ETP Pipeline Conference Features New Medications, New Devices, and Even A Few Sharks

[Lisa Boylan]

I am pleased to host a guest blog from Dr. Andrew Neff, an Epilepsy Foundation board member who was kind enough to share this report on a recent Epilepsy Therapy Project Pipeline conference he attended in San Francisco.

The Epilepsy Therapy Project (ETP) hosted its annual Pipeline Conference in San Francisco to present and assess treatments currently in development for epilepsy and choose the $50,000 Shark Tank grant winner.  The 3 day event was well attended by influencers in the epilepsy space and provided an enticing look at the most promising seizure treatments in development.  ETP co-founder Warren Lammert stressed the need for more effective medications with fewer side effects and more FDA-approved medical devices.  Overarching themes included:

• A Dire Need – Despite new treatments in the last 10+ years, efficacy rates for AED’s and most surgery types haven’t improved much.  One third of patients still have seizures despite available treatments, and add in those that struggle with side effects from multiple medications, and nearly half of epilepsy patients could benefit from new treatments.
• Collaboration - Synergistic collaboration between medical professionals, hospitals and universities, non-profits and the private sector is pushing progress in epilepsy even at a time when other neurological conditions such as pain and Alzheimer’s are getting more attention.
• Challenging FDA approvals – The FDA has raised the bar for approvals in recent years.  Intentions were good but there have been negative circumstances.  Approvals are challenging, so medical companies are focusing first on Europe first for new treatments and VC’s are investing in fewer companies with treatments without FDA approval. 

 

Anti-Epileptic Drugs (AED’s) Progress But No Major Breakthroughs In Sight

New medications include one approved (Clobazam), one close to approval (Retigabine), and 5 in late stage clinical trials such as Brivaracetam and Ganaxolone.  Challenges include increased cost for both development and testing, which combined with unpredictable efficacy rates in late stage trials, have narrowed the pipeline of medications in the later stages of development.  Larger pharmaceutical companies are now licensing more often from smaller companies and investing less in their own development.   Looking for upside, companies are favoring medications that show promise for multiple conditions vs. epilepsy alone. 

 

Early stage concepts were enticing and encouraged debate.  From neurotropins and genetics to biomarkers and receptor trafficking, new approaches are plentiful but still early stage.  New medications in testing include GABA inhibitors and Tonabersat.  One theme was new techniques for administering proven treatments, including:

• 'Midazolam 2.0': An inhalant version of the drug for patients with auras that could work faster.
• 'Diazepam 2.0': A new formulation that simplifies the tricky administration of a medication used to interrupt status epilepticus.
• 'Ketogenic 2.0': One company is trying to replicate the efficacy of the Ketogenic diet in a pill form.

 

Shark Tank Competition – Six New Ideas, Many Sharks, And One $50,000 Grant Winner

A commendable and let's say it, exciting new event was the Shark Tank competition.  The goal was to develop a novel concept to help people with epilepsy, with the winner receiving a $50,000 development grant.   Response was strong, and over 40 early submissions were narrowed down to 6 finalists who presented.  Five panelists, ranging from investors to patients, reviewed the ideas and voted, and a vote from attendees was needed to break the tie.  The six finalists included....

 

1. Video Monitor #1:  The father of a patient showed his home-developed solution for nighttime seizure monitoring.  Combine an iphone app, laptop SW, and one loud alarm and you can detect those seizures with  severe movements (e.g. tonic clonics) and notify/wake-up the parents. 
2. Decoding Signals to Prevent SUDEP: Sudden unexplained death due to epilepsy (SUDEP) remains an issue, and one company would use existing technologies and begin development of hardware to assess heart and brain signals that would theoretically reduce SUDEP.
3. Sleep Apnea T-Shirt: Poor sleep is a common seizure trigger, and sleep apnea is one known cause.  The patient would wear a special t-shirt with a electronic device that detects sleep apnea.
4. Sleek Helmet: Patients with tonic clonics and no aura can need helmets to prevent injury, but helmets create stress by drawing attention to the seizures.  A crowd favorite, this sleek head wrap would theoretically cushion the landing yet look sleek like funky headphones.
5. Eyes as a Window: A local epileptologitst and inventor felt that eye movement, when analyzed in details with special electronic glasses that conveyed results to custom software, can detect seizures.
6. Video Monitor #2:  The SmartWatch company would initiate development of custom software that, when combined with the watch and their video system, could alert parents of nighttime seizures.

 

The judges were top notch, complimenting efforts yet asking probing questions to assess the market need, feasibility, and tangible patient benefits.  Differing backgrounds for judges led to entertaining debate, and their final votes were split enough that an audience vote was needed to break the tie.  The $50,000 grant winner was the patient's father with Video Monitor #1, which was appealing with its limited development costs and  functional model.  

  

Implantable Devices for Seizures Await Approval

While only Cyberonics' Vagus Nerve Stimulator (VNS) has FDA approval, other companies are developing new implantable devices. While they work slightly differently, they are all intended for refractory patients, most with tonic clonics, who have had no success with medications. Several devices are still awaiting FDA approval in the US, with one already approved in Europe.  

• Cyberonics presented updates on AspireSR, a new version of the VNS now in tests that incorporates heart rate irregularities as an additional sensor.  
• Neuropace provided an update on the RNS, the cranial implant that provides neuro-stimulation, which has completed successful clinical trials and still awaits FDA approval. 
• NeuroVista presented positive clinical trial results for its' intracranial EEG device that, together with a chest implant, can theoretically warn patients of when they're prone to have a seizure. 
• Medtronic's Deep Brain Stimulator (DBS), approved for Europe, is awaiting US approval while the company develops the next version that combines brain and motion activity to detect seizures. 
• NeuroSigma's Trigeminal Nerve Stimulation (eTNS) features an external device that sends electronic signals to extracranial (vs. deep brain) electrodes.  It will first launch in Europe and will be submitted for US approval later in 2012.

 

Other devices included new electrode systems for improved or more portable EEG recording, but without FDA approvals in the US, not much has changed for patients in the US device market in the last year.

 

The takeaway from this key conference was that more progress is needed in the battle with epilepsy.  Attendees witnessed 23 medications and devices in development and the entertaining Shark Tank competition with a $50,00 grant to the winner. Collaboration is strong and must continue between all sectors, including non-profits like the host Epilepsy Therapy Project as well as the Epilepsy Foundation and CURE if we're going to find a much-needed breakthrough for patients struggling with seizures.  Remember, knowledge is power.

 

[bryan farley]

Southie,

I would like to know more about home monitoring... or other monitor that are similar to our normal environment. 

BF

[Southie]

I have done the video home monitoring; with my own personal computer - it was a personal agreement between my Neuro and myself; but that was some years ago, and seizures were captured (Nocturnal) in which gave him the idea exactly what type of EEG test to run up. Seeing myself in seizures was of no big deal, while some were what I would say "too mild or not of significance importance" - it's weird to post this that I knew what the Doctor wanted and needed and that he got... and exactly what he wanted!

I am a strong supporter of Home Video Monitoring, since Hospital VEEG's just "seems to be a cure-all" (as you can tell by posters who have gone through it) for many; while those at home suffers after having undergone days to weeks and weeks of waiting for a seizure. I believe the home environment is best suited.

One question however, would an AEEG be involved (mine was not) or included or would the whole situation be differing?

As for the FDA BAN - I just received a FDA Letter pertaining to Neurontin / Gabapentin and posted it on Living Well - they want to eliminate this AED drug... I called FDA and ... go read up on it ... self-sufficient right there and I am peeved because I cannot find anywhere or maybe I am looking in all the wrong places where the FDA wants to consider a ban on Neurontin / Gabapentin.

If we let the Congress/FDA/President have their way ... before you know it - we, the people with Epilepsy are not going to have any anti-epileptic medications available at all; so what options are going to be available for us - smuggling? (HONEST! Think about it ...)

[bryan farley]

@lisa,

I think I met him. I did not realize he was from EFA. On my personal blog, I might have a photo of him. Does he live out here or in the DC area?

bf

[Lisa Boylan]

I like the NFL helmet collaboration idea!

[Sherri Delmar]

Big fan of #1 Video home monitoring . I have noctornal seizures & done the 5 day stay in EMU twice now. I too considered having this kind of home device!! It has great potential & I'd like to see Easton Ridell (NFL helmets) based here in Ohio & the Cleveland Clinic colloborate on this project.    

[Lisa Boylan]

Hi Bryan--that is great to hear! Thanks for letting me know. Did you hapen to also see our new research director Joe Twist? He was there as well.

[bryan farley]

I was able to attend part of the conference. I live across the bay from San Francisco and the third day was open to the public. (Perhaps I could have attended the first two days too; I am not sure if I am the public.)

Andrew represented the Epilepsy Foundation of America well. He spoke on a panel with a few other people affected with epilepsy. Most of the people had epilepsy. Including our voices is important for many reasons, even for improving the effectiveness of research.

Thank you Andrew and Lisa.