I am a married father of a 12-year-old son Owen, and twin 9-year-old girls, Mimi and Piper, who were born on May 20, 2008. Piper was a healthy and happy baby until January 1, 2009, the day Piper had her first tonic-clonic seizure while vacationing on a small island in the Bahamas. It was a terrifying experience that no parent should have to experience. We were not sure Piper would survive. Unfortunately, this was only the beginning of many more horrifying and near-death experiences for Piper until she was introduced to GW Pharmaceuticals’ Epidiolex five years later.
Upon our return, we were fortunate to be introduced to UCSF’s Director of the Pediatric Epilepsy Center, Joseph Sullivan, MD. As Piper presented with more tonic-clonic seizures, Dr. Sullivan started her on Topamax and urged genetic testing. Fortunately, Topamax reduced the frequency of Piper’s seizures, but not the severity. Soon after, we learned Piper’s diagnosis: a de novo SCN1A mutation and Dravet.
Dr. Sullivan also suggested we consider the ketogenic diet to further reduce seizure activity. The combination of the keto diet and Topamax helped reduce Piper’s seizure burden to ~6 tonic-clonic seizures a year from ages 18 months to 4 years. Unfortunately, many of these seizures could not be stopped with the intervention of rescue medications from our local hospital’s emergency room, many lasting well over one hour.
At close to 4 1⁄2 years of age, the positive effects of the ketogenic diet began to wane and Piper began having tonic-clonic seizures with greater frequency. She also started presenting with myoclonic seizures for the first time. Over the next 15 months, Piper suffered over 1,000 tonic-clonic seizures with 7 admissions to the intensive care unit. During this period, Piper was prescribed a multitude of Dravet-specific anticonvulsants in a desperate attempt to reduce the steadily increasing number of seizures. None of the new medications helped, while one had a paradoxical effect. All the while, Piper’s cognitive development began to regress.
State of Science
In 2018, the U.S. Food and Drug Administration (FDA) approved Epidiolex (cannabidiol) as the first drug derived from marijuana for treating seizures in Dravet syndrome in patients aged two and older, marking the first FDA-approved treatment for Dravet syndrome.
Later in 2018, the FDA also approved Diacomit (stiripentol) for Dravet syndrome. The development and approval of stiripentol, from its discovery in 1978 to its FDA approval in 2018, has been supported by numerous clinical trials that demonstrated its effectiveness when combined with clobazam and valproic acid.
The stress inflicted on our family as a result of Piper’s prolonged seizures was overwhelming. Our son Owen began to have day terrors, like extreme nightmares but during the day while fully awake. My wife Ashley and I lay awake much of every night waiting and watching Piper in fear of another tonic-clonic seizure and everything that came with it.
This is in no way unique to us. Every family with a Dravet syndrome child is subject to immense suffering.
In late January of 2014, Piper was prescribed Epidiolex. Piper then went 7 months without a single seizure of any kind. Piper has now been on Epidiolex for over 4 years. She is averaging one tonic-clonic seizure a week. Immediately prior to starting on Epidiolex, Piper suffered an average of over 20 tonic-clonic seizures a week. Unlike many of Piper’s seizures prior to the introduction of Epidiolex, they all stop after a few minutes or less. Owen, Mimi, Piper, Ashley, and my quality of life has radically improved. We owe this all to GW Pharma’s Epidiolex, NDA 210365, cannabidiol oral solution.
Before DSF: Piper’s family endured years of terror and stress, with frequent, life-threatening seizures and failed treatments.
With DSF: Thanks to the support of research driven by organizations like DSF, Piper’s seizure frequency has drastically reduced, allowing her to thrive and seek independence.
Hope for the Future: Families like Piper’s are hopeful for even more treatment breakthroughs that will continue improving the quality of life for children battling Dravet syndrome.
Despite the many difficulties imposed on Piper’s life with Dravet syndrome, she is thriving as a result of Epidiolex. Since she began taking Epidiolex, Piper has been participating in equestrian therapy. She loves riding. Piper also enjoys riding her two-wheel bike to school every day, bouncing on our trampoline, taking gymnastics, dancing, and singing. Piper is an enthusiastic swimmer and can hold her breath the longest of her siblings. Last season, Piper had her first experience snow skiing. Piper is happy, engaged, and outgoing. She continues to gain confidence and seeks independence. Most people who meet Piper remark on her spirit and are instantly attracted to her charm, warmth, and openness to connect. I strongly believe none of this would be possible without the benefit of GW Pharma’s Epidiolex.
I urge you to consider recommending the FDA approve the new drug application (NDA) 210365, cannabidiol oral solution, sponsored by GW Pharmaceuticals, for the adjunctive treatment of seizures associated with Lennox-Gastaut syndrome or Dravet syndrome.
Sincerely,
Timothy M. Wood
Original letter addressed to the Peripheral and Central Nervous System Drugs Advisory Committee, March 2018
