A Breakthrough for Down Syndrome and Leukemia

Kids with Down syndrome are 20 times more likely to get a type of blood cancer called acute lymphoblastic leukemia (ALL). Sadly, they also have a harder time with the side effects from treatment. But thanks to early funding from Rally Foundation for Childhood Cancer Research, one scientist decided to change that and he’s making real progress.

John D. Crispino, Ph.D., was a researcher at Northwestern University when he received his first Rally Foundation for Childhood Cancer Research grant in 2011. Rally Foundation believed in his bold idea, and it paid off in a big way. Today, Dr. Crispino leads research at St. Jude Children’s Research Hospital, where he serves as Director of the Division of Hematological Malignancies.

Finding a Cancer’s Weak Spot

Dr. Crispino and his team discovered that a gene called DYRK1A acts like a light switch stuck in the “on” position. When this gene is always turned on, it helps leukemia grow. But in the lab, they found a way to turn it off and when they did, the cancer cells slowed down.

That discovery opened the door to even more support. Dr. Crispino’s lab went on to earn major funding from the National Cancer Institute and the Leukemia & Lymphoma Society (LLS), a proud partner of Rally.

Creating New Hope

Because of the progress sparked by Rally’s early funding, LLS awarded a special research center focused on leukemia in children with Down syndrome. Now, researchers are developing new drugs called DYRK1A inhibitors that may one day help kids in clinical trials.

And there’s more good news: these new medicines might also work for kids with other types of high-risk leukemia, not just those with Down syndrome.

Training Future Scientists

Rally’s impact didn’t stop with the discovery. Dr. Crispino used that early support to help train the next generation of cancer researchers, including:
 – Rahul Bhansali, M.D., who worked in the lab as a medical student and now treats leukemia patients in both the clinic and the lab.
 – Austin Boucher, Ph.D., who continues to study leukemia in children with Down syndrome as part of Dr. Crispino’s research team.

Why This Work Matters

This research is about more than science, it’s about real kids, like Jacob Moore, a Rally Kid who survived leukemia and has Down syndrome. His mom, Heidi, now serves on Dr. Crispino’s advisory team, helping guide this important work.

The Bottom Line

What started as one idea funded by Rally has turned into a powerful discovery with the potential to help children everywhere.

This is why early-stage research funding matters.

This is the power of philanthropic seed investing.

This is Rally.