On today’s episode of the 5 Things podcast: New gene therapy treatment promises to transform rare disease

Patient safety reporter Karen Weintraub explains. Plus, Yellowstone National Park remains closed amid severe flooding, Congress reporter Candy Woodall has the latest progress report on gun legislation, Trump’s endorsed candidates have mixed results in primaries and the Stanley Cup Final begins.

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Hit play on the player above to hear the podcast and follow along with the transcript below. This transcript was automatically generated, and then edited for clarity in its current form.

Taylor Wilson:

Good morning. I’m Taylor Wilson, and this is 5 Things you need to know Wednesday, the 15th of June, 2022. Today, the promise of a new gene therapy, plus the latest from a flooded Yellowstone and more.

Here are some of the top headlines:

  1. The federal reserve will reveal today whether it’ll raise interest rates more sharply than previously expected. The fed closes its two day meeting after a massive stock selloff last week, following a consumer price index report that revealed inflation had not yet peaked.
  2. Thieves have stolen 20 freight containers loaded with gold, silver, and televisions. They broke into a storage area at the Pacific Mexican seaport of Manzanillo.
  3. And Microsoft is shutting down Internet Explorer. Beginning today, those who try to open the application will be directed to the company’s more recent browser, Microsoft Edge.

A new gene therapy treatment is showing promise to transform a rare disease. Patient safety reporter, Karen Weintraub has the details.

Karen Weintraub:

Yeah, so the potential of gene therapy is incredible and we’re still just at the very, very beginning stages of it. The idea is that people born with genetic mutations that cause disease – sickle cell disease, cystic fibrosis, and this one that I talk about in this news story called beta thalassemia – can cause really terrible side effects, terrible diseases, and also can be fatal or cut lives short. So with gene therapy, they can literally go in, alter the genes and change the underlying disease so that theoretically it doesn’t exist anymore. Gene therapy hasn’t been around long enough to know if this is permanent, but it seems to be in this trial that I talk about in this story, a number of people have been essentially cured for seven years. They generally don’t study longer than that, but that’s certainly a long period of time.

Gene therapy has been a dream for at least 40 years, and there have been a lot of challenges in getting to this point. It’s still not perfect. It’s still challenging to make it work. And unfortunately, every disease sort of needs its own attention. It’s not like they can come up with one gene therapy that solves every disease.

So it’s going to be a lot of slow chipping away at a lot of these diseases. The disease I write about called beta thalassemia is a blood disorder. So they can take blood stem cells out of a person and alter them in a lab and then put them back. And so it’s easier, it’s safer in a lot of ways, they can check and make sure that the edits are correct before they put them back. Other genes are going to have to be corrected within the body. So that’s a little scarier, a little riskier, although they’re working on improving that technology as well.

The costs for this currently are astronomical. We’re talking $2 million a treatment. For something like beta thalassemia, where people need very expensive therapies to … It’s a blood disorder. You need transfusions in order … the blood doesn’t work properly, doesn’t carry oxygen. So, people get very exhausted. They get transfusions every month from other people to provide that oxygen they need in their blood, but it also provides too much iron. And so they need medication chelation therapy to get rid of that iron. That therapy is extremely expensive. And so a one shot gene therapy at $2 million sounds extremely expensive, but actually over somebody’s lifetime would be cheaper than paying for the chelation therapy. So, that’s the question. Can we afford these treatments? Can we afford if they’re … but in the end over decades, they may be cheaper. And hopefully over time it will also come down, the cost will come down as well.

Taylor Wilson:

For Karen’s full story, click a link in today’s episode description.

Yellowstone National Park remains closed today. Dangerous floods and mudslides have wiped out roads, destroyed homes and even ripped apart bridges around the park. Some 10,000 visitors have also been evacuated this week. The park superintendent, Cam Sholly, said officials are still assessing overall damage.

Cam Sholly:

All visitors are currently out of Yellowstone. Because it’s mid June, because Yellowstone has a lot of back country use, we had a large number of back country campers and users that we’ve tracked, have made contact with. We have put a full closure of the Yellowstone back country into place, and we have contacted or know the whereabouts of every back country user currently in Yellowstone. Right now there’s only one group remaining in the Northern range. We have made contact with them. We were prepared to do helicopter evacuations if necessary. That hasn’t been necessary up to this point. When the water subsides probably early next week, we will be pulling together a large number of people from different agencies around the country to come to Yellowstone and help us assess what the damage is to various infrastructure in the park.

Taylor Wilson:

Historic flood waters may have forever altered the park’s terrain. They pushed a popular fishing river off course, potentially permanently and may force roadways to be rebuilt in new places. Floods came from heavy rains and rapid snow melt over the past week.

What’s next for gun legislation? In the wake of the Uvalde Elementary School mass shooting, there’s been a renewed push for gun reform in both the House and Senate, but hangups on bipartisan progress remain. Congress reporter Candy Woodall has more.

Candy Woodall:

One of the hurdles in the House, as Majority Whip James Clyburn told me, there are members who are reticent because of the amount of time that would be allowed for background checks. The Senate framework enhances the background checks for 18 to 21 year olds. And there are Democrats in the House who want to know how long will people have to conduct those background checks.

One of the reasons that Rep. Clyburn raises a concern is because in a mass shooting in Charleston a while back, the gunman was able to get a gun because three days had expired on his background check time. And so the majority whip is saying we don’t want to create in a sense, another loophole that would allow people to acquire guns, even if they shouldn’t just because the clock ran out on a background check. That’s one hurdle.

Another hurdle that really both chambers are running against is the clock. Senate leaders have imposed their own deadline that they want to get this done before July 4th and Senate Leader Schumer said he wants to put this on the floor for a vote next week. They’re trying to do this before lawmakers go on recess for the July 4th holiday. And yesterday, Sen. Dick Durbin said they’re concerned there could be another national issue that could take attention away from the gun issue. And they don’t want to lose the moment on getting something done.

Right now they’re writing the legislative language. So this is essentially taking an idea that was the framework into an actual bill. The Judiciary Committee is helping them write that. And once they have that language done, they’re even talking about bypassing some of the committee processes and just getting it straight to the floor, which again, like I said, Majority Leader Chuck Schumer said he wants to get this bill on the Senate floor for a vote next week. After that, it would go to the House, and then, if the House passes it and there’s nothing else that needs done to it in the Senate, it would go to the President’s desk – is the best way I’m understanding it at this point, like I said, procedurally, what they’ve said so far, but there could be changes.

It was another primary Tuesday, yesterday as four states sent voters to the polls. In South Carolina, voters faced a pair of candidates endorsed by former President Donald Trump in congressional races. And they went one in one. Earlier results showed Republican Congresswoman, Nancy Mace beat out former State Rep. Katie Arrington, who was backed by Trump. But Congressman Tom Rice got thrashed by State Rep. Russell Fry. Rice was one of 10 house Republicans who voted to impeach Trump. Fry was endorsed by Trump. Elsewhere in Nevada, former State Attorney General Adam Laxalt won the Republican primary for a US Senate seat. Laxalt is supported by both Trump and Senate Republican Leader Mitch McConnell. His win sets up a major fall race with incumbent Democrat, Catherine Cortez Masto in a split 50/50 Senate.

The Stanley Cup Final begins today. The Tampa Bay Lightning travel to Denver to take on the Colorado Avalanche in Game 1 of the hockey finals. The Lightning are looking for their third consecutive Stanley Cup. They’ve become the first team to do so since the New York Islanders won four in a row in the early 1980s. As for the Avs, they’ve not won it all since 2001. The Lightning come into the series fresh off a 4-2 win over the New York Rangers in the Eastern Conference finals, while the Avalanche swept the Edmonton Oilers in four straight games out West. You can tune in tonight at 8:00 PM Eastern, 5:00 Pacific on ABC.

Thanks for listening to 5 Things. You can find us on whatever your favorite podcast app is, seven mornings a week. Thanks to PJ Elliott for his great work on the show, and I’m back tomorrow with more of 5 Things from USA TODAY.

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