Novel Therapeutics Remain a Primary Research Focus

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Leveraging Cutting-edge Technology

"Our goal is to identify pediatric- and congenital-heart disease specific targets of therapy and develop therapeutics for these targets, which will prevent the progression of heart failure and restore ventricular function once heart failure develops."

Through the combined expertise of researchers in the cardiovascular and omics fields, the Advanced Cardiac Therapies for Heart Failure Patients Frontier Program will leverage cutting-edge technology to realize several major advancements in the diagnosis and care of patients with heart failure, especially congenital heart disease. 

“Our goal is to identify pediatric- and congenital-heart disease specific targets of therapy and develop therapeutics for these targets, which will prevent the progression of heart failure and restore ventricular function once heart failure develops,” said Joseph Rossano, MD, program lead and principal investigator.

This program’s work to identify pediatric-specific heart failure biomarkers and molecular targets for precision therapeutics will lead to significant improvement in the clinical care of these patients through earlier recognition and institution of therapies. In addition, the Advanced Cardiac Therapies for Heart Failure Patients Frontier Program will consolidate and focus the multidisciplinary expertise needed to care for this complex population by creating a heart failure intensive care unit. 

Taking on Bleeding Disorders

"We aim to continue to build on these successes by developing the next generation approaches to realize the ultimate goal of hemophilia gene therapy to provide safe, predictable, and long-term benefit for all hemophilia patients."

Frontier Programs differentiate Children’s Hospital of Philadelphia because of their unique combination of translational research and exceptional clinical care of children with highly complex conditions. Hemophilia is the most common inherited bleeding disorder, but current standard of care is suboptimal and often comes with complications.

CHOP’s existing basic, translational, and clinical research expertise in coagulation and gene therapy ideally positions the new Novel Therapeutics for Bleeding Disorders (NoT Bleeding) Frontier Program to lead the clinical implementation of gene therapy as well as develop the next generation of therapeutics for bleeding disorders. The team’s clinical programmatic efforts serve as the basis for an expanded referral base, while the research efforts have marked capacity for the development of intellectual property and grant funding.

“Hemophilia gene therapy research at Children’s Hospital of Philadelphia provided the foundation for successful clinical trials in hemophilia A and B gene therapy,” said Lindsey George, MD, director of Clinical In Vivo Gene Therapy and an attending physician. “We aim to continue to build on these successes by developing the next generation approaches to realize the ultimate goal of hemophilia gene therapy to provide safe, predictable, and long-term benefit for all hemophilia patients.”

Potential to Change Standard of Care

"The results of this trial have the potential to change the standard of care for patients with T-LL and T-ALL."

By adding bortezomib, a proteasome inhibitor, to chemotherapy, the overall survival rate in children and young adults with newly diagnosed T-cell lymphoblastic lymphoma (T-LL) significantly improved, according to a Children’s Oncology Group study led by researchers at Children’s Hospital of Philadelphia. The findings, published in the Journal of Clinical Oncology, also showed that when chemotherapy was intensified, it eliminated the need for cranial radiation in 90% of children with T-cell acute lymphoblastic leukemia (T-ALL).

“The results of this trial have the potential to change the standard of care for patients with T-LL and T-ALL,” said the study chair David T. Teachey, MD, director of Clinical Research at the Center for Childhood Cancer Research at CHOP. “The data show that most patients with T-ALL no longer need cranial radiation for cure and also suggest bortezomib should be considered as part of the new standard of care for newly diagnosed patients with T-LL.”

New Treatment on the Horizon

"The FDA designation expedites the treatment’s track through the process, putting it on course to become the first therapy approved for Friedreich’s Ataxia."

The U.S. Food and Drug Administration granted Fast Track Designation and Orphan Drug Designation for a potential new therapy for the progressive neurological disorder, Friedreich’s Ataxia (FA).

The designation puts the treatment, called omaveloxolone, on an expedited course to become the first therapy approved for patients with FA. An oral, once-daily drug that works by activating Nrf2, a transcription factor that induces molecular pathways that promote the resolution of neuroinflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling.

David Lynch, MD, PhD, director of the FA Program at CHOP, collaborated with Reata Pharmaceutical and other scientists worldwide to evaluate omaveloxolone. They demonstrated the treatment has a potentially beneficial effect in slowing the progression of FA out to more than two years. The findings, which appeared in Neurodegenerative Disease Management, showed omaveloxolone modified the long-term behavior of FA, and when analyzed in a delayed-start fashion, each trial cohort benefitted equally from the drug no matter when they started omaveloxolone.

“The FDA designation expedites the treatment’s track through the process, putting it on course to become the first therapy approved for Friedreich’s Ataxia.” Dr. Lynch said.

Enhancing Data Usability

"Assembling a team across several CHOP centers has provided a perfect conglomeration of capabilities necessary for this large-scale project."

This year, John Maris, MD, and Deanne Taylor, PhD, began leading a team to facilitate the development of a platform that will enhance the usability of childhood cancer data. The Molecular Targets Platform will allow physician-scientists, advocacy groups, research communities and pharmaceutical industry partners to have easy access to childhood cancer data on a publicly available, computational platform, thus streamlining drug development for childhood cancers.

“Children’s Hospital of Philadelphia’s strength in genomics, computational biology, and drug development present the ideal environment for this endeavor,” said Dr. Maris, the Giulio D’Angio Chair in Neuroblastoma Research at CHOP, and professor of Pediatrics at the University of Pennsylvania Perelman School of Medicine.

Dr. Taylor, director of Bioinformatics in the Department of Biomedical and Health Informatics (DBHI) at CHOP, and assistant professor of Pediatrics at Penn Perelman School of Medicine, is leading the platform development. The dynamic platform will be continuously built upon and condense what is typically a few days’ computation work into a few clicks. The project is a collaboration between DBHI, the Center for Data Driven Discovery in Biomedicine, and the Center for Childhood Cancer Research.

“Assembling a team across several CHOP centers has provided a perfect conglomeration of capabilities necessary for this large-scale project,” Dr. Taylor said.