Acquiring Targets in the Fight Against AML

Acquiring Targets in the Fight Against AML

A childhood cancer diagnosis can turn complete strangers into intimate friends very quickly. Because each type of cancer is unique, parents tend to fragment by diagnosis to share knowledge, side-effect remedies, and treatment options. Three Atlanta families met that way; while their children were all fighting Acute Myeloid Leukemia (AML). Those beautiful children – Lake, Mary Elizabeth, and Melissa – fought bravely but ultimately succumbed to the disease. The bonds built by their families during treatment didn’t break after their deaths. They strengthened.

Together they formed a Named Fund at CURE called United for a CURE. They decided to work together in honor of their children with a vow to never stop fighting until there is a cure for children battling AML. Since then, they’ve worked tirelessly, and thanks to the support of friends, family, and colleagues they have raised over $350,000 in just two years. With that sum, they chose to support a project called Target Pediatric AML.

“We chose Target Pediatric AML because leading researchers agree that this project has the highest potential for a cure in the next five years,” said Joe Depa, Melissa’s father. “All children fighting AML have their tumor samples banked, and when we spoke with the project’s lead researcher, Dr. Soheil Meshinchi, he become emotional as he connected the samples he was studying with our families and our children. We could feel his passion immediately.”

Target Pediatric AML aims to facilitate genomic sequencing for every child fighting AML, searching for unique identifiers and vulnerabilities of the disease in young patients. Once these factors are identified, the hope is that the information will allow existing agents to be delivered in the right combination to effectively treat children fighting the disease. This knowledge will enable expansion of the treatment toolkit and jumpstart more informed, individualized therapy.

The project has already had some key wins:

  • The project has created the world’s largest database of genetic data for pediatric cancer patients. The team has provided genetic sequencing (mRNA) for more than 1200 AML patients.
  • A new therapeutic target has been identified and funding has been secured for clinical trial. Mesothelin was identified as a therapeutic target in AML. Typically found in lung cancer patients, genetic profiling revealed this protein in AML patients, as well. Bayer Pharmaceuticals has an anti-mesothelin drug for lung cancer and has agreed to participate in a clinical trial in 2018 to test its effectiveness against pediatric AML.
  • Additional targets are under development. Based on the RNA sequencing, over 100 potential targets have been identified and are undergoing verification for therapeutic development.
  • The information and database is shared publicly. All health institutions, pharmaceutical companies, and researchers can access and leverage the genetic data acquired with the hopes that collaboration will lead to provide more targeted therapies.

The underlying fact is that all children are unique and their cancers are also very different. If we can find common targets in the genomic make-up of AML, we might be able to fight these targets with existing therapies or therapy combinations.

“There are many different subtypes of AML,” explained Joe. “In fact, each of our three children had a completely different type. And yet, today’s treatments are basically the same for every subtype. This project is searching for individual therapies for every child to improve their survival rate. It’s too late for Lake, Mary Elizabeth, and Melissa. But we hope someday their names are attached to a cure for AML so that other families win their fight. We owe them that much.”

To learn more about the United for a CURE Fund, please click here:

 

 

 

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