Life Science Today 015 – CRISPR, Tinnitus, Takeda, and Political-Science
Originally Published as The Niche Podcast
Introduction
Welcome to The Niche Podcast – Your weekly 5-minute rundown of the biotech, clinical research, and applied science industries. I’m your host, Dr. Noah Goodson. This week, Nobel prizes, clinical trials for tinnitus, Takeda’s billion-dollar deal, and the White House keeps science political.
CRISPR/Cas9 Nobel Prize
The Nobel Prize in Chemistry was awarded to Dr Emmanuelle Charpentier and Dr Jennifer Doudna for their work discovering and developing CRISPR/cas9 gene editing technology. The breakthrough was made while exploring immune defense in Streptococcus pyogenes, a species of bacteria that cause human illnesses ranging from tonsillitis to scarlet fever.
In a world-shaking series of experiments, they modified the bacteria’s original defense system and showed that it could be fed specific coordinates to any DNA and create a slice at that position. Imagine having a pair of genetic scissors that can be precisely targeted to anywhere in the genomes to make edits. Most importantly, subsequent studies revealed that this technology could be applied to mammalian cells.
Their papers were first published in 2011 and 2012, but they have quite literally transformed almost every aspect of how scientists approach biological problems. My own PhD thesis was driven by the use of CRISPR/cas9 and without it, I would not have made the critical discoveries that drove my graduate work. For example. I was able to selectively delete two genes simultaneously and their upstream coding elements, uncovering novel insights into how cell fates are regulated in retinal development. These experiments would have taken a decade before CRISPR/Cas9, yet one graduate student was able to accomplish the work in just a couple of years.
My own experience with the technology is just a fragment of the developments that have occurred as a result, including massive discoveries in basic biology, fundamental shifts in how we design plants for human consumption, and a significant number of clinical trials and potential therapies that hope to permanently eliminate certain genetically inherited diseases.
In 2017, I was speaking with the professor who I worked for about this research and we agreed that it would one day win a Nobel prize. This is the first time in history that two women have shared a Nobel Prize in Chemistry. Dr. Charpentier and Dr. Doudna could not be more deserving. Their work is incredible and personally shaped the course of my life. Thank you for what you gave the world.
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The Buzz About Tinnitus
Tinnitus is a phantom ringing in the ears that effects as many as 1/5 individuals. It can result through head trauma, adverse reactions to certain drugs, normal age-related hearing loss, or by unknown causes. For those who experience severe tinnitus, the condition can be debilitating. Currently there is no cure, but a new clinical trial suggests a potentially effective therapy may be on the way.
The treatment is a medical device that coopts the tendency of the brain to re-program. The product is simple. A pair of Bluetooth headphones and a small strip that is held on the tip of the tongue. The therapy works by presenting auditory signals through the headphones and variable vibration stimuli to the tongue. Through some rather complex back-end biology, this basically confuses your brain about the tinnitus and causes it to pay more attention to other stimuli. Think of it like watching a movie on a long plane ride. The movie doesn’t take you to a first-class seat, but it does distract you from being uncomfortable.
The amazing finding was that through using the device long term decreases in tinnitus are observed, even after the therapy has stopped. Individuals took the therapy daily for 12 weeks and their tinnitus systems were measured at the end of that period and for the next year. Even a full year after therapy had stopped patients had a significant decrease in tinnitus. In effect, a therapeutic device can reprogram your brain to ignore tinnitus.
Takeda's Billions
Takeda made a billion-dollar deal with Arrowhead this week. The agreement sees Takeda pay $300 million upfront and up to $740 million in milestones for an agreement to license Arrowhead’s ARO-ATT, an RNAi therapy to treat a liver disease. ARO-ATT had promising results in a phase II clinical trial. With this kind of money on the line, Takeda sees this as a potentially life changing and wildly successful gene therapy. RNAi therapies work by interfering (hence the i) with RNA before it can be translated into a DNA. Effectively it is analogous to a gene therapy but without modifying the genome. There are currently only two RNAi therapies approved by the FDA and each run close to half a million dollars per patient. You don’t need a degree in economics to see the potential ROI on this deal is significant.
Political-Science
We told you last week that the president’s experience with COVID would doubtless shape the drug approval processes. That appears to be the case this week as the administration blocked an attempt by the FDA to employ more stringent safety guidelines for the approval of a COVID19 vaccine. The motivation for Trump to resolve the COVID19 crisis is clear, however, pushing a less than rigorously approved vaccine to the public could pose significant risks. With more than 200,000 Americans dead, the need for a vaccine is obvious, but this must be balanced with appropriate safety measures in a country rife with anti-scientific sentiment and suspicion. Now that the White House is directing American institutions for electoral purposes, we are facing a new kind of political-science.
Closing Credits
Thanks for joining me on The Niche Podcast; your weekly 5-minute summary of top news in the biotech, clinical trials, and applied science industries. You can find us on your favorite podcast app. Like, comment, subscribe, and most of all share with your friends. If you like what you hear, please rate and review, it really helps us. Once again, I’m Dr. Noah Goodson, I’ll see you next week.
Sponsors
https://www.thescopemethod.com
Story References
https://www.nobelprize.org/prizes/chemistry/2020/summary/
https://stm.sciencemag.org/content/12/564/eabb2830
https://www.nytimes.com/2020/10/05/us/politics/coronavirus-vaccine-guidelines.html
https://www.businesswire.com/news/home/20201008005340/en/
Music by Luke Goodson