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Chronic Myeloid Leukemia (CML)

Chronic myeloid leukemia (CML) is also known as chronic myelogenous leukemia. CML begins in the blooding-forming cells of the bone marrow. It is most common in adults, but can occur in children.

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Timsi Rao, PhD, Principal Investigator
Yale School of Medicine
Young Investigator Grants, Awarded 2016
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Chronic Myeloid Leukemia (CML) Heroes

Latest Chronic Myeloid Leukemia (CML) blog posts

December 17, 2018

by Trish Adkins

For Eden, the trouble began when she was 10 years old. The trouble had a name: pain. It seemed normal at first—maybe a side effect from dancing or growing. But then it never went away and then, suddenly, Eden could not dance anymore.

“I knew in my heart it was more than growing pains,” said Eden’s mom, Shannon. 



A MRI revealed the source of the pain: a tumor on her leg. And not just any tumor, it was a tumor so rare that it had never been seen before. They nicknamed it Eden’s Tumor. Eden’s doctor, Dr. Jennifer Foster at Texas Children’s Cancer Center turned to research to find a treatment plan for Eden. Eden went through treatment once and then her cancer returned, this time in her shoulder. 

Eden is now enrolled in a clinical trial for children with relapsed tumors. We spoke with Dr. Foster about Eden’s tumor, her treatment and how innovative clinical trials are propelling the world closer to cures. 

ALSF: What type of cancer does Eden have?
Dr. Jennifer Foster (JF): Eden has a tumor that has not been described before. Our team ended up calling it a “primitive round blue cell primitive neural tumor.” But, really, we just refer to it as Eden’s Tumor, since it is the only one of its kind anyone has ever described. 

ALSF: Wow, walk me through what happens when a tumor like Eden’s lands on your desk?
JF: Until we can describe the tumor, we cannot treat it. This is true for both rare and less-rare tumors. We tapped into our large team of pathologists, solid tumor oncologists, radiologists and other childhood cancer experts across the country to come up with a description of the tumor. 

ALSF: What are the features that make Eden’s tumor so rare?
JF: Eden’s tumor has several features that make it so unusual. First, the tumor did not pick up on an MIBG or bone scan, which are scans used to detect neuroblastoma cells. Yet, the pathologists could tell the tumor had some characteristics of neuroblastoma. The PET scan did pick up the tumor cells in Eden’s leg, shoulder and throughout her body. 

Pathologists looked at samples of Eden’s tumor under the microscope and not only saw the neuroblastoma features, but also saw some features of the common bone cancer Ewing’s sarcoma. When we sequenced the tumor to study its biology, there were no genetic hits against a wide panel of known markers of cancer. 

ALSF: So, how do you treat something that isn’t anything that has existed before?
JF: We heavily relied on the pathology report to create a treatment plan that we knew to be effective against both neuroblastoma and Ewing sarcoma. We chose chemotherapy drugs known to work on both cancers as well as radiation, which we know kills both of these types of cancer cells. The treatment was effective and the tumors responded. Unfortunately, Eden relapsed 1 year off of therapy, this time in her opposite shoulder. 

ALSF: What happens when a child like Eden relapses?
JF: When any child relapses, we know that the standard of care did not work and we have to try something new. Often, tumors are harder to treat after relapse.  So, we turn to clinical trials and research to find potential solutions. For Eden, the biopsy of her relapsed disease showed that the tumor looked exactly the same under the microscope as it did at diagnosis. Since the first round of treatment did not work,  we had to come up with a new plan. For Eden, that plan was a new clinical trial opening both at Texas Children’s Cancer Center and at other select institutions across the country.

ALSF: What is the name of the trial Eden is on?
JF: The official title of the study Eden is a part of is “ADVL1615, A Phase 1 Study of Pevonedistat a NEDD8 Activating Enzyme (NAE) Inhibitor, in Combination with Temozolomide and Irinotecan in Pediatric Patients with Recurrent or Refractory Solid Tumors”

ALSF: How does the drug work?
JF: Pevonedistat is a novel agent that we use in combination with two standard chemotherapy regiments (Temozolomide and Irinotecan). Pevonedistat may work in combination with Irinotecan and temozolomide by blocking some of the enzymes needed for cell growth, thus stopping the growth of cancer cells. 

The pre-clinical lab testing of this combination has shown great promise and this drug has also showed success in clinical trials for adults with cancer. As long as Eden is responding to treatment, she will have up to 17 cycles of therapy over about a year. 

ALSF: Why are childhood cancer research and clinical trials so important?
JF: The trial Eden is on represents 10 years of process—pre-clinical experiments in the lab, adult clinical trials, and the building of the infrastructure necessary to support a clinical trial. We don’t have the full story for most types of childhood cancer. Continued research and clinical trials will help us learn the full story, so we can start making the drugs and agents that can cure future children diagnosed with cancer. 

ALSF: How can studying a rare, one-of-a-kind tumor help other kids battling cancer?
JF:  Every piece of knowledge we gain from rare tumors like ‘Eden’s tumor’ will build and help guide us in future treatment decisions for other children; just like how the knowledge has guided us to this point of being able to help Eden.

Our Q&A with Dr. Jennifer Foster is the second in a series about Eden’s incredible story.  If you missed the first installment, an interview with Eden, just head here.
 

YOU CAN MAKE A DIFFERENCE for children, like Eden, who want more opportunities to receive potentially lifesaving treatments and hope for a brighter, healthier future.
YOUR GIFT will fund the most promising, innovative scientific projects with the likelihood of making an impact.
YOUR SUPPORT allows Alex’s Lemonade Stand Foundation to continue to fund researchers around the country to develop less-toxic treatments and more cures for kids with cancer.
DONATE, today. 

 

 

 

 

 

 

 

 

 

December 14, 2018

by Trish Adkins

Children with certain types of hard-to-treat childhood cancers just got another huge dose of hope. Last week, the U.S. Food and Drug Administration (FDA) granted accelerated approval to a drug called Vitrakvi (also known as larotrectinib), making the treatment available to children with cancers that are NTRK fusion-positive. 

NTRK is a gene that is present in certain types of pediatric cancers, as well as some adult cancers.

ALSF funded-researcher Dr. Steven DuBois at Dana-Farber Cancer Institute played a critical role in the research that led to the breakthrough. In a clinical trial led by Dr. DuBois over 75-percent of patients treated with larotrectinib responded positively to the drug and their tumors either shrunk or disappeared. 

“Our goal is to make discoveries and disseminate these discoveries, especially when there is a therapy with such a high response rate and tolerability,” said Dr. DuBois.

The trial was held at multiple sites, including Dana-Farber Cancer Institute, which ALSF funds through its Center of Excellence (COE) program. Dr. DuBois, together with Dr. Wendy London, managed the ALSF grant funds to help sustain the infrastructure of the Developmental Therapeutics Center, collaborate with other institutions, train physician scholars in drug development and build a pediatric oncology developmental therapeutics program. The center actively participates in over 100 oncology studies at any given time.

Each of the patients in the larotrectinib trial had cancers that had the NTRK gene present. NTRK occurs when genes fuse together abnormally, resulting in the growth of abnormal cells, which become cancer. While NTRK is rare, it does occur across a range of cancer types including infantile fibrosarcoma, soft-tissue sarcoma and types of colon, lung and thyroid cancers. 

Prior to the FDA approval of larotrectinib, children with this type of cancer had no effective options for treatment.

This is the second time the FDA has approved a cancer treatment based on a common genetic feature across different types of cancer rather than the specific tumor type. The approval marks a continued effort to develop treatment protocols that could work for several different types of cancer—versus the old paradigm where cancer was treated based on the type rather than the genetic marker.

In Dr. DuBois’ trial, a total of 55 patients, ranging in age from 4 months to 76 years were treated. His findings were published in the February 22, 2018 edition of the New England Journal of Medicine. The article, “Efficacy of Larotrectinib in TRK Fusion-Positive Cancers in Adults and Children,” concluded that larotrectinib, had marked durable anti-tumor activity in patients with TRK fusion-positive cancer, regardless of the age of the patient or of the tumor type. The findings led to the recent FDA approval of the drug. The full article is available here

In addition to the support from ALSF, the trial was supported by Loxo Oncology and by grants from the National Institutes of Health, the Cancer Prevention and Research Institute of Texas and the National Center for Advancing Translational Sciences.


About the ALSF Center of Excellence grants program 
ALSF supports the advancement of clinical trials through its entire research grant program. By funding all stages of research from early career to advanced laboratory studies to our clinical trial programs, ALSF ensures that the breakthrough science happens now, so children can have cures for tomorrow.

Center of Excellence grants are given to institutions over a 5-year period to support research as well as the acceleration of clinical trials through funding for infrastructure needs and training the next generation of research scholars. In addition to Dana-Farber Cancer Institute, ALSF funds Children’s Hospital of Philadelphia, University of California San Francisco and Baylor College of Medicine through the Center of Excellence grant program.

This year, the four-Center of Excellence institutions announced the opening of a multi-site phase 1 clinical trial for a drug that shows promise for reactivating a gene that typically acts as a tumor suppressor. Called p53, the gene is often missing or mutated in several types of resistant childhood cancers including lymphoma, refractory leukemia, Ewing sarcoma, rhaboid tumors, retinoblastoma, hepatoblastoma and other cancers.  Subscribe to the ALSF blog for more details on this Phase 1 study. 

December 3, 2018

by Adam Paris

Eden is a 13 year old who loves to shoot photos and smile for her own snapshots. After being unable to walk or practice her favorite activity, dancing, for nearly a year, she was diagnosed at age 10 with a cancer no doctor had ever seen before. They tried standard chemotherapies for two different types of solid tumors similar to her cancer and after many months doctors deemed her cancer-free in March 2016.

Two years later, the cancer relapsed in her shoulder. Now, she and her tight-knit family continue to fight the cancer they named after her, Eden’s tumor, while waiting for a potential cure. 
As researchers learn more about Eden’s cancer, we wanted to give you a glimpse into Eden, the person. Check out our conversation below to learn more about this inspirational cancer fighter. 

ALSF: If you could have a superpower, what would it be and why?
Eden Green (EG): I would like to fly and be invisible. Flying is cool, but also scary. I like invisibility because I could hear conversations without being noticed in the other room. 

ALSF: What’s your go-to song to sing?
EG: I like to sing Francesca Battistelli. She’s a Christian artist so I sing her songs a lot, and then Lauren Daigle too. 

ALSF: I know you love taking photos! What do you like to photograph? 
EG: I like shooting nature a lot. I do a lot of flowers and nature shots and they turn out pretty good actually. 

ALSF: What is your favorite 2018 memory so far?
EG: My family went on a cruise in January. We went to Cozumel, Jamaica and Puerto Rico. I didn’t get to do as much this year because of my relapse. 

ALSF: What do you want to be when you grow up?
EG: It changes, but I kind of want to be a travel agent like my mom. I changed my mind from nursing because I don’t really want to do that anymore. Maybe a social worker at the hospital, but I don’t even know if I want to work at the hospital.

ALSF: What did you miss most while you were in treatment?
EG: I missed church, and we go to church a lot, so I missed going there. I like to see my friends and everything, so I didn’t get to see them really when I was diagnosed again. That was hard. I got to spend a little bit of my summer with my friends though, especially my one friend Rachel. She’s at my house like every day. I also missed going on trips that I was supposed to go on. 

ALSF: How has your family supported you?
EG: I don’t know, I’m asleep most of the time! My whole family is really funny though, but my dad he’s really funny. He’ll pull up things like the In My Feelings challenge videos to cops lip syncing stuff so he’ll watch those and start dying from laughter!
My sister Mya provides entertainment and my mom does too. Mom has to get her Fitbit steps in. When doctors come to help me I say, “Okay mom, you get your Fitbit steps in.”

ALSF: What does Mya do that helps you out? 
EG: She’s a competitive gymnast, so that helps me because I’m at the gym almost every day with her, watching their whole team. They practice every day for three hours. Most of the time before I relapsed, I was on the floor encouraging them during their routines. I actually got an award for being their team mascot. I’m like their group’s big sister because their siblings usually don’t come and watch them, so I’m always there. 

ALSF: What advice do you have for kids with cancer in the hospital?
EG: Stay strong and you’ll get through it. I’ve done it once and I’m doing it a second time so you can do it, I know you can. Keep having your faith and keep doing what you’re doing to help get through it.  

Our Q&A with Eden is the first in a series about her incredible story. Stay tuned next week for a discussion with her doctor, ALSF-funded researcher Dr. Jennifer Foster. 
 

YOU CAN MAKE A DIFFERENCE for children, like Eden, who want more opportunities to receive potentially lifesaving treatments and hope for a brighter, healthier future.

YOUR GIFT will fund the most promising, innovative scientific projects with the likelihood of making an impact.

YOUR SUPPORT allows Alex’s Lemonade Stand Foundation to continue to fund researchers around the country to develop less-toxic treatments and more cures for kids with cancer.

DONATE, today.