Newborn babies requiring critical care have gained a big ally in the William, Jeff and Jennifer Gross Foundation. Formed by asset manager Bill Gross, his son and daughter, the foundation today donated $2 million to CHOC Children’s neonatal intensive care unit (NICU) on its main campus in Orange. The recent gift rounds the Foundation’s support of CHOC’s neonatal services to $7 million in the past 12 months.
Many hospitals offer intensive care units but only a select few are rated by the American Academy of Pediatrics as Level 4 – the highest rating available – and even fewer are ranked among the best in the nation, according to U.S. News & World Report. CHOC’s program features three NICUs, a team of board-certified neonatologists and special units for the smallest preemies, infants who need complex surgery, and babies who have neurological and cardiac concerns.
“CHOC’s neonatal services are unlike anything else offered on the West Coast, providing the highest levels of care and tremendous hope to families in the region. We are honored to continue our commitment to CHOC and the care of newborn babies,” says Jeff Gross.
Two years ago, CHOC expanded neonatal services on its main campus with the opening of a new NICU. Located in the Bill Holmes Tower, the 25,000-square-foot unit features 36 private rooms in a homey atmosphere with family sleeping quarters and storage spaces outfitted in warm colors and wooden accents to help parents feel more comfortable. Private NICU rooms are a new standard for improved patient outcomes. Benefits for babies include higher weight at discharge and more rapid weight gain. Also, they require fewer medical procedures and experience less stress, lethargy and pain. Researchers attribute these findings to increased maternal involvement.
CHOC’s NICUs include the 91-bed unit on its main campus, a 13-bed unit at St. Joseph Hospital and a 22-bed NICU at CHOC Children’s at Mission Hospital.
The newborn baby girl arrived at CHOC Children’s Hospital with a
life-threatening irregular heartbeat. Thwarting what could have become a
medical odyssey to find a diagnosis, rapid whole genome sequencing (rWGS)
pinpointed her condition within two days: Timothy Syndrome, an extremely rare
cardiac condition that put her at risk for sudden death.
With the genetic diagnosis in hand, CHOC physicians were able to
treat the infant with a medication normally contraindicated for her condition.
Her heartbeat was restored to normal, significantly reducing the risk of sudden
cardiac death. Her physicians, secure in her diagnosis, implanted a pacemaker,
dramatically improving her chances for a happy and healthy childhood.
Now almost 1, this baby is one of nearly 150 critically ill
infants who have undergone rapid whole genome sequencing (rWGS) since fall 2018
as part of Project Baby Bear, a pilot project to save babies’ lives – in
addition to healthcare costs.
With five participating California children’s hospitals, including
Project Baby Bear has proven the medical and economic benefits of this most
advanced and comprehensive diagnostic method.
Led by Rady
Children’s Hospital-San Diego, the program helps
infants who are undergoing intensive care and covered by Medi-Cal. By
pinpointing the cause of rare disease with rWGS, physicians can customize
treatment. Having a genetic diagnosis can eliminate the need for futile tests
and procedures while decreasing hospital stays. For parents and children, getting
a fast answer means less suffering and more peace of mind.
CHOC Children’s has been recognized by Press Ganey in multiple categories for the 2019 fiscal year. The three awards include:
The Pinnacle Award of Excellence recognizes organizations that have maintained consistently high levels of excellence over multiple years in patient experience, workforce engagement or clinical quality performance and is awarded to the three top-performing organizations in each category.
Survey Solutions Workplace of the Year is a title given to the top 20 organizations recognized for their associate engagement efforts out of over 1000 considered.
The NDNQI Outstanding Nursing Quality Award is presented annually to the best performing hospital in each of seven categories, including academic medical centers, teaching hospital, community hospital, pediatric hospital, rehabilitation hospital, psychiatric hospital and international. Seventeen quality measures are used to evaluate scores by unit type. Combine scores across the units to produce an overall score. The highest-ranking hospital in each category receives the award.
The gift from the Cherese Mari Laulhere Foundation will:
Endow CHOC’s mental health inpatient center. Opened in April 2018 for children ages 3 to 17, the center is the only inpatient facility in Orange County that offers specialized programs for kids younger than 12. The center will now be named the Cherese Mari Laulhere Mental Health Inpatient Center.
Establish the Cherese Mari Laulhere Young Child Clinic for children ages 3 to 8 who are experiencing behavioral and emotional challenges, mental health issues and school readiness challenges. Filling a gap in care, the clinic’s goal will be to help prevent long-term developmental problems for these young children.
Expand CHOC’s Intensive Outpatient Program, a mental health treatment program for high-schoolers with moderate to severe symptoms of anxiety, depression or other symptoms related to mental health conditions. The program will be expanded to middle school-aged children to help address the risk of suicidal ideation that can occur among pre-teens in grades sixth through ninth.
Advance trauma-informed care, including providing tools to pediatricians to help in identifying adverse childhood experiences, and connecting patients and families with resources. The support from the Cherese Mari Laulhere Foundation will enable CHOC to catalyze systems-level change in the treatment of pediatric trauma in the county to improve the long-term health and well-being of children.
“Although her life ended far too soon, Cherese left a lasting impact on people because of her passionate commitment to make a difference in the world. The gift from the foundation named in her honor will help advance her dream by bringing newfound peace into the lives of children with mental illnesses and their families,” says CHOC President and CEO Kimberly Chavalas Cripe. “I can’t imagine a more fitting way to remember such a bright, beautiful and giving spirit.”
Established to honor Cherese Mari Laulhere, who died in a tragic accident when she was 21, the Cherese Mari Laulhere Foundation provides funds to charitable organizations that reflect Cherese’s dedication to enriching the lives of children. In 2017, the Cherese Mari Laulhere Foundation donated $5 million to endow the child life department at CHOC Children’s Hospital. In recognition of the foundation’s generosity and to honor the memory of Cherese, the department was named The Cherese Mari Laulhere Child Life Department.
“Our donations are gifts from our daughter, who brought so much light and love into this world. As someone who advocated for the underserved, Cherese would be very proud of her role in supporting CHOC’s mental health efforts and helping change the trajectory of thousands of young lives,” says Cherese’s parents, Chris and Larry.
CHOC Children’s Hospital recently administered the first ever in-human dosing
of gene therapy for Hurler Syndrome, the severe form of mucopolysaccharidosis
type I (MPS I), a rare and progressive lysosomal storage disease.
successful, the gene therapy could present an alternative treatment for Hurler
Syndrome, which currently calls for a stem cell transplant for children younger
than 2 ½. While stem cell transplants are well-proven to help prevent most of
the neurologic decline that happens to untreated MPS I patients, the procedure
poses significant risks.
Patients with MPS I have a
genetic mutation that leads to a deficiency in alpha-L-iduronidase. This enzyme
helps break down chemicals called glycosaminoglycans, specifically dermatan and
heparin sulfate, the buildup of which can ultimately cause enlarged organs and
tissues, heart valve thickening, spinal cord compression, hydrocephalus and progressive
loss of intellectual milestones.
The hope is that the gene therapy – RGX-111, which is produced by REGENXBIO Inc. – will equip the patient’s brain cells with the information needed to make working alpha-Liduronidase enzyme to stop the glycosaminoglycans from building in the brain, says Dr. Raymond Wang, a CHOC pediatric metabolic disorders specialist and the study’s lead investigator.
The therapy was
administered through a cervical puncture in the neck. With three-dimensional
visualization and guidance from a computed tomography scanner, CHOC
interventional radiologist Dr. Tammam Beydoun carefully inserted the needle into
the fluid-filled space at the junction of the spinal cord and brain stem. Then,
Dr. Wang administered the gene therapy.
“I could not have
asked for a better implementation of everything we had planned for many months,”
Dr. Wang says. “I am so grateful for such an awesome team of people working
together for one common goal.”
Dr. Wang says the patient will be monitored indefinitely, with clinicians tracking the child’s cognitive and physical development, as well as measuring the quantity of alpha-L-iduronidase in the child’s body and spinal fluid to determine whether glycosaminoglycans dermatan and heparin sulfate are being broken down.
“I cannot guarantee if the
gene therapy is going to work, but the alternative was to watch and let this
patient get worse and worse and worse,” said Dr. Raymond Wang. “We’ve got to
Babies with MPS I show no
signs of the condition upon birth. To this end, MPS I was added to the list of
conditions tested for in California’s Newborn Screening Program in 2018. But
even with enzyme replacement therapy treatment, recommended to begin before age
2, the disease continues to progress, Dr. Wang says.
“Patients will reach a plateau in developmental milestones, and then they’ll start losing milestones,” he says. “Once they could speak, they lose the ability to speak. Once they were able to walk, they lose ability to walk. Then it gets to be really heartbreaking: Once they could eat, then they can’t eat anymore.”
Many patients ultimately rely
on feeding tubes, while often enduring airway problems, cardiac disease and
hydrocephalus. Quality of life is typically poor, Dr. Wang says.
Not only could a
successful outcome in this investigation dramatically change the course of a
patient’s life, but it could also pave the way for future gene therapy for
additional patients with rare diseases at CHOC, Dr. Wang says.
“This is an opening chapter for a very cool story,” Dr. Wang says.