America’s Charities Celebrates CureSearch Day!

Making Treatment Possible & a Cure Probable for Children Diagnosed with Cancer

What We Do:

CureSearch

CureSearchCureSearch for Children’s Cancer is a national non-profit foundation that accelerates the cure for children’s cancer by driving innovation, eliminating research barriers, and solving the field’s most challenging problems. We fight every day to make treatment possible and a cure probable for the 42 children diagnosed with cancer daily. We do this in three ways:

We accelerate the search for cures for children at greatest risk of losing their battle with cancer by posing essential challenges to scientists and inviting teams to overcome them with novel research approaches.
We support children’s enrollment in clinical trials that have the potential to save their lives today.
We provide resources and education, so no child faces a cancer diagnosis without a fully equipped support team behind them.

Ultimately, CureSearch is working to change the odds for those children most at risk.

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Mattel to Produce More Bald Barbie ‘Ella’ Dolls for Kids with Cancer

EllaFor the second year, CureSearch is honored to partner with Mattel to celebrate children with cancer through meaningful play. Mattel will once again produce Ella, friend of Barbie, to provide children with cancer a Barbie doll that is just like them. CureSearch is excited to work with hospitals across the country to host Ella Parties and give kids an opportunity to engage in meaningful play this September, Childhood Cancer Awareness Month.

“CureSearch is honored to be partnering with Mattel to ensure children across the country are able to have Ella, friend of Barbie,” says Kelli Wright, National Director of Strategic Partnerships. “Losing your hair at any age is hard; having a doll that looks just like them is invaluable to children during their cancer journey.”

To learn more about Ella Parities, please contact Shelby.Hammond@CureSearch.org, or read more at The Huffington Post.

Meet the Children: Maggie Sarbora

Maggie-Sarbora-Web1Maggie was an energetic and enthusiastic 2 1/2 year old when her parents noticed that she had become very tired. Instead of running around with her friends, she was more content to watch.

Her parents took her to her to the doctor where they performed blood work. Much to Maggie’s mother, Stephanie’s, surprise their doctor told them to head to the hospital right away. Once they arrived, they received news that they never expected, Maggie was diagnosed with low-risk b-cell leukemia.

They started treatment immediately, but it wasn’t always easy. The first month was hard for Maggie and her family. She had to be hospitalized for weeks while her body got used to the chemotherapy. Stephanie was happy when they were finally able to bring Maggie home and away from the hospital.

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What is Translational Research? Explaining the “Valley of Death’”

research2In our last post, we explored the different phases of cancer research. One of these phases—translational research—is sometimes referred to as the “Valley of Death.”

Translational research got this reputation because the process of translating early discoveries into effective treatments for patients is time-consuming, costly, and often unsuccessful. A discovery in basic science—for instance, a new way of monitoring how a certain tumor spreads through the body—must be followed by years of additional work before it becomes a treatment.  One recent article estimated that the rate of success in translational science is less than 1%. This means that if 5,000-10,000 compounds are tested, only about 5 will end up in Phase 1 studies, and only a single drug is likely to survive to become approved by the FDA.

If we follow the tumor discovery down the drug pipeline, we begin with researchers trying to figure out what proteins, enzymes, or other targets will accompany the tumor they can now track, and which of them might be susceptible to treatment. Perhaps after much testing, researchers discover that a single protein is instrumental to the growth of the tumor. The next step is to find compounds that will inhibit this protein, thus inhibiting the spread of the tumor. They might test hundreds or even thousands of compounds to see which ones work to inhibit this protein. Then, they need a good model of the tumor to test these potential compounds. Once they have narrowed down the compounds to the hundred or more most effective drugs, they will test them on animal models.  Both tumor models and animal models take time and expertise to develop to ensure they are accurate and behave like a tumor in a human.

The handful of potential treatments that make it through these initial phases and through pre-clinical testing may go on to Phase I studies in humans, the next stage of the research pipeline. If researchers are incredibly lucky, one of these treatments will be effective in treating the disease and patients will be able to tolerate it. After several more rounds of clinical trials, the drug can move on to the expensive and lengthy process of receiving FDA approval for wider use.

Although translational research is a crucial step for discovering new cancer treatments, it is an under-funded research area, in part because the risk of failure is so high. While a discovery in basic science has the potential to impact hundreds of disease areas, translational research is the crucial bridge between the laboratory and improving outcomes for patients today. In addition, because early research is so often funded by government and clinical trials are often funded by pharmaceutical companies, it can be difficult to move between these different environments and funding models. You can find more information about translational research here, here, or here.

The Challenging Road to New Childhood Cancer Therapies

research1Even though cancer is responsible for more deaths in children than all other diseases combined, the road to developing new treatments for pediatric cancer is long and slow. There are roughly four phases in the development of new therapies: basic research, translational research, clinical trials, and FDA review and approval.

Basic research is motivated by scientific curiosity without a specific end goal or product in mind. In contrast to applied research, which focuses on the practical use of scientific discoveries, basic research is undertaken for the advance of scientific knowledge and often serves as a foundation for the applied work that follows. One example of basic research is the development of recombinant-DNA techniques in the laboratory. This discovery has allowed scientists to study human genetics in much greater detail, so that scientists can now understand the precise coding of cancer mutations.

The second phase of research, translational research, takes discoveries in basic science and tries to apply them to treating and preventing diseases. This phase of research has been nicknamed the “valley of death” because it is incredibly difficult to move a discovery successfully from basic science to clinical application. Sometimes, researchers will test a compound unsuccessfully on one disease and find years later that this same compound is effective for treating a completely different disease.  This is true for raloxifene, a drug developed for osteoporosis that is actually more effective at breast cancer prevention. Translational research includes identifying cancer biomarkers and targets, and developing early testing in animal models. Even though translational research is often not successful, it is the crucial bridge between early discoveries and new drugs for patients.

Once researchers have found compounds that work on animal models, they move into the third phase of research: clinical trials. Clinical trials test new treatments on human patients. Clinical trials also have phases, which you can read more about here. Once a new treatment has gone through clinical trials, it must also go through FDA review and approval, another lengthy process in which the FDA determines whether the treatment is effective and safe for all patients.

The funding model for the current pipeline is piecemeal, contributing to the slow pace of drug development. The early stages of the pipeline are primarily funded by public sources like the National Institute of Health, while the latter stages of clinical development and FDA review are chiefly funded by for-profit industry like pharmaceuticals. Translational research is often a mix of government funding, private funding, and pharmaceutical development. CureSearch works to accelerate this process by funding research that is moving into clinical development to speed the translation of promising discoveries into potential new therapies to treat children’s cancers. By providing flexible, large-scale funding for researchers with project support and firm research deadlines, CureSearch tries to move translational discoveries into clinical application faster.

Meet the Children: Wesley Zablocki

Wesley was diagnosed with osteosarcoma (bone cancer) on March 29, 2011, after a chance fall and lucky x-ray. He was immediately thrust into the world of hospitals, doctors and the unknown. His parents had no idea what to expect with the treatments, how he would react or what they would do to their otherwise healthy son. After nine months of chemo treatments and a really-really long limb salvage surgery he was pronounced “No Evidence of Disease” for nine months. Then in October of 2012 there was a recurrence of thecancer in his lungs. Immediately they were again thrown into a life they did not choose.

Wesley had six more months of chemo and surgery on his lungs. His first set of post treatment scans, in April 2013, showed one small spot on his right lung that was too small to do anything about so the doctors just watched it for six months until it grew some and they could remove it. Another lung surgery. In the meantime it was discovered that his leg had not completely healed as they thought and in January of 2014 he had another lengthy surgery on his leg to fix the issues. Just when the family thought there might be a break in the action his scans in February displayed more cancer in his left lung. A third lung surgery.

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Six Young Investigator Grants Now Available!

$1.3 million in research funding to be provided by CureSearch
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 will be 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 available on proposalCENTRAL.altum.com.