All posts tagged: Drug Development

FDA Public Meeting: Rare Disease Day 2022

FDA will host a virtual public meeting on March 4, 2022 to join the global observance of Rare Disease Day. The theme for FDA’s Rare Disease Day is “Sharing Experiences in Rare Diseases Together.” Patients, patient advocates, researchers and medical product developers may benefit from attending this public meeting on rare disease product development. During presentations and panel discussions various stakeholders will share their perspectives on and experiences in rare disease product development.

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CAC2 Webinar–Why Childhood Cancer Foundations are Adopting the Venture Philanthropy Approach

In our January CAC2 All-Member webinar CAC2 invited the following panelists to speak on Venture Philanthropy: Dean Crowe - Rally Foundation For Childhood Cancer Research, Steve Giusto - Pediatric Cancer Research Foundation, Joe McDonough - The Andrew McDonough B+ Foundation and Dena Sherwood - Arms Wide Open Childhood Cancer Foundation. Venture Philanthropy is a growing trend across many important social issues. For the childhood cancer community, one case study of venture philanthropy is with Oncoheroes Biosciences. Since October 2019, 12 childhood cancer nonprofits have invested in this biotech company that is developing new therapies for children with cancer. In this webinar, [...] Read more

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

Guest Blog–How Collaboration is Advancing Research and Clinical Care in Pediatric Cancer

By Guest Blogger Dr. Lynne Davies, Operations Manager, International Cancer Research Program   It is with great excitement that I write to let you know about the upcoming webinar scheduled for Monday, April 19th at 10am ET, which aims to capture the very essence of why the International Cancer Research Partnership (ICRP) was created.   ICRP is a unique alliance of cancer organizations working together to enhance global collaboration and strategic coordination of research.  This webinar, entitled “How collaboration is advancing research and clinical care in pediatric cancer,” is jointly organized by ICRP, National Cancer Research Institute Children’s Group and Events […]

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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

CAC2 Member Blog–Joining Forces to Bring New Therapies to Children with Cancer

By 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 approved by [...] 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

CAC2 Webinar–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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