It is said that to defeat an enemy, you must know them — and that’s exactly what St. Baldrick’s Scholar Dr. Grzegorz Nalepa is doing. The enemy? Childhood cancer. His weapon? Genomics — the study of all the genes within an organism, like a human child with cancer for instance. Who is winning? Read on to find out. (Hint, hint…it’s the good guy.)
St. Baldrick’s Scholar Dr. Grzegorz Nalepa amuses a young patient by making her the doctor, instead of him.
For St. Baldrick’s Scholar Dr. Grzegorz Nalepa, childhood cancer treatment can’t be one-size-fits-all. To be successful, it needs to be personal.
That’s why this physician-scientist studies what makes everyone unique, including kids with cancer – genes.
With support from St. Baldrick’s and a deep passion for the field of genomics, Dr. Nalepa is studying how the genetic changes, or mutations, present in kids with leukemia can be exploited to create targeted therapies against that cancer, with few side effects.
Last week, in the pages of the medical journal Cancer Cell, St. Baldrick’s researchers announced a discovery that could radically transform treatment for kids with neuroblastoma – a new immunotherapy drug candidate that harnesses the immune system to fight cancer.
Neuroblastoma is a cancer that begins in the nerve tissue outside the brain, usually in a child’s abdomen. It strikes very young children, up to about age 7, and is the most common cancer diagnosed in infants. Only about 50% of patients survive the high-risk form of neuroblastoma.
All of this makes this new targeted immunotherapy for neuroblastoma especially good news, but it gets even better.
Investing in the next generation of childhood cancer researchers — like researcher and St. Baldrick’s International Scholar Dr. Joseph Lubega – can change the lives of kids with cancer all over the world. Read on (and watch a video shot at the Uganda Cancer Institute) to learn more about the huge impact Dr. Lubega is making on kids with cancer in East Africa.
When Belinda heard, ‘leukemia,’ she was terrified.
She feared cancer. Belinda saw the impact it had on her family. In fact, she’d attended her cousin’s funeral in the spring. But cancer striking her 5-year-old daughter, Christa? How could that happen?
What is DIPG?
DIPG stands for diffuse intrinsic pontine glioma. It is a type of high-grade glioma, a brain tumor that comes from cells called glia that surround, protect, and otherwise support the nerve cells in the brain.
DIPG is always found in the brainstem. This part of the brain controls many basic functions like breathing and swallowing, as well as muscles that help with speech and eye movements.
It is most common in elementary school-aged children, but it can affect children of any age.
Learn more about childhood cancer >
About 250 kids in the U.S. are diagnosed with DIPG each year.
In the world of childhood cancer research, the St. Baldrick’s Foundation and the Children’s Oncology Group (COG) are two superheroes, teaming up to use their complementary strengths to save lives.
Dr. Mary Lou Schmidt decorates pumpkins with her patient, Isaac, and his mom. Isaac is on a Children’s Oncology Group clinical trial that’s part of a unique tri-institutional clinical trial program in Chicago that’s supported by St. Baldrick’s.
We sat down virtually with St. Baldrick’s researcher, Stephan A. Grupp, MD, PhD, for a Q&A on immunotherapy. Dr. Grupp, located at Children’s Hospital of Philadelphia, is a key member of the Stand Up to Cancer – St. Baldrick’s Pediatric Dream Team. He treated the first child with CAR T cell therapy, which was recently approved by the FDA for acute lymphoblastic leukemia (ALL). Check out our Twitter chat with Dr. Grupp and see what he thinks the future is for immunotherapy and childhood cancers.
St. Baldrick’s Scholar Dr. David Barrett gives us an inside look at immunotherapy, and we meet the first pediatric patient to ever receive this new cancer treatment.
Emily Whitehead, the first pediatric patient to be treated with immunotherapy, 18 months post-treatment and starting third grade.
For most of us, our immune systems fight off disease every day. A healthy immune system can even fight off life-threatening diseases like cancer. And when we do get sick, a healthy immune system attacks sickness and help us get back to feeling like ourselves again.
It is a different story for kids with cancer. A child with cancer has a suppressed immune system. It’s as if a blindfold has been pulled down, and the body is unable to target invasive cells. You might say the immune system is asleep.
What if doctors could wake up a sleeping immune system?
Where is our very first St. Baldrick’s Fellow now? Still in the lab, that’s where! Meet Dr. Sharon Singh, the physician-scientist who was given the inaugural St. Baldrick’s Fellow Award in 2005. What does that monetary vote of confidence do for a new researcher and for the childhood cancer research field? It’s been 12 years and the results are in – read on to find out.
Dr. Sharon Singh currently works as a clinician, researcher and assistant professor with the Western Michigan University Homer Stryker M.D. School of Medicine.
At the heart of it, Dr. Sharon Singh is a problem solver and for the last decade, the pediatric hematologist-oncologist has been working on the problem of childhood cancer. But she knows that the big problem of kids’ cancer can’t be solved only at the bedside of a patient – problem solvers like her need to be in the lab too.
And that is exactly what Dr. Singh is doing, thanks to a St. Baldrick’s grant given more than 10 years ago.
When Honored Kid Hazel H. relapsed, there was no clear path forward. There was no tried-and-true roadmap to survival. There was no cure. But now, spunky, precocious little Hazel is cancer free for the second time. How? Three words that bring kids with cancer and their families hope – childhood cancer research.
Diagnosed with neuroblastoma in 2013, Hazel relapsed after two and a half years cancer free. Now, in a turn of events rare for kids with relapsed neuroblastoma, she’s cancer free once again.
It was the summer of 2016 when Hazel and her family went on vacation to Yosemite National Park. For once, they were all together – Lauren and Aaron, plus the five kids: Micah, Elizabeth, Hazel, Jonah and Zoe. It felt normal. Like a regular family vacation.
For some families that might sound typical, like something every family does every summer. But to this family – which had spent the past few years in and out of hospitals, making life-or-death decisions – typical was wonderful. It was a strange, unfamiliar kind of bliss.
St. Baldrick’s supporters, this is a day to celebrate! You have helped make history. Today, the FDA approved the first gene therapy available in the United States. Called Kymriah, it’s an entirely new way of treating cancer and it’s saving lives.
This “living drug” is for patients with a type of acute lymphoblastic leukemia (ALL), a cancer of the blood and bone marrow and the most common form of childhood cancer. Scientists genetically modify a patient’s own immune cells in the lab, then infuse these new cells back into the patient’s body. These modified cells – called CAR T cells or chimeric antigen receptor T cells – then prompt the child’s own immune system to attack and kill leukemia cells.
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