Childhood Cancer

Leukemia

Childhood leukemia is a cancer that starts in the blood-forming cells in the bone marrow. Leukemia is most often found in the white blood cells; but  it can develop in other blood cell types. It is the most common form of childhood cancer and there are several types and sub-types.

Latest Leukemia grants

Adolfo A. Ferrando, MD/PhD, Principal Investigator
Columbia University
Innovation Grants, Awarded 2017
Seth Corey, MD & Marek Kimmel, PhD, Principal Investigator
Virginia Commonwealth University
Innovation Grants, Awarded 2017
Steven Jonas, MD/PhD, Principal Investigator
University of California, Los Angeles
Young Investigator Grants, Awarded 2017

Latest Leukemia blog posts

December 1, 2017

by Adam Paris, ALSF

John Szigety was diagnosed at age 10 with Hodgkin lymphoma and underwent treatment at Memorial Sloan Kettering Hospital and Hackensack University Medical Center. After eight months of treatment, he suffered a relapse in early 2006, but completed treatment that June. Today, he is 11 years cancer-free. 

Amjad Shaikh was diagnosed just before his 9th birthday with leukemia and went through his entire treatment schedule at Children’s Hospital of Philadelphia (CHOP) for four years, before entering remission. Today, he is cancer-free. Amjad Shaikh

Both are childhood cancer survivors and medical school students who participated in Alex’s Lemonade Stand Foundation’s (ALSF) Pediatric Oncology Student Training (POST) grant program this past summer. They each felt the immense pride of being able to give back and help kids fight for their lives just like they did. They talked about the experience of beating cancer as kids and now, as researchers, searching for a cure.

Can you describe your work this summer as a POST student?
John Szigety (JS): I was looking at the effect of a drug on tumor progression and cell replication. The doctor I worked with at CHOP, Dr. Sarah Tasian, was investigating how to treat cancers with a specific mutation that makes the disease especially aggressive.
Amjad Shaikh (AS): I looked at CHOP and found an opportunity to study pediatric leukemia. I worked on studying mortalities associated with hospital characteristics and what a hospital can do to improve treatments or survival rates in kids fighting the same kind of cancer that I did.

 

Pictured: Amjad Shaikh

What was most meaningful about this experience?
JS: It was meaningful to see the different roles that a pediatric oncologist plays. As a patient, I only saw my oncologist, Dr. Steven Halpern, as a clinician. Dr. Tasian showed me how big of a role research plays in her life as a physician. That was new information to me.
AS: Part of it was that I got to see a lot of pediatric patients. The fact that I was on the other side and I understood from a clinical and medical level what was going on, I felt that maybe given a couple years I could be the person who was helping them out.

Was there a particular individual who inspired you to pursue pediatric oncology? 
JS: Dr. Halpern. When I was feeling miserable or having a bad day, he was receptive to me about how to change my regimen to make it more manageable. He would stop in my room during treatment to say hello or play board games. The cliché is that he treated me like a person and not an illness, but it's true. He's a big reason why I want to go into this field.
AS: My oncologist, Dr. Stephen Grupp. A few years after treatment, he told me he had been diagnosed with cancer and was undergoing treatment. It was a bit shocking because I had never seen an adult go through chemo. It left an impression that despite the fact he was going through chemo, and knowing what that was like, he still came in and treated kids. That meant a lot to me. It guided my philosophy in med school and framed my outlook for how a doctor should be. 

What did this POST opportunity and the chance to help other kids fight cancer mean to you?
JS: It was an honor. Since my diagnosis, I have wanted to help children affected by cancer. I've worked at Camp Kesem (summer camps hosted for kids whose parents have cancer), fundraised for various charities and pursued an education that brought me to medical school. The POST grant helped me contribute in another important way by investigating potential new treatments. While I didn't cure cancer or even come close, I am proud to say that I helped.
AS: It meant the world. Seeing Dr. Grupp and the team of physicians at CHOP, that’s my vision for the kind of doctor I’d like to be. 

As someone who survived childhood cancer, what does ALSF represent to you?
JS: In a word, ALSF represents hope. By supporting research across the country, ALSF represents the future of medical treatments and all the advances that are yet to come. 
AS: I think it’s a great organization for helping to develop a new generation of physicians and researchers who are going to continue these advancements. 

What are your goals and aspirations going forward?
JS: My goals are to become a pediatric oncologist and help treat children who have to fight for their lives. As a survivor, I have an understanding of how these pediatric cancer patients feel and I hope to help my future patients through the frightening and overwhelming experience of battling cancer.
AS: Especially after this summer I’m definitely set on a career in oncology. I’m not 100% sure how I want to go about it, since I'm still exploring all the specialties, but pediatric oncology is at the top of my list.

Each year, ALSF provides grants opportunities to medical students through the POST grant program. Read more about our POST grants here. 

June 30, 2017

by Trish Adkins

ALSF-funded researcher (Innovation Grant 2009 and Epidemiology Grant 2013) Dr. Richard Aplenc of Children’s Hospital of Philadelphia is working to understand the underlying causes of cardiac dysfunction in children battling acute myeloid leukemia (AML). Five ALSF –funded POST Grant students have also worked on this research in the Aplenc Lab: Yang Ding, Alexandra Blumer, Joe Horowitz, Julianna Mann and Elizabeth Goodman. 

In the treatment of acute myeloid leukemia (AML), most cardiac side effects come from a type of chemotherapy called anthracyclines. Anthracyclines are used pretty extensively in treating pediatric cancer. In general, the more doses of anthracyclines a child receives, the greater the risk of long-term side effects, such as cardiac dysfunction. These long-term side effects of anthracyclines have been researched extensively. However, the short-term cardiac side effects, which can affect some children during treatment, have been less researched. 

ALSF-funded researcher and Scientific Review Board member Dr. Richard Aplenc is working to understand the underlying causes of short-term cardiac dysfunction. These side effects could happen immediately or within the first year of treatment with anthracyclines in children battling AML, a form of leukemia that requires more intensive chemotherapy.

Dr. Aplenc’s lab is working on a genome-wide genotyping effort to discover the genetic variations that change the risks of relapse, life-threatening infections and heart complications in these children. He also leads several efforts to use administrative data and sets out to improve the care of children with AML, particularly focusing on antibiotic and intensive care use.

Long-term cardiac side effects of anthracycline treatment can include an increased risk of heart attack and cardiomyopathy. In the short term, heart attacks do not happen, but some children experience cardiomyopathy, which is a dysfunction of the heart muscle. The heart muscle does not squeeze effectively, which can lead to a myriad of problems including congestive heart failure. This short-term side effect can lead to treatment delays, jeopardizing its effectiveness.

Dr. Aplenc is also studying the link between blood infections and short-term side effects of anthracycline treatment in children battling AML. Blood infections can also trigger cardiomyopathy. However, this link is poorly understood. 

The first part of his research will:

  • Define the clinical experience of children with AML who experience a decrease in heart functions
  • Define the cardiac risk factors in kids who have a blood infection versus those who do not have a blood infection
  • Understand the pathophysiology that leads children with an infection to have a worse infection
  • Determine if decreased heart function affects the risk of AML relapse 

The second part of his research is using DNA sequencing to understand if changes in a patient’s DNA could be related to their risk of cardiac side effects. 

“I want to be able to provide patients and their families with accurate estimates of cardiac toxicity risks in AML, so when a physician sits down with a family, they can accurately predict treatment outcomes and manage risk,” said Dr. Aplenc. 

April 28, 2017

by Trish Adkins

When a child is first diagnosed with leukemia, the goal is to force the disease into remission. The treatment protocol is long and grueling—at least 2 1/2 years of chemotherapy, lumbar punctures and clinic visits. Today, children diagnosed with the most common form of pediatric leukemia—acute lymphoblastic leukemia (ALL)—have a high cure rate. The discovery of genetic differences that can increase a child’s risk of relapse has helped some of the highest risk patients reach remission. 

But, not all children with ALL reach remission. When they relapse, the second round of treatment is much more intense than the first says Dr. Todd Druley, ALSF Scientific Advisory Board Member and Assistant Professor of Pediatrics, Developmental Biology and Genetics at Washington University.  

Dr. Druley says the reason lies within the genetic drivers of the child’s specific type of leukemia. Better outcomes and cures will be found with continued genetic studies, innovation in technology and targeted treatments.

Understanding Leukemia
A diagnosis of leukemia is suspected after a blood test and confirmed by a bone marrow biopsy. The cells that make blood reside primarily in the bone marrow and when a child has leukemia, one of those cells becomes cancerous and overruns the other healthy cells.

While most childhood leukemia diagnoses are ALL, children are also diagnosed with acute myeloid leukemia (AML), chronic lymphoblastic leukemia (CLL), chronic myeloid leukemia (CML), and juvenile myelomonocytic leukemia (JMML). The acute form of the disease will grow suddenly—meaning the leukemia is spreading rapidly and outnumbering healthy cells and a child can become very sick quickly. 

Managing Risk Factors
One of the early breakthroughs in the treatment of childhood leukemia was to better understand the genetic differences between adult and childhood forms of the disease. Now, researchers have deconstructed the disease even further, giving them the ability to manage the risk factors for newly diagnosed patients. 

“Ten years ago, we did not appreciate the genetic differences inherent to leukemia and we thought most children had a standard risk. Now, we can see the subtypes of leukemia more precisely and provide the correct intensity of treatment,” said Dr. Druley

This helps children with both low and high-risk leukemia. Children with lower risk factors can receive a less intense treatment and therefore minimize long term side effects (which can include cardiac damage, developmental delays and fertility issues). 

If a relapse occurs, doctors now have more tools to battle the disease including CAR T cell immunotherapy, which works to harness the body’s immune system to attack the cancer cells. This therapy uses a patient’s own genetically engineered T cells (an immune cell that attacks things that are foreign to the body) to attack cancer cells that have been hiding from the immune system. 

Hope in the DNA
Genetic studies have also helped doctors understand infant ALL and AML (leukemia less than 12 months of age), which has significantly lower cure rates compared to leukemia in older children. Dr. Druley’s research suggests that babies who have leukemia appear to have inherited a genetic predisposition that makes them highly susceptible to developing the disease. 

Dr. Druley’s research continues to focus on determining the genetic drivers that predispose children to cancer and how to mitigate the effects of these genetic mutations and stop cancer formation. Understanding these genetic markers can also open the door to understanding how to treat other types of childhood cancer.

Scientists have discovered the same mutations in blood cells, also exist in solid tissue tumors, which helps provide critical clues into what makes a variety of childhood cancer types tick. 

“Science is telling us that treating cancer by its tissue or origin ( blood, lung, brain, etc) is often less effective than treating the genetic type of the tumor, guiding us to tailor therapy in whole new and exciting way,” said Dr. Druley.

Read more about Dr. Druley’s research here.