Life with hypoparathyroidism has been a challenging journey for a Texas Diabetes patient,
who for the purposes of this blog will be referred to as “the patient”. Diagnosed at the age of
23 after their parathyroid glands were removed during thyroid cancer surgery, the 54-year old
Frisco resident has lived with the condition for over three decades.
Hypoparathyroidism is a rare condition where the parathyroid glands, located near the
thyroid in the neck, produce insufficient amounts of parathyroid hormone (PTH). PTH plays
a critical role in regulating calcium, phosphorus, and vitamin D levels in the blood and
bones.
“When I was diagnosed, there was no treatment for the disease, just medications to
manage the symptoms,” the patient recalls. Over the years, they relied on an exhausting
regimen of medications—up to 64 pills a day, taken every three hours—to keep their
calcium levels stable and avoid severe symptoms. Despite their best efforts, they faced
over 200 hospitalizations due to low calcium levels.
In 2019, the patient learned about a clinical trial for TransCon PTH, a new hormone
replacement therapy designed to restore physiologic levels of PTH. After speaking with
their endocrinologist, they applied to participate in the trial the day it went live on
ClinicalTrials.gov.
Within a week, they were contacted by Texas Diabetes & Endocrinology Research team to
begin the screening process. “The first four weeks were spent getting my blood levels
optimized for the trial,” they explain. The staff at Texas Diabetes played a critical role in
guiding them through this phase. “They communicated frequently and really walked me
through the process. It felt like I was the only patient they were working with.”
After successfully completing the optimization phase, the patient began the daily
injections, not knowing at the time if they were receiving the actual drug or a placebo.
Although the treatment regimen wasn’t easy at first, the team at Texas Diabetes was there
to support them.
Seeing Results
Soon, the patient experienced results beyond what they could have imagined – confirming
in their mind that they were receiving the actual drug. For the first time in decades, their
calcium levels stabilized, allowing themto gradually reduce and ultimately stop taking
daily pills.
“In less than six months of starting the trial, I was able to come off all my medications,”
they said. “I hadn’t been in range in over 30 years, and suddenly, I was.”
As their body adjusted to the new treatment, the symptoms that had plagued them for
years—numbness, tingling,muscle cramps, heart palpitations, fatigue and brain fog—
began to disappear.
“I didn’t realize how much brain fog I was living with until it lifted. I couldn’t remember the
names of everyday objects, and I would struggle to getmy thoughts out. But now, I feel like I
can think clearly again.”
Advocating for Change
With their newfound energy, the patient began volunteering for the HypoPARAthyroidism
Association, a nonprofit dedicated to raising awareness and support for those living with
the condition. Passionate about helping others, their volunteer work quickly turned into
accepting a position on the board and eventually a full-time role with the organization.
One of their proudest accomplishments is spearheading an Externally Led – Patient
Focused Drug Development meeting with the FDA for PTH.
“You can read about symptoms on a page, but that doesn’t tell you how it impacts
someone’s daily life,” they explain. “I wanted the FDA to understand how
hypoparathyroidism affects real people.”
The meeting brought together patients and experts to share their experiences with the FDA.
Looking Forward
The FDA approved TransCon PTH and it is expected to come to market in early 2025. The
patient is grateful to Texas Diabetes for applying to be an early access provider so they can
continue to take the medication while waiting for it to become available.
Reflecting on their decades long journey, the patient emphasizes the importance of clinical
trials in advancing treatment options for rare conditions. “There’s a misnomer that you only
do a clinical trial when it’s your last hope, but that’s not the case” they say. “The drug has
given me a chance at life again, and I can’t thank Texas Diabetes enough for giving me the
opportunity to feel like a new human being.”
The patient hopes their story will inspire others to consider participating in clinical trials
and raise awareness of the transformative power they hold. “If I had to do it all over again, I
would, in a heartbeat. It has been life changing.”
You can learn more about our research department and currently enrolling clinical trials here. Please feel free to contact us at (512) 334-3505, option 4, to speak to a recruiter about our enrolling studies.
Don’t forget to follow us on Facebook and Instagram and check back with us eachmonth as we provide you helpful wellness and health information.
