What We’re Thankful for This Year: 2016

In celebration of Thanksgiving, members of the CHOC Children’s family express what they’re most grateful for this year.

thanksgiving at chocMary Green 

Registered nurse in the Hyundai Cancer Institute at CHOC Children’s

“I could list 100 reasons why I am thankful for CHOC. I’m thankful to work at a place with such visible growth: in the number of available treatments, in the percentage of children that are surviving cancer, in relationships between patients, family members and staff; and growth visible in children as they begin to believe how strong they truly are. Even more so, I am thankful that CHOC is passionate about celebrating growth and takes pride in celebrating all of the little things.”

thanksgiving at chocDr. Joanne Starr

Medical director, cardiothoracic surgery

“I’m grateful to be part of an innovative pediatric hospital and for CHOC’s commitment to providing patients and families with access to the best neonatal and open-heart surgery in Orange County.”

thanksgiving at chocDana Sperling

Social worker, NICU

“I am thankful for two amazing teams I am privileged to be a part of:  the social services team and the Neonatal Intensive  Care Unit (NICU) team.  The compassion and dedication of both teams makes me proud to work along side them day after day, delivering outstanding care to patients and families.”

 

thanksgiving at chocDr. Kenneth Grant

Chair of gastroenterology 

“I am thankful to be working for an organization that creates an environment where our patients become our family. I am also grateful that CHOC Children’s has the foresight to invest in the innovative ideas we have to improve the health care we provide. ”

thanksgiving at chocDr. David Gibbs

Medical director of trauma services

“I am thankful for the trust of our patients and families. With the strong support of the hospital and the community, our Level 2 Trauma Center is proud to care for children in Orange County.”

thanksgiving at chocJoani Stocker

Volunteer

“I am so thankful for the opportunity to bring smiles and laughter to our patients through Turtle Talk and the playrooms. Laughter is medicine to the bones, and I am humbled to be a part of the healing. My cup is overflowing with joy when I see a patient giggle and play.”

thanksgiving at chocDr. Daniel Mackey

CHOC Children’s pediatrician

“I am thankful for the opportunity to be partnered with an excellent children’s hospital. I am also thankful for the pleasure of working with other positive people who provide outstanding care to the children of Orange County. Together we work to improve the care and services we deliver to our most important resource…our children.”

thanksgiving at chocDr. Gary Goodman

Medical director of the Pediatric Intensive Care Unit, CHOC Children’s at Mission Hospital

I am most grateful to the people behind the scenes at the hospital that do all the invisible jobs that are so important to keep CHOC Children’s running: the housekeepers, lab and x-ray technologists, bio-medical engineers, pharmacy technicians, scrub technicians, security guards and maintenance staff that work tirelessly, 24-hours a day.”

thanksgiving at choc

Dr. Raymond Wang

Metabolic disorders specialist

“I am thankful that CHOC cares for families and children with rare disorders by supporting clinical trials and translational research, and the staff who care for these families, to find treatments and cures for their conditions.”

thanksgiving at chocEric Mammen

Lead music therapist

“I am grateful that I get to witness the transformative powers of music with amazing patients and families everyday here at CHOC. So very grateful for the generous donors that continue to support our growing music therapy program. Without them we would not be able to impact the families and help them face incredible challenges with courage, smiles, and a song. Super grateful to be apart of writing a powerful song with a patient in response to his medical diagnosis- “To Life Live To The Fullest!” Happy Thanksgiving and I hope you get to spend some extra time with your loved ones around you.”

Matt Gerlachwhat choc is thankful for

Executive vice president and chief operating officer

“At this time of Thanksgiving, I am thankful for CHOC Children’s and the wonderful service we are privileged to provide for the communities we serve. I am thankful for the dedication and commitment of our physicians, associates and volunteers, who give the very best they have to give— their knowledge, skills, abilities, care and compassion— to make CHOC’s mission to nurture, advance and protect the health and well-being of children a reality for so many in need, every day. I am also thankful for those that stand behind our physicians, associates and volunteers— their loved ones, who support our CHOC Children’s team to be the best that they can be, both at work and at home. I wish all of our CHOC Children’s family a happy and healthy Thanksgiving.”

Related posts:

CHOC’s Specialized Metabolics Team Treats Rare Genetic Disorder

CHOC Children’s highly specialized metabolics team identifies rare and dangerous genetic disorders before it’s too late. And early, non-invasive screening is a big reason CHOC metabolicswhy Henry Louderback celebrated his fourth birthday last year.

Henry was one of 100,000 kids diagnosed each year with tyrosinemia type I, a deficiency of an enzyme that, if untreated, causes liver failure and death. As one of the largest designated newborn screening centers in California, CHOC was able to save Henry, whose medication and diet will prevent the serious and life-threatening complications of the disease.

“He was losing some weight but no huge alarms went off,” Nicole Louderback, Henry’s mother, says of his condition shortly after he was born on Sept. 20, 2010. Newborn screening tests detected the disorder before he was a week old, allowing the metabolics team to start life-saving treatment before Henry’s liver went into failure.

Henry’s now a happy, normally developing child — thanks to what Nicole calls the “incredible” work of Dr. Raymond Wang, a CHOC metabolic disorders specialist, and other members on the metabolics team.

Learn more about metabolic disorders services at CHOC.

Related posts:

  • Physician Tenacity, Experimental Treatment Help Baby With Rare Disease
    A toddler with a devastating rare condition has a chance for health, thanks to an experimental treatment and the tenacity of a CHOC Children’s physician. Galya Chan, 2, is showing marked improvement ...
  • Resources, Tools for Parents of Children with Rare Diseases
    Parents often struggle with navigating how to raise a child with rare or genetic diseases. CHOC Children’s provides multiple channels of support including family-centered care, which empowers parents to become ...
  • Investing in Care
    Stephen and Cynthia Fry of Newport Coast are longtime philanthropists in Orange County who were introduced to CHOC Children’s three years ago when their younggranddaughter was diagnosed with a rare ...

Physician Tenacity, Experimental Treatment Help Baby With Rare Disease

A toddler with a devastating rare condition has a chance for health, thanks to an experimental treatment and the tenacity of a CHOC Children’s physician.

Galya Chan, 2, is showing marked improvement after nearly nine months of regular infusions of cyclodextrin, a compound that has helped mice with Niemann Pick C disease, the condition also affecting Galya.

Dr. Raymond Wang, a CHOC metabolic disorders specialist, says Niemann Pick C affects one in every 150,000 people and causes cholesterol to accumulate in the brain, lungs, liver and spleen, leading to deterioration and early death.

Even worse, there is no Food and Drug Administration-approved treatment for the condition.

A sick preemie

After a premature birth, Galya developed an extremely enlarged liver and severe jaundice. A battery of tests led to a full exome sequence, which revealed Niemann Pick C – and no available treatment plan.

“The diagnosis is horrible, but it’s better to know than to not know,” says Brian Chan, Galya’s father.

But Dr. Wang was aware of researchers studying the effects of cyclodextrin in animals with Niemann Pick C. The compound had shown to reduce cholesterol levels in their bodies, help animals survive without symptoms longer and have more overall longevity.

“The problem is there’s no company that’s willing to get behind the treatment for humans,” Dr. Wang said. “There aren’t enough patients with this condition for companies to see the potential and profit for this. It comes down to individual physicians who want to make a difference.”

Taking another route

So, with Galya’s parents in agreement, Dr. Wang began the long process to seek special permission to treat the baby with cyclodextrin.

Writing a treatment protocol especially tailored for Galya, Dr. Wang filed an investigational new drug application with the FDA. CHOC’s Institutional Review Board, a body that examines proposed research, also reviewed the proposed use.

Four months later, Galya became the youngest of just 11 patients nationwide to undergo this treatment.

Currently, Galya undergoes weekly intravenous cyclodextrin infusions. She also receives monthly intrathecal infusions, which are administered through a lumbar puncture to reach her central nervous system.

Each time, Galya is admitted to CHOC’s pediatric intensive care unit. Infusions last six hours, and she stays 15 more hours for observation.

Encouraging outcomes

But the time and effort is paying off: markers of Galya’s cholesterol storage levels have dramatically decreased since she began treatment nearly nine months ago, data shows.

Before receiving the cyclodextrin, those markers were tremendously elevated, more than six times the normal level. Today, her storage levels have dropped more than 80 percent and now hover just above normal, with progress expected to continue. Also, Galya’s liver softened and its volume decreased by about 10 mL.

Galya’s results are so good that Dr. Wang is working to adjust her treatment protocol to receive intrathecal infusions twice monthly.

Moving forward, Dr. Wang and his colleagues will continue to study Galya to determine if the treatments are also healing her lungs, and it’s likely the cyclodextrin treatment will continue indefinitely – or until a different treatment is developed.

“Once we found out the diagnosis, it was sad,” says Brian, Galya’s father. “But now we can put our energy into helping Galya and working to find a cure.”

Related posts:

  • Resources, Tools for Parents of Children with Rare Diseases
    Parents often struggle with navigating how to raise a child with rare or genetic diseases. CHOC Children’s provides multiple channels of support including family-centered care, which empowers parents to become ...
  • Investing in Care
    Stephen and Cynthia Fry of Newport Coast are longtime philanthropists in Orange County who were introduced to CHOC Children’s three years ago when their younggranddaughter was diagnosed with a rare ...