-text version of article. Abstract Summary: Epilepsy is the most common serious neurological disorder among children. Figure 1 Open in figure viewer PowerPoint. Children with more than one seizure type had their most clinically significant seizure type estimated from medical records by the study pediatric neurologist.
Reproduced with permission from reference 2. Epilepsy syndrome or seizure type AED no. Studies of primarily children birth to 18 years that included small numbers of adults are included. Apparent efficacy only in partial seizures. Rapid evolution of different seizure types and epilepsy syndromes Some of the most intractable of epilepsy syndromes have variable patterns of evolution that sometimes include rapid changes in seizure type and seizure frequency over a few weeks to months.
Rapid change of baseline neurological status The natural history of coexisting neurological, developmental, and metabolic disorders also confound the interpretation of efficacy studies and the interpretation and reporting of adverse events in studies of pediatric epilepsy. Financial disincentives, regulatory policies, and the absence of a plan The financial disincentives for pharmaceutical companies to fund RCTs of AEDs among children early in drug develop should be obvious to even the casual observer.
Incidence and prevalence. Epilepsy: a comprehensive texbook.
Dravet Syndrome Treatment Granted Orphan Drug Designation by FDA
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