The promise of gene therapy looms large for families dealing with rare, genetic disorders. Such treatments offer the possibility of one-time cures.
But families and researchers worry such therapies will remain out of reach.
Collectively, about 350 million people worldwide suffer from rare diseases, most of which are genetic. But each of the 7,000 individual disorders affects perhaps a few in a million people or less. So there’s little commercial incentive to develop or bring to market these one-time therapies to fix faulty genes or replace them with healthy ones.
The Associated Press examined what this means for families, scientists and the nascent field of gene therapy.
Here are key takeaways from AP's report.
Camden Alderman was diagnosed as a baby with a rare disease called Wiskott-Aldrich syndrome, which is caused by a mutated gene on the X chromosome. It primarily affects boys – up to 10 out of every million — and can cause frequent infections, eczema and excessive bleeding.
When he was a toddler, doctors removed his spleen because of uncontrolled bleeding. As a young boy, he wound up in the hospital many times and was told he couldn’t play baseball.
His mother Robin Alderman recalls one doctor saying: “Basically, your son’s only chance at a cure is going to be gene therapy.”
He also told her researchers weren’t then accepting U.S. residents into a clinical trial for the treatment, which “just kind of broke my heart,” she said. There's still no clinical trial he can join, and London-based Orchard Therapeutics stopped investing in an experimental treatment for the condition in 2022.
Lacey Henderson’s daughter, 5-year-old Estella, has alternating hemiplegia of childhood, a neurological condition that affects 300 people in the U.S. Estella is cognitively delayed, has limited use of her hands and suffers episodes that temporarily paralyze part or all of her body, Henderson said. Medications can curb symptoms, but there’s no cure.
Her Iowa family raises money through a GoFundMe and a website to develop a gene therapy. They’ve brought in around $200,000.
“We have three different projects with various researchers,” Henderson said. “But the problem is everything is underfunded.”
The amount of work it takes to get from a lab to human testing and through the drug approval process is “incredibly expensive,” said Dr. Donald Kohn, professor of microbiology, immunology and molecular genetics at the University of California, Los Angeles.
In the last couple of years, he said, investment in gene therapy has largely dried up.
“If you have to spend $20 million or $30 million to get approval and you have five or 10 patients a year, it’s hard to get a return on investment,” Kohn said. “So we have successful, safe therapies, but it’s more the financial, economic elements that are limiting them from becoming approved drugs."
Ultimately, most biotechnology companies become public and must focus on shareholder profit, said Francois Vigneault, CEO of the Seattle biotech Shape Therapeutics.
“The board is the thing that gets in the way; they’re trying to maximize gain,” said Vigneault, whose company is privately held. “That’s just greed. That’s just incentive misaligned between corporate company structure and what we should do that’s good for the world.”
In the U.S., for example, The Bespoke Gene Therapy Consortium was organized by the Foundation for the National Institutes of Health and includes the FDA, various NIH institutes and several drug companies and nonprofits. Its goals include supporting a handful of clinical trials and streamlining regulatory processes.
Researchers are trying to address the problem scientifically. Dr. Anna Greka said the Broad Institute of MIT and Harvard has launched an effort to look at the commonalities behind various conditions — or nodes, which can be likened to branches meeting at a tree trunk. Fixing the nodes with gene therapies or other treatments, rather than particular “misspellings” in DNA responsible for one disorder, could potentially address multiple diseases simultaneously.
“What this does is it increases the number of patients who can benefit from the therapy,” said Greka, a Broad member.
Still, scientists say these efforts don’t negate the larger financial quandary surrounding therapies for rare diseases, and it may be a while before such gene therapies are available to patients worldwide.
“This is a massive challenge, and I’m not entirely sure we’re going to be able to overcome it,” said Claire Booth of University College London. “But we have to give it a go because we’ve spent decades and millions making these transformative treatments. And if we don’t try, then it feels like the end of an era.”
The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute’s Science and Educational Media Group. The AP is solely responsible for all content.
FILE - This undated image made available by the National Human Genome Research Institute shows the output from a DNA sequencer. Collectively, about 350 million people worldwide suffer from rare diseases, most of which are genetic. But each of the 7,000 individual disorders affects perhaps a few in a million people or less. So there’s little commercial incentive to develop or bring to market these one-time therapies to fix faulty genes or replace them with healthy ones. (NHGRI via AP, File)
The satirical news publication The Onion won the bidding for Alex Jones' Infowars at a bankruptcy auction, backed by families of Sandy Hook Elementary School shooting victims whom Jones owes more than $1 billion in defamation judgments for calling the massacre a hoax.
The purchase turns over Jones’ company, which for decades has peddled in conspiracy and misinformation, to a humor website that plans to relaunch the Infowars platform in January as a parody. Within hours of the sale’s announcement Thursday, Infowars’ website was down and Jones was broadcasting from what he said was a new studio location.
“The dissolution of Alex Jones’ assets and the death of Infowars is the justice we have long awaited and fought for,” Robbie Parker, whose daughter Emilie was killed in the 2012 shooting in Connecticut, said in a statement provided by his lawyers.
The Onion acquired the conspiracy theory platform’s website; social media accounts; studio in Austin, Texas; trademarks; and video archive for an undisclosed sales price.
The satirical outlet — which carries the banner of “America’s Finest News Source” on its masthead — was founded in the 1980s and for decades has skewered politics and pop culture, including making Jones a frequent target of mocking articles. Mass shootings in the U.S., such as the Sandy Hook attack, are often followed by The Onion publishing slightly updated versions of one of its most well-known recurring pieces of satire: "'No Way to Prevent This,' Says Only Nation Where This Regularly Happens.”
“No price would be too high for such a cornucopia of malleable assets and minds,” The Onion said in its satirical post on the sale. “And yet, in a stroke of good fortune, a formidable special interest group has outwitted the hapless owner of InfoWars (a forgettable man with an already-forgotten name) and forced him to sell it at a steep bargain: less than one trillion dollars.”
On his live broadcast, Jones was angry and defiant, vowing to challenge the sale and auction process in court. He later announced his show was being shut down. Jones, who had told listeners for days that he had a new studio already set up nearby, then resumed his broadcast on a different social media account.
“This is a total attack on free speech, the deep state is completely out of control,” Jones told his listeners, referencing conspiracy theories. “This the tyranny of the New World Order, desperate to silence the American people."
He claimed the takeover by The Onion was premature because the bankruptcy judge had not yet signed off on the winning bid.
A Jones-affiliated company named by the bankruptcy trustee as the backup bid requested an immediate status conference, citing “the apparent defects in the sale process, including changing the procedures, lack of transparency, and inaccurate disclosures to interested bidders." A hearing was scheduled for Thursday afternoon in Houston.
The Onion, based in Chicago, consulted on the bidding with some of the Sandy Hook families that sued Jones for defamation and emotional distress in lawsuits in Connecticut and Texas, lawyers for the families said.
“Our clients knew that true accountability meant an end to Infowars and an end to Jones’ ability to spread lies, pain and fear at scale,” said Christopher Mattei, a lawyer for the families.
Ben Collins, CEO of The Onion’s parent company, Global Tetrahedron, told The Associated Press in a video interview that it will relaunch the Infowars website in January with satire aimed at conspiracy theorists and right-wing personalities, as well as educational information about gun violence prevention from the group Everytown for Gun Safety. Collins would not disclose the sale price.
“We thought it would be a very funny joke if we bought this thing, probably one of the better jokes we’ve ever told,” Collins said. “The (Sandy Hook) families decided they would effectively join our bid, back our bid, to try to get us over the finish line. Because by the end of the day, it was us or Alex Jones, who could either continue this website unabated, basically unpunished, for what he’s done to these families over the years, or we could make a dumb, stupid website, and we decided to do the second thing.”
Sandy Hook families sued Jones and his company for repeatedly saying on his show that the shooting that killed 20 children and six educators in Newtown, Connecticut, was a hoax staged by crisis actors to spur more gun control. Parents and children of many of the victims testified that they were traumatized by Jones’ conspiracies and threats by his followers.
The Onion bills itself as “the world’s leading news publication, offering highly acclaimed, universally revered coverage of breaking national, international, and local news events” and says it has 4.3 trillion daily readers. Recent headlines have included, “Trump Boys Have Slap Fight Over Who Gets To Run Foreign Policy Meetings,” “Oklahoma Law Requires Ten Commandments To Be Displayed In Every Womb” and “Man Forgetting Difference Between Meteoroid, Meteorite Struggles To Describe What Just Killed His Dog.”
Sealed bids for the private auction were opened Wednesday. Both supporters and detractors of Jones expressed interest in buying Infowars, and Jones had told listeners the outcome would determine whether he stayed on the Infowars platform.
The bankruptcy trustee named First United American Companies, which is affiliated with one of Jones’ product-selling sites, as the backup bid should the sale to The Onion fall through.
Associated Press writer Ken Miller contributed from Oklahoma City.
Global Tetrahedron, LLC, owner of The Onion, is displayed on the entrance screen to the office building that is headquarters to the satirical publication, Thursday, Nov. 14, 2024 in Chicago. The Onion won the bidding for Alex Jones'Infowars at a bankruptcy auction, backed by families of Sandy Hook Elementary School shooting victims whom Jones owes more than $1 billion in defamation judgments for calling the massacre a hoax. (AP Photo/Teresa Crawford)
This undated photo provided by ThreeSixty Asset Advisors shows the Infowars set. (ThreeSixty Asset Advisors via AP)
A copy of the satirical outlet The Onion is seen Thursday, Nov. 14, 2024, in Little Rock, Ark. (AP Photo/Jill Bleed)
FILE - Right-wing conspiracy theorist Alex Jones speaks to the media after arriving at the federal courthouse for a hearing in front of a bankruptcy judge, Friday, June 14, 2024, in Houston. (AP Photo/David J. Phillip, File)
