In her lab at Children’s Hospital of Philadelphia, Dr. Yael Mossé leads an international team of researchers studying MYCN, a treatment-elusive mutated gene that drives cancer. At City of Hope in Los Angeles, Dr. Linda Malkas is working on a liquid formulation of promising cancer drug she developed, so that drug can be trialed in children as well as adults. In Memphis, Dr. Rebecca Gardner is working to make CAR T immunotherapy more effective, more accessible, and more equitable for kids with cancer. In her Vienna, Austria lab, Dr. Anna Obeneuf combines biology with cutting-edge technology to identify what makes childhood cancer spread. From her Dana-Farber Cancer Institute lab in Boston, Dr. Rani George is leading a multi-disciplinary team that is working to understand gene behavior in osteosarcoma with the hope of finding this bone cancer’s weakness, curing it once and for all. 

These women are bringing their own expertise and passion to the fight for cures for childhood cancer, using grant funding from Alex’s Lemonade Stand Foundation (ALSF) to power their research. They also have one other secret ingredient in their labs: hope. 

“Hope is what gets us up in the morning. It’s what gives us the passion, the motivation, the resilience to keep going, even when we fall or when there are hard moments. That hope is what really gives us the strength and the courage to believe that we are going to make a big difference,” said Dr. Mossé. 

Meet five women who are leading the search for childhood cancer cures:

1.    Dr. Yael Mossé

Dr. Mossé has dedicated her clinical and research work to treating kids with neuroblastoma, the third most common type of childhood cancer. Neuroblastoma is easy to treat, but tricky to cure. Her research has led to breakthroughs in treatment for kids with inherited neuroblastoma. Now, Dr. Mossé has turned her attention to study MYCN, a well-known mutated gene that been long-considered undruggable. Funded by an ALSF Crazy 8 Initiative grant, she is leading an international team working together to understand this gene and find a targeted drug. For Dr. Mossé, enormous discoveries in science, new technologies, and now, the collaboration made possible by the Crazy 8 has given her hope that she will find a drug to treat children with this type of neuroblastoma. As a result of her work studying neuroblastoma, Dr. Mosse was recently awarded a Cancer Grand Challenges grant by Cancer Research UK. 

2.    Dr. Linda Malkas 

Dr. Malkas began her research career studying breast cancer, but then a chance meeting with a photographer changed the entire trajectory of her career. Inspired by the photographer’s story of his daughter, who was battling cancer, Dr. Malkas pivoted her research from breast cancer to pediatric cancer, by studying a mutated protein that drives solid tumor development in both children and adults. Malkas received an ALSF Reach Grant that supported the gathering of preclinical data about this protein and a drug that was effective at treating it. That grant led to the opening of an early phase clinical trial at City of Hope for a pill called AOH1996. Named for that photographer’s daughter, Anna Olivia Healey, the pill is given twice a day, and in lab studies it decimated cancer cells and left healthy cells alone. It’s being called “the holy grail” of cancer treatments. It is also Malkas’s very first clinical trial. The next stop: a formulation and trial for children. 
 

3.    Dr. Rebecca Gardener

ALSF-funded researcher Dr. Gardner was a young scientist and clinician leading a then-cutting edge CAR T immunotherapy trial at Seattle Children’s Hospital. 

“I still remember the first patient we infused, and in my heart I wanted it to work so badly and in my head I was like, ‘There’s no way this is going to work,’” recalls Dr. Gardner, who is now at St. Jude Children’s Hospital in Memphis. CAR T immunotherapy, which has since been approved by the FDA, makes use of a child’s own immune system cells. The cells are harvested, engineered to recognize cancer cells and then returned to the child. All of this requires children to travel to CAR T treatment centers, making the treatment hard for some families to access. Dr. Gardner is now studying the immunotherapy can be more accessible to more kids and also following its long-term effectiveness in treating leukemia. 

4.    Dr. Anna Obenauf

In her Vienna lab, Dr. Obenauf developed CaTCH, a molecular time machine that allows researchers to trace cells as they undergo change and mutation. This technique has helped isolate and identify how cancer cells become resistant to treatment. Dr. Obenauf brings this technology to the Crazy 8 Grant Initiative team that is working to understand, and then inhibit, the mechanisms of metastatic spread of osteosarcoma. She works with a multi-disciplinary team in Europe and the United States to study this type of bone cancer, which has an extremely low cure rate after spread. Dr. Obenauf draws inspiration from families and her own daughter. “We have a very big potential to make a difference for these kids and their families,” she said. 
 

5.    Dr. Rani George

Dr. Rani George equates the work of a scientist to that of a detective in a mystery novel. In her lab, she works with a multi-disciplinary team to solve the mysteries of solid tumors. She is also leading an international team, funded by the Crazy 8 Grant initiative that is specifically working to understand the gene behavior in osteosarcoma, a type of bone cancer that most often spreads to the lungs. The challenge, according to Dr. George, is three-fold: the common genetic changes in osteosarcoma are known but not understood; the steps that drive the development of metastatic lesions are not known; and lastly, the few identified drivers of metastatic osteosarcoma are not druggable with available therapies.

Her team, hailing from five different institutions, will use innovative technology to rewire specific cancer gene connections making them more susceptible to targeted treatments, with the hope of ultimately leading to cancer cell death and a cure.

Fund Research. Find Cures. Help Kids. 
All of this, from research breakthroughs to cures, is made possible by generous supporters, who were inspired by ALSF founder Alex Scott’s dream of finding cures for all children. 

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