Angelica Mendoza can rattle off the names of her best friends with pride. Despite struggling with Morquio, she’s kept up a thriving social life at school, where she has formed a bond with some of the younger students on campus. She wants to be a teacher when she grows up.
“I’m really good with kids,” says Angelica. “I put them in time out.”
Jackie Mendoza was 15-years-old when she had Angelica. A few years after giving birth, she observed as the baby’s chest grew while the other parts of her body did not.
“I was scared. I was just 18 at the time and I wasn’t sure how to tell my parents something was wrong,” Jackie said.
Having moved to Arizona to be with the father of her second child, Jackie disconnected from her family, who didn’t support the relationship, and stopped following up with Angelica’s doctors.
“I was hot, pregnant, I wasn’t going to school, I was miserable” Jackie said. “So we came back to California.”
Back home, things started getting better. Jackie earned her GED and began studying to become a nurse. She moved in with her dad, who was able to help her take care of Angelica. But still, there was a sense of hopelessness surrounding Angelica’s illness.
“Several doctors said there was no cure, no treatment. There was nothing we could do about her condition,” Jackie said.
It was through a Morquio support group that she heard about the Vimizim trial at Children’s Hospital Oakland. Angelica, now 12, has been participating in the trial for over a year.
“She does have a lot more energy,” Jackie said. “She’s able to walk and dance. She loves to dance on the Wii.”
Jackie and Angelica travel to Oakland from Bakersfield every Thursday for treatment. Jackie, now a registered nurse on the night shift, comes home from work at 4am on Thursday mornings, scoops up Angelica and hauls off to the airport for a 6am flight.
“It’s hard, what we do. But there are a couple families who have siblings both with Morquio. It could be worse,” says Jackie.
As she sits on the edge of the hospital bed, her heart rate pinging steadily on a machine in the background, Sadie O’Donnell fingers the apple embroidered on her pink pajama shirt, shyly averting the doting attention from the others in the room. Her father runs a hand over the top of her head of wispy blonde curls.
“That’s her mother’s hair,” he says.
At just three years old, it’s hard to tell Sadie has Morquio Syndrome. The disease hasn’t stunted Sadie’s growth or contorted her limbs and joints - at least not yet. And because her parents, Michael and Erin O’Donnell, started Sadie on the new experimental drug, Vimizim, when she was just nine months old, doctors are hopeful that they might be able to keep the disease at bay.
Had it not been for her older sister, Evie, who also has Morquio, Sadie might not have been so lucky. After the O’Donnells spent over a year looking for a diagnosis for Evie, they knew there was a distinct possibility that Sadie would also have inherited the gene for the disorder.
“They tell you there’s no treatment. No cure. That’s one of the scarier things,” Michael said.
Growing up, Evie went through a series of tests and corrective surgeries meant to keep her bones from growing in on one another and blocking blood to her spine. “Then we heard about the trial,” Michael said.
Two years later, Sadie and Evie O’Donnell are waking up at 6am every Thursday morning to hop a plane to Oakland from their home in Huntington Beach. Once they arrive at Children’s Hospital Oakland nurses set them up with an IV and the girls are rewarded with unlimited TV and iPad time- a luxury reserved for the hospital stay.
But Michael and Erin are still on the fence about whether the new drug can help their daughters. Erin said they actually took Evie off of the drug for six months because they weren’t sure it was helping her. They came back to the trial when they noticed that Evie’s energy level was going down while her pain level was going up. Erin said things stabilized once Evie was back on the drug, but not for long. After a few months, Evie’s energy levels began to drop again and she began experiencing more pain.
“We’ve been really conflicted about how we feel about the drug,” Erin said. “We do feel it’s beneficial in some ways but not as much as we were hoping.”
When Ariana Kallas underwent surgery to have metal plates installed on her knees and ankles, it was supposed to increase her mobility by strengthening the joints and ligaments weakened by Morquio Syndrome. Instead, the metal plates made walking so painful that Ariana resorted to a wheelchair full time. She hasn’t been able to walk since. To top it off, she had an allergic reaction to the adhesive used to help close her wounds and her lower body broke out in a rash.
“It was a tough recovery,” recalls her mother, Bertha Kallas.
Luckily, the Kallas’ are local- they live in South San Francisco. So when Bertha, who once raised $65,000 by organizing a 500-person banquet dinner for Morquio Syndrome research, heard about the Vimizim trial, she enrolled her daughter immediately. Ariana became the first patient in the U.S. to receive the new drug.
Since joining the study, Ariana has become best friends with Angelica Mendoza from Bakersfield. When they aren’t together in Children’s Hospital Oakland, they’re face-timing on their moms’ cell phones, playing Minecraft on their iPads, or connecting via Facebook. They share the same love of all things pink and differ on one main point: Angelica wants to be a teacher, but Ariana wants to be a fashion designer.
“I want to get a toy sewing machine to practice on,” says Ariana.
Today, at 8 years old, Ariana’s lung capacity has improved. As part of the study, nurses periodically have Ariana breathe into a set of tubes that register the strength of her lungs into a computer, which in turn simulates an image of candles going out on a birthday cake, or a harmonica whistling a tune.
At the beginning of the study, Ariana’s breath barely registered on the computer. But now, “I can blow a harmonica,” said Ariana with pride.
Bertha credits the improvements to Vimizim. She calls the study, “a blessing.”
“This has been awesome. We’re very hopeful,” she said.
Sarah Morrell moved halfway around the world from New Zealand with her father, Ben Morrell, to take part in the Vimizim trial at Children’s Hospital Oakland. They rented an apartment in Walnut Creek and bought a small cheap van to shuttle Sarah and her wheelchair back and forth between the apartment and the hospital for her weekly infusions. Ben has improved his cooking, adjusted to Bay Area traffic, and extended the mortgage on his house. He says the hardest part about the move has been leaving his wife and son back home.
For Sarah, the move meant saying goodbye to her mother and older brother, her friends and her school. Initially, it wasn’t an easy decision.
“I mean, we have a life, you know, on the other side of the world,” said Sarah, who is 18.
Despite the apprehension, Sarah wasn’t left with many other options. Like other Morquio patients, Sarah has gone through a series of hip, knee and ankle surgeries that were largely unsuccessful. Only one procedure delivered results: a back surgery that corrected the curve in her spine.
Since then, both Sarah and her dad have noticed some improvements in her condition.
“No major improvements but small things. I can use scissors. Even though it’s small, it’s exciting. I can clip my own fingernails,” said Sarah.
Ben also credits the drug for reducing Sarah’s joint pain.
“Before, we constantly were rubbing cream on her joints,” Ben said. ”But since the trial, the joint pain has gone down probably 80-90%.”
Because Sarah’s visa allows her to be in the country only for medical purposes, her schooling was cut off at the age of 17. She was able to attend high school in Walnut Creek, but missed the standardized exams necessary to apply for college back home. She’s concerned about being behind other students when she finally does manage to apply.
The Morrells are also unsure whether they will be able to receive the treatment back in New Zealand. Even if the drug is approved by the US Food and Drug Administration, they would still have to navigate costs with their insurance companies and find a doctor who could administer the treatment.
Lying on a hospital bed with an IV attached to his arm, Justin White closes his eyes and tries to sleep over the voice of his sister, Kianna, who sits across from him. Now and then he raises an eyelid and laughs at something she has said, or adds a detail to a story she is telling. He says the experimental treatment, Vimizim, makes him sleepy. “I’m always sleepy, though,” he admits. Fatigue is a common symptom among Morquio patients.
His many tattoos peek out from under his white t-shirt. One reads “FOE,” an acronym for “Family Over Everything.” Another says “Hungry but humble.” Justin swears they didn’t hurt.
On days when he and his sister Kianna come to Children’s Hospital Oakland for treatment, Justin wakes up at 3am to catch a plane into Oakland from Gilbert, Arizona. He usually goes without sleep the night before, since he stays up late working on his music. Justin, aka: J-Weezy, harbors hopes of becoming a famous rap artist.
“Ever since I saw Tupac and TI, I felt inspired. It became something that I loved and could put my feelings into,” he said.
Justin says he draws from his 18 years living with Morquio to find inspiration for his music. But if the rapping career doesn’t work out he has a back-up plan: a reality show, dubbed “The Life of J-Weezy.”
“I wanna be ballin. I wanna be rich,” Justin says with a laugh. “I want to donate to charity and help my family out. We’ve struggled our whole lives so I want to make our life easier.”
When he comes to the hospital, Justin says he tries to just relax and enjoy the moment.
“We can come here and we don’t have to do anything. We can just sit here and be happy, have fun with each other,” he said.
Having started the drug late in life, it’s hard for Justin to know how the drug will affect his body; most of his growth has already happened. He says, he can feel the drug’s benefits for anywhere from a couple hours to several weeks at a time.
“Sometimes I feel more energized,” he said. “They say my heart is doing well so I think it’s helping with that.”
Eighteen-year-old Kianna White has big ambitions, the gift of chatter and a thing for bright red Chuck Taylors.
“I like learning new things and I like English and psychology,” says Kianna before rattling off a few to-do items on her lengthy list of dreams.
Along with going to college, there is starting a nonprofit, writing a book (or two) and “Basically just changing the world!” She graduates from high school this May.
The Gilbert, Arizona native travels to Children’s Hospital Oakland each week with her older brother, Justin White. They are both Morquio Syndrome patients who heard about the study just a little over a year ago - giving them a late start on the experimental drug, Vimizim. But this doesn’t seem to temper Kianna’s optimism for the future. Despite limited mobility and an onslaught of related health problems connected with the disease, Kianna is quick to dispel any idea that she is living in self-pity because of Morquio syndrome.
“I go to football games, I go shopping and I hang out at home with my family,” said Kianna. “(Morquio) is something I’ve always had so I don’t know what I’m missing.”
As she sits on her hospital bed with her IV attached, Kianna fills out a worksheet for school while intermittently checking her iPhone - a tool she uses to keep in touch with friends back home and other patients in the Vimizim trial.
Before the trial, Kianna hadn’t received any treatment for the disease, which has twisted her left wrist and completely flattened her feet and toes – something that makes walking difficult. Yet she says the most valuable part of the study has been the relationships she’s built with other Morquio patients.
“We just click because it’s hard to find other people who have (Morquio). I know in Arizona there’s just 4 or 5 of us,” said Kianna.
“Right now it’s pretty chill because some of the people are gone,” said Kianna, describing the mood of the hospital room. “It’s usually pretty fun. There’s a big table with glitter and markers and crayons. But when one member is gone, it throws us way off.”
As for the medicine itself, “Hopefully it’s working. We don’t know yet. But so far it’s been good. I mean, nothing bad has happened,” she said with a shrug.