Tag Archives: FDA

Cord Blood, a life line

Posted on August 31, 2016 by Team Captain

We are living in truly amazing times as scientists are just beginning to understand the power of stem cells. In particular, cord blood stem cells are providing new hope to cancer patients throughout the world. At Save the Cord Foundation, … Continue reading →

Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease, Uncategorized, young adults | Tagged #SAVEtheCORD, Acute Lymphocytic Leukemia, ALL, autism, BeTheMatch, bone marrow, cerebral palsy, Cord blood registries, cord blood stem cells, donor, Dylan Praskins, FDA, Graft Versus Host Disease, GVHD, HIV, Leukemia, lymphoma, match, MDSacquired hearing loss, Myelodysplastic Syndrome, Neutropenia, Noah Swanson, perinatal stroke, public donation program, Save the Cord Foundation, sickle cell anemia, stem cells, transplant | 1 Comment

Immunotherapy

Posted on December 21, 2014 by Team Captain

I am not sure how many people outside of the cancer research community know this, but it is well recognized that the human body is equipped to fight off cancer on its own. The same immune system that we have … Continue reading →

Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease, Uncategorized | Tagged acute lymphoblastic leukemia, ALL CLL, American Society of Hematology, autoimmune disease, biotech, Bristol-Meyers Squibb, CAR, CAR T, CART, checkpoint inhibitor, chemotherapy, Children's Hospital of Philadelphia, chimeric antigen receptor, cytokines, DNA, Dr. Stephen Grupp, EGFG, FDA, Gardasil, HIV Aids, immunosupressive, immunotherapy, interferon, interleukins, Keytruda, Laurie Orloski, Leukemia, Melanoma, Merck, metastatic disease, molecularly targeted agents, New England Journal of Medicine, nivolumab, Novartis Pharmaceuticals, Opdivo with r symbol, PD-1, pembrolizumab, personalized medicine, pharma, pharmaceutical, small-molecule inhibitors, spontaneous remission, T-cells, toxicity, tyrosine kinase inhibitors (TKIs), University of Pennsylvania, vaccines | 1 Comment

Compassionate Use

Posted on July 30, 2014 by Team Captain

You may remember the #SaveJosh news articles in the spring of 2014 where little Josh Hardy’s family mounted, as a last ditch effort, a social media campaign to get him into a clinical trial that St. Jude’s doctors said was … Continue reading →

Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease, Uncategorized | Tagged Brain cancer, Compassionate use, Ellyn Miller, FDA, Grabriella Miller, Joe Baber, Nathalie Traller, NCI, NIH, pediatric diseases, Pharna, Smashing Walnuts | 4 Comments

The Fault in Our Systems

Posted on July 29, 2014 by Team Captain

by Nathan Traller, Nathalie’s father Editor’s Note: Nathan’s daughter was diagnosed with Aveolar Soft Part Sarcoma (ASPS) two years ago and he has been fighting to get her into a possible life saving clinical trial for PD1 and PDL1. Time … Continue reading →

Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease, Uncategorized | Tagged 2.0 clinical Trials, ASCO, ASPS, Aveolar Soft Part Sarcoma, Bristol-Myers Squibb, Clinical Trials, Compassionate use, FDA, Genentech Inc., genomic profile, Lung MAP trial, Merck, metastatic cancer, Nathan Traller, National Cancer Institute, National Institutes of Health, NCI, NIH, PD1, PDL1, pediatric trials, rare disease, tumor bank | 3 Comments

Thank You, American Cancer Society

Posted on March 3, 2014 by Team Captain

In the past, I have been a huge critic of the American Cancer Society and in September, I wrote a very inflamed blog about all that I felt was wrong with the American Cancer Society’s (ACS) involvement with childhood cancer. … Continue reading →

Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease, Uncategorized | Tagged ACS, American Cancer Society, FDA, Joe Baber, NCI, NIH | 9 Comments

Let’s Push Buttons

Posted on December 5, 2013 by Team Captain

Would you believe?: 96% of all clothing sold is for adults. 96% of all breakfast cereals are consumed by adults. 96% of all bicycle helmets are worn by adults. 96% of all cancer research conducted by the National Cancer Institute … Continue reading →

Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease, Uncategorized | Tagged American Cancer Society, FDA, gold ribbon, Joe Baber, National Cancer Institute, NCI | 4 Comments

We need more than just research

Posted on May 2, 2013 by Team Captain

A mother noticed that her six year-old son’s eyelids were not closing normally and when he walked, he was slightly off balance. A few days later, his parents’ worst nightmare became a reality; they were told that their son has … Continue reading →

Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease, Uncategorized | Tagged Anaplastic Astrocytoma, Beth Anne Baber, chemothrapy, childhood cancer, Compassionate use, FDA, NIH, pathologists | 1 Comment

The FDA and Childhood Cancer – Part 2

Posted on March 30, 2013 by Team Captain

This is part 2 of the Jonathan Agin series, “Childhood Cancer – My Road Map” Being sent to an emergency room to have a CT scan performed on your two-year-old child is frightening. Being brought into a small cramped room … Continue reading →

Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease, Uncategorized | Tagged clinical trial, Compassionate use, FDA, funding, Jonathan Agin, patient advocate, patient representative, pediatric oncology | Leave a comment

Engaging the Pharmaceutical Industry – Part 1

Posted on March 15, 2013 by Team Captain

This is beginning of a four part series by Jonathan Agin, an attorney and well known childhood cancer advocate (www.jonathanagin.com) in Washington, DC. He is married to Neely Agin and is the father of Alexis, Gabriel Agin and Trevor Agin. Alexis was … Continue reading →

Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease, Uncategorized | Tagged childhood specific, compounds, Congress, Creating Hope Act, DIPG, DIPG Collaborative, FDA, Food and Drug Administration, harold V arms, Jonathan Agin, lobby, M.D., Nancy Goodman, National Cancer Institute, pediatric cancer drugs, pharmaceutical companies, profit model | Leave a comment

The Funding Conundrum for Pediatric Cancer

Posted on March 6, 2013 by Team Captain

My grandson, at 15 months of age, was diagnosed with high risk neuroblastoma. He is a survivor. Although, he endured an incredible six year long journey, this story is not about him, his family, the changes in their lives, or … Continue reading →

Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease, Uncategorized | Tagged Best Pediatric Exclusivity Provision, BPCA, Carolyn Pryce Walker, CDC, childhood cancer, Congress, Deborah Pryce, Dr. Jessica Boklan, FDA, Jack Reed, Joe Baber, Michael McCaul, NCI, neuroblastoma, Norm Coleman | 3 Comments

Tissue Donation – The last thing any parent wants to give

Posted on January 15, 2013 by Team Captain

Progress, unfortunately is sometimes forged from the fires of pain mixed with strength, courage, passion and determination. Nancy Goodman has such a story. Nancy made a donation that no parent ever wants to give. “My son, Jacob, was a beautiful, … Continue reading →

Posted in Cancer, Childhood Cancer, Pediatric Cancer, Rare Disease | Tagged autopsy, Brain cancer, Congressional Childhood Cancer Caucas, Creating Hope Act, DIPG, donation, Dylan Jewett, FDA, four square clobbers cancer, Hope Act, Jacob Goodman, Joe Baber, medulloblastoma, Morquio A syndrome, Nancy Goodman, President Obama, Rare Pediatric Disease Priority Review Voucher, tissue, tumor, Vimizim | Leave a comment

Cancer Drug’s Valley of Death – Where is it, What is it?

Posted on January 13, 2013 by Team Captain

To truly understand the problems with development of drugs specific to childhood cancer, and rare disease in general, examine the FDA’s Therapeutic Development Pipeline below. The pipeline represents the normal development of an average drug. The average successful drug will … Continue reading →