The FDA approved labeling changes for elivaldogene autotemcel (eli-cel; Skysona bluebird bio), a one-time gene therapy for patients with early, active cerebral adrenoleukodystrophy (CALD), to reflect new safety information about hematologic malignancy risk, theagency announced
The update restricts the eli-cel indicationopens in a new tab or window to patients without an available human leukocyte antigen (HLA)-matched allogeneic hematopoietic stem cell donor. Given the risk of blood cancer, eli-cel should be used only in CALD patients without suitable alternative treatment options, the agency said.
The notice follows an FDA investigation of reportsopens in a new tab or window of blood cancers -- including life-threatening cases of myelodysplastic syndrome and acute myeloid leukemia -- in patients with early, active CALD who were treated with eli-cel.
In 2022, the FDA granted accelerated approval to eli-celopens in a new tab or window to treat CALD in boys ages 4 to 17 years. Childhood CALD is a rare debilitating form of adrenoleukodystrophy that generally occurs in young boys. It's caused by a mutation in the ABCD1 gene that leads to the overproduction of very long-chain fatty acids primarily in the white matter of the brain and spinal cord that destroys myelin.
When eli-cel was approved, hematologic malignancy was identified as a serious risk and myelodysplastic syndrome was reported in three of 67 patients (4%) across clinical studies. Since then, the FDA received seven more blood cancer reports from clinical trial participants. As of July, hematologic malignancies have been diagnosed in 10 of 67 (15%) participants.
Reports suggest that blood cancer diagnoses occurred from 14 months to 10 years after eli-cell administration, the FDA said. Nine of the 10 patients with blood cancer were treated with allogeneic hematopoietic stem cell transplantation, with or without chemotherapy.
"The malignancies are life-threatening conditions, and one death related to treatment for malignancy has occurred," the agency reported. "One patient developed recurrence of myelodysplastic syndrome after initial treatment, which required re-treatment." Some patients developed cancer before eli-cel had time to provide therapeutic benefit for CALD.
Patients and clinical trial participants receiving eli-cel treatment should be monitored for life for hematologic malignancy, the FDA stated. Patients should complete blood count checks at least every 3 months and have thorough assessments for evidence of clonal expansion or predominance at least twice in the first year after eli-cel administration and annually thereafter. Bone marrow evaluations should be considered as clinically indicated.
Eli-cel's approval included a postmarketing requirement for a 15-year follow-up prospective study to assess long-term safety and the risk of secondary malignancies occurring after treatment. The study includes monitoring for clonal expansion.
A New York billionaire has become obsessed with saving hundreds of rare ostriches from being slaughtered by Canadian officials because he believes they are crucial to groundbreaking medical research.
Supermarket mogul John Catsimatidis, owner of D’agostino and Gristedes, told Daily Mail he is 'outraged' at what is happening in Canada and is trying to prevent the flock of birds from being slaughtered by the Canadian Food Inspection Agency (CFIA).
Ostrich eggs contain high concentrations of IgY antibodies, which are being studied for their potential to provide immunity from, and treatment of, diseases and viral infections.
'These are ancient animals, beautiful animals that are one million years old and could hold the key to medical breakthroughs,' Catsimatidis told Daily Mail.
The nine foot African natives have become a passion project for the supermarket mogul after he heard about a family's plight to save the animals on their farm.
Katie Pasitney, whose family owns Universal Ostrich Farm in Edgewood, British Columbia, recently shared her plight on WABC AM radio.
'We have millions of people in North America who have a heart and feel,' he said.
The supermarket mogul brought in Dr. Mehmet Oz, the head of Medicare, and Robert Kennedy Jr., Trump's health secretary, to help with his mission.
He also wrote a personal letter to the Canadian Prime Minister Mark Carney citing an 'urgent humanitarian appeal regarding the ostriches'.
He is yet to here back from the PM's office.
'Canada and the United States are partners. We are part of the same America. We're part of North America together. Right? And I appeal to the prime minister for a little bit of common sense,' Catsimatidis said.
The ostriches at the Universal Ostrich Farm in Edgewood, British Columbia
Katie Pasitney calls the ostriches 'family' and says that each ostrich has their own name and personality
What's happening with these iconic ancient animals is a travesty, 400 innocent souls who'll be lost due to lack of common sense,' he said.
The trouble on the farm started in mid-December when 69 ostriches died from what was believed to be H5N1 virus, or bird flu.
Pasitney said that someone made an anonymous call to the Canadian Food Inspection Agency claiming that their birds had the avian influenza.
Officials from the agency came down to their farm and tested two dead birds. After the results came back positive for the H5N1 or HPAI virus the farm was put under quarantine.
Ostrich eggs (pictured) are believed to carry antibodies that could provide valuable scientific insight into immunity in helping to cure disease
'They only tested two deceased birds based on a PCR test not all the others so we don't know what they died from,' she said.
She explained that a blood test or tissue test on the deceased birds were never performed which would have given them a more accurate reading.
However, the test results never reached the accredited lab in Winnipeg, Canada because officials already signed the kill order to kill every animal on the property.
To make matters worse, she said they were ordered to kill all the animals themselves and dispose of them on their own.
If the farm goes against the quarantine rules and conducts any of their own testing they will get fined $200,000 per bird and would face up to six months in jail, per animal.
Pasitney said it has been 195 days since their last bird died, all the ostriches on the farm have been healthy and they are still doing business with their trading partners.
Pasitney said the ostriches all have their own unique personalities
'When we asked them (CFIA) to test our healthy animals they denied us that right. We have been fighting with them for seven months,' she said.
'And, in the last four-and-a-half months they have not been here to check on the animals though they want to say we have H5N1 and they are worried about public health and safety.
'We are 135 kilometers away from any major city. We are not near any poultry facilities, and we have not stopped trading with our different countries.'
Catsimatidis, who also owns WABC Radio and Red Apple Media, understands the clock is ticking. He has been lobbying aggressively on the matter calling it 'a moral crisis', and explaining that 'lives are on the line'.
'The U.S. government has offered every recourse to test these birds and see if they pose any threat before they’re unnecessarily slaughtered,' he said.
'This is a death sentence for 400 innocent animals who could be saved with a simple test.'
Pasitney, who like other farmers are concerned about their future and expressed her gratitude towards Catsimatidis who brought her family's farm to the attention of Dr. Oz and RFK.
'We developed a relationship over saving animals,' she said. 'If I didn't have this strength and support and his voice I don't know if we would still be here.'
Supermarket mogul John Catsimatidis has been lobbying to save the ostriches from slaughter
The letter that Catsimatidis sent to the Prime Minister of Canada
The second page of the letter that the supermarket mogul sent. He has not heard back from Canadian Prime Minister Mark Carney
Pasitney grew up with the ostriches and said they have been part of her family for decades.
She shared that each bird has their own name and unique personality. Some bear the more traditional names such as, Frank, Betsy, Bernie, Troy, and Connor. While others bear a quirkier moniker.
'Regretta because you will regret when she pecks you,' Pastiney said. 'She has a really hard peck.'
'Then there is Q-Tip. He's got a really long neck and a really white fluffy head so he looks like a Q-Tip.'
The birds possess a distinctive long neck, long legs and a small head. They stand at at towering 9 feet high, and have two toes on each foot.
They are known to be very fast runners - they can run up to 45 miles per hour and they peck all day long, Pastiney shared.
'They peck an average of 4,000 times a day and mostly do it out of curiosity, and to also taste and to feel,' she said.
On July 15, the Federal Court of Appeal heard arguments in the case and the court will soon determine if the birds will live or die.
The wait has been agonizing for Pasitney and her family.
'We are waiting for a verdict right now. It can be any day and it has been emotional for our whole family.
'This is our livelihood these animals. They have been raised on the farm for the past 35 years.'
She added: 'We would never be able to start over.'
Iran threatened on Saturday to block a corridor planned in the Caucasus under a regional deal sponsored by U.S. President Donald Trump, Iranian media reported, raising a new question mark over a peace plan hailed as a strategically important shift.
A top Azerbaijani diplomat said earlier that the plan, announced by Trump on Friday, was just one step from a final peace deal between his country and Armenia, which reiterated its support for the plan.
The proposed Trump Route for International Peace and Prosperity (TRIPP) would run across southern Armenia, giving Azerbaijan a direct route to its exclave of Nakhchivan and in turn to Turkey.
Brazilian President Luiz Inacio Lula da Silva spoke with Russia's President Vladimir Putin on Saturday for about 40 minutes, the Brazilian presidential palace said, adding that Putin shared information about his discussions with the United States and "recent peace efforts between Russia and Ukraine."
The leaders also discussed their cooperation in the BRICS group of emerging countries and "discussed the current international political and economic scenario," according to the statement.
The conversation with Lula is the latest of a flurry of calls between Putin and foreign leaders in recent days ahead of the Russian president's expected meeting with U.S. President Donald Trump next week.
Putin spoke to the leaders of China and India, both also part of the BRICS group of developing nations, and other presidents from Central Asia and Europe on Friday to brief them on his contacts with the United States about the war in Ukraine.
Lula has been in a public spat with Trump since the U.S. imposed a 50% tariff on the imports of Brazilian goods, which Trump linked to an alleged "witch hunt" against his ally and Brazil's former right-wing President Jair Bolsonaro. U.S. imports of some Brazilian products, such as orange juice and aircraft, received a lower rate.
Lula told Reuters on Wednesday he planned to call the leaders of the BRICS countries, which also include South Africa, to discuss a joint response to Trump's tariffs on U.S. imports. The Brazilian leader spoke with Indian Prime Minister Narendra Modi on Thursday.
Trump has threatened BRICS nations with additional 10% tariffs last month, as the group gathered in a summit in Rio de Janeiro in July.
Vinay Prasad is returning to his role overseeing vaccine, gene therapy and blood product regulation at the U.S. Food and Drug Administration a little more than a week after he left the agency.
"At the FDA's request, Dr. Vinay Prasad is resuming leadership of the Center for Biologics Evaluation and Research," U.S. Health and Human Services spokesperson Andrew Nixon said in an emailed statement to Reuters.
Prasad left the agency on July 30 after just a few months as director of the center. STAT News first reported the return of Prasad.
Prasad, an oncologist who was a fierce critic of U.S. COVID-19 vaccine and mask mandates, was named the center's director by FDA Commissioner Marty Makary in May.
Criticism of Prasad's tenure intensified around the agency's handling of a gene therapy for Duchenne muscular dystrophy from Sarepta Therapeutics. The FDA-approved therapy played a role in the death of two teens who had advanced DMD.
After a third death in a separate experimental gene therapy from the company, the FDA asked Sarepta on July 18 to stop all shipments of the approved DMD therapy, saying it had safety concerns.
The FDA changed course on Sarepta on July 28 and said shipments to the main group of patients for the drug could restart.
Laura Loomer, a far-right activist and an ally of U.S. President Donald Trump, posted a blog on July 20 in which she called Prasad a "progressive leftist saboteur" who was undermining the agency's work.
Prasad was a physician who joined the agency from the University of California, San Francisco.
He has had stints at the National Cancer Institute and the National Institutes of Health.
The FDA and other health agencies have seen multiple shake-ups in recent months under the leadership of U.S. Health Secretary Robert F. Kennedy Jr.
The EPA is ending its contract with unionised employees, aiming to weaken collective bargaining, fulfilling President Trump's executive order from March. The union, representing 8,000 EPA employees, plans legal action, arguing the move violates free speech and bargaining obligations. EPA aims to cut its workforce by at least 23% and close its scientific research office; unions are challenging Trump's actions in court.
The US Environmental Protection Agency (EPA) moved to end its contract with unionised employees, according to the union’s president, the latest action in President Donald Trump’s push to weaken collective bargaining across the federal government.
The union, which represents 8,000 EPA employees, is planning a legal response to the decision, said Mr Justin Chen, president of the agency’s chapter of the American Federation of Government Employees, in a statement on Aug 8.
The decision gets Mr Trump closer to his goal to strip hundreds of thousands of federal workers of the ability to collectively bargain with US agencies.
Eliminating union deals would allow agencies to more easily fire or discipline employees, according to attorneys representing federal workers.
“EPA is working to diligently implement President Trump’s Executive Orders with respect to AFGE, including ‘Exclusions from Federal Labor-Management Relations Programs’, in compliance with the law,” an EPA spokesperson said in a statement, referring to the executive order Mr Trump issued in March.
The order involves removing collective bargaining rights at more than 30 federal agencies, including the EPA, and is currently being challenged in court by unions who say it violates free speech and obligations to bargain with workers.
The EPA had more than 16,000 employees as of March 2025, according to federal HR records. That figure does not include employees that accepted buyouts. The agency said in July it would cut the size of its workforce by at least 23 per cent and close its scientific research office as part of Mr Trump’s
Under a new ordinance in a New Jersey town, parents can be fined and even charged for their children’s unruly behavior.
The Township of Gloucester Minors and Parents Responsibility Ordinance stipulates consequences, including jail time, for parents and guardians who “fail to prevent their children from breaking the law,” according to a news release from police.
The ordinance was passed by the Township Council July 28 and was introduced after incidents of public disturbance involving kids at events like last year’s Gloucester Township Day and Drone Show.
During that event, a large brawl involving teens broke out, and 11 people were arrested, including nine teenagers, according to NJ.com.
The Township of Gloucester Minors and Parents’ Responsibility Ordinance stipulates consequences, including jail time, for parents and guardians who “fail to prevent their children from breaking the law.”Gloucester Township Police
Three police officers were also injured during the melee.
The event was postponed this year, the outlet reported, because social media posts suggested similar behavior was planned.
The ordinance includes 28 different offenses that range from felonies to minor infractions such as loitering, breaking curfew or chronic truancy.
The Gloucester Township Police Department is located at the township municipal building.Google Maps
“If a child is repeatedly found guilty in juvenile court, their caretaker could face up to 90 days in prison and/or a fine of up to $2,000,” the department said.
Guardians will be issued a warning before a citation, Gloucester Township Police Chief David Harkins said, according to NewJersey.com.
“Our ordinance was actually sampled from other towns,” he explained. “We’re not necessarily the first, but we’re probably the first bigger town to adopt it.”
Fox News Digital has reached out to the Gloucester Township Police Department for comment.
Gloucester Township is about 15 miles from Philadelphia.
