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The Buzz Around Gene Therapy

Over the last several weeks, results from gene therapy trials have created a lot of excitement in our community.

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By Melody Bertrand

AG Bell recently attended the Association of Research Otolaryngology (ARO) Midwinter Meeting February 2 to 7 in Anaheim, Calif. During a two-hour President’s Symposium, scientists from bio-pharmaceutical organizations in China, France and the United States shared early results of clinical trials in gene therapy targeted to the replacement of the Otoferlin protein.

[Pictured: AG Bell’s Gayla Guignard with Sunil Puria, Ph.D., President of the ARO, taken just following the Presidential Forum focused on Gene Therapy.]

Of the 34 million children with sensorineural hearing loss, an estimated 200,000 children have Otoferlin–induced auditory neuropathy resulting in profound hearing loss. Auditory scientist John Tilak Ratnanather, PhD, who also attended the ARO meeting, said that these early results suggest a “game changer” for those children with hearing loss who might qualify for this therapy if it is approved in the future.

“One child in one of the clinical trials had no response while all of the others who received the otoferlin gene therapy went from having a profound hearing loss to a mild-to-moderate hearing loss, or even closer to normal hearing in some cases,” said Gayla Guignard, AG Bell’s Chief Strategy and Programs Officer. “All of those directly involved in the clinical trials are careful to caution that we are still at a very early point in the process. Still, after both listening to these findings and viewing pre- and post-videos of a few participating children, one can’t help but sense that we have reached the edge of a new frontier.”

Recently, the New York Times published an article highlighting the change in one boy’s life after receiving gene therapy. This young man went from having a profound hearing loss to a mild one, hearing sounds for the first time in his life and even beginning to use words to communicate as well.

With so much to gain from such an amazing breakthrough, Guignard had the opportunity to comment after the President’s Symposium during the ARO meeting. She thanked the scientists and represented organizations for their innovative work and for their care of patients/those who participated. She also highlighted how much children, their families, and the professionals who work with them will benefit from this ongoing research and development.

This is all just the beginning. This summer, the AG Bell Global LSL Virtual Symposium will feature sessions related to this groundbreaking science. Please visit www.agbellsymposium.com to learn more and register to attend.

La expectación que genera la terapia génica

En las últimas semanas, los resultados de los ensayos de terapia génica han suscitado una gran expectación en nuestra comunidad.

AG Bell asistió recientemente al congreso de mediados de invierno de la Association of Research Otolaryngology (ARO, Asociación de Investigación en Otorrinolaringología), celebrado del 2 al 7 de febrero en Anaheim, California. Durante un simposio presidencial de dos horas de duración, científicos de organizaciones biofarmacéuticas de China, Francia y Estados Unidos compartieron los primeros resultados de ensayos clínicos de terapia génica dirigida a la sustitución de la proteína otoferlina.

De los 34 millones de niños con pérdida auditiva neurosensorial, se calcula que 200 000 padecen una neuropatía auditiva inducida por la otoferlina que provoca una pérdida auditiva profunda. El científico especializado en audición John Tilak Ratnanather, PhD, que también asistió a la reunión de la ARO, afirmó que estos primeros resultados suponen un «cambio radical» para los niños con pérdida auditiva que puedan optar a esta terapia si se aprueba en el futuro.

«En el caso de un niño, en uno de los ensayos clínicos, no se obtuvo respuesta, mientras que el resto de los niños que recibieron la terapia génica con otoferlina pasaron de tener una pérdida auditiva profunda a una pérdida aditiva de leve a moderada, o incluso más cercana a la audición normal en algunos casos», señala Gayla Guignard, directora de Estrategia y Programas de AG Bell. «Todas las personas que participan directamente en los ensayos clínicos advierten que todavía estamos en una fase muy temprana del proceso. Aun así, tras conocer estos resultados y ver los videos previos y posteriores de algunos niños participantes, no se puede evitar tener la sensación de que hemos llegado al borde de una nueva frontera».

Recientemente, el New York Times publicó un artículo en el que se destacaba el cambio en la vida de un niño tras recibir terapia génica, ya que pasó de tener una pérdida auditiva profunda a una leve, oyendo sonidos por primera vez en su vida e incluso empezando a utilizar también palabras para comunicarse.

Ante este avance tan favorable, la Sra. Guignard tuvo la oportunidad de facilitar algunos comentarios al final del simposio presidencial durante la reunión de la ARO. Dio las gracias a los científicos y a las organizaciones representadas por su trabajo innovador y por su atención a los pacientes/participantes. También destacó lo mucho que los niños, sus familias y los profesionales que trabajan con ellos se beneficiarán de esta investigación y desarrollo en curso.

Esto es solo el principio. Este verano, el Simposio Global Virtual de AG Bell de LSL incluirá sesiones relacionadas con esta ciencia de vanguardia. Visite www.agbellsymposium.com para obtener más información e inscribirse para asistir.

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