Current:Home > ScamsFDA approves gene-editing treatment for sickle cell disease-LoTradeCoin
FDA approves gene-editing treatment for sickle cell disease
View Date:2024-12-23 19:49:24
The U.S. Food and Drug Administration on Friday approved a landmark gene-editing treatment for sickle cell disease, a painful condition that affects approximately 100,000 people in the United States, predominantly people of color. The innovative therapy promises to repair the gene responsible for the disease.
The breakthrough offers a beacon of hope for Johnny Lubin, a 15-year-old from Connecticut who has lived with the debilitating effects of the disease. He inherited the sickle cell gene from both of his parents and has experienced severe pain and health complications since infancy.
Red blood cells, which are normally donut-shaped, bend into inflexible sickle shapes, causing them to pile up inside blood vessels and prevent the normal delivery of oxygen in the body. Complications include bone deterioration, strokes and organ failure.
Doctors told Lubin he would not live past 40.
"I was starting to get a little bit scared. Like I actually did want to live past 40," he said.
For more than a decade, Lubin was in and out of the hospital. He said he would count how many times he had been in each hospital room and at one point he realized he had been in every room on the floor.
Johnny's parents, Fabienne and J.R. Lubin, were desperate for a solution when they learned about a cutting-edge clinical trial involving gene editing, a process not requiring a donor.
First, stem cells were removed from Lubin's bone marrow and he was given chemotherapy to help wipe out the abnormal cells.
Then, in a laboratory, the editing technology called CRISPR was used to increase the amount of a protective form of hemoglobin, a protein that picks up oxygen from lungs and delivers it throughout the body — that protective form usually diminishes after birth. The cells were then infused back into Lubin's bloodstream.
Dr. Monica Bhatia, who is Johnny's doctor and the chief of pediatric stem cell transplantation at NewYork-Presbyterian/Columbia University Irving Medical Center, said by editing the cell, you're reprogramming cells to produce fetal hemoglobin.
"It's been widely known that fetal hemoglobin is somewhat protective and those who have higher levels of fetal hemoglobin tend to have less severe symptoms of sickle cell disease," she said.
"You're changing somebody's DNA. So obviously you wanna make sure that the corrections you're making are, are the ones you want," said Bhatia.
After a challenging five weeks in the hospital and a six-month absence from school, Lubin has drastically improved health and prospects for a longer life.
"I thought that was pretty cool how I have like new cells and I honestly hoped, you know, I could get, you know, some super powers from it, you know, maybe become a superhero, you know, like genetically engineered," Lubin said.
The treatment, called Casgevy, was developed by the Boston-based Vertex Pharmaceuticals and CRISPR Therapeutics.
Patients will have to be followed long-term before the experts call this a cure. Gene editing is expected to cost several million dollars per patient and may not be appropriate for everyone who has sickle cell disease. It would also not prevent the gene from being passed down to future generations.
Jon LaPookDr. Jonathan LaPook is the chief medical correspondent for CBS News.
TwitterveryGood! (2)
Related
- Ryan Reynolds Clarifies Taylor Swift’s Role as Godmother to His Kids With Blake Lively
- North Korea makes first comments on U.S. soldier who crossed the border
- Family of pregnant mother of 3 fatally shot by police in Denver suburb sues
- 166-year-old San Francisco luxury store threatens to close over unsafe street conditions
- Jared Goff stats: Lions QB throws career-high 5 INTs in SNF win over Texans
- Britney Spears’ Lawyer Previously Detailed Plan for Sam Asghari Prenup to Protect Her “Best Interests”
- Biden will use Camp David backdrop hoping to broker a breakthrough in Japan-South Korea relations
- Stock market today: Asia follows Wall Street lower after Fed’s notes dent hopes of rate hikes ending
- Richard Allen found guilty in the murders of two teens in Delphi, Indiana. What now?
- Nicaraguan government seizes highly regarded university from Jesuits
Ranking
- Michael Jordan and driver Tyler Reddick come up short in bid for NASCAR championship
- South Dakota state senator resigns and agrees to repay $500,000 in pandemic aid
- Tampa Bay Rays' Luke Raley hits unique inside-the-park HR, ball bounces off top of wall
- Ex-Anaheim mayor to plead guilty in federal corruption case over Angel Stadium sale
- Eminem, Alanis Morissette, Sheryl Crow, N.W.A. and Janet Jackson get Songwriters Hall of Fame nods
- Activists campaign for shackled elderly zoo elephants to be released in Vietnam
- Our favorite product launches from LG this year—and what's coming soon
- Michael Parkinson, British talk show host knighted by Queen Elizabeth II, dies at 88
Recommendation
-
SNL's Chloe Fineman Says Rude Elon Musk Made Her Burst Into Tears as Show Host
-
'Suits' just set a streaming record years after it ended. Here's what's going on
-
Firefighters battling lightning-sparked blazes in Northern California get help from light rain
-
Family of U.S. resident left out of prisoner deal with Iran demands answers from Biden administration
-
Shel Talmy, produced hits by The Who, The Kinks and other 1960s British bands, dead at 87
-
Study finds ‘rare but real risk’ of tsunami threat to parts of Alaska’s largest city
-
Woman sentenced to 25 years in prison for murdering victim whose headless body was found in a park
-
Mississippi issues statewide burn ban at state parks and fishing lakes