Prof Lieven Lagae, ICNA Executive Board Member & Director of the Childhood Epilepsy Program at the KUL University Hospitals is the recipient of "Cures within Research" 5th Annual Global Health Repurposing Awards. Prof Lagae has been awarded the new Industry Patient Impact Clinical Award.
Award recipients will be recognized at the Global Health Repurposing Awards on June 27, 2017 at the 6th Annual Global Health Drug Repositioning and Repurposing Conference in Chicago, IL.
We are very proud to acknowledge the achievements of our fellow ICNA Executive Board member, Dr. Lieven Lagae of KUL University Hospitals in Belgium, for his "Cures Within Reach Industry Patient Impact Clinical Award 2017" which has been given in recognition of his pioneering research and world-wide efforts to repurpose the drug fenfluramine for Dravet syndrome patients.
Dravet syndrome is a rare, catastrophic, lifelong form of epilepsy that begins in the first year of life with frequent and/or prolonged seizures. Previously known as Severe Myoclonic Epilepsy of Infancy (SMEI), it affects 1:15,700 individuals, 80% of whom have a mutation in their SCN1A gene.
Fenfluramine is an amphetamine-like drug that has been used in the past as a part of antiobesity treatments. Because of the possible cardiac adverse effects (valve thickening, pulmonary hypertension) associated with use of fenfluramine, it was withdrawn from the market in 2001. Dr. Lagae has doggedly fought to bring Dravet patients access to this drug, and is leading a worldwide effort to secure regulatory approval for fenfluramine so that it will help stop these debilitating seizures.
In Belgium, a Royal Decree permitted examination of the potential anticonvulsive effects of fenfluramine in a clinical trial consisting of a small group of patients diagnosed with Dravet syndrome.
The results of a prospective, open-label study by Prof Lage and colleagues assessing the safety and effectiveness of low-dose fenfluramine in a new cohort of Nine patients (aged 1.2-29.8 years) with Dravet syndrome has been recently published in the European Journal of Neurology in February 2017. In this study following a 3-month baseline period, fenfluramine was added to each patient's current antiepileptic drug regimen at a dose of 0.25-1.0 mg/kg/day (max. 20 mg/day) for a median duration of 1.5 (range, 0.3-5.1) years.
All patients demonstrated a reduction in seizure frequency during the treatment period with a median reduction of 75% (range, 28-100%). Seven patients (78%) experienced a ≥50% reduction in major motor seizure frequency. The most common adverse events were somnolence (n = 5) and anorexia (n = 4). No evidence of cardiac valvulopathy or pulmonary hypertension was observed.
The effectiveness and safety of low-dose fenfluramine as an add-on therapy for Dravet syndrome in this new prospective cohort supported previous findings by the same researchers in smaller observational studies.
Cures Within Reach
Cures Within Reach, is a leading global nonprofit focused on repurposing research as a fast track to saving patient lives. Cures Within Reach (http://www.cureswithinreach.org) works to catalyze repurposing research to quickly and affordably improve patient lives. We accomplish this by connecting funders with researchers to jumpstart repurposing research clinical trials, by providing collaboration tools so repurposing stakeholders can work together more easily, and by pioneering alternative finance engines and incentives for repurposing research. Cures Within Reach's repurposing research projects have generated over a dozen "new" treatments making patient impact through off-label use in clinical practice or through a commercialization track.
Low-dose fenfluramine significantly reduces seizure frequency in Dravet syndrome: a prospective study of a new cohort of patients.Schoonjans A, Paelinck BP, Marchau F, Gunning B, Gammaitoni A, Galer BS, Lagae L, Ceulemans B.Eur J Neurol. 2017 Feb;24(2):309-314. doi: 10.1111/ene.13195.
Five-year extended follow-up status of 10 patients with Dravet syndrome treated with fenfluramine.Ceulemans B, Schoonjans AS, Marchau F, Paelinck BP, Lagae L.Epilepsia. 2016 Jul;57(7):e129-34. doi: 10.1111/epi.13407. Erratum in: Epilepsia. 2017 Mar;58(3):509-510.
Low-dose fenfluramine in the treatment of neurologic disorders: experience in Dravet syndrome.Schoonjans AS, Lagae L, Ceulemans B.Ther Adv Neurol Disord. 2015 Nov;8(6):328-38. doi: 10.1177/1756285615607726.