Cancer Research and Clinical Trials

At the Hyundai Cancer Institute at CHOC, we are dedicated to providing patients the very latest break-through treatments available to children, adolescents and young adults with pediatric cancers. As part of our mission, we are continuously actively engaging in robust research into tomorrow’s treatments and the very latest clinical trials. Our efforts to provide the very latest treatments can bring hope to patients with rare cancers or cancers not responding to traditional treatments.

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Clinical Trials: The very latest treatments for pediatric cancer patients.

The Hyundai Cancer Institute at CHOC is a member of the Children’s Oncology Group (COG) and has received a prestigious Phase I clinical trial designation, making us one of only 21 facilities in North America offering COG’s groundbreaking alternative treatments. The Children’s Oncology Group (COG) is the most experienced organization in the world when it comes to the research and development of new therapeutics for children and adolescents with cancer.

Access to these trials means our patients are able to receive the very latest research-based treatments before they are available in most other hospitals in the country.

For more information on participation in clinical trials, contact the CHOC Research Institute at (714) 509-4341.

What is a Phase 1 Clinical Trial?

A clinical trial is a research study that aims to determine the most effective treatment for a particular disease. Each trial is a result of a very detailed study of factors associated with the most current standard treatment and variables that may lead to an improvement in survival rates or a reduction in side effects or late effects of treatment. (Late effects are side effects of treatment a patient experiences later in life.) Clinical trials are conducted in “phases,” with the earliest research of the use of a new treatment being “Phase I.”

Genomic Research: Unlocking the building blocks to tomorrow’s cures today

As part of a generous gift of $10 million from Hyundai Motor America, the Hyundai Cancer Institute at CHOC is conducting incredible genomic research using the very latest technology to better understand the cancers that occur in children and teenagers. Whole genome (DNA) sequencing of both tumor and non-tumor tissue and transcriptome (RNA) sequencing of tumor tissue is being conducted to identify the molecular profile of cancers occurring in pediatric and adolescent patients.

This sequencing is helping researchers determine the best drugs currently available to treat those cancers. These treatments can be used to extend life, increase survival, and improve the patient’s quality of life. Even if the tumor board is unable to identify a treatment that is available now, the information learned may be used to help researchers better understand what causes cancer and how it may be treated or prevented in the future. The research also provides scientists information on why some cancers become refractory (do not respond to therapy) or recurrent (come back after treatment).

Genomic Research and Cancer Treatment

The goal in the Cancer Institute is to collect tumor and non-tumor specimens from each and every child that is diagnosed with cancer at CHOC. At this time, specimens are not sequenced unless the child ‘s cancer is refractory or recurrent. The DNA and RNA is extracted from the tissues in the Molecular Laboratory at CHOC and sent to an off-site facility where the whole genome and transcriptome sequencing procedures are performed.

Once the sequencing is complete, the information is returned and analyzed by a multidisciplinary team of scientists including oncologists, cancer epidemiologists, cancer biologists and bioinformaticists. The goal of this specialized tumor board is to identify treatments that may be beneficial for the child or teenager with cancer based on the molecular profile of the cancer.

The Science of Genomic Research

Deoxyribonucleic acid (DNA) is a large molecule present in each cell of most living organisms that contains the instructions needed by every cell to function. In humans, the DNA molecule contains roughly three billion base pairs that are divided into 20,000 to 25,000 functional units called genes. By determining the order of the base pairs, researchers can identify genetic variations (mutations) that may be responsible for a child’s cancer. Usually, more than one mutation must be present before a cell becomes malignant (cancerous) and the presence of a mutation does not automatically mean that it will cause cancer.

In this very specialized form of cancer research, the DNA from the tumor is compared with the DNA from the non-tumor to identify the differences and help researchers identify what make the cancer cells special. The DNA is also compared with the reference human genome to determine if the differences seen are expected (normal variation) or unexpected (potentially disease-causing). Mutations that are known to cause cancer are also evaluated.

There are many different types of ribonucleic acid (RNA), which is different than DNA. RNA are much smaller molecules that carry out the instructions present in the DNA. By studying each patient’s RNA, researchers can tell which genes are working, which genes are not working and which genes are not working as expected (such as working too much or not enough).

Precision Medicine and Pediatric Cancer Treatment

Smiling cancer patient

Until recently, precision medicine – personalized medicine using genetics and a patient’s health information to design a targeted treatment for a person’s specific disease – seemed like science fiction. Today, new technologies, more affordable human genome sequencing and advanced computer capabilities have made this a reality.