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Precision Medicine

It’s in the Genes

Ally has been through a lot in her eleven years. When she was two years old, she fell off a stool while playing with her brother. A large bruise appeared on her stomach almost immediately, and her parents took her to their local hospital. After blood work, they learned that Ally had B-cell acute lymphoblastic leukemia. “We were told that within days, she would have had uncontrollable bleeding without treatment,” said Ally’s mother, Amber. “Her body was working so hard to fight the cancer that her heart would have given out. It is hard to be glad for your child to fall, but that fall saved her life.”

Ally received her first dose of chemotherapy within 16 hours of diagnosis. Over the two years of treatment that followed, Ally struggled with almost every possible side effect. She suffered three life-threatening infections, temporarily lost the ability to walk, and struggled to rebuild her immune system after every round of chemo.

“We were given a bleak prospect of survival during her second fight with an infection,” shared Amber. “We prayed for a miracle and finally got one when her body started to respond to antibiotics.”

Her treatment finally ended on August 24, 2018, but her battle was far from over because her immune system did not recover as expected. Even years later, her doctors were concerned because she was still living with depleted immune function. In 2022, they decided to do genetic sequencing on Ally and her family to see if a disorder was causing her immune system deficiency. What the sequencing uncovered has lifelong implications for Ally and her family.

“My nephew was diagnosed with leukemia after Ally,” said Amber. “So I expected that if there was an inherited genetic trait linked to her cancer or her body’s response to treatment, it would be on my side. We found out that the opposite was true.”

Ally shares a genetic condition called Lynch syndrome with her father, Justin. This condition increases the risk of many kinds of cancer, including colon cancer, endometrial cancer, and other types. Knowing this will allow Justin and Ally to be monitored for early signs of cancer, giving treatment a greater chance of success. Ally sees a doctor with expertise in genetic conditions every year, and her parents are updated on new testing and protocols. Ally is doing great today. She is in her first year of middle school and enjoys competing in pageants. Her parents are grateful for her health and credit CURE’s investment in precision medicine as a big part of it.

“I have a friend who just went through breast cancer treatment, and her genetic testing cost $4,000 out of pocket,” Amber said. “The cost of repeated gene sequencing for us would have been staggering. But it didn’t cost us a dime because of CURE’s funding. We wouldn’t have known about the syndrome without it. With this knowledge, we can be prepared and should be able to detect any cancer in Ally or Justin early.”

2024 Research Awards

We are excited to share a significant step forward in CURE’s 49-year effort to find cures for childhood cancers as we proudly announce a $5.6 million investment in innovative research studies – the largest amount we’ve ever committed in a single year.

This record-breaking investment represents an important advancement in our ongoing work. It reflects the growing support of our community, the persistent dedication of researchers, and our unwavering commitment to finding better treatments for children with cancer.

These studies are led by top scientists at leading pediatric cancer research institutions nationwide and focus on the most critical needs. Here’s what makes this investment so impactful:

  • Advancing Precision Medicine: We’re awarding $2 million to the Aflac Precision Medicine Program, reinforcing our leadership in this crucial field. This funding expands access to genetic sequencing for young patients. It accelerates research into targeted therapies, bringing us closer to truly personalized treatments and giving hope to many children with aggressive or recurring cancers.
  • Funding Diverse, Innovative Research: The remaining $3.6 million funds 11 promising projects carefully selected from over 90 applications. These studies address some of the most challenging childhood cancers with limited or outdated treatments, employing innovative approaches that could significantly improve treatment outcomes.
  • Bridging Research and Treatment: Time is precious in the fight against childhood cancer. That’s why we’ve prioritized studies with strong potential for rapid clinical translation, potentially bringing new treatments to children faster than ever before.
  • Harnessing Immune Power: Within every child’s body lies an army of potential cancer-fighting cells. Several projects focus on enhancing cutting-edge immunotherapy approaches, unlocking the full potential of the body’s own defense mechanisms, and offering new possibilities for children battling resistant forms of cancer.
  • Looking Beyond Remission: The fear of relapse looms heavy for patients and families. By investigating mechanisms of recurrence, these studies aim to improve long-term outcomes and quality of life for survivors – and offer children a chance at a cancer-free future.

As we make these awards, we’re filled with a sense of purpose and hope. While the road ahead may be challenging, each step forward brings us closer to our goal of conquering childhood cancer.

Thank you for being an essential part of this journey.

CURE’s 2024 RESEARCH AWARDS

Early Investigator Awards

Rula Green Gladden, MD, Fred Hutchinson Cancer Center
Redefining residual disease detection in pediatric AML

Elizabeth Young, MD, University of California, San Francisco
Defining determinants of a cGAS-STIGN-mediated anti-tumor inflammatory response in osteosarcoma

Translation to CURE Awards

Eric Sweet-Cordero, MD, University of California, San Francisco
Defining replication stress and DNA damage as a therapeutic vulnerability in osteosarcoma

Pavithra Viswanath, PhD, University of California, San Francisco
Targeting and imaging serine metabolism in the tumor microenvironment in pediatric brain tumors

Michael Andreeff, MD, PhD,  University of Texas, M.D. Anderson Cancer Center,
c-MYC protein degradation in therapy-resistant pediatric leukemias

Eugenie Kleinerman, MD, University of Texas, M.D, Anderson Cancer Center
Metabolic reprogramming of the Ewing Sarcoma tumor microenvironment using pramlintide to augment NK cell immunotherapy

Kristopher Bosse, MD, Children’s Hospital of Philadelphia
Development of a GPC2 CAR T cell amplifying RNA vaccine

Alex Huang, MD, PhD, Case Western Reserve University
Effective TGF-beta signaling blockade synergizes cryoablation-induced STING activation in treating refractory and metastatic sarcoma

Jason Yustein, MD, Emory University
Dissecting and targeting PAK4-mediated signaling in Ewing Sarcoma development and metastasis

David Robbins, PhD, Georgetown University
Defining the druggable GLI Interactome in medulloblastoma

Soheil Meshinchi, MD, PhD, Fred Hutchinson Cancer Center
Rapid Transition of B7-H3 Targeted Therapies to High-Risk Childhood AML

Precision Medicine Program, Children’s Healthcare of Atlanta
A program leveraging genomic sequencing for pediatric patients with high-risk tumors, with the goal of identifying alterations that can impact therapies and improve outcomes.

We Finally Have Hope

young boy named Easton, a recipient of personalized cancer medicine

Easton has been fighting cancer for most of his twelve years. When he was 21 months old, he was constantly sick. His pediatrician noticed that his sickness was abnormal – he vomited more frequently in the mornings and while sitting in his car seat. A scan revealed a large tumor on Easton’s brain stem near the area that controls swallowing and nausea.

“The fact that Easton would get sick after being in his car seat made our pediatrician suspicious,” recalled his mother, Jill. “If the car seat hadn’t put pressure on the tumor, it might have taken longer to find.”

Easton had surgery to remove as much of the tumor as possible, followed by eight weeks of proton radiation in Jacksonville, Florida. His family was thrilled when a follow-up scan showed no presence of the tumor.

“We thought it was over,” said Jill. “We were told that if we had five years of clear scans, we wouldn’t have to worry about it again. Easton had four years of clear scans. But the cancer came back during the fifth year.”

Easton started treatment all over again in 2017 and experienced horrible side effects from the chemotherapy and radiation. Because the tumor grows around the area of the brain that controls swallowing, he has always had challenges eating. The awful mouth sores from his chemo caused him to be on a mostly liquid diet, and he had trouble maintaining weight.

In May 2022, Easton underwent a very risky surgery to try and remove the last part of the tumor. The surgery was unsuccessful. As he started recovering, Easton fell out of bed in the middle of the night. His parents rushed him to the emergency room, where doctors found that Easton’s brain was having a reaction to the glue that was used on his skull after surgery. He would need another risky brain surgery.

“This time, I felt like it was getting away from us and it was the beginning of the end,” said Jill. “He had been on some form of treatment for five years, and the tumor always found its way around it.”

But thanks to funding from CURE Childhood Cancer, Easton’s doctors have a new tool in their toolbox. CURE’s funds would pay for Easton’s tumor to be genetically mapped to see if his cancer involved any genetic mutations that could be targeted.

The genetic mapping revealed that a protein was feeding Easton’s tumor, causing it to grow. Doctors found an open clinical trial using a chemotherapy to inhibit this specific protein to prevent further tumor growth. Easton was immediately enrolled in the trial, and the results have been astounding. After four months, a scan showed that the inside of Easton’s tumor appeared to be dying. Three months later, the tumor is much smaller and is collapsing in on itself.

“The best news is that the tumor is dying. But also, he has no side effects. He can do the things a twelve-year-old should do while on this treatment,” shared Jill. “We’re still early in the process, but it has saved his life – at least at this point. I finally have hope for the first time in years. We are so encouraged and thankful to his doctors and to CURE for investing in precision medicine.”

Take the next step to support research that will help save kids with cancer… kids like Easton.

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