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Te contamos qué hemos aprendido durante el primer día del congreso EILAT XVI

Hoy comienza oficialmente el congreso EILAT XVI, un evento bianual que reúne en Madrid a profesionales en neurología, investigación e industria de todo el mundo para presentar las novedades en investigación clínica y preclínica de fármacos y terapias en desarrollo específicamente destinados a pacientes con epilepsia.

Sabéis que ayer atrajimos a la audiencia de este congreso a nuestro simposio satélite sobre el síndrome de Dravet. Hoy nos toca a nosotros aprender sobre qué está ocurriendo en la investigación internacional, y asegurarnos de que todos estos expertos cuentan con nosotros para acelerar la llegada de fármacos a los pacientes con Dravet. Os contamos a continuación los temas tratados durante el congreso en lo que concierne al síndrome de Dravet.

Stella Ngo, directora senior de asuntos médicos en Eisai y con quien hemos tenido el placer de reunirnos en persona, presentó resultados sobre los ensayos clínicos en personas con síndrome de Dravet de la lorcaserina, una molécula que actúa selectivamente sobre el receptor 2C de la serotonina (5-HT2C). Esta molécula, que estuvo en el pasado indicada para el control de la obesidad, parece estar involucrada en la inhibición de corrientes eléctricas en el cerebro, hiperactivadas durante las crisis epilépticas, y su selectividad sobre el receptor 5-HT2C la hace más segura frente a posibles efectos alucinógenos o cardiovasculares observados con el uso de moléculas que actúan a través de los receptores de la serotonina 2A o 2B. La lorcaserina, la cual reduce la frecuencia de crisis en un modelo de pez cebra del síndrome de Dravet, está siendo actualmente investigada en dos ensayos clínicos en E.E.U.U. y Canadá: MOMENTUM 1, ensayo de fase 3 en pacientes Dravet de 2 años o más de edad y MOMENTUM 2, un programa de acceso continuado a la lorcaserina para aquellos pacientes Dravet y con otras epilepsias refractarias que han encontrado beneficio en dicho fármaco. ¿Se podría convertir así en una opción terapéutica para aquellos pacientes que no responden bien a fenfluramina, la cual también actúa a través de la vía de la serotonina pero afectando a varios receptores a la vez? Por supuesto, siempre habrá que tener en cuenta y controlar su posible efecto en el peso corporal del paciente. Estaremos pendientes de si Eisai considerara abrir ensayos clínicos en España o Europa.

Mahnaz Asgharnejad, vicepresidenta del programa global de Takeda Pharmaceuticals, nos actualizó sobre los resultados de los ensayos clínicos con soticlestat (TAK-935), un inhibidor selectivo de la 24-hidroxilasa de colesterol (CH24H). Podéis encontrar más información sobre su mecanismo de acción en nuestro resumen anual de fármacos en camino de fin de 2021. En la fase 2 de estos ensayos (ELEKTRA) han participado nuestros pacientes con Dravet, y los resultados muestran, tras las 20 semanas de tratamiento, una reducción del 46% en la frecuencia de las crisis convulsivas. Parece que soticlestat muestra además un buen perfil de seguridad y tolerabilidad, con limitada o nula interacción con otros fármacos antiepilépticos. Como os contamos hace poco en nuestro blog, ahora están realizando en nuestro país la fase 3 (SKYLINE), para la que aún se encuentran reclutando pacientes.

Javier Avendaño, director médico de Stoke Therapeutics y al que ya conocéis puesto que estuvo con nosotros en la pasada reunión annual de familias del día 2 de abril, ha vuelto a hablar sobre su terapia potencial de regulación génica para el síndrome de Dravet. Su oligonucleótido antisentido STK-001 actúa aumentando la producción de canales de sodio Nav1.1, recuperando el defecto provocado por la presencia de una copia defectuosa del gen SCN1A en Dravet. Javier nos ha recordado que, en ratones Dravet, una única dosis de STK-001 recuperaba la producción de Nav1.1 hasta niveles casi normales (los que producirían dos copias sanas de SCN1A) por al menos 14 semanas, y reducía la frecuencia de crisis epilépticas y el riesgo de mortalidad prematura. Stoke Therapeutics también observó aumento de la producción de Nav1.1 por STK-001 en primates. Los primeros resultados del ensayo MONARCH de STK-001 en pacientes Dravet parecen indicar que esta terapia es segura y bien tolerada por los pacientes. Además, 12 de los 17 pacientes analizados hasta la fecha con edades comprendidas entre los 2 y los 18 años experimentaron una reducción de la frecuencia de crisis convulsivas tras 1 a 3 meses de tratamiento. Estos datos favorecen la continuación de los ensayos de la que pueda convertirse en la primera terapia de regulación génica para el síndrome de Dravet.

Además de asistir a las excelentes charlas presentadas durante el día de hoy y comentar los resultados y perspectivas de futuro directamente con los ponentes, hemos tenido también la oportunidad de charlar con algunos de los expertos presentes en el evento como, entre otros, Meir Bialer, profesor emérito de farmacia en la Universidad Hebrea de Jerusalén en Israel y uno de los organizadores del EILAT XVI, Matthias Koepp, profesor de neurología en el Instituto de Neurología UCL Queen Square de Londres, el Prof. Roland Reed, del departamento de farmacia clínica de la West Virginia University, y Konrad Werhahn, líder médico del equipo global de epilepsia de UCB Pharma, empresa que ya sabéis acaba de adquirir la empresa Zogenix, propietaria de Fintepla (fenfluramina).

Mañana volveremos con más detalles sobre la segunda jornada del EILAT XVI.

Un sueño, una meta.

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¿Te has descargado ya tu Dorsal Solidario 2022?

TERMS & CONDITIONS

The European Dravet Syndrome Advanced Therapies (EDSAT) Meeting 2022 is organised by Dravet Syndrome Foundation Spain, whose registered office is located at Calle Doctor Fleming, 30, 28036 Madrid, Spain.

The conference shall take place at the Illustrious Official College of Physicians of Madrid, at Calle de Santa Isabel, 51, 28012 Madrid, Spain, on Friday 1st of April 2022. Attendance to this conference is free of charge, but mandatory for organisational reasons. Participants must register online at www.dravetconference.com. Upon registration, each participant will receive an electronic confirmation by email. On-site registration will not be be possible. If a participant has not received the official email registration confirmation within 3 days, they must contact the conference secretariat at research@dravetfoundation.eu.

The conference registration includes: Attendance to all sessions, oral and visual presentations, all coffee and lunch breaks, and conference documentation with all information about the event. The registration does not comprise travelling and accommodation costs or any other personal expenses.

Admittance to the workshop without registration cannot be granted.

Conference Attendance
Upon arrival at the Illustrious Official College of Physicians of Madrid the day of the event, all participants have to register at the onsite Conference Registration Desk. The desk will open on 1st of April at 8:30 a.m. Upon registration onsite, the conference team will provide participants with information about the event and a badge, which should be worn visibly at all times. The personalised badge authorises participants to attend all conference sessions, and lunch and coffee breaks.

Cancellation Policy
Dravet Syndrome Foundation Spain reserves the right to cancel any registration forthwith and without liability on its part in the event of any damage to, or destruction of the venue by fire, shortage of labour, strikes, industrial unrest, public health measures, or any other cause beyond the control of the Dravet Syndrome Foundation Spain, which shall prevent it from performing its obligations. In these circumstances, every effort will be made to find an alternative format/venue or propose a new date for the conference. Should the conference be cancelled for any reason, airfares, hotels, or other costs incurred by the participants are not reimbursed by the Dravet Syndrome Foundation Spain.

Any participant is allowed to cancel his/her registration but must notify this to the conference organisers by e-mail at research@dravetfoundation.eu within a minimum of 10 days
prior the event. Failure to attend the event without prior written notification
by email may incur a no-show fee, which shall cover individual organizational,
material and catering costs.

Name Change
All registrations are only valid for the respective person mentioned in the form. A registered participant unable to attend the conference may nominate (free of charge) a substitute participant by notifying the conference organisers by email at research@dravetfoundation.eu before 24th of March 2022. The procedure allows giving the last name, first name, and email address of the new participant.

Personal Data
Upon registration, the participant authorises Dravet Syndrome Foundation Spain to use their personal data stated above to inform the participant of any further conferences, events, or information concerning the conference and Dravet Syndrome Foundation Spain’s activities. The conference booklet online will only contain authors, titles, and affiliations of submitted abstracts, but no full abstract texts. The conference printed booklet will contain full abstract texts from all participants. Conference certificates will be sent to the participants as a PDF document upon request at research@dravetfoundation.eu. Only the name & affiliation will be stated in the certificate, not the email addresses of the participants.

Information is collected by an automated computer system that manages registrations. Security and confidentiality are guaranteed by state-of-the-art technical tools. However, Dravet Syndrome Foundation Spain declines any responsibility concerning data security transmitted through the internet.

Photography
The conference is held in public; therefore, we do not ban participants, exhibitors, partners and other companies from taking photographs of conference activities and participants, and share it via social media. By participating in the conference, you approve that general pictures will be taken and used for our Social Media output, our websites and other communication purposes. You hereby waive any right to oppose, and expressly authorise the organiser to make them available on the Internet or as it may seem fit. Author’s presentation slides shall not be photographed by any conference participant.

Disclaimer
The conference organiser cannot be held responsible for any loss, injury or damage to any property, whatever the cause may be. Should, for any reason outside the organiser’s control (e.g., political or economic circumstances or a case of ‘force majeure’), the venue or speakers change, or the event be cancelled, the organiser will endeavour to reschedule, but shall not be held responsible for any costs, damages or expenses incurred by the participants. If for any reason the organiser decides to make material changes to this conference, the organiser is not responsible for airfares, hotels or other costs incurred by the participants. The participant takes part in the conference, all tours, and trips at his own risk.

Copyright
The participant is allowed to use the conference documentation exclusively for his/her personal needs.

TERMS & CONDITIONS

The Dravet Syndrome Conference 2022 is organised by Dravet Syndrome Foundation Spain, whose registered office is located at Calle Doctor Fleming, 30, 28036 Madrid, Spain.

The conference shall take place at the Illustrious Official College of Physicians of Madrid, at Calle de Santa Isabel, 51, 28012 Madrid, Spain, on Thursday 31st of March 2022. Attendance to this conference is free of charge, but mandatory for organisational reasons. Participants must register online at www.dravetconference.com. Upon registration, each participant will receive an electronic confirmation by email. On-site registration will not be be possible. If a participant has not received the official email registration confirmation within 3 days, they must contact the conference secretariat at research@dravetfoundation.eu.

The conference registration includes: Attendance to all sessions, oral and visual presentations, all coffee and lunch breaks, and conference documentation with all information about the event. The registration does not include travel and accommodation costs or any other personal expenses.

Admittance to the conference without registration cannot be granted.

Conference Attendance
Upon arrival at the Illustrious Official College of Physicians of Madrid the day of the event, all participants have to register at the onsite Conference Registration Desk. The desk will open on 31st of March at 8:30 a.m. Upon registration onsite, the conference team will provide participants with information about the event and a badge, which should be worn visibly at all times. The personalised badge authorises participants to attend all conference sessions, and lunch and coffee breaks. 

Cancellation Policy
Dravet Syndrome Foundation Spain reserves the right to cancel any registration forthwith and without liability on its part in the event of any damage to, or destruction of the venue by fire, shortage of labour, strikes, industrial unrest, public health measures, or any other cause beyond the control of the Dravet Syndrome Foundation Spain, which shall prevent it from performing its obligations. In these circumstances, every effort will be made to find an alternative format/venue or propose a new date for the conference. Should the conference be cancelled for any reason, airfares, hotels, or other costs incurred by the participants are not reimbursed by the Dravet Syndrome Foundation Spain.

Any participant is allowed to cancel his/her registration but must notify this to the conference organisers by e-mail at research@dravetfoundation.eu within a minimum of 10 days prior the event. Failure to attend the event without prior written notification by email may incur a no-show fee, which shall cover individual organizational, material and catering costs.

Name Change
All registrations are only valid for the respective person mentioned in the form. A registered participant unable to attend the conference may nominate (free of charge) a substitute participant by notifying the conference organisers by email at research@dravetfoundation.eu before 24th of March 2022. The procedure allows giving the last name, first name, and email address of the new participant.

Personal Data
Upon registration, the participant authorises Dravet Syndrome Foundation Spain to use their personal data stated above to inform the participant of any further conferences, events, or information concerning the conference and Dravet Syndrome Foundation Spain’s activities. The conference booklet online will only contain authors, titles, and affiliations of submitted abstracts, but no full abstract texts. The conference printed booklet will contain full abstract texts from all participants. Conference certificates will be sent to the participants as a PDF document upon request at research@dravetfoundation.eu. Only the name & affiliation will be stated in the certificate, not the email addresses of the participants.

Information is collected by an automated computer system that manages registrations. Security and confidentiality are guaranteed by state-of-the-art technical tools. However, Dravet Syndrome Foundation Spain declines any responsibility concerning data security transmitted through the internet.

Photography
The conference is held in public; therefore, we do not ban participants, exhibitors, partners and other companies from taking photographs of conference activities and participants, and share it via social media. By participating in the conference, you approve that general pictures will be taken and used for our Social Media output, our websites and other communication purposes. You hereby waive any right to oppose, and expressly authorise the organiser to make them available on the Internet or as it may seem fit. Author’s presentation slides shall not be photographed by any conference participant. 

Disclaimer
The conference organiser cannot be held responsible for any loss, injury or damage to any property, whatever the cause may be. Should, for any reason outside the organiser’s control (e.g., political or economic circumstances or a case of ‘force majeure’), the venue or speakers change, or the event be cancelled, the organiser will endeavour to reschedule, but shall not be held responsible for any costs, damages or expenses incurred by the participants. If for any reason the organiser decides to make material changes to this conference, the organiser is not responsible for airfares, hotels or other costs incurred by the participants. The participant takes part in the conference, all tours, and trips at his own risk.

Copyright
The participant is allowed to use the conference documentation exclusively for his/her personal needs.