The university celebrated the work of scientists Frank Bennett and Adrian Krainer on Thursday Oct. 21 at the 23rd annual Jacob and Louis Gabbay Award ceremony. Dagnar Ringe, Professor Emeritus of Biochemistry, administered the award and gave congratulatory remarks at the ceremony. Bennett and Krainer received the award based on their research, which has led to a novel approach to treating a rare genetic disorder afflicting young children.
Bennett is the current senior vice president of Ionis Pharmaceuticals, a company that specializes in discovering and developing RNA therapeutics, according to their page. Krainer is a professor at the Cold Spring Harbor Laboratory—a lab focused on biomedical research, according to their page—with a focus in molecular biology and genetics. The two scientists collaborated in order to produce the therapeutic treatment Spinraza, an injection-administered treatment for SMA, or spinal muscular atrophy.
The Gabbay Award recognizes research that adds value to the field of biomedical science. Ringe clarified the distinction between the recently awarded Rosenstiel Award and the Gabbay award in an email with The Brandeis Hoot. The Rosenstiel Award focuses primarily on basic research, or research that aims to understand natural phenomena. On the other hand, the Gabbay award targets research that has a distinct application from its basic science roots. Recipients of the Gabbay Award win either $25,000 if there is a sole winner or $30,000 dollars split between multiple winners.
SMA is a genetically transmitted neuromuscular disease that weakens voluntary muscular movement by weakening the neural connections in the spinal cord, according to the United States Library of Medicine. It is typically apparent in children below the age of two. Spinraza treats this condition by correcting gene expression of a crucial gene SMN2.
Spinraza became the first FDA approved drug to treat all types of SMA: Type 1, Type 2 and Type 3, according to a SMA News today article. While the disease is more common and appears in children, it can also be used to treat adult cases. The treatment is approved in more than 50 countries, including China, Canada, Japan, Israel, Turkey and Korea.
Spinraza is an antisense oligonucleotide drug that targets protein production in the gene of interest. Humans have two genes encoding the gene pertinent to SMA called SMN1 and SMN2. In cases of the SMA disorder, the SMN1 gene contains mutations that lead to little to no protein production. While the other SMN gene, SMN2, can produce protein, it forms a protein that is easily degraded in the cell. Spinraza works by increasing the SMN2 gene’s ability to produce a viable protein by attaching molecular tags that indicate the cell machinery not to break it down.
Ringe asserts that “basic research drives the innovations that allow new technologies to be developed, thereby improving our lives.” He cites the example from last year’s Rosenstiel Award winners, who won for their pioneering work on mRNA vaccine technology. This technology proved to be crucial for the development of the COVID-19 vaccines.
Ringe also comments on why awards and prize money from foundations like the Gabbay Foundation and the Rosenstiel Award are important to science. “Any award is important: recognition of unique and singular achievements is a statement of the importance of the work being recognized. In addition, an award is a form of a ‘thank you’ to the researchers for their efforts on behalf of the global community,” he wrote in the email to The Hoot.
Ringe imparts the following notion to students pursuing degrees in the sciences: “Science is a unique opportunity to learn about us, our planet and our universe. Understanding our environment is essential if we are to appreciate it, protect it and make it safe for generations to come. Many disciplines are necessary for that end: biology, chemistry and physics.”
