St. Baldrick’s Fellow Dr. Elliot Stieglitz is a big reader, but not in the way that you might think. Over three years, he read the DNA of one hundred children with JMML, a rare leukemia, and he discovered something major. Read on to learn how his discovery could lead to better treatments for kids with this rare disease.
RESEARCH DEVELOPMENT: Dr. Elliot Stieglitz has brought his St. Baldrick’s-funded research to a Phase 2 clinical trial for kids with relapsed JMML. In this trial, which is first of its kind in the United States, researchers will be testing whether an oral targeted medication used in the treatment of melanoma in adults slows or even kills leukemia cells in kids with persistent JMML. In addition, Dr. Stieglitz developed a test that predicts which JMML patients have the best prognosis and therefore need less intense therapy. He’s now in the process of establishing a clinical test that will eventually be available to patients. This test will help kids get just the treatment they need and avoid damaging long-term effects from harsh therapies. Keep up the great work, Dr. Stieglitz!
For St. Baldrick’s Fellow Dr. Elliot Stieglitz, being a pediatric oncologist is the perfect blend of emotional satisfaction and intellectual stimulation.
His heart is with the kids and their families, guiding them through the toughest time in their lives. His head is in the lab, trying to find better treatments for childhood cancer.
Dr. Elliot Stieglitz is a St. Baldrick’s Fellow at the University of California, San Francisco. He’s researching ways to help kids with JMML who don’t respond to standard treatment. He explains JMML symptoms, treatment options, and how your support is moving research forward.
What is JMML?
Juvenile myelomonocytic leukemia (JMML) is a type of blood cancer that affects young children.
Why do kids get cancer? That’s the question we asked Dr. John Maris, who co-leads the St. Baldrick’s Foundation – Stand Up to Cancer Pediatric Cancer Dream Team. Researchers like Dr. Maris are working hard to find the answer to this question because it could hold the key to cures for kids’ cancer.
Why do kids get cancer? In short, there’s no single, easy answer.
The answer is complicated, said Dr. Maris.
Honored Kid Micah is ready for his close-up during a hospital stay in 2015, when he was taking part in a St. Baldrick’s-funded clinical trial at Children’s Hospital of Los Angeles (CHLA) for neuroblastoma, a type of childhood cancer.
For kids with cancer and their families, clinical trials mean hope. Clinical trials offer a chance for survival when all other options are exhausted. They offer kids in treatment and survivors the possibility of a better future with fewer side effects. Most of all, for all kids with cancer today and in the future, clinical trials help scientists get closer to cures.
With your help, St. Baldrick’s has been an essential part of this lifesaving research phase since 2005 — every St. Baldrick’s donor has helped make these trials possible for more than 100,000 children with cancer.
Honored Kid Madelyn was diagnosed with DIPG in 2010. A fashionista with a big spirit and an equally big heart, Maddy was an inspiration to those around her. She passed away in November 2011 after an 18-month battle with DIPG.
No child has ever survived a diffuse intrinsic pontine glioma brain tumor, commonly called DIPG.
This aggressive pediatric brain cancer multiplies in the brainstem, which controls some of our most basic functions for living – our breathing, heart rate, blood pressure and ability to swallow and speak. And because the cancer is so intertwined with its delicate surroundings, DIPG is often inoperable.
A DIPG diagnosis and a zero percent survival rate has remained the reality for kids and families for decades.
But what if there might be hope? What if that hope came from within the child’s own body?
Dr. Joseph Lubega has big news — he’s bringing specialized pediatric cancer training to his home country of Uganda, thanks to his St. Baldrick’s International Scholar Grant and a partnership between Baylor College of Medicine and Texas Children’s Hospital. Read on for more about the pioneering program and why it will be a lifesaver for kids with cancer in the region.
EXCITING UPDATE: The first class of the East African Pediatric Hematology and Oncology Fellowship Program has officially graduated from the unique two-year program, which is the first of its kind in the region. “This is the most exciting point in my entire medical career,” said Dr. Joseph Lubega, a St. Baldrick’s International Scholar and leader of the fellowship program. “The realization that we have a critical mass of specialists to take care of children with cancer in this region of the world, and that they will train others going forward to infinitely multiply the specialist workforce — it is truly a momentous day.” The graduates are Drs. Barnabas Atwiine, Fadhil Geriga, Philip Kasirye, and Ruth Namazzi. Congratulations, grads!
Dr. Lubega speaks at the launch of the fellowship program in Uganda earlier this month.
Lack of diagnosis, poor care, staggering drug costs, a deficit in specialized medical training for doctors — all of these factors make survival rare for a kid with cancer in Africa.
But St. Baldrick’s researcher Dr. Joseph Lubega hopes to change that with a pioneering program that will train a new wave of East African pediatricians in children’s oncology and hematology.
New St. Baldrick’s researcher Dr. Rintaro Hashizume in the lab at Northwestern University.
For a child diagnosed with an atypical teratoid rhabdoid tumor or AT/RT, the options for treatment can be sparse and survival uncertain. This rare, aggressive tumor generally strikes very young kids and though research has progressed, many of these kids live less than a year after diagnosis.
As the father of a kindergartener, this breaks Dr. Rintaro Hashizume’s heart.
Recently awarded a St. Baldrick’s Research grant, Dr. Hashizume wants to change that reality for kids with AT/RT and their families.
Remember that check you wrote months ago to that foundation with the funny name? The donation bucket you hauled around the neighborhood? That epic shave? Now, all that hard work has paid off! Buckle your seat belts, ladies and gentlemen, because you funded childhood cancer research! Say hello to the newest grants of 2018 …
In our biggest grants cycle of 2018, St. Baldrick’s is awarding $19.1 million in funds to researchers and institutions dedicated to helping kids with cancer live long, healthy lives – and it’s thanks to your hard work!
Just mentioning herpes might make some people a little nervous, but in this story, herpes is the good guy. Read on for more about St. Baldrick’s Scholar Dr. Gregory Friedman’s work on a genetically altered version of the virus that could be the next targeted therapy for kids with brain tumors.
BREAKING NEWS: An innovative therapeutic tool developed by Dr. Friedman has proven to be safe in kids with high-grade gliomas, according to recently released clinical trial findings. Crafted from the herpes virus that causes cold sores, the genetically modified virus has already shown promise in killing cancer cells and stimulating the immune system to attack the brain tumor – with one patient still showing progress more than a year after treatment! In the next phase of research, Dr. Friedman will study how safe the viral tool is when combined with one low dose of radiation, which is expected to boost the immune system and help the virus replicate.
Dr. Friedman smiles with a young patient in an exam room at Children’s of Alabama.
St. Baldrick’s Scholar Dr. Gregory Friedman discovered that the herpes simplex virus, with a few modifications, will kill pediatric brain cancer cells — without causing cold sores.
For childhood cancer survivors, treatment helps them to survive, but often that survival comes at a cost. But what are these costs? And how big is the problem? That’s what St. Baldrick’s Fellow Dr. Nickhill Bhakta wanted to figure out. And as it turns out, that data could be a lifesaver.
St. Baldrick’s Fellow Dr. Nickhill Bhakta works at his desk in St. Jude Children’s Research Hospital. With a portion of the grant supported by the St. Baldrick’s Morgan and Friends Fund, he developed a special statistical tool to help capture the true volume and complexity of chronic health conditions faced by childhood cancer survivors because of the long-term consequences of their treatment — something that hadn’t been done before. Photos courtesy of St. Jude Children’s Research Hospital
Over the years, researchers have discovered that because of their treatment, childhood cancer survivors can be at risk of everything from heart attacks to secondary cancers to stroke. That’s helpful to know, but Dr. Bhakta recognized that something was missing from the data that was available on survivorship. It wasn’t painting the complete picture. It was just capturing the first big health scare, instead of following the survivor through the multitude of chronic, often recurring conditions.
The scope simply wasn’t big enough.
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