Spectrum Health West Michigan

Nathaniel’s Story

Like pieces of a puzzle, everything finally came together for Nathaniel Bailey.

Darlene Bailey, a nursing home aide, had been raising her nephew, Nathaniel, since he was a baby. His health seemed fine until, early in 2012, Darlene noticed that Nathaniel, 5, was “walking funny, almost on the side of his feet. I thought he had tripped or hurt his foot somehow but it just didn’t get better.”

When he started complaining of pain, Darlene sought medical help. After an orthopedic specialist found lesions on his knees, Nathaniel spent months in casts. Once out of the casts, Darlene – who, along with working, was busy raising her own three young children – took Nathaniel to weekly physical and occupational therapy appointments.

In the summer of 2012, Darlene noticed a bump starting to form on Nathaniel’s back. “He told me he had fallen out of bed at his grandmother’s, so I wasn’t worried,” she recalled. However, the bump got bigger and bigger and it became hard for him to walk.

A trip to a Spectrum Health emergency department led to a referral to Deanna Mitchell, MD, a pediatric oncologist with Helen DeVos Children’s Hospital in Grand Rapids. Dr. Mitchell ordered a MRI that identified an aneurysmal bone cyst located in Nathaniel’s sacrum, the lowest part of the spine.

“This type of tumor is benign, rather than cancerous,” said Mitchell. “However, it grows aggressively and eventually eats away at the bone causing fractures. Few treatment options exist for these tumors apart from surgery.”

Dr. Mitchell connected the family with Matthew Steensma, MD, an orthopedic oncological surgeon with Spectrum Health Medical Group. Dr. Steensma, who practices at Lemmen-Holton Cancer Pavilion and Helen DeVos Children’s Hospital, is also an assistant professor at the VanAndel Institute (VAI) Center for Skeletal Disease Research, oversees VAI’s Laboratory of Musculoskeletal Oncology and has a position at the nearby Michigan State University College of Human Medicine.

Dr. Matthew Steensma is the only pediatric orthopedic oncological surgeon in West Michigan.

“When I first met Nathaniel, his aunt basically carried him in to the appointment. He was unable to walk, had lost control of his bladder and was requiring frequent catheterizations,” explained Steensma. “In Nathaniel’s case, because of where the tumor was located and how much destruction it had caused, the normal treatment – surgery – was not a realistic option. There was no bone left in his sacrum, and he faced the risk of life-threatening bleeding because of the vascular nature of this type of tumor.”

Without surgery, the kindergartner – who was desperately missing school and his friends – faced permanent paralysis. Steensma and Mitchell considered other treatment options to help Nathaniel, but no effective therapy could be found.

Dr. Steensma researched and found that the protein called RANK ligand (RANKL) was present in the cyst, specifically in cells that eat away at bone. He learned that the FDA had approved a specific therapy for dealing with RANKL, although it had never been used to treat an aneurysmal bone cyst. This involved monthly injections of the drug Denosumab, which is commonly used for treating cancer metastasis (growth) in bone.

Dr. Mitchell agreed to administer the drug to Nathaniel at Helen DeVos Children’s Hospital and worked with the appropriate people to get the treatment approved.  After spending a week in the hospital in August of 2012, Nathaniel returned for monthly treatments. After only a few weeks of Denosumab treatment, Nathaniel’s pain control improved. Within four months, his tumor shrank by 30 percent and his bladder function began to normalize. The fracture that was present originally healed. Nathaniel was walking better and even running occasionally. After eight months of treatment, the tumor shrank to half of its original size and was filling in with normal bone.

Today, after 15 months of treatment, Nathaniel’s tumor continues to respond. Fortunately, he has not experienced any of the side effects associated with this drug. He is a happy, active 7-year-old, back in school and enjoying life.

“Nathaniel may ultimately require more treatment, but we are delighted that he is doing well today,” said Steensma. “We were all impressed by Nathaniel’s resiliency during treatment. He was very determined to get better and received tremendous support from his aunt, as well as Dr. Mitchell and the oncology team at Helen DeVos Children’s Hospital. Nathaniel’s response to this type of treatment was truly remarkable, particularly given the lack of effective treatment options.”

VIDEO LINK: Dr. Steensma explains the challenges of orthopedic oncology http://youtu.be/6Am3agyeQNU

In order to determine if the drug would benefit other patients with similar tumors, Dr. Steensma turned to his lab at the Van Andel Institute. He was assisted at VAI by Dominic Pelle, MD, who was involved in orthopedic research.

“We studied a number of other aneurysmal bone cysts and found that the same protein – RANKL – was being produced in all of the tumors. This finding suggests that other unresectable tumors like Nathaniel’s can be treated similarly. Certainly, more research is needed in this area but we are very encouraged by Nathaniel’s case,” Steensma said.

VIDEO LINK: Dr. Steensma discusses what this discovery may mean for other patients. http://youtu.be/Fggw3z9pFG4

Dr. Steensma presented his findings at the national meeting of the Musculoskeletal Tumor Society in the fall of 2013. His work will be published by Translational Research later this year. The research is currently posted at www.ncbi.nlm.nih.gov/pubmed/.

VIDEO LINK: Dr. Steensma explains the role organizational relationships played in helping Nathaniel. http://youtu.be/P-HN93AoAkY

While it took many people to put together the pieces of the puzzle to help Nathaniel, “Darlene is the real hero in this,” explains Dr. Mitchell. “She made sure that Nathaniel got the help he needed when he needed it, no matter what.”

According to Darlene, “Nathaniel is my hero. He gives me hope – he is so strong. And he is happy that what he went through may help other people. Helping other people is the best.”

UPDATE:

In May of 2014, Nathaniel experienced a significant side effect after discontinuation of the medication when he developed a high level of calcium in the blood. He was admitted back to Helen DeVos Children’s Hospital for treatment and, to date, this issue has resolved.

Spectrum Health is a not-for-profit health system, based in West Michigan, offering a full continuum of care through the Spectrum Health Hospital Group, which is comprised of 11 hospitals including Helen DeVos Children’s Hospital; 170 ambulatory and service sites; 1,080 employed physicians and advanced practice providers including members of the Spectrum Health Medical Group; and Priority Health, a 575,000-member health plan. Spectrum Health is West Michigan’s largest employer with 21,300 employees. The organization provided $250 million in community benefit during its 2013 fiscal year.