Finding the Cure for Childhood Cancer and Rare Diseases

While cell and gene therapies continue to hold immense promise for children with cancer or rare diseases, there are many diagnosed every year that are still in need of access to life-saving treatments.

According to the American Childhood Cancer Organization, approximately 1 in 285 children in the US will be diagnosed with cancer before their 20th birthday. And while about 85% of children with cancer live five years or more after being diagnosed, it remains the most common cause of death by disease for children in America.

When it comes to rare diseases, it's estimated that 1 in 10 Americans are living with a rare disease, and about half of those are children. Although gene therapies offer an opportunity to stop or slow down the disease, there are many rare diseases still without a cure.

As a leader in research, development, and the manufacture of novel cell and gene therapies, Charles River is also an advocate for finding cures for childhood diseases.

In addition to numerous partnerships with companies, organizations, and non-profits to advance pediatric treatments, we are a proud supporter of the Emily Whitehead Foundation and a Believe Ball sponsor.

Blazing the trail with CAR-T therapies

Tom and Kari Whitehead started the foundation in 2015 after their daughter Emily was the first pediatric patient to successfully undergo treatment for acute lymphoblastic leukemia (ALL) using a CAR-T cell therapy.

While experimental at the time, Emily has now been cancer free for 10 years and CAR-T cell therapies are considered a curative treatment for many childhood cancers, especially because they lack the long-term side effects or secondary cancer that can follow chemotherapy or radiation.

The foundation works to raise awareness and funds to continue research and treatment options for impacted families, as oftentimes the costs for CAR-T therapies is prohibitive.

The foundation's signature event, Believe Ball, is taking place this year on September 27 in Philadelphia, bringing together patients, or "warriors" as they are called, their families, and leaders in the field.

These events serve as critical reminders of the importance of drug developers and contract manufacturing and development organizations (CDMOs) like Charles River to improve pediatric patient access through the support of clinical trials and innovative CGT therapies.

Integrated Discovery and CDMO Capabilities Support New Partnerships

To help expedite the cure for childhood cancers in particular, Charles River is part of ITCCP4, a non-profit initiative that aids the fight by providing a database of patient-derived pediatric tumors and their corresponding characterization.

With that, Charles River provides over 200 annotated patient derived xenografts (PDX) models that are Research to Accelerate Cure and Equity (RACE) compliant to accelerate drug developers' time to clinic and FDA approval.

Helping to fight childhood cancer is just one side of the coin. Our commitment to find cures for rare disease in children continues to evolve. Most recently, it has included development and manufacturing support, including plasmid DNA critical starting materials and viral vector production for therapies to treat neurodegenerative diseases, for foundations including Genetic Cures for Kids, Inc. (GC4K) and FOXG1 Research Foundation.

This work highlights the importance that gene therapy plays in finding the treatment and cure to rare diseases for children.

For more information on our integrated plasmid DNA, viral vector, and cell therapy development and manufacturing platforms and services, please visit Cell and Gene Therapy CDMO Solutions.

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