Department of Biology Davidson Lecture: "From bench to bedside: Spinraza, the first approved treatment for spinal muscular atrophy"

DateSeptember 27, 2018Time6:30 - 8:00 pm
LocationBaylor Sciences Building, room B.110
Description

From bench to bedside: Spinraza, the first approved treatment for spinal muscular atrophy

Speaker

Adrian Krainer, Ph.D.
St. Giles Foundation Professor
Program Chair of Cancer & Molecular Biology
Cold Spring Harbor Laboratory

Abstract

Dr. Krainer's lab studies the mechanisms of RNA splicing, ways in which they go awry in disease, and the means by which faulty splicing can be corrected. In particular, they study splicing in spinal muscular atrophy (SMA), a neuromuscular disease that is the leading genetic cause of death in infants. In SMA, a gene called SMN2 is spliced incorrectly, making it only partially functional. The Krainer lab found a way to correct this defect using a powerful therapeutic approach. It is possible to stimulate SMN protein production by altering mRNA splicing through the introduction into cells of chemically modified pieces of RNA called antisense oligonucleotides (ASOs). Following extensive work with ASOs in mouse models of SMA, one such molecule, known as nusinersen or Spinraza, was taken to the clinic, and at the end of 2016 it became the first FDA-approved drug to treat SMA, by injection into the fluid surrounding the spinal cord.

Faith Fortenberry of Waco, Texas lives with SMA type 2, and received her first treatment of Spinraza in May 2017 with remarkable results. Faith is currently a National Ambassador for the Muscular Dystrophy Association (MDA.org), which provided significant funding for the development of Spinraza. In Faith's case, Spinraza is already having a tremendous impact on her life. "It has been a game-changer," says her mother, Leeann. "She can now lift things, hold a glass, and brush her own teeth, which she was not able to do before. More importantly, she is able to breathe better."

Please join us in the Baylor Sciences Building (BSB) on September 27, 2018 for a celebration of science and society. We will bring Faith and Dr. Krainer together for the first time and hear an extraordinary story of scientific discovery and healing. There will be a public reception with hors d'oeuvres in the BSB Atrium from 5:30-6:30 pm followed by public remarks, lecture, and question/answer session in lecture hall B.110 from 6:30 - 8:00 pm.

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