This is a feature story assignment I wrote while I was a journalism student at Ryerson University 14 years ago. My Newspaper/Print professor liked it and gave it a high grade. We even talked about getting it published in one of the major newspapers or magazines. Unfortunately this didn't happen because I ended up losing the work, or so I thought at the time. This weekend I found it again while going through old items in my apartment. Now I want to share this story with everybody. Though some of the participants may not be involved with this treatment today, the content remains relevant and timely. - DB
Story on Bisphosphonate therapy (1999)
Donald Barrie
Carlos Medina is a happy six-year-old, but unlike most
children his age, his bones break easily. Carlos was born with osteogenesis imperfecta,
or brittle bone disease, which affects about one in nearly 20,000 children born
each year. In addition to frequent fractures, it causes progressive deformities
of the arms and legs, and chronic bone pain. Lately, however, Carlos has been
feeling stronger physically, thanks to bisphosphonate therapy.
Bisphosphonates are a group of synthetic compounds that are
most widely used in the treatment of post-menopausal osteoporosis. They reduce
bone turnover and suppresses bone resorption (a normal breakdown process that
is excessive in some diseases including osteoporosis and OI). The most common
bisphosphonates being administered to these children with OI is pamidronate,
which is closely related to alendronate (Fosamax), used to treat osteoporosis
in adults.
At Sunny View Public School in Toronto, physiotherapist Lynn
Mueller takes Carlos out of his electric wheelchair and puts him on the floor.
Mueller, skeptical of his mother’s claims about his increased mobility, asks
him to crawl on the floor. Lying on his back, Carlos turns over on his stomach,
and crawls gently on his hands and knees. Carlos then pushes himself off the
ground, and sits in a kneeling position.
Mueller’s eyes light up, and so do those of the kids looking
on. Another therapist watching him from a distance rushes to get her camera. “I
can’t believe it!” the two therapists exclaim. “This is the first time I have
seen him do that!” Mueller adds ecstatically.
“When Carlos arrived at Sunny View, he was very weak,”
Mueller says. “Since the start of this school year, he has gotten stronger, and
his mobility has improved. However, he has only been doing the treatment since
August, and sometimes he gets too brave.” She notes that Carlos recently
suffered a skull fracture when he tried to get off the toilet on his own.
“I feel less pain now since I started the therapy,” he says,
as he gently touches the small bump on the left side of his head where he had
the skull fracture. “It still hurts a little though. If I was not doing the
drug treatment, it would probably hurt more.”
Prior to taking this drug, Carlos was unable to crawl or sit
up on his own, and would get fractures at least every one or two months. Now he
can use a walker in therapy, and he can grip a pencil more securely when
writing.
Bisphosphonate therapy is still at the experimental stage in
the treatment of OI. Carlos is receiving treatment as part of a research
project being conducted by Dr. Francis Glorieux and colleagues at the Shriners
Hospital in Montreal.
Last October, the New England Journal of Medicine (NEJM)
published results of their initial findings, arising out of an uncontrolled
observational study on bisphosphonate therapy. The study, one of the first to
investigate the use of bisphosphonates in this patient group, involved 30
children who were three to 16 years old and had severe osteogenesis imperfecta.
The children were administered the drug pamidronate intravenously. The results
of the study found that chronic bone pain decreased, motor function improved,
bone mineral density increased, and bone resorption decreased.
Dr. Glorieux has extended the study of the effect of
pamindronate to other children with OI. “We are currently following more than
170 patients suffering different types of OI,” Dr. Glorieux says. “It includes
all the departments involved in the care of patients with OI. [We] aim to
respond to all their needs, not only the medical management of their condition.”
As director of research at Montreal’s Shriners Hospital and
professor of surgery, human genetics and pediatrics at McGill University, Dr.
Glorieux has treated people with OI for the past 25 years. He found other
medical treatments for OI unsuccessful, so he turned to bisphosphonates after
seeing their effects in animal studies and in adults with osteoporosis.
The main concern against the use of these drugs in children
was that it could affect growth. So he started treating very severe patients
for whom height was not an issue. He discovered that growth was not arrested.
When a person like Carlos goes to Montreal for the
treatment, it is for a three-day period every four months. During his first
visit, the doctors made a small cut inn his hip to get a sampling of his bone
structure. For the next three days, Carlos had pamidronate taken intravenously
for six to eight hours each day.
Dr. Horacio Plotkin, one of Dr. Glorieux’s colleagues and
co-author of the report in the NEJM, said in a phone interview from Montreal
that the only side effect to pamidronate found is an “acute phase reaction.” It
is a flu-like syndrome patients often experience the first time they receive
the drug. This reaction resolves in 24 hours, and usually does not reappear
with new infusions.
There are no other similar treatment facilities in Canada. Dr.
William Cole, chief of Orthopedic Surgery at Toronto’s Hospital for Sick
Children, says he is supportive of Dr. Glorieux’s study, and has referred
patients to him. At this time, however, there are no plans to open a similar treatment
facility in Toronto, as the treatment is still experimental. The Shriners
Hospitals in Los Angeles and in Springfield (Massachusetts) are providing the treatment
to selected cases.
Since Dr. Glorieux began his bisphosphonate therapy clinic
in 1992, OI children from the United States, France, Brazil and Kuwait have
come to Montreal for the procedure once every four months.
“There is no plan to end the study. All patients are
improving, so we do not want to discontinue the treatment,” Dr. Plotkin
explains.
The long-term benefits of bisphosphonate therapy are not
known, and the knowledge of side effects is limited to seven years at the
Montreal clinic. “It is probably the only treatment so far that is able to
produce significant and positive changes in the quality of life of the
patients,” says Dr. Plotkin, currently co-chair of the Pediatric Committee of
the American Society of Bone and Mineral Research. “It is not the ideal
treatment, and active research is taking place to find better treatments. Currently,
we are only accepting patients under four years of age in Montreal, due to the overwhelming
demand.”
As a 25-year-old adult with OI, I expressed personal
interest in bisphosphonate therapy to Dr. Plotkin. But he cautioned me that
bisphosphonate therapy may be less effective for anyone whose bones have
stopped growing. Alendronate would be most appropriate for adults with OI,
according to Dr. Plotkin.
At present, there are no reports that confirm the benefits
of bisphosphonate therapy for adults with OI. In the long term, Dr. Plotkin
believes it could help strengthen bones and reduce the likelihood of recurrent fractures
later in life, when bone loss occurs with age. One serious side effect of
alendronate is that it may cause esophagitis (i.e. an inflammation of the
esophagus).
Both Dr. Glorieux’s paper and the editorial commentary in
the NEJM make clear that bisphosphonate therapy is not a cure for OI. It reduces
the rate of fractures, and improves bone mineral density as seen on X-rays. According
to the editorial commentator in the NEJM, this treatment provides cause for cautious
optimism. It strengthens the bones, but may not enable the more severely
affected patients to walk. For some patients, walking is not their primary
concern.
“[Dr. Glorieux] seemed to be so determined that I would walk
and that’s not a big priority for me. It took a while for them to understand
that,” insists Kristin Hayes, a 15-year-old who has been having bisphosphonate
therapy since 1997. “I wanna get stronger but walking [to me] is not that
important… After my first two treatments, my swimming coach noticed my
endurance ad speed had suddenly improved, and, of course, bone density
improved.”
As she hadn’t had bone pain or a fracture for four years
before starting the treatment, the effects were less dramatic than those seen in
younger, more severely affected patients.
In Dr. Plotkin’s view, bisphosphonate therapy has opened the
door for new treatments, has increased the interest of the medical community in
the disease, and he hopes it will lead to a change in the lives of patients
with OI.
It has already given hope and strength to Carlos and his
mother Eusemia, who feels more confident knowing her son is benefiting from the
treatment. “The doctors told us that we could stop the treatment at any time,
but we will keep going with it, because it is making his bones stronger,” she
says as Carlos sits in his chair watching television at home after a long
school day. “Carlos has become so confident since starting the therapy that he
thinks he can do anything. He is not strong enough yet to walk on his own, but
the doctors said someday that may happen.”
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