1. Catherine Fairchild Calhoun, J.D.
July 12, 2011 (Tuesday)
FINAL Notes
Inclusive Conference Workshop: The Voice of the Caregiver – Building Relationships and Brand Advocacy through
Relationship Marketing Programs
Workshop Objectives: The key influencers with whom you must connect, communicate and build trust with in the rare disease
space is not limited to physicians and patients. An educated, persuasive and empowered influencer, unique to the rare disease
space, is the caregiver. Caregivers lobby, start foundations and drive research efforts. Building relationships and brand
advocacy through relationship marketing programs can ultimately create trust and value with this group. Join this engaging,
interactive workshop and leave with the foundation for an integrated continuous relationship marketing plan for the caregiver
target market, complete with goals, strategy and tactics which can be implemented right away.
Key Questions to Be Addressed:
What is ―Continuous Relationship Marketing?‖
How can social media and online tools be used to create trusted relationships with caregivers?
Workshop Outline:
:: Understand the Caregiver’s Journey from Overwhelmed to Empowered
Hear personal stories from caregivers
:: Learn the Four Key Principles to Adding Value in the Orphan Drug Market
Learn strategies to continually engage patients and caregivers
Collaborate with patient organizations as a trust agent
:: Interactive Exercise — Create the Foundation for an Integrated Relationship Marketing Plan
Create a plan with goals, strategy and tactics for immediate implementation
Systematically build relationships with critical influencers
Utilize online tools and social media to create trust and value
12:00 Close of Workshop (There will be a 30-minute networking and refreshment break at 10:00 a.m.)
Wendy White
Featured Session
Founder and President
Siren Interactive
William (Bill) Cusick
Featured Session
Vice President of Creative Services
Siren Interactive
Catherine Calhoun
Featured Session
Writer and Mother of 8yr old son Billy who was diagnosed with a complex health condition
www.supercatcalhoun.com
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2. “Caregiving: Buckle Up for a Rare Disease Adventure”
By Catherine Fairchild Calhoun, J.D.
Hi, my name is Catherine Calhoun. I am a “recovering” environmental law attorney from
Louisiana. My son Billy now 9 years old was diagnosed with a complex rare condition called
MCCUNE ALBRIGHT syndrome almost 5 years ago, in October 2006.
This is a photo of Billy with his big sister Ella.
When Billy was diagnosed in 2006, I stepped away from law practice and conquering MCCUNE
ALBRIGHT became my new profession.
My presentation today will focus on my caregiver experience. And hopefully provide some insight
for you on how caregivers in the rare disease community work through the health care process.
We usually begin the process scared but over time we reach a point where we are quick
effective communicators and whipass patient advocates.
A little about me, I sit on many boards and commissions. I’m a trained policy advocate for
disability issues. I’m totally addicted to social media. I’m a graduate of Georgetown’s Kennedy
Institute Bioethics Course. I’ve been interviewed by the WSJ and featured on NPR’s Morning
Edition. I have my congressman’s personal email and correspond in initials only.
I probably know more people with MCCUNE ALBRIGHT and working on MCCUNE ALBRIGHT than
anyone other than Dr. Mike Collins, the clinician and researcher at the National Institutes of Health
(NIH) working on an assay right now to target MCCUNE ALBRIGHT. I’ve fought with insurance
companies and billing departments. I’ve essentially stalked researchers and clinicians. I’ve started
a bone research fund. And I’ve begged for narcotics. I’ve been busy.
It’s been exhausting, yet an exhilarating almost 5 years. I’ve always been a curious learning sort
of person, adding the motivation of my love for Billy (and sometimes guilt, I so wish I could do
more for my sweet boy) has been very powerful. One person, building relationship after
relationship, can really make a difference.
MCCUNE ALBRIGHT is a spontaneous genetic mutation related to a methylation error within days
of conception, one letter goes wrong. Billy should be “CGT” and instead is either “CAT”, “TGT” or
“AGT”. This “mistake” causes big endocrine and bone troubles (the bone disease is called fibrous
dysplasia, it makes bones fragile, deformed and painful). It’s a gain of function mutation which
means that any Gs alpha process touched by the mutation does “crazy” things, it’s a “too much”
situation:
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3. Too much growth hormone,
thyroid hormone,
cortisol,
puberty hormones,
too much bone turnover
This is a poster prepared by researchers at the National Institutes of Health that shows the
spectrum of trouble related to MAS.
Actually, MCCUNE ALBRIGHT is kinda like a genetic cholera (arg201 is the site of ADP-
ribosylation by the cholera toxin). But unlike cholera there is no medication to stop the hyper
process. Hormone problems are treated with various medications in a guessing or artful cat and
mouse game. Bone problems are treated with surgery, with very limited success.
Billy’s first set of rods.
Billy has had 3 bone surgeries so far, custom titanium blend nails placed in each femur. We
expect to do this every 2 to 3 years as he grows.
There is no true fix for the bones, Billy uses a wheelchair and walker. He suffers with significant
bone pain, intermittently. My best estimate is that 80 percent of the time he does alright. The
other 20 percent, that time is spent crying all night curled up in a ball of pain. It’s very hard to
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4. see as a mom, harder still as an attorney mom used to fixing things with hard work and the right
connections.
Experts guess the prevalence of MCCUNE ALBRIGHT as 1 in 100K to 1 in one million. If severe,
MCCUNE ALBRIGHT can be fatal. If moderate, MCCUNE ALBRIGHT can be very physically
disabling and significantly deforming. It is possible to have a mild case of MCCUNE ALBRIGHT
and not really even know you have it, maybe catch a small spot of fibrous dysplasia with a
random x-ray later in life.
It is a lifelong condition, progressive through the growing years with lifelong complications,
lifelong medications. It’s a condition that qualifies for Make-a-Wish.
* Caregivers in rare disease worry, I worry about what will happen to my sweet boy living with a
lifelong expensive complicated health condition. This worry forces me to acknowledge the
fundamental fact that I’m alive but will die before long and “who will care for my baby like I
do?” This worry is palpable and something we rare moms just whisper about when talking things
like special needs trusts and long term care options.
MCCUNE ALBRIGHT also includes “café au lait” spots like the ones in this photo of Billy taken
when he was about 2 years old (one of my absolute favorite ages for little boys).
These spots don’t really cause trouble per se but are something that an astute doctor might
appreciate early on (Billy’s appeared by age 2 weeks old). I worried that they were a marker
for neurofibromatosis 1 and went down that path for several years, trying to rule NF1 out for
Billy. I bought the $80 book on NF1 and carted it with me from doctor appointment to doctor
appointment, certain that something was medically wrong with my sweet boy.
Billy was diagnosed with MCCUNE ALBRIGHT at age 4.5, the average age of diagnosis. Genetic
testing is available but the mosaic nature of the condition makes a genetic test catch tricky, false
negatives.
DIAGNOSIS:
We figured it out because he was limping one Monday morning.
He played rough on a Sunday. Limping that Monday. X-rays Monday afternoon, lesions covering
the femurs, no clear diagnosis. Bone doctor in New Orleans on Tuesday, still not certain on what it
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5. was. Bone doctor number 2 in New Orleans on Wednesday. Of course, when I pulled out the NF1
book, there was MAS listed as a possible alternate diagnosis, I just never thought bone trouble so
completely missed it as a true possibility for Billy until we learned of the fibrous dysplasia.
Thinking of that Wednesday still gives me chills, the doctor and medical students had just studied
MCCUNE ALBRIGHT and with one look at the café au laits, there was no question, MCCUNE
ALBRIGHT for sure.
Billy had fractured his right femoral neck. He was ordered into a wheelchair and spent the next 4
months wheeling or scooting around the house like a baby monkey while his bone doctor planned.
Billy was this doctor’s first MCCUNE ALBRIGHT patient. We kind of laugh about this now. It just
makes more sense with his bones to have someone near, the true “expert” is in Pensacola. When
Billy’s doctor said he would work with the Pensacola doctor, run the x-rays and details by him, we
said “you’re the guy.” Billy had his first big bone surgery in February 2007 and then again in
February 2009 and most recently February 2011.
I send patients to Billy’s doctors all the time, mostly his bone doctor and endocrinologist. They call
these “consults for Dr. Calhoun” (which I not so secretly love). They also describe me simply as “she
is a character.” Moms talk so we know these things.
I’m a medical minimalist to the core. Before Billy’s diagnosis I barely wanted to give him an
antibiotic or an allergy pill despite recurring ear infections with 4 sets of ear tubes and a
tonsillectomy by age 4. But medicine is not optional for him now.
This is a photo that Billy’s dad texted me while I was out of town, he was confirming that the
nighttime meds had indeed been remembered and were about to be given. The other photo is the
pill case I found at a local pharmacy. When I bragged about it on FB, several other moms
commented that they had the same, one friend uses it to organize jewelry.
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6. (Thyroid)
Once we got a grip on the bone situation we moved into endocrine testing. Billy came back as
hyperthyroid (a scan showed one side of his thyroid was “sleeping” and the other side was really
cooking with 2 or 3 hot nodules pumping out hormone). He was started on methimazole. He took
methimazole from January 2007 to November 2008.
I was never comfortable with the methimazole because I know a MCCUNE ALBRIGHT kid who
took it for years and then woke up one day with a horrible reaction and ended up in the ICU
unable to breath and then doing an emergency thyroidectomy. Knowing this kid made the risk
real to me.
You know, once your kid is diagnosed with something rare like MCCUNE ALBRIGHT, I think you no
longer respect statistics the same way, you know that you could be that 1, you’ve been that 1, it’s
irrational but true still.
Practically speaking, controlling the thyroid levels was challenging. And with each bone surgery I
worried about the unlikely but possible “thyroid storm”. You read about one MCCUNE ALBRIGHT
kid having thyroid storm, it seems like a real risk.
As soon as Billy’s first femur surgery was settled, I started researching the best thyroid surgeons in
the U.S. I read journal articles on the procedure and found my place, Yale. We flew to New
Haven and did the surgery in November 2008. He did great, no calcium problems and was able
to leave the next afternoon.
He has taken Synthroid since. We often go back and forth with the pharmacy on brand versus
generic, his endocrinologist stands firm, brand only for Billy on the Synthroid. Perhaps because I
am worried about weight gain, a real issue for a kid who spends lots of time immobilized, I am
picky on this one.
(Stomach)
In the spring of 2008, Billy started complaining of stomach pain. By the end of the summer, he
had vomited blood in his sleep three times. I talked with the pediatrician with no success. I found
Billy a pediatric GI doctor and took him in. Because he had recently done the femur surgery the
doctor held off on doing a scope or anything, and went straight to prescribing Prevacid, melting
tablets. Within weeks his stomach was pain free with no more vomiting blood. After awhile he
switched to capsules because he doesn’t like liquid or melting medicines. He prefers to swallow
pills, since age 4, seriously.
Now for fun we have contests at our house on who can swallow the most pills at once. Probably
that means we are freaks, maybe. I’m the current winner with like 8 pills including vitamins and
fish oil type pills.
I’ve tried to reduce his Prevacid dose, he takes 30 mg every morning, with no success, within one
week of a lower dose, he was miserable. It was like pouring gasoline into his stomach. His doc did
an upper GI scope in December 2010, he has some gastritis and some past damage to the
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7. esophagus, nothing shocking. His GI doctor (Billy’s second), is recommending a Nissen
fundopolication. He’s convinced that the Prevacid will worsen Billy’s already problematic bones.
He says: “If this were my kid, I would do the surgery. Get Billy off the Prevacid.”
Thing is that I have done a lot of reading and still am not convinced that surgery is the way to go
on this. Plus, I know another MCCUNE ALBRIGHT kid who outgrew the stomach troubles. So for
now, we are ignoring the surgery recommendation and sticking with Prevacid (generic form). I
suppose we might have to find a new GI doctor because the current doctor was clearly angered
that I haven’t done the surgery at Billy’s last check-up.
I know this guy is smart but wonder if his young age means that maybe he has awhile to go
before he has that same patina that our favorite doctors have, the patina of humility and
compassion for moms just trying to keep their sweet boys in one piece.
(Allergies)
Billy sees an allergist/immunologist. He has really sensitive skin so we’ve done the whole gamut of
pokey things in his back to test for triggers. The only hit is a simple histamine excess, a big one.
I promise, you’ve not lived until you too have this written on your hand. Until you have collected urine
from a 9 year old for 48 straight hours while touring around DC.
In the past Billy has hived with Septra and Morphine by IV post bone surgery. His allergist
recommends a Claritin in the morning and a Zyrtec at night. We do generic for both. We also like
Clarinex but think it’s too expensive since the other medicines seem to work okay enough. Billy
also takes plenty of Benadryl here and there, generic also.
(Pain)
The hardest part of MCCUNE ALBRIGHT?
Pain.
Billy suffers from what I call “chronic intermittent acute bone pain in his hips and femurs.” He is ok
most of the time, but when he is not ok, it is really bad. Like really really bad. The bone scan in
June 2011 at the National Institutes of Health showed an active “hot” spot of fibrous dysplasia in
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8. Billy’s left leg. This is the exact spot that causes him the most grief. The doctors can’t really explain
why Billy’s fibrous dysplasia is as bad as it is, as painful as it is.
Pediatric pain is complicated. A lot of doctors we’ve seen over time are very uncomfortable with
believing that Billy has pain, are uncomfortable prescribing medication, even medication like
Tylenol 3. They say things like “but he is going to get addicted.”
And I say: “But I am the dispensary and will not allow him to get addicted, his pain is real (would
you like to comfort him at midnight, 2 a.m. and again at 5 a.m. as he cries in his sleep curled into a
tight ball of sadness?),
And when Billy feels good he wants to live his life, not take pain meds for some sort of life
escape.”
I wouldn’t recommend these types of conversations.
Almost 5 years into this, I have worked out a process in which I don’t have to beg for narcotics
anymore, thank goodness. Billy switched from a pediatrician to a family practice doctor who
believes that a child can have pain. When his pain is really bad, this doctor will prescribe Tylenol
3 or Lortab, pills (Billy refuses liquid medicine, hates it).
A recent arm break is a good vignette of the pediatric pain world, at least in our area.
On the Saturday afternoon of Memorial Day weekend:
Billy and his big sister, Ella, were having an exciting water gun fight, mega big water guns. Billy
was going in and out filling the gun, holding his walker with one hand and the water gun with the
other hand (probably not an approved use). Things did not end so great. Billy ended up on the
floor screaming with his arm visibly broken. I thought Ella was gonna vomit. I immediately
snapped into crisis super mom mode, barked for the screaming and freaking out to stop so I could
assess the situation and call for help.
I called the number for the closest not scary emergency room to find out which bone doctor was
on call. Billy’s local bone doctor was on call, the operator connected me through to him on his cell
phone. I explained the deal. He said: “I will meet you there. Dr. T, an ER doctor and personal
friend, is there now. I will call and let him know you’re on your way.”
I gave Billy a Tylenol 3 and then lifted him off the floor. And then got him into the car, dripping
wet. We got to the ER, Dr. T almost immediately gave Billy Stadol for pain. And offered more if
needed. After several hours NPO, the doctors sedated Billy to reduce the break and splint it. It
was broken into several pieces, right through a big area of fibrous dysplasia. When Billy was
ready to leave they insisted I take a prescription for Tylenol 3. And the discharge nurse totally
got it when I joked that we collect orthopedists.
On Monday, I drove Billy to New Orleans to see his regular bone doctor, his surgeon. His surgeon
wasn’t satisfied with the bone alignment. So we whiled away hours, NPO for another sedation
and reduction in the ER of the Children’s Hospital. It took his surgeon and a resident almost an
hour to straighten the bones. They had expected it to take 15 minutes. They came out sweating.
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9. When Billy was ready to leave, I asked the ER doctors to give him something good for pain
because transferring him into the car was going to be a challenge. They said: “He had Versed
during the procedure, twice.” I said: “Not gonna be good enough.” They said: “Let us ask him
about his pain.”
Doctor: “Billy, do you have any pain?”
Billy: “Yes, my arm.”
Doctor: “Which arm, can you point to where it hurts?”
Billy: “My whole right arm hurts.”
There was some more hemming and hawing. And then I stated as clearly and not crazy as
possible, “I will not leave with this child until you give him some Dilaudid.” They offered half a
dose, and we hit the road home.
Not over yet.
Billy didn’t get better. Cried and cried, no sleep. I texted his PCP from a conference in DC and
asked for something better than Tylenol 3, some Lortab. Done.
And then when Billy’s dad took him in for a checkup the next week, well, Billy lifted his arm to
reveal a blackness of pressure sores under his arm from the splint rubbing. This was the pain, not
the bones, but the pressure sores.
The splint was removed and replaced by many fancy silver bandages and a different splint type
thing. Also, a Levaquin prescription. Now that the wounds are healing, his pain is minimal.
In addition to over the counter things like regular Tylenol, Advil and Aleve (generics), and
narcotics, Billy does infusions of bisphosphonates for pain, specifically Aredia. Usually he will sit
for 2 days at a local infusion clinic, 4 to 6 hours each day. The medicine is ordered with his
endocrine doctor. He does this every few months. Now that he is part of the NIH MCCUNE
ALBRIGHT study, we will likely fly to DC for Zometa the next time he needs an infusion. The
Zometa is more potent and should last longer with a faster infusion time as well.
There is a new medication that people in the MCCUNE ALBRIGHT community are talking about,
Denosumab. It’s very strong as I understand it. We will not try that one until at least 5 other
pediatric patients try it with good result. This is information that I expect we will gather simply
through word of mouth channels. An adult friend, a bone doctor with fibrous dysplasia, has
volunteered to try Denosumab, I will most certainly tap him for information too.
(So where am I headed?)
As I mentioned at the beginning, Billy has pretty much been my profession for the past almost 5
years. I’m so happy to share with you that seeing the results of all the efforts of everyone who
loves him (which is lots of people) is even better than I would have ever imagined.
He had the most wonderful year in school, moving onto the fourth grade this fall. His teachers are
tough on him but compassionate. By the end of the year he has them wrapped around his sweet
little pinky, of course. All of the teachers are always glad to have him in their classes because his
fragile bones mean no classmates with severe behavior problems, it’s just too risky for the school
system. When someone says that having Billy for a student is a “blessing,” well, I am just not sure
that life gets better than that. He has a fantastic medical team and the sweetest possible friends.
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10. This is a card that his best friend Jarrett made for him for his most recent surgery.
Seeing Billy in such good shape means I sleep better at night and am turning my attention to
reinventing my professional hopes, I’m pursing work now in health administration/policy/law.
And working on a bioethics project with Michael Malinowski, an LSU Law professor. Taking care
of Billy has given me a unique and powerful perspective of health care, and a passion for
bioethics.
So how do I decide what medications for Billy? Brand or generic? It really depends on each
situation. Do I know any kids with a specific experience? I will call the parents to ask for details.
Have I heard a good or terrible story about it? When I mention it to a trusted nurse or clinician,
how does he or she respond? I watch for body language clues on this.
I don’t think that I am unusual in this for the rare disease community. We really rely on each other
and want to know. In the rare disease community you really can know everyone who wants to be
known or found. We share our stories at conferences and online. We make ourselves available to
each other in a way that is hard for people outside this community to believe. It’s like a secret
club, a powerful, empathetic, “no bullcrap” (as my Truly Mom in East Texas likes to say) secret club.
In October 2006, I set an attorney mom type goal, I told myself: “Billy will be the kid in this world
with MCCUNE ALBRIGHT to get the absolute best possible medical care.”
I now believe in my heart and soul that Billy is as ok as he can be, and he is going to be as ok as
he can be, so the goal now is less about “best possible” and more about “good enough” and
getting back to living our lives.
I’ve been so busy. I’ve been so humbled. I’ve been so amazed by the kindness and grace of
people, health care providers and strangers even. I’m like the Grinch, caring for Billy, my heart
has grown 3 sizes, maybe more. I believe so strongly now that you just never know, you never
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11. know when pure awesome will appear, to open a door for you, to create an appointment for
you, to respond to a stressed out text message or email, you just never know.
This slide is a visual of “pure awesome”, this is what it feels like, the feeling of humility and
amazement and rare disease community.
I will close with my favorite quote:
Guillaume Appollinaire, and I hope this one will inspire you like it inspires me to find a way to
better support and serve the rare disease community,
“Come to the edge of the cliff,” she said.
“We’re afraid,” they said.
“Come to the edge of the cliff,” she said.
“We’re afraid,” they said.
“Come to the edge of the cliff,” she said.
They came.
she pushed.
They flew.
When you partner with caregivers like me, and I am not all that unique, we all fly. You can find us
– online, at conferences, in doctor offices and in hospitals, we’re busy keeping our kids in one
piece, you might have to come to us, but know that it will be worth your time and effort.
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