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NEW TREATMENTS FOR EPILEPSY

Did you know how long it takes for a new treatment for epilepsy to be tested and approved?  The average for neurological drugs is almost 13 years from the first testing in humans to FDA approval and availability at your local pharmacy.  Why does it take so long?  We understand that testing requires very careful evaluation of safety as well as determination that the treatment is effective.  This requires several stages of clinical trials, each lasting several years.

Did you know that you can improve this timeline?  The biggest roadblock in testing new treatments is finding patients willing to participate in clinical trials. It’s not easy to find patients who are eligible for these studies so the recruitment phase often lasts more than two years for each clinical trial.  Testing new treatments usually is done by asking patients who have many seizures and have already tried many drugs to enroll with the opportunity to receive either the new treatment added onto current therapy or a placebo added onto current therapy.  Most studies are brief, lasting only three to four months, after which all patients are offered the new treatment.  Therefore, people who received the placebo initially can have a new therapy after this brief introductory phase.

Why does it take so long to find people who are having many seizures when there are millions of people with epilepsy?  Clinical trials must be limited to people with specific types of seizures, minimum frequency of seizures (usually at least four partial-onset seizures per month), and be in excellent health.  In addition, patients must be willing to visit the study center frequently during the follow-up period for free care.  Not every doctor can and enroll patients into every clinical trial, so people willing to participate in studies must be referred to the doctor in their area who is conducting the clinical trial.  Although patients are followed by the study doctor for the duration of the clinical trial, they return to their own doctor at the conclusion of the study.

What can you do to help speed the development of new treatments for epilepsy?  Enrolling in a clinical trial for which you are eligible might provide you with improved seizure control and certainly will help others when we understand the usefulness of a new treatment. The first place to look for a clinical trial is on our website at http://my.epilepsy.com/clinical_trials where you can find studies being conducted in your area. A video in which I interview Dr. Morrell provides a description of a clinical trial of the RNS Neurostimulator may be seen at http://my.epilepsy.com/node/976507 . This brain stimulating device is being tested with adults who have frequent partial-onset seizures at 29 centers across the USA.

Success! Retigabine is an example of a new antiepileptic drug that has been tested in clinical trials since 1997 over the years: Enough testing has been completed with retigabine to be ready to request approval by the FDA. Almost 1600 patients participated in these studies over the years.  It will take another year before we take a being is in your pharmacy, if approved as expected.

I am excited to see another antiepileptic drug move toward approval so the epilepsy community will have another treatment for seizures.   I also am pleased to see that two companies (Valeant and Glaxo Smith Kline) will share in developing this compound to make it available around the world.  I look forward to additional epilepsy treatments (drugs and brain stimulating devices) being approved in the near future.  We need even more therapies to help those who have not yet achieved seizure control.

Wishing everyone “freedom from seizures” as soon as possible,

 

Joyce Cramer

President, Epilepsy Therapy Project

 

Comments

Re: NEW TREATMENTS FOR EPILEPSY

 FDA Review of vigabatrin (Sabril®)     I am starting the year by participating in the FDA Advisory Board review of vigabatrin (VGB)(Sabril®).  The FDA expert panel considered whether the drug is effective and safe for treatment of (a) partial onset seizures and (b) infantile spasms, on sequential days.It is important to note that VGB has been in use in countries for more than a decade around the world except USA. When first tested for partial onset seizures, it was considered highly valuable at a time when few other drugs were available in the early 1980s. Doctors soon noticed that it also was highly effective in treating babies with infantile spasms, a severe form of epilepsy that often leads to slow development and lifelong seizures.

However, after several years, doctors discovered that some patients had decreases in peripheral (side) vision.  When this happens, people lose the ability to see around the edges of their eye; their range of vision becomes more central. However, people often do not notice the changes because they accommodate by turning their heads slightly to see what is on the side. That news greatly decreased the number of patients starting VGB, largely restricting its use to people who had not be controlled on several other drugs (considered treatment-resistant).

Over the years, VGB has been reviewed and disapproved three times by FDA for use in the USA. Nonetheless, some people have been able to import the drug from Canada or Europe to continue to use it or to start VGB when other treatments for partial onset seizures or infantile spasms failed. This situation brought a useful treatment to a select few – how unfair! I asked the Advisory Committee to give people freedom of choice.

The discussion for treatment of partial-onset epilepsy centered on the risk of visual field deficits that occur in more than a quarter of patients who take the drug long-term.  Despite years of experience with VGB, we do not know who will be affected or how rapidly it occurs. Testing requires a lot of cooperation by the patient making it impossible for those have limited capacity to understand the instructions and stay still throughout testing. Although visual field deficits also are a concern for babies with infantile spasms, the urgency for rapid treatment may outweigh the risk for limited vision in later life. Babies also are treated for shorter periods than adults with partial-onset seizures.  I noted suggested that adults should be given the opportunity to try vigabatrin for 3 months and babies for 2-3 weeks to determine whether their seizures improve. If not, the drug would be stopped with little risk of visual problems after brief treatment. Give people freedom to choose!

 The Advisory Committee discussed who should be eligible for VGB, trying to define “severe” epilepsy. Dr. Weinstein (New York) noted that a history of poor seizure control with 2 or 3 drugs seemed too early, considering the dozen drugs widely available. He felt that VGB should be reserved for use after multiple drugs had been ineffective. Yet, no specific definition could be made. Dr. Mizrahi (Houston) said: “Intractability is a state that can’t be defined, but you know it when you see it.”

After extensive discussion of the risks and benefits of treatment, the Advisory Committee voted unanimously to recommend approval of vigabatrin (Sabril) for the treatment of refractory partial-onset seizures and infantile spasms, with a special safety monitoring program. I am delighted that Americans will have access to this useful medication.

This is one of the ways in which Epilepsy Therapy Project supports new treatments for people with epilepsy. We start with grant support to scientists, seed investments to start-up companies, advising investors, matchmaking between entrepreneurs and venture capital groups, and presentations of therapies in development (the “pipeline”) to showcase the future.  When you make a donation to ETP, these are the activities you support. Thank you!

Wishing you a seizure-free year in 2009,Joyce CramerPresident, Epilepsy Therapy Project 

Re: NEW TREATMENTS FOR EPILEPSY

When 2009 got under way, the optimism and confidence about our economic future that prevailed just a year ago has given way to anxious concern and uncertainty.  As spring and summer emerge from winter, the economic downturn will play out, and we will again see better conditions.  But the more than 1 million people with epilepsy uncontrolled by available therapies have been frozen in a winter marked by uncertainty for more than three decades.  New therapies working their way through the pipeline offer the promise of seizure control without unacceptable side-effects but only with continued support and nurturing.  With financial markets in turmoil and financing hard to come by, the opportunity and need to help those living with epilepsy by supporting promising new therapies as a friend of the Epilepsy Therapy Project has never been greater.   That’s why we remain deeply committed to helping scientists translate innovative ideas from the laboratory to people with epilepsy.  We are the ONLY national nonprofit epilepsy organization focused on accelerating the development of new therapies so they reach people with epilepsy in the near future.   We firmly believe that people suffering from seizure disorders that impair their lives will benefit from our efforts, which is why we concentrate our resources on making a difference today.   Following are recent highlights of how ETP is moving towards our goal:  Freedom from Seizures (and side effects): New Therapy Development That’s our name, and that’s where our Board, advisors and I spend significant energy and time working with start-up companies and investors to bring them together.  ETP does not have the resources to fund every great idea, but we can knowledgably serve as a “match-maker” to bring innovators to funding.  Every few weeks I learn of another new company that has a molecule with potential anti-seizure properties, or a technology that could detect or stop seizures.  With support from our Board, I follow every lead to learn about the background of the company, their understanding of epilepsy field, patent protection and access to seed funding. This helps guide the advice we constantly impart on basic research, clinical strategy and resources to support the fastest and safest path to helping people with epilepsy.    An ETP Success Story We proudly announce the successful milestone for ganaxolone, a new epilepsy therapy. ETP was an early supporter of Marinus Pharmaceuticals, Inc., providing seed funds and introductions that helped the company engage venture capital groups to provide the $30 million needed to reach this stage. 
Our seed funding is an example of ETP’s venture philanthropy program: Our goal is to assist start-up companies to move to the next step leading toward a new therapy for epilepsy. Since inception, ETP has provided 33 grants for specific, short-term project, one matching grant, and three seed-stage investments to support start-up companies. We can do more with more donations! We support new therapy development by providing advice throughout the stages of clinical development, explaining the best approaches to clinical trials and understand the treatment outcomes needed by people with epilepsy: seizure control with few side effects. The new emphasis on personalized medicine and comparative effectiveness of treatments is a welcome advancement in the standard approach to therapy development. I am particularly pleased to see this because it draws on my many years of experience in health outcomes research, including assessments of quality-of-life, seizure severity, and treatment satisfaction. These are values for patients that are now being recognized by the Food and Drug Administration (FDA) as part of new therapy approval. What’s Next?Yes, we are making headway.  But we can’t do this alone - we need your donations this year more than ever to maintain the momentum.  With more support, we can move at a faster pace.  We also appreciate your thoughts and ideas on how you may prefer to designate or structure a gift toward a particular program.   People with epilepsy can’t wait and often can’t stop having seizures and living with personal and physical losses.  We also cannot afford to let a visionary developer stop working on epilepsy therapies for lack of support.  This is our responsibility and will be to our advantage when better treatments become available. Wishing everyone freedom from seizures,Joyce Cramer

President, Epilepsy Therapy Project

Re: NEW TREATMENTS FOR EPILEPSY

I love this website and love reading about new treatments for people with Epilepsy, one I am waiting for is something in place of Zonegran. I have to be on that medication to control grand-mal seizures, one of the side effects to that med is kidney stones. I have had 4 surgeries to remove kidney stones in 2 years now. I am preying that doctors or researchers can come of with a new medication otherwise my kidneys with fail by the time I am 40 and I am 26 now.

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