Baby saved by gene-editing therapy ‘graduates’ from hospital, goes home
Children’s Hospital of Philadelphia
(PHILADELPHIA) — KJ Muldoon, a 10-month-old baby who sparked nationwide headlines after receiving a first-of-its kind gene-editing treatment, was released from the hospital this week.
KJ has spent the majority of his life at Children’s Hospital of Philadelphia after being diagnosed with a one-in-a-million, deadly genetic disease shortly after birth. Working quickly, his doctors were able to use new gene-editing technology called CRISPR, designing a bespoke treatment just for him.
The treatment, first infused into his body at seven months old, seems to have worked. KJ’s body, which was fighting a toxic buildup of ammonia, began to thrive and he quickly gained weight appropriate for a baby of his age.
Wearing a cap and gown to symbolize his “graduation” from the hospital, baby KJ was discharged home to his parents and siblings on Tuesday after spending 307 days at Children’s Hospital of Philadelphia. Doctors and nurses gathered for a “clap out” on his way out the hospital doors, and he was escorted home by local law enforcement.
KJ’s metabolic condition, called carbamoyl-phosphate synthetase 1 deficiency, affects about 1 in 1.3 million people. The disease kills 50% of babies by early infancy.
KJ “had the most severe variant,” Dr. Ahrens-Nicklas, one of KJ’s doctors at Children’s Hospital of Philadelphia, told ABC News.
“This meant that we had to expedite the pathway for personalized therapy we were already working on,” he said.
Gene therapy treatments have already been approved for more common genetic diseases, including the blood disorders sickle cell disease and beta thalassemia, which affect tens of thousands of patients in the U.S. Those treatments are sold by major pharmaceutical and biotechnology companies.
KJ’s disease is so rare that his doctors were on their own. But thanks to the technology available at Children’s Hospital of Philadelphia, his doctors believed they could use a streamlined approach to make their own bespoke therapy, in-house.
CRISPR gene-editing technology was the perfect tool for a rare genetic disease like KJ’s, and potentially future babies born with slightly different genetic errors.
“Think of it like a GPS signal,” Dr. Kiran Musunuru, director of the Penn Cardiovascular Institute’s Genetic and Epigenetic Origins of Disease Program, told ABC News. “You can change where the GPS is going depending on what specific sequence of genes you want to change.”
Musunuru says there is still a lot of work to be done on this bespoke treatment to make it feasible, but he is hopeful that more babies with ultra-rare conditions can be treated this way.
ABC’s Dr. Keerthana Pakanati contributed to this report.
(WASHINGTON) — In a major policy change, the Food and Drug Administration has announced a plan to limit access to future COVID-19 shots only to people over 65 years old or those with an underlying health condition.
This change would apply to any future updated versions of the vaccine, a spokesperson at the department of Health and Human Services told ABC News. For the past several years, COVID shots have been updated ahead of the winter respiratory virus season to better match the evolving virus.
But FDA Commissioner Dr. Martin Makary and FDA vaccine head Dr. Vinay Prasad argued this week that annual booster shots are not worth the risk for otherwise healthy people.
The FDA plans to impose these restrictions on future shots for younger, healthy people, unless vaccine makers are willing to pay for newer and and lengthy placebo-controlled trials.
“We are evaluating the details shared today and discussions with the FDA are ongoing,” Pfizer told ABC News in a statement.
“We appreciate the FDA’s clear guidance and remain committed to working with the Agency to provide the data they need to ensure access for Americans,” Moderna told ABC News.
Since the COVID-19 virus can mutate quickly, a lengthy clinical trial may result in a vaccine that no longer protects against currently circulating variants, experts cautioned.
“By the time you finish the trial, the strain that’s out there in the community is probably long gone. So, they’re basically saying, unless you’re in those higher-risk groups, you can forget about getting the COVID vaccine,” former acting CDC director Richard Besser, now president and CEO of the Robert Wood Johnson Foundation, told ABC News.
A spokesperson for HHS, which oversees FDA, told ABC News, “The COVID-19 public health emergency has officially ended, and we are entering a new phase in our response to the virus. A rubber-stamping approach to approving COVID boosters in perpetuity without updated clinical trial data under the Biden administration is now over.”
Last month, more than 300 people died each week from COVID, according to the latest CDC data. Death rates were even higher earlier this year as the virus was spreading, with nearly 1,000 Americans dying weekly in January.
The new policy comes as the FDA’s vaccine committee of independent advisers are set to meet on Thursday to discuss the latest data on vaccine safety and efficacy, including COVID shots. Typically, FDA leadership waits until after hearing the advice of its advisers before implementing a new approval or policy change.
Previously, updated COVID vaccines had been recommended annually by the CDC for everyone over the age of 6 months. The CDC has historically set vaccine recommendations, while the FDA determines which vaccines to authorize or approve after being deemed safe and effective.
“The FDA has kind of usurped the CDC’s recommending capacity. Usually, the FDA is a regulatory body. They say the vaccine can be sold, and the CDC says, ‘Jere’s the groups who should get it,’” Dr. Paul Offit, a vaccine expert at Children’s Hospital of Philadelphia, told ABC News.
Added Besser: “It’s also not something the FDA would normally do. It’s something CDC would do. And so that’s a really important issue here.”
The new COVID vaccine framework would allow an estimated 33% of the U.S. population to be eligible for the shots, or at least 100 million people, FDA officials noted. It matches recommendations from other countries such as the U.K., Canada and Australia.
Experts say there is a real scientific debate about whether annual boosters are necessary for otherwise young and healthy children and younger adults, especially now that most Americans now have some immunity gained from past vaccination and infections.
In mid-April, the CDC’s vaccine committee of independent advisers had discussed whether to keep the recommendation that everyone over 6 months receive an annual COVID vaccine.
Some experts have argued an FDA restriction could create insurmountable barriers for those who no longer qualify but want to get vaccinated anyway to protect vulnerable family members. Officials at the FDA have called for more evidence on whether vaccines prevent transmission before providing that option.
“To date, there is no high-quality evidence that you getting a booster protects your grandma, beyond your grandma getting the booster herself,” Prasad said at an FDA press conference Tuesday. “Does it lead to less transmission? Does it lead to fewer instances of severe disease? Again, we are interested in evidence to inform this claim.”
Insurance coverage of future COVID vaccines will likely be determined based on recommendations from the CDC and the agency’s independent panel of advisers later this summer. A final recommendation on who should be eligible for updated shots will likely come from the CDC director later this year.
Insurance companies will “continue to monitor the forthcoming recommendations and guidance from ACIP and CDC. As of today, there is no change in how plans cover the existing vaccines for the previously recommended populations,” according to AHIP, a national organization representing insurance companies.
“The ultimate goal of the vaccine is to keep people out of the hospital. But people could reasonably say, moderate infections aren’t fun either, so I’m going to choose and get this vaccine,” Offit noted. “I think that’s fine. I think it’s a reasonable choice. I just hope that we’re not restrictive.”
(WASHINGTON) — The Advisory Committee on Immunization Practices (ACIP), made up of members recently hand-selected by Health and Human Services Secretary Robert F. Kennedy Jr., voted 5-1 on Thursday to recommend against flu vaccines containing the preservative thimerosal.
One committee member, Vicky Pebsworth, abstained on each vote.
A few moments before, the CDC’s vaccine advisory committee voted 6-0 to recommend all Americans aged 6 months and older receive an annual influenza vaccine.
This is a developing story. Please check back for updates.
Raquel Natalicchio/Houston Chronicle via Getty Images
(NEW YORK) — Measles cases are continuing to spread throughout the U.S. with outbreaks in at least six states.
Public health experts have previously said lagging vaccination rates are to blame for the rise in cases, at least partly due to vaccine hesitancy and vaccine fatigue left over from the COVID-19 pandemic.
However, even a small uptick in MMR (measles, mumps and rubella) vaccination could prevent millions of infections, according to new research.
The study, published in JAMA last week, used a model to simulate the spread of vaccine-preventable infectious diseases across the U.S., evaluating different scenarios with different vaccination rates over a 25-year period.
The Centers for Disease Control and Prevention (CDC) currently recommends that people receive two vaccine doses — the first at 12 to 15 months and the second between 4 and 6 years old. One dose is 93% effective and two doses are 97% effective against measles, the CDC says. Most vaccinated adults don’t need an additional dose.
At current vaccination rates, the model predicts measles could once again become endemic — constantly present — in the U.S. with an estimated 851,300 cases over 25 years. If vaccination rates decline by just 10%, the model estimates 11.1 million cases of measles over 25 years.
CDC data show vaccination rates have been lagging in recent years. During the 2023 to 2024 school year, 92.7% of kindergartners received the MMR vaccine, according to data. This is lower than the 93.1% seen the previous school year and the 95.2% seen in the 2019 to 2020 school year, prior to the COVID-19 pandemic.
“That result — that we’re already at this tipping point for measles in the U.S. — was a really striking finding that somewhat surprised us as well,” Dr. Nathan Lo, study co-author and an infectious diseases physician at Stanford Medicine, told ABC News.
“We continue to see measles outbreaks, but, by doing the study … you start to crystallize that result that even under the current levels of vaccine decline, there is a very feasible scenario where, over time, our country has measles return where it’s a common household disease again.”
Dr. William Schaffner, a professor of preventive medicine at Vanderbilt University Medical Center in Nashville, who was not involved in the study, said the findings are a “well-thought-out worst-case scenario.”
“I’m not entirely sure that would actually happen because … the general level of vaccination remains pretty high, but there are pockets of unvaccinated children, and what you’re seeing now is the fact that this virus has been reintroduced several times from other parts of the world,” he told ABC News. “I think it’s a bit of a worst-case scenario but, as a worst-case scenario, it’s scary and, in that sense, reasonable.”
However, the model estimated that just a 5% increase in vaccination would lead to only 5,800 cases over the same 25-year period.
This is because when more than 95% of people in a community are vaccinated, most are protected from measles through community immunity, also known as herd immunity, according to the CDC.
“My hope is that this study can provide that data to parents to say, ‘This is the benefit of continuing to vaccinate your child. This is the kind of alternate reality that we’re preventing. And yes, the risks aren’t here right now, but perhaps not too far off,'” Lo said.
Schaffner said there are pockets of the U.S. where MMR vaccination rates are 80% or less and vaccination rates would need to increase substantially in those areas to reduce the number of cases long-term.
However, “if we increase the level of vaccination by a small amount that would reduce the risk of other small outbreaks here and there,” Schaffner said.
The study also found that a 50% drop in vaccinations would lead to an estimated 51.2 million cases over 25 years, but Lo thinks that would only occur if there were a large-scale policy change by the Department of Health and Human Services or the CDC to reduce or remove the childhood vaccine recommendation.
As of Friday, the CDC has confirmed nearly 900 measles cases in at least 29 states. That number is likely an undercount due to delays in states reporting cases to the federal health agency.
In western Texas, an outbreak has been spreading with 663 reported cases of measles, according to new data published Tuesday by the state Department of State Health Services. At least 87 people have been hospitalized over the course of the outbreak.
Measles was declared eliminated from the U.S. in 2000 due to the highly effective vaccination program, according to the CDC. However, an outbreak lasting 12 months or more would threaten to end measles elimination status in the U.S. The Texas outbreak began in January of this year.
To drive vaccination rates up, Lo and Schaffner recommend that parents who haven’t vaccinated their children yet speak to their pediatrician to address their concerns.
“We have to provide recommendations and let people know the facts that these vaccines are very effective and that they are safe,” Schaffner said. “But we have to do more to try to reach out and provide reassurance, and this has to be done very much on a local basis.”
For example, when it comes to the community affected in Texas, Schaffner said they need local influencers they trust to turn to.
“Speak with your local physician, health care provider, the person who cares for your children,” Schaffner said. “Have a conversation with them, and that’s our best hope for regaining some of this trust which public health had and which has slipped away.”
