In a milestone year, gene therapy finds a place in medicine

TECH

In a milestone year, gene therapy finds a place in medicine

2017-12-29 11:09 Last Updated At：18:11

After decades of hope and high promise, this was the year scientists really showed they could doctor DNA to successfully treat diseases. Gene therapies to treat cancer and even pull off the biblical-sounding feat of helping the blind to see were approved by U.S. regulators, establishing gene manipulation as a new mode of medicine.

Almost 20 years ago, a teen's death in a gene experiment put a chill on what had been a field full of outsized expectations. Now, a series of jaw-dropping successes have renewed hopes that some one-time fixes of DNA, the chemical code that governs life, might turn out to be cures.

"I am totally willing to use the 'C' word," said the National Institutes of Health's director, Dr. Francis Collins.

Gene therapy aims to treat the root cause of a problem by deleting, adding or altering DNA, rather than just treating symptoms that result from the genetic flaw.

The advent of gene editing — a more precise and long-lasting way to do gene therapy — may expand the number and types of diseases that can be treated. In November, California scientists tried editing a gene inside someone's body for the first time, using a tool called zinc finger nucleases for a man with a metabolic disease. It's like a cut-and-paste operation to place a new gene in a specific spot. Tests of another editing tool called CRISPR, to genetically alter human cells in the lab, may start next year.

"There are a few times in our lives when science astonishes us. This is one of those times," Dr. Matthew Porteus, a Stanford University gene editing expert, told a Senate panel discussing this technology last month.

It's a common path for trail-blazing science — success initially seems within reach, setbacks send researchers back to the lab, new understandings emerge over years, and studies ultimately reveal what is safe and effective.

Here is a look at what's been achieved and what lies ahead.

A STRING OF FIRSTS

The year started with no gene therapies sold in the U.S. and only a couple elsewhere. Then the Food and Drug Administration approved the first CAR-T cell therapies, which alter a patient's own blood cells to turn them into specialized cancer killers. They're only for certain types of leukemia and lymphoma now, but more are in the works for other blood cancers.

Last week, the FDA approved Luxturna, the first gene therapy for an inherited disease, a form of blindness. People with it can't make a protein needed by the retina, tissue at the back of the eye that converts light into signals to the brain, enabling sight. The therapy injects a modified virus containing a corrective gene into the retina so the cells can make the protein.

Children who received the treatment told what it was like to gain vision.

"Oh yikes, colors. Colors are super fun," said 13-year-old Caroline Carper of Little Rock, Arkansas. "And the sunshine is blinding."

Gene therapies also showed some promise against a variety of diseases including hemophilia, a blood clotting problem; "bubble boy" disease, where a flawed immune system leaves patients vulnerable to fatal infections, and sickle cell disease, a serious and painful blood disorder common among black people.

It's not all good news, though. The therapies don't work for everyone. They're shockingly expensive. And no one knows how long some results will last, though scientists say the aim is a one-time repair that gets at the root cause.

"The whole promise ... is to cure diseases. It's based on the rationale of fixing the problem," not just improving treatment, said Dr. Carl June, a University of Pennsylvania scientist who pioneered CAR-T therapy.

A NEW FRONTIER: GENE EDITING

In mid-November, Brian Madeux, a 44-year-old Phoenix man with a metabolic disease called Hunter syndrome, had just become the first person to try an experimental gene editing treatment.

"I believe in science," he texted The Associated Press after doctors sent viruses containing a corrective gene and an editing tool through an IV into his body. The hope is that the gene and the editing tool would enter some of his liver cells and insert the instructions needed to start making an enzyme he lacks.

It's not known yet if it worked. Sangamo Therapeutics is testing its therapy in several studies, and independent monitors will help decide when results are released.

"It's a pretty exciting milestone," Collins said, because it shows a way to treat more diseases than ones that can be addressed now by altering blood cells in the lab or injecting genes into the eye.

"You can imagine having a scalable approach to thousands of genetic diseases," he said.

WHAT'S NEXT

Top of Collins' list: muscular dystrophy and sickle cell.

There's been so much progress that the NIH has modified an oversight panel that just a few years ago reviewed every gene therapy experiment in the U.S. Most are considered safe enough to go ahead without the Recombinant DNA Advisory Committee's review. The panel hasn't even met for a year.

When the panel was formed decades ago, "there was a lot of concern that a graduate student could take some of this home and create a monster in his basement," said one panel member, Boston scientist Dr. Howard Kaufman.

Those fears have eased, he said.

"There's no monsters that have materialized from this."

A massive Powerball win draws attention to a little-known immigrant culture in the US

2024-05-02 06:36 Last Updated At：06:41

PORTLAND, Ore. (AP) — Cheng “Charlie” Saephan wore a broad smile and a bright blue sash emblazoned with the words “Iu-Mien USA” as he hoisted an oversized check for $1.3 billion above his head.

The 46-year-old immigrant's luck in winning an enormous Powerball jackpot in Oregon earlier this month — a lump sum payment of $422 million after taxes, which he and his wife will split with a friend — has changed his life. It also raised awareness about Iu Mien people, a southeast Asian ethnic group with origins in China, many of whose members fled from Laos to Thailand and then settled in the U.S. following the Vietnam War.

“I am born in Laos, but I am not Laotian,” Saephan told a news conference Monday at Oregon Lottery headquarters, where his identity as one of the jackpot's winners was revealed. “I am Iu Mien.”

During the Vietnam War, the CIA and U.S. military recruited Iu Mien in neighboring Laos, many of them subsistence farmers, to engage in guerrilla warfare and to provide intelligence and surveillance to disrupt the Ho Chi Minh Trail that the North Vietnamese used to send troops and weapons through Laos and Cambodia into South Vietnam.

After the conflict as well as the Laotian civil war, when the U.S.-backed government of Laos fell in 1975, they fled by the thousands to avoid reprisals from the new Communist government, escaping by foot through the jungle and then across the Mekong River into Thailand, according to a history posted on the website of Iu Mien Community Services in Sacramento, California. More than 70% of the Iu Mien population in Laos left and many wound up in refugee camps in Thailand.

Thousands of the refugees were allowed to come to the U.S., with the first waves arriving in the late 1970s and most settling along the West Coast. The culture had rich traditions of storytelling, basketry, embroidery and jewelry-making, but many initially had difficulty adjusting to Western life due to cultural and language differences as well as a lack of formal education.

There are now tens of thousands of Iu Mien — pronounced “yoo MEE’-en” — in the U.S., with many attending universities or starting businesses. Many have converted to Christianity from traditional animist religions. There is a sizeable Iu Mien community in Portland and its suburbs, with a Buddhist temple and Baptist church, active social organization, and businesses and restaurants.

Cayle Tern, president of the Iu Mien Association of Oregon, arrived in Portland with his family in 1980, when he was 3 years old. He is now running for City Council. His father and uncle assisted American forces in Laos and he was born as his mother fled to a refugee camp in Thailand.

Many Iu Mien in the U.S. have similar stories, and Saephan’s Powerball win sheds light on the new lives they have made in Oregon and elsewhere after such trauma, he said. Tern knows all three of the Powerball winners, he said.

“You know, I think for me it’s more than just about the money. ... We’ve been here since the late '70s, but very little is known of us," he said while sitting in his uncle’s restaurant in Troutdale, a Portland suburb.

“This attention that we’re getting — people are interested in what the community is, who we are, where we came from. That is to me is equally special.”

Saephan, 46, said he was born in Laos and moved to Thailand in 1987, before immigrating to the U.S. in 1994. He graduated from high school in 1996 and has lived in Portland for 30 years. He worked as a machinist for an aerospace company.

He said Monday that he has had cancer for eight years and had his latest chemotherapy treatment last week.

“I will be able to provide for my family and my health,” he said, adding that he’d “find a good doctor for myself.”

Saephan, who has two young children, said that as a cancer patient, he wondered, “How am I going to have time to spend all of this money? How long will I live?”

He said he and his 37-year-old wife, Duanpen, are taking half the money, and the rest is going to a friend, Laiza Chao, 55, of the Portland suburb of Milwaukie. Chao had chipped in $100 to buy a batch of tickets with them.

Chao, was on her way to work when Saephan called her with the news: “You don’t have to go anymore,” he said.

In the weeks leading up to the drawing, he wrote out numbers for the game on a piece of paper and slept with it under his pillow, he said. He prayed that he would win, saying, “I need some help — I don’t want to die yet unless I have done something for my family first.”

The winning Powerball ticket was sold in early April at a Plaid Pantry convenience store in Portland, ending a winless streak that had stretched more than three months. The Oregon Lottery said it had to go through a security and vetting process before announcing the identity of the person who came forward to claim the prize.

Under Oregon law, with few exceptions, lottery players cannot remain anonymous. Winners have a year to claim the top prize.

The jackpot had a cash value of $621 million before taxes if the winner chose to take a lump sum rather than an annuity paid over 30 years, with an immediate payout followed by 29 annual installments. The prize is subject to federal taxes and state taxes in Oregon.

The $1.3 billion prize is the fourth largest Powerball jackpot in history, and the eighth largest among U.S. jackpot games, according to the Oregon Lottery.

The biggest U.S. lottery jackpot won was $2.04 billion in California in 2022.

Johnson reported from Seattle.