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The Quest for a Cure


Joseph O'Brien

Despite having a rare neurodegenerative disease called Niemann-Pick Type C, the two oldest Hadley children, Peyton and Kayla, enjoy staying active and participating in events with their family at their Catholic parish in Medford, Ore. (Photos by Jared Cruce)

Born three years apart, Peyton and Kayla Hadley began their lives as normal, healthy children. For their first seven years, both progressed normally as they began elementary school, scoring high on tests with excellent reading skills. Then, when Peyton turned 8, things began to change. One of the first signs that something wasn't right began with Peyton's tendency to cock his head while reading. "It was gradual at first," said Bryan Hadley, Peyton and Kayla's father and a member of Rogue River Council 1594 in Medford, Ore. "We'd notice that he was having a harder and harder time tracking down or up with his eyes." After a battery of tests administered by medical doctors, eye doctors and physical therapists, the Hadley family finally received an explanation of their son's problems on Nov. 9, 2007. Peyton had a neurodegenerative disease known as Niemann-Pick Type C (NPC). It is almost always fatal, and it has no cure. Although tempered by the news that the Hadleys' third child, Jonah, tested negative for the disease, Kayla was also diagnosed with NPC about a year later. PROMOTING ETHICAL RESEARCH Bryan and his wife Laura recovered from the shock and, putting their faith in God and science, began researching the disease. They discovered NPC was among more than 5,000 "orphan diseases" — rare disorders that often affect children and, because they are largely ignored by the pharmaceutical industry, have little hope for cures. A metabolic disorder that prevents cells from properly processing cholesterol, NPC is so rare that there are only about 500 diagnosed cases worldwide. NPC patients commonly suffer from an enlargement of the liver and spleen, where cholesterol-glutted cells accumulate. Affected cells also corrode the central nervous system, leading to debilitated motor skills. Another telltale sign of the disease is vertical gaze palsy — which Peyton exhibited in his reading habits. Because it usually leads to the same mental regression, NPC is often called "juvenile Alzheimer's." Once they knew what they were up against, the Hadleys created a non-profit organization, Hadley Hope (hadleyhope.com), to help find a cure. In addition to raising awareness about NPC, which often goes undiagnosed, the organization has raised more than $211,000 in research funds since 2007. "The only way to do anything about the disease was to come up with the money for research," Laura explained.

"We thought early on about what would happen if scientists found a cure for NPC that uses embryonic stem cells," Bryan said. "Would we use that cure with our children? The answer is no. It would be the taking of another life to save our children, and that can never be justified in our faith." Not that the decision was easy, Laura added. "If there was a cure in front of us involving embryonic stem cells," she said, "it would be heart-wrenching, but I know what our faith tells us — and we believe it to the core." THE PROGRESSION OF HOPE The Hadleys believe that adult stem cells not only offer an ethical basis for research, but also hold one of the greatest hopes for finding a cure. Most credible estimates, they said, put any real cure about 10 years away, but they are working to revise that forecast with help from groups such as the John Paul II Stem Cell Research Institute in Iowa City, Iowa. Dr. Alan Moy founded the institute in 2006 in part to make up for the lack of ethical stem-cell research at secular institutes and the poor government funding that is typically directed toward adult stem cells. As a first step toward a cure for NPC, Moy and his colleagues were able to harvest and cultivate adult stem-cell lines from Peyton and Kayla's fat tissue. They are now working to develop methods to generate induced pluripotent stem cells — omnibus cells with the same potential as embryonic stem cells to differentiate. "We want to take the adult stem cells and genetically reprogram them back to the most primitive state," Moy explained, adding that such cells are not ethically controversial. Once the methods are successfully developed, Moy said, doctors can use the resulting cells to battle NPC as a tool or a therapy. "As a tool, we'll take a cell from a patient with the disease, create a stem cell model and then take an already approved drug," explained Moy. "The hope is that the drug will enter into the cell and attach to the genetically mutated protein. It will then help restore fully or partially the mutated protein to get it to function more normally." More directly, Moy added, stem cells could be used as a form of therapy. "To some diseases, the adult stem cell might be able to partially or fully regenerate organ function," he said. Because of the progress that has already been made, researchers at the National Institute of Health Chemical Genomics Center in Bethesda, Md., announced in September that they would use the cell lines obtained from the Hadley children to begin identifying drugs to treat NPC. Ultimately, by working with other patients who have orphan diseases, the scientists at the John Paul II Stem Cell Research Institute plan to create a bank of disease-specific stem cells and provide hope for many other families like the Hadleys. LIVING WITH FAITH With a little more than 100 cases of NPC in the United States, the Hadleys have found it easy to network with other families struggling with the disease. "We have talked about it with other families. They share our opinion on the issue, and are assisting in pushing adult stem-cell research," Bryan said. The Hadleys are aware that others are looking to embryonic stem cells for a cure to NPC, but knowing that they and other families are witnessing to the truth serves as yet another sign of hope in their ordeal. "Based on recent history, adult stem cells are not only the ethical choice for stem-cell research, but also have proven to hold the greatest promise to treat and cure rare diseases," Laura said.

So far, Kayla shows no perceptible signs of her disease except for an enlarged spleen. Peyton, though, is "much farther along," according to the Hadleys. "He has many of the main hallmarks," Laura said. "He has the slow gait, and without a cure, eventually he'll be in a wheelchair and then bedridden with a gastric tube inserted to help him eat." Nonetheless, Peyton still attends the Hadleys' parish middle school and plays on the school's basketball team. "He's not able to take tests, but he still enjoys learning and paying attention," Laura said. "He especially likes to learn about history and his faith. There's not a day that goes by that he doesn't tell us about the saint of the day." Kayla is currently in fifth grade and stays busy playing the saxophone and attending dance class. Sometimes, it can be a real struggle working through her condition to maintain a mostly normal existence. "We can't have milk and have to drink rice milk," she said, describing their strictly fat-free diet. She added that she doesn't find school as difficult as the medical tests that she and her brother have had to undergo. "The IVs we have to do aren't hard, but they have to stick a needle in each time and it hurts," she said, adding that when things get tough, she asks her favorite saint, St. Thérèse of the Child Jesus, to intercede. Joseph O'Brien writes from Wisconsin.