Why Even ‘Failure’ Is Important

Most of us probably learned about the scientific method in grade school: ask a question, do research, construct a hypothesis, test it to see if it works, analyze results, and communicate the conclusion. We also probably learned that even when your experiment doesn’t turn out the way you expected, the results are still important!

This is also true in academic medicine. When researchers work to develop an effective new treatment, they often start by testing out specific chemical compounds and their effects on cancer cells. Researchers have to rule out hundreds or even thousands of compounds before they find one that works. This is a very long process, and from the outside, it can sometimes seem like nothing is getting done. However, the process of elimination is crucial to discovering new treatments! Every compound crossed off the list gets researchers one step closer to their ultimate goal: finding effective, safe treatments for childhood cancer.

One example is the discovery of All-Trans-Retinoic-Acid (ATRA), which is used to treat Acute Promyelocytic Leukemia (APL), which is a unique subtype of Acute Myeloid Leukemia (AML). In the 1970’s, children diagnosed with APL were considered very high-risk cases. Only about 50% made it to complete remission, and many relapsed or suffered significant side effects. Researchers who were studying the cellular structure of APL discovered that APL had a high number of “immature” cells, meaning they replicated very quickly. They realized that certain compounds might be able to make these cells “differentiate,” or mature, so they behaved more like normal cells. After testing hundreds of compounds like butryrate, hypoxanthine, and dimethyl sulfoxide, they landed on ATRA. ATRA was a vitamin-A derivative that caused APL cells to “differentiate,” dramatically reducing the spread of the cancer. ATRA has far fewer side effects than conventional chemotherapy, and it was nearly miraculous in treating the disease. Adding ATRA to standard treatment increased the remission rate from about 50% to almost 90%.

In order to find ATRA, researchers first had to eliminate hundreds of other compounds that failed to work with APL. Researchers could have considered these other compounds “failures,” but we prefer to think of them as necessary steps along the way toward the development of a new treatment. At CureSearch, we know that the road to a life-saving discovery is paved with these failures.

Sources:

Breitman, T. R., Stuart E. Selonick, and Steven J. Collins. “Induction of differentiation of the human promyelocytic leukemia cell line (HL-60) by retinoic acid.” Proceedings of the National Academy of Sciences 77.5 (1980): 2936-2940.

Breitman, T. R., Steven J. Collins, and B. R. Keene. “Terminal differentiation of human promyelocytic leukemic cells in primary culture in response to retinoic acid.” Blood 57.6 (1981): 1000-1004.

CureSearch Hosts Second Annual Research Symposium!

Topics will focus on emerging themes in laboratory science
Symposium

On October 17-18, CureSearch will host its second annual research symposium in Washington DC. This year’s Symposium, entitled “Translational Oncology: Emerging Targets in Childhood Cancer” will focus on emerging themes in laboratory science with potential for early implementation. Featured speakers include Malcolm Smith, James Bradner, and John Maris.

The Symposium offers special networking opportunities for young investigators seeking careers in clinical or translational oncology. For more information about the event, including registration and hotel information, please visit www.curesearch.org/Symposium. We hope to see you there!

Meet the Children: Cora Johnson

Cora JohnsonIn seventh grade, Cora found a lump in her breast. She and her mother Martha went to the doctor to see what was causing the lump. Cora’s doctors performed an ultrasound, but the results were inconclusive. After another ultrasound her team of doctors determined that she had a tumor. They weren’t sure what type of cancer Cora had, but they knew that they had to remove it.

Cora had surgery at Children’s Hospitals and Clinics of Minnesota, where doctors discovered that she had a rare form a skin cancer called Dermafibrosarcoma Puterberan. Another surgery was needed to ensure that clear margins were obtained. Since then Cora continues to be monitored by her oncologist and surgeon.

While they were at the hospital, Cora saw a poster for the CureSearch Walk and told her mom that she wanted to get involved. They formed a team – Cora’s Pink Posse – and started fundraising right away. Cora has taken the lead on organizing events and motivating her teammates to continue fundraising. Helping other children with cancer has become her passion. “We love seeing people come together to support survivors and those who have lost their battles,” says Martha. “Seeing people come together is really moving. The CureSearch Walk has become a great community.”

Read more…

Meet the National CureSearch Walk Committee

We are very pleased to introduce the inaugural National CureSearch Walk Committee (NCWC). The NCWC is a group of volunteers from across the country who have been involved with the CureSearch Walk for 2+ years and have a desire to help shape the program by lending their collective ideas, opinions, and expertise to the national platform. NCWC were nominated and chosen for their individual contributions to their local Walk and their ideas to help the national CSW program grow.

Below you will find brief bios about each member, their family and their involvement with CureSearch for Children’s Cancer.

NationalCommitte-Home

Update: Researcher to Study Bone Fractures in Pediatric and Adolescent Cancer Survivors

Dr-Vrooman-home

Researchers and physicians know that children who undergo cancer treatment are at risk for decreased bone mineral density because many cancer treatments negatively affect bone health. It is particularly important that children and adolescents develop strong bone density because bone strength decreases in adulthood. Despite this knowledge, little is known about the rate at which fractures actually occur in survivors of childhood cancer and if they can be prevented.

Lynda Vrooman, MD, MMSc of Dana-Farber Cancer Institute is a CureSearch Young Investigator interested in better defining the risks and long-term consequences of fracture in survivors of childhood cancer, and in intervening to minimize long-term complications. During her first year of research, Dr. Vrooman completed enrollment of 193 patients in a study of reported bone fracture. Her findings demonstrate that approximately 25% of cancer survivors experienced at least one fracture after cancer treatment. Of those with fracture, 35% experienced more than one fracture after treatment. In addition, survivors treated with corticosteroids, commonly used in the treatment of many types of children’s cancer, experienced significantly higher rates of fracture after cancer treatment. These results highlight the importance of minimizing the bony complications associated with corticosteroids and suggest that a treatment-associated fracture risk may extend beyond cancer therapy completion.

In the next year of her work, Dr. Vrooman will conduct detailed bone density testing in childhood cancer survivors with a history of fracture. Dr. Vrooman anticipates that this work will inform future interventional studies aimed at decreasing skeletal toxicity in survivors of childhood cancer.

Read more…

Meet the Children: Margo Miller

Margo Miller21-month-old Margo Miller hadn’t been herself for weeks. She had a fever on and off, was cranky at daycare, and wasn’t eating well. Her mother, Gina, took her to their pediatrician to see what could be causing her behavior.

Her pediatrician couldn’t find anything that could be causing her pain, but suggested that Gina bring Margo back on Monday so that they could run some additional tests. They went home and tried to enjoy a normal weekend.

On Monday, Gina and Margo headed back to the pediatrician, where her they noticed red dots on her legs. Gina was told that Margo had to have a blood test done immediately. Once the blood work came back, their pediatrician told them news that they never hoped to hear, Margo had acute lymphocytic leukemia (ALL).

Six Young Investigator Grants Now Available!

CureSearch for Children’s Cancer is now accepting applications for six (6) Young Investigator Grants. These “Young Investigator Pathway to Independence Grants” are funded at $225,000 each over three years and are available in the areas of neuroblastoma, palliative care, rhabdomyosarcoma, and three “open” areas. “Open” applications may be in disease or discipline areas of pediatric oncology and will be based on the merits of the research proposed, the productivity of the applicant, mentorship, and environment.

Awards will be announced in October, 2014 and funding will begin in January, 2015.

For those interested in applying, RFAs are posted onproposalCENTRAL.altum.com.