Make-A-Wish Foundation says cystic fibrosis patients will no longer automatically get a wish
The Make-A-Wish Foundation offers children with life-threatening illnesses a chance to live their dream: be a firefighter, adopt a puppy, go on a wild shopping spree. But in an era of rapid medical advancements, what counts as life-threatening is changing.
A case in point: the charity has announced that, beginning next year, people with cystic fibrosis, a genetic disease that primarily affects the lungs, will no longer qualify automatically for a wish. The good news is that that is because the illness is, for many people, far less of a threat than it once was.
“Given the ongoing life-changing advances in cystic fibrosis research and treatment, Make-A-Wish is transitioning our CF eligibility to qualify for a wish on a case-by-case basis,” the foundation said in a statement. The illness takes a range of forms, and if a case is life-threatening, the child will remain eligible for a wish.
“This decision was not made lightly, and we understand it may result in some frustration and disappointment,” the statement continued.
But for Dr Meghan McGarry, a pediatric pulmonologist and associate professor at UC San Francisco who is an expert on cystic fibrosis, it is ultimately a good sign. “I think it’s exciting. It does reflect that cystic fibrosis has really changed,” she said. Dr Bruce Marshall, chief medical officer of the Cystic
Fibrosis Foundation, agreed in an email, calling the Make-A-Wish decision – on which the CFF was consulted – “a reflection of the tremendous story that is cystic fibrosis”. Similar decisions have been made in the past, including for patients with HIV/Aids, the foundation said in an email.
Still, the advancements in cystic fibrosis treatment have not benefited everyone equally.
The illness, which affected children are born with, has an impact on organs throughout the body – in particular the lungs and gastrointestinal system. A gene normally makes a protein that helps prevent the accumulation of mucus in the lungs and intestines; with cystic fibrosis, that protein does not work as it should. Children with the condition “lived in and out of a hospital, did hourly treatments every single day, and despite all that, their lungs were filled with mucus”, which could often lead to respiratory failure.
Over the past three years, however, drugs called CFTR modulators that tackle this protein problem have become more available. “For people who qualify for that drug, within days, they start getting dramatic results, where their lungs feel much better if they’re not making nearly as much mucus. I’ve had patients stop coughing and really go on to live much more normal lives.”
Indeed, life expectancy has been increasing for people with CF, jumping from 38 years a decade ago to 53 years for babies born between 2017 and 2021, according to Make-A-Wish. “We’re starting babies on these drugs, and we really expect that they’re going to have a normal, full life,” said McGarry, noting that another recent paper projects patients living into their 70s.
But there are many ways the genetic issue behind CF can manifest, and not all of them respond to the drug. Much of the early research on the illness focused on non-Hispanic white patients, McGarry says, and genetic testing often misses variants in Black, Asian, Hispanic and Ashkenazi Jewish populations.
Now, McGarry said, “we’ve turned it into two different diseases”. Ninety per cent of patients have variants of CF that will respond to the drugs. The other 10% continues to grapple with severe illness.
“And of course, because the mutations [that qualify you for the drugs] are mainly in people who are non-Hispanic white, then the people who are in that 10% group left over are much more likely to be Black or Hispanic or Asian or mixed race,” McGarry said.
Other treatments, including gene therapy, are being developed that may treat these mutations. But for now, “I think that’s where we’re going to have to work with Make-A-Wish to make sure that they understand about the patients who don’t qualify [for the drugs] – that cystic fibrosis is still a devastating disease for them,” McGarry said.