William, Jeff and Jennifer Gross Family Foundation donates $2 million to CHOC’s Neonatal Intensive Care Unit

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.

Offering answers, hope through rapid whole genome sequencing

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 CHOC Children’s, 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.

The other participating hospitals include: UCSF Benioff Children’s Hospital Oakland, UC Davis Children’s Hospital in Sacramento, and Valley Children’s Healthcare in Madera.

Here’s a breakdown of each site’s cases and diagnoses, as of August 2019:

Here’s a look at Project Baby Bear by the numbers:

  • July 2019: project funded
  • $2 million: California state funding
  • 5: Number of participating hospitals statewide
  • 150 approximate infants sequenced
  • 182 hospital days avoided
  • 3 to 4 days: Ultra-rapid results turnaround
  • 3 to 7 days: Rapid results turnaround
  • 6 million children on Medi-Cal
  • 150,000 children could benefit from rWGS

Learn more about the CHOC Children’s Research Institute.

Press Ganey recognizes CHOC Children’s with three awards

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.
Kathryn Van Dijk, CHOC Children’s Director of Talent Management and Engagement, and Randi McCarthy, CHOC Children’s Organizational Effectiveness Specialist

Read more about awards CHOC Children’s has received.

CHOC Children’s Receives Transformational Gift from Cherese Mari Laulhere Foundation to Expand Pediatric Mental Health Care

CHOC Children’s announced it has received a transformational gift from the Cherese Mari Laulhere Foundation to enhance and expand its pediatric mental health system of care. The announcement comes on the heels of the Conditions of Children in Orange County report, which highlights an alarming increase in the number of children hospitalized in the county for mental illnesses.

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.”

Cherese Mari Laulhere

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.

For information about Cherese and photos, visit www.cherese.org.

CHOC performs first in-human gene therapy dosing for MPS I

Physicians at 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.

If ultimately 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 procedure required five months of planning across disciplines at CHOC, including pediatric metabolic disorders, radiology and imaging, nursing and pharmacy, the pediatric intensive care unit and the CHOC Children’s Research Institute, as well as additional radiology expertise from another institution and support from REGENXBIO.

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.

(From top left, clockwise) Investigational pharmacist Winnie Stockton, radiology technologist Lesley Mercado, radiology technologist lead Nadia Casarrubias-Garcia, clinical research coordinator Eric Rodriguez, clinical research supervisor Nina Movsesyan, director of imaging services Windy Broking, pediatric metabolic disorders specialist Dr. Raymond Wang, interventional radiologist Dr. Tammam Beydoun and radiology medical director Azam Eghbal are part of the large team at CHOC Children’s who facilitated the procedure. The team also includes members of the radiology scheduling, information systems, health information management, research and nursing teams.

“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 try something.”

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.

Learn more about research at CHOC Children’s.