All posts tagged: Drug Development

CC Community News Digest (May 25-31)

Assorted news from the last week: FDA issues its final guidance on the RACE Act and specifies when sponsors must conduct pediatric studies for new cancer drugs as well as when those requirements can be waived or deferred. CAC2 Supporting Organization Day One Biopharmaceuticals, Inc. (Nasdaq: DAWN), a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for patients of all ages with genetically defined cancers, went public and announced the pricing of its initial public offering of 10,000,000 shares of its common stock. Findings from an analysis of patients’ cognitive functioning after treatment of specific extracranial solid tumors [...] Read more

CC Community News Digest (December 21-January 3)

Assorted news from the last two weeks: NCI unveiled two new resources for young people with cancer: tips to help young people facing end-of-life decisions talk with their doctors and make choices about palliative care, advance directives, living wills, and how they want to be remembered. resource to help young people find support after a cancer diagnosis and during cancer treatment. It includes advice on talking with mental health experts, spotting signs of depression, and finding support groups. Study suggests “that children in the U.S. may be treated more intensely compared to children in the U.K., which led to a decreased […]

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CC Community News Digest (week of August 24-30)

Assorted news from the last week: Cancer and its treatment may accelerate the aging process in young patients Research has identified a small molecule compound that may make some medulloblastoma patients respond better to treatment Childhood Cancer virtual Action Day planned for September 9 Chadwick Boseman, who died of cancer Friday at age 43, inspired many in roles as King T'Challa, Jackie Robinson and James Brown, but also used his prominence to lift up children with cancer. CAC2 salutes Members Solving Kids’ Cancer, Momcology, Ty Louis Campbell Foundation, and the Max Cure Foundation who worked collaboratively with Friends of Karen [...] Read more

Joining Forces to Bring New Therapies to Children with Cancer

By Guest Blogger, CAC2 Member Cesare Spadoni (aPODD).   “Pharma and biotech companies do not develop drugs for kids with cancer!” How many times have we heard these words within the childhood cancer community? Over the past several decades we have witnessed unprecedented progress in oncology, with an array of innovative and more targeted treatments reaching cancer patients. However, these scientific advances have only partially benefited younger patients. The pharmaceutical industry, in strategic pursuit of bigger financial returns, has traditionally focused on adult oncology indications. As a result, over the past 30 years more than 200 new drugs have been [...] Read more

FDA Approves Gemtuzumab Ozogamicin for CD33-Positive AML in Pediatric Patients

On June 16, 2020, the Food and Drug Administration extended the indication of gemtuzumab ozogamicin (MYLOTARGTM, Wyeth Pharmaceuticals LLC) for newly-diagnosed CD33-positive acute myeloid leukemia (AML) to include pediatric patients 1 month and older.   Read More Here On June 16, 2020, the Food and Drug Administration granted accelerated approval to pembrolizumab (KEYTRUDA®, Merck & Co., Inc.) for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB H) [≥10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. Read [...] Read more

What is a Pediatric Study Plan?

Our May CAC2 All-Member webinar focused on how pediatric cancer study plans are developed.  CAC2 Member Kelli Wright (CureSearch) introduced Dr. Brenda Weigel from University of Minnesota’s Masonic Cancer Center and Dr. Samuel Blackman of Day One Biopharmaceuticals as they addressed: What is a Pediatric Study Plan (PSP)? How is it created and by who? How does FDARA/RACE for Children impact PSP development? Where in the process are the opportunities to create better PSPs? Currently, regulatory agencies require that a pharmaceutical or biotechnology company submit a Pediatric Study Plan (PSP) to confirm the suitability of drug usage in the pediatric [...] Read more

Day One Biopharmaceuticals

Day One Biopharmaceutical, a start-up biotech company founded with a focus on developing therapies for children with cancer through drug repurposing announced its debut in the market last week.  Its first agent, DAY101, licensed from Takeda Pharmaceuticals, is a targeted treatment for children with low grade glioma, a type of brain cancer. DAY101 is a Phase 2-ready RAF inhibitor that also shows strong potential for other pediatric and adult indications. The company is co-founded by Chief Medical Officer Dr. Sam Blackman (who offered our May webinar) and Acting CEO Julie Grant from Canaan Partners.   You can read more here.   Please look for a […]

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Accelerating Drug Development for Children and Adolescents with Cancer

We were pleased to invite CAC2 members, associate members, student members, supporting organizations to our December CAC2 All-member webinar where Susan Weiner (Children's Cancer Cause) hosted Dr. Gilles Vassal, chair of ACCELERATE, an international nonprofit. Dr. Vassal discussed ACCELERATE and its fresh approach to addressing the complex challenges of developing innovative pediatric cancer therapies in the new regulatory environment. View the webinar below. Read more

Understanding and Enhancing the Role of Children with Cancer and their Parents in Pediatric Oncology Drug Development

This month's webinar was presented by Stacy Crane, Predoctoral Fellow at Indiana University School of Nursing.  She described the experience of participating in a Phase I clinical trial from the perspective of the parents of children battling cancer. While overall, parents do not regret their child participating in a phase I clinical trial and would recommend participation to other parents of children with cancer, there are implications for clinicians and researchers who work with children with cancer and their families. One way to improve experiences in early phase clinical trial participation is to ensure that the side effects that impact the patients during these [...] Read more

Non-Profit Driven Pediatric Oncology Drug Development

In this All-Member webinar for May 2016, CAC2 welcomed Dr. Cesare Spadoni, Chairman and Co-Founder of Accelerating Paediatric Oncology Drug Development. Parents and non-profit entities can play a strong role in driving drug development and thus accelerate the availability of more innovative drugs for children with cancer. But to do, so they must adopt new operating models and collaborate more effectively with other stakeholders. Read more