New Clinical Manufacturing Facility Launches to Develop Novel Gene Therapy Tools

New Clinical Manufacturing Facility Launches to Develop Novel Gene Therapy Tools

The Raymond G. Perelman Center for Cellular and Molecular Therapeutics (CCMT) at Children’s Hospital of Philadelphia unveiled the Clinical Vector Core’s new manufacturing facility to produce biotechnology tools that will deliver cell and gene therapy for difficult-to-treat diseases.

CHOP partnered with the CEO Council for Growth of the Chamber of Commerce for Greater Philadelphia to officially open the facility in October 2018 and highlighted the city’s position as an important hub of scientific and medical innovation.

“Philadelphia is a city of breakthroughs,” said Madeline Bell, CHOP President and CEO, during the event. “In this facility, we make the tools — the vectors — that scientists use to deliver cell and gene therapies, bringing dramatic precision medicine treatments to patients.”

CHOP researchers developed two vectors that are the among the first gene therapies approved by the U.S. Food and Drug Administration: CAR T-cell therapy (Kymriah®) to combat an aggressive form of pediatric acute lymphoblastic leukemia and voretigene neparvovec (Luxturna™) to treat a form of inherited blindness.

The Clinical Vector Core focuses on tapping into how viruses efficiently infect and transfer their genes to host cells. Core Director Johannes van der Loo, PhD, leads a team dedicated to producing high-quality viral vectors for numerous preclinical and clinical trials at CHOP and other facilities.

The Clinical Vector Core was established as a Core facility within the CCMT more than a decade ago, and it began at a time when treatment of patients through gene therapy at CHOP was just getting underway, Dr. van der Loo noted.

At first, the Clinical Vector Core developed one clinical product a year, but it now works on approximately 10 each year, according to Director of Quality Olga Zelenaia, PhD. It has also expanded from delivering products primarily for CHOP and the University of Pennsylvania to national and international clients.

Congenital Hyperinsulinism Center Celebrates 20th Anniversary and 500 Surgeries

Congenital Hyperinsulinism Center Celebrates 20th Anniversary and 500 Surgeries

The Congenital Hyperinsulinism Center at Children’s Hospital of Philadelphia celebrated its 20th anniversary in October 2018 and marked its 500th surgery, which saved the life of a baby girl named Alaya from North Carolina.

Congenital HI is a life-threatening, genetic disorder in which the insulin cells of the pancreas secrete too much insulin. Fortunately for Alaya, doctors discovered her low blood sugar just moments after she was born. She was later diagnosed with HI and brought to CHOP, where the HI team determined Alaya had focal disease, and a PET scan found a legion on the head of her pancreas. It took two surgeries for Surgeon-in-Chief N. Scott Adzick, MD, to remove the entire lesion.

Established in 1998, CHOP’s HI Center was the first of its kind in the United States. Today, it is the largest and most active HI Center in the world and has evaluated, diagnosed, and treated more than 1,000 children with congenital HI from around the nation and the globe. The 500 pancreatectomies performed at CHOP far exceeds the number performed by any other hospital.

Building on the momentum of the clinical program, Diva De León-Crutchlow, MD, MSCE, director of the Congenital Hyperinsulinism Center, saw an opportunity to expand and integrate the combined scope of the clinical and research programs under the auspices of the new Frontier Program for the Advancement of Hyperinsulinism Care and Research.

Co-principal investigator Dr. Adzick and a multidisciplinary team of collaborators are working toward a future where novel ways to treat, monitor, and support young patients with HI throughout their lifespan will improve long-term outcomes. They anticipate that Frontier Program support will give rise to the discovery of new genetic forms of hyperinsulinism and broaden scientists’ understanding of disease mechanisms that will help to develop effective targeted therapies.

Karabots Pediatric Care Center in West Philadelphia Marks Fifth Anniversary

Karabots Pediatric Care Center in West Philadelphia Marks Fifth Anniversary

The Nicholas and Athena Karabots Pediatric Care Center in West Philadelphia celebrated five years of providing innovative community healthcare and programs for thousands of children and their families.

The Karabots Center opened in 2013, funded by a gift from philanthropists Nicholas and Athena Karabots to provide primary care services and programs for West Philadelphia families. The 52,000-square-foot environmentally sustainable building contains 56 child-friendly exam rooms and dedicated space for radiology, hearing and vision testing, and a phlebotomy laboratory.

The center works closely with West Philadelphia community leaders to best meet the needs of families in the area. Over the past five years, it has served 32,000 patients, with 85 percent enrolled in medical assistance, and held more than 700 community events on site.

The Karabots Center additionally serves as a base of operations and research initiatives for 16 diverse community programs, including the Center for Grieving Children, Community Asthma Prevention Program, Homeless Health Initiative, Keystone First Cares Program, Kids Smiles, Lutheran Settlement House-Domestic Violence, Pediatric Research Consortium, PriCARE Parenting Program, Reach Out and Read, Refugee Health Program, and Violence Prevention Initiative.

One of the center’s most unique outreach efforts, the Karabots Garden, brings together more than 1,300 corporate and community volunteers to cultivate a wide variety of fruits and vegetables, which are then distributed to local families through the Early Head Start and Healthy Weight programs. The Karabots Garden has grown 4.2 tons of fresh produce in the past five years to share with the community. 

<em>Parents</em> Magazine Honors CHOP as One of the Most Innovative Children's Hospitals

Parents Magazine Honors CHOP as One of the Most Innovative Children's Hospitals

Parents magazine named Children’s Hospital of Philadelphia one of the 20 most innovative children’s hospitals in the nation in 2018.

The magazine surveyed members of the Children’s Hospital Association who identified hospitals with a proven track record of innovations that lead to medical advances. The survey also asked members about children’s hospitals that have adopted the latest technologies and led the way in sharing its innovations with other pediatric centers.

Parents specifically recognized CHOP for its significant contributions to the development of a groundbreaking treatment for advanced acute lymphoblastic leukemia (ALL) and its efforts to help other hospitals offer the new therapy to patients. CHOP researchers helped develop the first national and international clinical trials for an immunotherapy known as chimeric antigen receptor (CAR) T-cell therapy for children, a first-of-its-kind treatment approved by the U.S. Food and Drug Administration.

Parents magazine focuses on helping mothers and fathers navigate parenthood from pregnancy to school, including children’s health, safety, nutrition, and behavior issues, and is read by 11.8 million subscribers.

CHOP-Based Pediatric Device Consortium Expands With New Partnerships

CHOP-Based Pediatric Device Consortium Expands With New Partnerships

The Pennsylvania Pediatric Medical Device Consortium (PPDC), which is based at Children’s Hospital of Philadelphia, now spans from Philadelphia to Pittsburgh.

The PPDC announced a new partnership with two programs at the University of Pittsburgh to continue supporting the development of promising medical devices that address unmet clinical needs in children. The expansion follows the award of a five-year, $5 million grant renewal from the PPDC’s funder, the U.S. Food and Drug Administration. It is one of only five consortia in the nation funded by the FDA through the Pediatric Device Grants Consortia Program.

“With genetic diseases and congenital malformations, there are many circumstances where off the shelf products just don't work,” said Robert Levy, MD, William J. Rashkind Endowed Chair in Pediatric Cardiology and PPDC principal investigator. “The opportunity for us to set up a model to show how you can create personalized pediatric medical devices on a routine basis is one of the main goals I would like to accomplish over the next five years.”

Formerly known as the Philadelphia Pediatric Medical Device Consortium, the PPDC’s recent name change reflects its new, statewide scope. The PPDC’s new University of Pittsburgh partners are the McGowan Institute for Regenerative Medicine and sciVelo, which advances the university’s translational research in brain health, cell therapy, and digital health to create market-ready solutions.

The PPDC has assisted more than 150 innovative projects over the past six years and awarded more than 20 seed grants of up to $50,000 each to companies through a competitive application process. It has provided both funding and guidance for pediatric products including an airway clearance system, a powered arm brace, a speech-generating communication system, and a vision acuity test for preverbal children.

In addition to awarding grants to support pediatric device advancement and regulatory approval, the PPDC has launched a new early-stage development program in partnership with the Philadelphia-area medical device design company, Archimedic, to develop innovative pediatric medical device ideas.

The PPDC guides applicants through every step of the process, from building partnerships within the business community, matching inventors with funding, and introducing them to a medical device developer, to assisting inventors through the FDA submission process.

CHOP Receives Recognition as Center of Excellence for Rare Bladder Disorder

CHOP Receives Recognition as Center of Excellence for Rare Bladder Disorder

The Association for the Bladder Exstrophy Community (A-BE-C) named Children’s Hospital of Philadelphia one of eight Centers of Excellence in the United States that provides complete, effective care for children born with bladder or cloacal exstrophy.

Bladder or cloacal exstrophy is a rare disorder that occurs while a fetus is developing. The condition results in the abdominal wall not fully forming, leaving the pubic bones separated and the bladder exposed to the outside skin surface through an opening in the lower abdominal wall.

A-BE-C determined that CHOP and seven other hospitals meet the organization’s rigorous criteria for the treatment of bladder or cloacal exstrophy in infants and children. The criteria includes that the program is led by a physician/medical director who has at least five years post-fellowship training in pediatric urology/surgery; the hospital follows a sufficient number of patients with diverse manifestations of bladder and cloacal exstrophy to offer wide array of treatment options; and the hospital engages in single or multicenter research to share their patient information and program results.

The criteria also requires that the care team includes a pelvic floor physical therapist, and inpatient nursing and outpatient staff who have received specialized training. The care team must additionally collaborate with orthopedic surgeons, pediatric anesthesiologists, radiologists, plastic surgeons, and when necessary, neurosurgeons and gastroenterologists who have expertise in bladder and cloacal exstrophy.

Additional requirements are that board-certified behavioral health specialists are included in the care team to provide support to patients and families throughout treatment, and offer resource interventions, education, and individual, family and group therapy.

A-BE-C will bring all Centers of Excellence together at least once a year to share updates on care activities and research initiatives. Each Center must re-apply every three years to renew the designation.