Would You Use Crowd Funding for a Chance to Save Your Child’s Life?
In the summer of 2016, Kaisy Knott, a funny, feisty 11-year-old, had surgery to straighten a lazy eye. A few months later, she began complaining of headaches so excruciating that she ended up at Johns Hopkins Hospital, in Baltimore. Her parents assumed the headaches were related to her recent surgery.
The real source of the headaches turned out to be far more malicious. The Knotts were told that Kaisy had diffuse intrinsic pontine glioma, or DIPG, a rare pediatric brain tumor diagnosed in 300 to 350 children each year in the United States, usually under age 10.
Brain tumors are the leading cause of cancer-related deaths for children and teens under age 20.
Among the array of possible types of brain tumors that can affect children, the outlook for DIPG is the grimmest of the grim. It is inoperable, and has a less than 1 percent survival rate. Children with DIPG usually have nine months to a year to live.
The only treatment is radiation, which doesn’t cure DIPG but can provide a temporary reprieve, a “honeymoon period,” during which the tumor often shrinks before coming back with a vengeance. Although there are a few clinical trials for therapies to treat DIPG in the U.S. and other countries, many children don’t meet the eligibility criteria. Many trials are only for newly diagnosed patients, or require that the child not undergo other treatments, or else the size, location, and extent of the tumor is incompatible with the trial.
Parents of kids with DIPG are usually advised to take their children home and “make memories.”
“We were told that children with this type of tumor are robbed of their essential bodily functions such as eye movement, eyesight, hearing, ability to use limbs, breathing, and ultimately stopping of the heart,” Kaisy’s mother, Melany Knott, of Mt. Airy, Maryland, recalls.
Kaisy, who shows cattle and hogs and loves horses, started radiation therapy. She experienced severe side effects, like thrush on the inside of her mouth so excruciating it caused her skin to peel. The steroids she was given to decrease the swelling in her brain also caused her pancreas to stop working, leaving her insulin dependent and with a weakened immune system.
Melany decided they needed to try something, anything, to fight back against the tumor. “The thought of going home and ‘making memories’ in the next six to nine months before my baby girl died was crushing,” says Melany, who has three other daughters. “I couldn’t live with myself if I didn’t look into every treatment option available.”
The problem was, there was nothing else to try.
Childhood Cancer: Rare — and Underfunded
Nearly 16,000 children in the U.S. between birth and age 19 are diagnosed with cancer each year. But childhood cancer is categorized as a “rare” disease, and has been funded accordingly. Only 4 percent of federal cancer research funding is solely dedicated to childhood cancer.
The reasons why are largely economic. Childhood cancer is much rarer than adult cancers. It’s also hard to conduct studies on children. In fact, most medications prescribed for kids with cancer haven’t been tested on them. One big reason? Money. Childhood cancers make up less than 1 percent of all cancers diagnosed each year, according to the American Cancer Society. Since fewer children get cancer, the market simply isn’t big enough to reap a big payday.
It’s also challenging to conduct studies on children, who need specially targeted equipment and environments.
Since 1980, fewer than eight drugs have been developed for use in children with cancer, compared with hundreds that have been developed specifically for adults only, according to the National Cancer Institute.
“More than 99 percent of the pharmaceutical industry’s cancer dollars are directed to adult cancers,” says Kathleen Ruddy, the CEO of the St. Baldrick’s Foundation, a nonprofit organization that raises funds for children with cancer. Pharmaceutical companies don’t want to invest in treatments if they can’t make money.
Parents of young cancer patients are enraged when they discover this. Especially, as in the case of DIPG, when almost no therapies have been developed. “I was most upset by the marginalization, the treatment of our family and others as ‘rare’ and our children’s lives as ‘irrelevant’ to investors,” says Janet Demeter, who runs Jack’s Angels Foundation named in memory of her 3-year-old son, Jack, who died of DIPG in 2012.
“The fury came when I found out that brain tumors are the leading cause of death in children with cancer in the U.S., and that DIPG is responsible for most of those deaths. There’s been no change in standard treatment nor terminal prognosis in over 50 years,” says Demeter. “DIPG exemplifies, in a powerful way, the neglect of funding and activity for pediatric cancer research — pediatric disease research, really.”
A Ray of Hope in Mexico
Through an online support group for DIPG families, Melany heard about Instituto de Oncologia Intervencionista, a medical practice in Monterrey, Mexico, that specializes in brain tumors. The facility, which is run by Alberto Siller, MD, a pediatric oncologist, and Alberto Garcia, MD, a radiologist with expertise in brain tumors, is located within a private hospital that caters to some of Mexico’s wealthiest families.
Using a drug cocktail specifically tailored to each patient — in some instances as many as 18 different drugs — the doctors administer what’s known as intra-arterial chemotherapy (IAC), which delivers chemotherapy drugs via a catheter in the groin directly into an artery that feeds in to the area in which the tumor resides, bypassing systemic circulation.
The treatment isn’t cheap: Each visit runs as high as $17,000, a price tag that includes the drugs, MRIs, doctors’ fees, and the hospital stay (but not transportation or lodging for families). Immunotherapy, which is only an option after the child has undergone a few IAC infusions and is entirely off steroids (which suppress the immune system and may ruin the effect of immunotherapy), runs another $15,000.
Families usually repeat this routine every 17 to 21 days until the tumor shows evidence of shrinking, whereupon treatments may take place every four to six weeks. But since it’s a relatively new protocol, the doctors have no idea how many treatments a patient may need. In theory, it could go on for y
ears. And the doctors are quick to say that it’s not a “cure.” Rather, it’s an attempt to prolong patients’ lives — maybe long enough so a cure will be found.
“No parent is down there expecting to cure the kid,” says Ashley Gregg Bandavanis, whose 7-year-old son, Deany, was diagnosed with DIPG in March of 2017. “There is nowhere in the world that has a cure. The only thing that we have ever asked these doctors to do — and they are willing to do — is try.”
The treatment is not without controversy. Dr. Siller and Dr. Garcia haven’t published any studies, nor do they share information about their protocol with other doctors, though they will give parents a list of the medications they use. “We don’t publish because we are too busy working,” says Siller.
Many American doctors don’t buy it. “It’s not the first time I’ve seen rogue physicians charge a lot of money for what they call a therapy and capitalize on hope,” says Michelle Monje, MD, an assistant professor of neurology and pediatrics and a pediatric neuro-oncologist at Stanford University in California.
David S. Terman, MD, an adjunct professor of biochemistry and molecular genetics at the University of Alabama at Birmingham, and a former professor and director of the cancer biology program at Baylor College of Medicine in Houston, expresses his concerns even more vehemently.
“Publication and peer review or presentation of the data in a major forum is the established way to achieve scientific credibility for a new therapy,” says Dr. Terman.
“We do not know the drugs used by the Monterrey group, so it’s impossible to speculate on which if any deployed in their intra-arterial therapy actually access the tumor,” he says. “The enormous cost of the Monterrey treatment would seem to confer a moral responsibility upon the Mexican physicians to validate and confirm their findings in an open forum before offering [the treatment] to parents desperately foraging for a cure for their dying children.”
Despite all that, Melany, who had never left the United States before her daughter got sick, didn’t think twice. She and her husband, David, were on a mission to save their daughter. And there was no other choice.
“I knew this treatment was a huge risk, but I decided that this was the only option we had aside from doing nothing — which was never an option for my child,” she says.
Kaisy’s doctor in the United States, Roger Packer, MD, of the Children’s National Health System in the District of Columbia, was remarkably supportive, given the lack of evidence. “For this particular treatment, there is no research or data that supports its effectiveness,” he says. “But with a diagnosis like DIPG that will likely be fatal, unless there is evidence to show that an alternative therapy will be detrimental, I’m inclined to support families with their wishes.”
The Knotts were in good company. By the end of 2017, more than 50 families from around the globe had traveled to Mexico for the treatment. A website sprang up, Making DIPG History in Monterrey, chronicling the children’s stories.
The problem all the families faced: how to pay for it.
High Costs, and Health Insurance Is No Guarantee
The average cost of a hospital stay in the U.S. for a child with cancer was about $40,400 as of 2009. Health insurance typically covers traditional cancer treatments, like chemotherapy, radiation, and surgery, as well as immunotherapy and some clinical trials.
In May, President Trump signed the so-called Right to Try Act, which legalizes someexperimental drugs in the United States. Forty-one states already had similar laws in place, but now it’s the law of the land.
But insurance, Medicaid or Medicare aren’t required to pay for drugs used in this way. Nor does the law cover the use of experimental treatments outside the U.S.
“Insurers would say, ‘There’s no evidence this works for every child, so we have a responsibility to other policy holders to use the funds we have in the most responsible way,” says Margaret Comeau, the senior project director at the Center for Innovation in Social Work and Health at Boston University. “Having health insurance doesn’t automatically protect families of children and youth with special needs from financial hardship.”
Parents who wanted their children to try the Monterrey protocol have generally had to kick into fund-raising mode to finance the treatment and the travel. Among the ways parents have raised money have been via bake sales, community-wide contests, grants, selling trinkets outside supermarkets, and launching Go Fund Me campaigns. Almost all of them have Facebook pages and Instagram accounts to solicit funds.
Tammi Faultersack Ramirez, of Las Vegas, has been auctioning makeup, iPads and jewelry and holding monthly car washes and bake sales to cover her 11-year-old son Jacob’s expenses in Mexico. Ashley Gregg Bandavanis, who lives in Pickerington, Ohio, started a YouCaring page, set up fund-raisers, and began raffling off items like a trip to Disney World after Deany was diagnosed.
“It’s a constant struggle to not only do everything in our power to save our children, but have to fight doctors, big pharma, and insurance companies, all while begging for money to keep them in treatment,” says Bandavanis. “My days are consumed by money. I’ve pretty much done everything but rob a bank and prostitute myself.”
Manisha Modi and Milan Mehta, of Fremont, California, have collected around $150,000 for their 14-year-old son, Ronil, through Brave Ronil. Ronil has had 15 IAC and two immunotherapy treatments in Mexico. While he did manage to complete his freshman year of high school, he hasn’t seen much clinical improvement. “He cannot walk, talk, or swallow on his own. He can’t even use his hand to use his phone,” says his mother. “But he’s still here with us, 29 months past diagnosis.”
Manisha left her full-time job as a software engineer during the first year of her son’s illness, and now works part-time at Wells Fargo.
The Family and Medical Leave Act allows 12 weeks of unpaid time off for employees to care for a sick family member. But 12 weeks is seldom enough. And disability only pertains to the employee, not anyone else in his or her orbit.
Even though Milan earns a good salary at his job at PlayStation, the couple feels the financial strain. Manisha imagines they’ll use some of their investments to pay for treatment.
“Insurance should be totally paying for this!” she says. “I keep telling them, ‘You don’t have to pay for doctors,’ but the IAC is not new — it’s done in the U.S. At least pay us for the hospital fees.”
Trying to Buy Time
Thirteen treatments and about $200,000 later, Deany Bandavanis’s tumor shrank by 80 percent, with little evidence of cancer activity. But then he started having difficulty with his right side; an MRI confirmed that the cancer had spread. He’s now doing 10 rounds of radiation in the States, and may return to Mexico afterward.
Some other children, like Kaisy Knott, are doing well. Shortly after her first IAC infusion, her parents noticed that her left eye, which had turned inward, had straightened out, and she was able to get off steroids. After 20 treatments, Kaisy’s doctor in the states, Dr. Packer, acknowledged that her tumor looks “a little bit smaller.” Although her words are somewhat slurred and she is slightly unsteady on her feet, she was able to go back to school (“Unfortunately,” Kaisy says.)
Melany estimates that she and her husband have spent about $450,000 on Kaisy’s IAC infusions. She raised about $200,000 through her housecleaning business and personal donations, and paid the other $250,000 out of pocket. “We have no health insurance because I have to decide between a roof over the kids’ heads, or expensive and not useful health insurance,” she says.
In April, Melany and Kaisy joined Janet Demeter and other activists in Washington, DC, in support of H.Res.69, which raises awareness of pediatric cancer research and designates May 17 as DIPG Awareness Day nationwide. So far, 31 states are on board.
In June, Congress passed the Childhood Cancer Survivorship, Treatment, Access, and Research (STAR) Act into law, which, among other things, provides $30 million a year from 2019 to 2023 for pediatric cancer research and grants.
Parents are optimistic that this legislation will have an impact. It’s not a solution. But it’s a start.
“If we can get a House vote on H.Res.69, and the public learns more about the neglect of pediatric research and the realities we are expected to endure as a result — it will be much easier to get the allocations for STAR and other important childhood cancer legislation,” says Demeter. “But again, it’s just another example of a much larger fight. I’m optimistic because we need to start with our children.”
Abby Ellin is an award-winning journalist, former columnist for the New York Times, and author of Teenage Waistland: A Former Fat Kid Weighs in On Living Large, Losing Weight and How Parents Can (and Can’t) Help. Her work has appeared in a range of publications, including The New York Times, Newsweek, Time, ABC News, The Daily Beast, The Village Voice, Marie Claire, More, Self, Glamour, and Spy (RIP). But her greatest claim to fame is naming “Karamel Sutra” ice cream for Ben and Jerry’s.
Co-published with Everyday Health.