Lauren Black, Distinguished Scientist in the Scientific advisory services at Charles River Labs, shares the story of how doctors came up with a customized drug to treat one specific patient with Batten disease. Plus: learn about how you really can tell if someone’s sick just by looking at them. In this podcast, Cody Gough and Ashley Hamer discuss the following story from Curiosity.com about how you really can tell someone’s sick just by looking at them: https://curiosity.im/2D6XTo8 Additional sources: Drug Regulation in the Era of Individualized Therapies | The New England Journal of Medicine — https://www.nejm.org/doi/full/10.1056/NEJMe1911295 Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease | The New England Journal of Medicine — https://www.nejm.org/doi/full/10.1056/NEJMoa1813279 Amazon smart speaker users: you can listen to our podcast as part of your Amazon Alexa Flash Briefing! Just click or tap “enable” here: https://curiosity.im/podcast-flash-briefing.
Lauren Black, Distinguished Scientist in the Scientific advisory services at Charles River Labs, shares the story of how doctors came up with a customized drug to treat one specific patient with Batten disease. Plus: learn about how you really can tell if someone’s sick just by looking at them.
In this podcast, Cody Gough and Ashley Hamer discuss the following story from Curiosity.com about how you really can tell someone’s sick just by looking at them: https://curiosity.im/2D6XTo8
Additional sources:
Amazon smart speaker users: you can listen to our podcast as part of your Amazon Alexa Flash Briefing! Just click or tap “enable” here: https://curiosity.im/podcast-flash-briefing.
Find episode transcript here: https://curiosity-daily-4e53644e.simplecast.com/episodes/customized-medicine-w-lauren-black-and-how-to-tell-someones-sick-by-looking-at-them
CODY: Hi! You’re about to get smarter in just a few minutes with Curiosity Daily from curiosity-dot-com. I’m Cody Gough.
ASHLEY: And I’m Ashley Hamer. Today, you’ll learn about how doctors came up with a drug to treat one specific patient with a rare genetic disease; and, how you really can tell if someone’s sick just by looking at them.
CODY: Let’s satisfy some curiosity.
Lauren Black, Charles River Labs — Customized drugs [4:34] (Cody)
Doctors recently developed a customized drug to save a little girl’s life. And the story of how it happened could offer a possible template for the rapid development of patient-customized treatments. Joining us today to share the story is Lauren Black, Distinguished Scientist in the Scientific advisory services at Charles River Labs. And the story starts in December 2016, when a couple in Colorado learned that their 6-year-old daughter Mila had a rare neurodegenerative disorder called Batten disease. It’s important to understand what this disease is and what it entails, so here’s Lauren Black with the story.
[CLIP 1:00]
Mila had been showing symptoms since she was about 3 years old, but it took doctors a while to diagnose since it’s an ultra-rare disease — in the US, it affects an estimated 2 to 4 births out of every hundred thousand. Here’s Lauren with details on Mila’s diagnosis and treatment.
[CLIP 2:56]
Obviously this is a relatively speedy process for drug discovery and development. But Lauren told us this kind of thing is not without precedent. During the AIDS epidemic in the early 1990s, laws were passed and the building blocks were put into place for a policy that the FDA calls expanded access, sometimes called “compassionate use.” According to the FDA, expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease to gain access to an investigational medical product. That can be a drug, biologic, or medical device, and under the right conditions, patients can get access outside of clinical trials when there aren’t really any alternatives available. Mila’s story is extraordinary because it demonstrates that now, doctors can use designer DNA drugs for individualized therapies in situations like these.
[CLIP 0:38]
And that’s the story of why doctors have high hopes for the future of tailored medicine. Again, our guest was Lauren Black, Distinguished Scientist in the Scientific advisory services at Charles River Labs. You can read all the details of Mila’s story in a paper published in October in the New England Journal of Medicine , and we’ll put a link to that and many other relevant sources in today’s show notes.
[NHTSA]
ASHLEY: Today’s episode is sponsored by the National Highway Traffic Safety Administration. CODY: Everyone knows about the risks of driving drunk. You could get in a crash. People could get hurt or killed. But let’s take a moment to look at some surprising statistics. Almost 29 people in the United States die every day in alcohol-impaired vehicle crashes. That’s one person every 50 minutes. Even though drunk-driving fatalities have fallen by a third in the last three decades, drunk driving crashes still claim more than 10,000 lives each year.
ASHLEY: Many people are unaware that driving while high can be just as dangerous. In 2015, 42% of drivers killed in crashes tested positive for drugs. Not so harmless after all, is it? And get this, from 2007 to 2015, marijuana use among drivers killed in crashes doubled. The truth is driving while high is deadly. So, stop kidding yourself. If you’re impaired from alcohol or drugs, don’t get behind the wheel. If you feel different you drive different. Drive high get a DUI.
CODY: Drive sober or get pulled over.
You Really Can Tell Someone's Sick Just By Looking at Them — https://curiosity.im/2D6XTo8 (Ashley)
ASHLEY: [ad lib it’s sick sesaon] Science says you can trust your instincts: you really can tell if someone’s sick just by looking at them.
For a 2018 study, researchers injected a panel of 16 Caucasian participants with either a placebo or something called a lipopolysaccharide [LIP-puh-POLLY-SACK-a-ride]. That’s a molecule found in the outer membrane of bacteria that can trigger an immune reaction without infecting them — basically, it could make the participants feel sick without actually making them sick.
Those participants’ photos were shown to a much larger group of people, and that group was able to guess whether the people in the photos were sick or healthy a whopping 81 percent of the time. And even though participants did such a good job, the researchers believe this effect is probably stronger in person, since you’ll be able to hear if they sound stuffy or complain about how sick they feel.
So what does a sick face look like? According to the study: pale skin and lips, droopy eyelids and corners of the mouth, a puffy face, red eyes, and the appearance of fatigue. The biggest giveaways were the pale skin and drooping eyes, so if you're trying to dodge a bug, then stay away!
The researchers say that in the future, they'd like to pinpoint early cold and flu warning signs, so people can stay at home before they get too contagious.
CODY: So what was so cool about today’s episode?
[ad lib optional]
CODY: Today’s story about sick faces was written by Reuben Westmaas, and edited by Ashley Hamer, who’s the managing editor for Curiosity.com.
ASHLEY: Today’s episode was scripted, produced, and edited by Cody Gough.
CODY: Join us again tomorrow to learn something new in just a few minutes.
ASHLEY: And until then, stay curious!