There is nothing more heartbreaking than learning your child is diagnosed with a serious disease like cancer. When such a crisis happens, parents want the best care and treatment to help their child get back to being a kid as quickly and safely as possible.
As a pediatric oncologist, I have seen many forms of cancer in children from newborns to young adults. I have seen common conditions like leukemia and very rare childhood cancers like adrenocortical tumors.
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In fact, the Centers for Disease Control (CDC) estimates about 12,500 children and adolescents under the age of 20 years are diagnosed annually with cancer nationwide.
To commemorate Childhood Cancer Awareness Month, we have an opportunity to focus on the latest breakthroughs in eradicating this dreadful disease as new advancements in research and treatments bring hope. We have come a long way in the battle against cancer and improving outcomes for our kids.
Fifty years ago, many childhood cancers were simply incurable. Now, average cure rates for certain pediatric cancer conditions have increased to 75-80 percent, with cure rates for some conditions at 98 percent. We see more children surviving cancer now than any other time in history.
Boosting survival rates
At Valley Children’s, we actively care for about 120-150 children newly diagnosed with cancer every year along with several hundred previously diagnosed, ranging from common to extremely rare conditions.
We are actively involved with an organization that has made the biggest impact in boosting survival rates, the Children’s Oncology Group (COG), the world’s largest organization devoted exclusively to childhood cancer research and treatment.
As a member of COG, we provide patients access to 90 open protocols for treating childhood cancer.
The organization has made tremendous strides in developing advanced treatments to improve quality of care and patient outcomes. The organization continually develops clinical trials, including front-line therapies, studies for understanding the hidden biology of pediatric cancers, evaluation of new emerging treatments, supportive care, stem cell transplantation and survivorship.
Existing cancer treatments such as chemotherapy and radiation therapy carry risks of adverse reactions. Treatments designed to destroy fast-growing cancer cells can potentially also kill normal body tissues and cells – blood cells, cells in the mouth, hair cells and many more.
More recently, innovative treatments called “targeted therapies” launched a new era in medicine and in management of childhood cancers. These therapies are designed to precisely identify and attack cancer cells and minimize effects to healthy tissues.
The newest “precision medicine” developments, such as DNA sequencing and gene therapy are revolutionary. We are now able to evaluate detailed sequencing of a patient’s genes, identify where a gene is mutated and provide a solution to specifically treat that disease safely and effectively.
This means that a subspecialist knowing your child’s genetic information can prescribe a drug smart enough to home in on a specific gene mutation. This particular medication hinders cancer cell growth by cutting off the supply that feeds the cancerous cell and/or kills it entirely without affecting normal cells around it.
The federal Food and Drug Administration recently approved the first gene therapy treatment for acute lymphoblastic leukemia (ALL), available in the United States. Called Chimeric Antigen Receptor (CAR) T-cell therapy, this transformational treatment uses the power of a patient’s immune system to fight off cancer cells – making it “a living drug.”
The therapy involves collecting a type of white blood cell called T-cells from a patient, and genetically modifying them to recognize a specific protein on the cancer cell. They are then infused back into the patient to destroy cancer cells.
For now, it has been approved primarily for children, adolescents and young adults with ALL who have not responded to conventional cancer treatments, including chemotherapy, radiation or stem cell transplant, and relapsed multiple times.
Madera patient treated in Philadelphia
Valley Children’s has a patient from Madera who underwent the CAR T-cell therapy at Children’s Hospital of Philadelphia earlier this year. He is a long-term survivor, first detected with cancer in 2011 when he was 2 years- old. He has had two relapses after being treated with chemotherapy and getting a stem-cell transplant.
He had the CAR T-cell therapy in May. So far, our 8-year- old patient is doing well.
A second patient, a 6-year-old from Winton, was diagnosed in 2016 and underwent a bone marrow transplant. He relapsed in August. He will get genetically modified T-cells in October at the University of California, San Francisco.
In early-stage clinical trials, more than 90 percent of patients achieved complete remission one month after receiving the therapy. Research efforts are now underway to use the same type of technology to attack other childhood cancers – solid tumors and lymphoma.
CAR T-cell therapy has the potential to completely change cancer treatment and possibly become the new standard of care. No doubt we will see childhood cancer cure rates improve as medicine becomes more precise and more personalized. Valley Children’s looks forward to offering this treatment within a few years.
Ultimately, this means providing tremendous hope for our children’s health and future.
Vinod Balasa, M.D, is the medical director and division chief of hematology and oncology at Valley Children’s. He also is director of the Hemophilia Treatment Center at Valley Children’s and a clinical professor of pediatrics at UC San Francisco. Connect with him at: 559-353-5480 or firstname.lastname@example.org.