Pediatric Hematology and Oncology

US WorldMeds Announces FDA Approval of IWILFIN™ (eflornithine) to Strengthen Fight Against Aggressive Childhood Cancer

Retrieved on: 
Thursday, December 14, 2023
Children, FDA, Health, General Health, Pharmaceutical, Oncology, Research, Family, Consumer, Science, Parenting, Neuroblastoma, Tenderness, Urine, Pneumonia, Huber loss, Somatic symptom disorder, Patient, Otitis media, Diagnosis, Tiffany Aching, Bruise, Liver, Chills, Hospital, Hearing loss, Vomiting, Food, Chair, American Cancer Society, EFS, Nose, Mortality, Multimedia, Division, Hematology, Pennsylvania State University, Blood, Diarrhea, Kool-Aid, Bleeding, Nausea, Development, Skin infection, Shivering, Urinary tract infection, Cough, Consortium, Risk, Sinusitis, Humble, Texas, Pain, Skin, Eflornithine, Hope, Upper respiratory tract infection, Disease, Abdomen, Tablet, Pediatric Hematology and Oncology, Sneeze, MD, Death, Immunotherapy, Pharmaceutical industry, Medical device, Nursing, Birth control, Phosphorus, Fever, USWM

High-risk neuroblastoma is a challenging disease, with a high mortality rate driven primarily by the risk of relapse after achieving remission.

Key Points: 
  • High-risk neuroblastoma is a challenging disease, with a high mortality rate driven primarily by the risk of relapse after achieving remission.
  • US WorldMeds partnered with the Beat Childhood Cancer Research Consortium at Penn State University, which conducted the preclinical and clinical research to help advance this vital therapy.
  • The Consortium represents a group of over 50 hospitals that offer collaboration through a network of childhood cancer clinical trials.
  • “We are thankful for the dedication of our partners, specifically the Beat Childhood Cancer Research Consortium, who work tirelessly to improve treatment outcomes for pediatric cancer patients.

Vertex Receives CHMP Positive Opinion for the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Retrieved on: 
Friday, December 15, 2023
Research, FDA, Genetics, Clinical Trials, Stem Cells, Biotechnology, Health, Pharmaceutical, Science, Quality, Pediatrics, European Commission, Sickle cell disease, European Medicines Agency, HSC, Bambino, Vertex, Civil service commission, Patient, European, Pediatric Hematology and Oncology, CHMP, Gene, Committee, Advanced Therapy Medicinal Product, Hematology, VRTX, CRISPR, TDT, Beta thalassemia, Volatile organic compound, Catholic higher education, SCD, Quality of life, Vertex Pharmaceuticals, Pharmaceutical industry

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the conditional approval of CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the conditional approval of CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
  • An approval decision by the European Commission is expected in February 2024.
  • “This positive opinion is yet another important regulatory milestone underscoring the potentially transformative benefit of CASGEVY for eligible patients with sickle cell and transfusion-dependent beta thalassemia,” said Nia Tatsis, Ph.D., Executive Vice President and Chief Regulatory and Quality Officer at Vertex.
  • “As an investigator, I have witnessed first-hand the transformative impact exa-cel can have on patients’ lives and I eagerly await the approval in the European Union.”

Scenic Biotech Announces Positive Preclinical Data for its QPCTL Inhibitor SC-2882 as Potential New Therapeutic Approach for Diffuse Large B-Cell Lymphoma

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Saturday, December 9, 2023
Oncology, Health, Genetics, Managed Care, Clinical Trials, Pharmaceutical, Biotechnology, Tumor-associated macrophage, Associate, Toxicity, Weill Cornell Medicine, Progression-free survival, ASH, IND, Immunosuppression, Clinical trial, B-cell lymphoma, Patient, Tumor microenvironment, Pediatric Hematology and Oncology, Pharmacology, Lymph, Lymphatic system, TME, Therapy, Cancer, CD47, CMO, Drug resistance, 65th Street Yard, Doctor of Philosophy, Chemokine, Society, Overalls, Pharmaceutical industry, Vaccine, DLBCL, Hematology, Lymphoma, MD

Scenic Biotech , a pioneer in the discovery of genetic modifiers developing therapeutics to treat severe diseases, today announced positive preclinical data for its lead small molecule QPCTL inhibitor, SC-2882.

Key Points: 
  • Scenic Biotech , a pioneer in the discovery of genetic modifiers developing therapeutics to treat severe diseases, today announced positive preclinical data for its lead small molecule QPCTL inhibitor, SC-2882.
  • The new preclinical data covers results for SC-2882, Scenic’s first-in-class QPCTL inhibitor, as a potential new treatment approach for diffuse large B-cell lymphoma (DLBCL).
  • SC-2882 targets QPCTL and thereby inhibits the CD47-SIRPα “don’t eat me” checkpoint and lowers immune suppression in the tumor microenvironment.
  • Analysis of DLBCL patient datasets revealed that QPCTL expression negatively correlates with overall and progression-free survival.

Onconova Expands Leadership Team with Two Key Appointments

Retrieved on: 
Tuesday, October 24, 2023
Drug development, Research, Bone marrow, Ovarian cancer, Nottingham Trent University, Medicine, Internal medicine, Bachelor, Biomedical sciences, George Washington School, Pediatric Hematology and Oncology, Medical Affairs Bureau, Pharmacy, European Society for Clinical Investigation, Pharmaceutical medicine, University of Zimbabwe, Postgraduate diploma, Epidermolysis bullosa, University, NDA, Cancer, Therapy, Patient, Myelodysplastic syndrome, Merrimack Pharmaceuticals, GSK plc, Medication, CMO, Nottingham, Johns Hopkins School of Medicine, King's College London, University of Surrey, IND, Consultant, Outline of health sciences, Patent, Rare, Inflammation, R, EB, Johnson & Johnson, Pharmaceutical industry, Nursing, Biotechnology, Science, Hematology, Honours degree

NEWTOWN, Pa., Oct. 24, 2023 (GLOBE NEWSWIRE) -- Onconova Therapeutics, Inc. (NASDAQ: ONTX), (“Onconova”, the “Company”), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, today announced that the Company expanded its leadership team with the naming of Dr. Victor Moyo as Chief Medical Officer (CMO), and the addition of Meena Arora as Vice President, Global Medical Affairs and Research and Development (R&D).

Key Points: 
  • Both are accomplished experts in their fields and bring significant and wide expertise in drug development.
  • I look forward to working with my colleagues at Onconova and our clinical collaborators to contribute to our mission of bringing narazaciclib and rigosertib to patients with cancer.
  • He has also held various leadership roles as Vice President of Clinical Investigations at Merrimack Pharmaceuticals and the Centocor Ortho Biotech Services, LLC division of Johnson & Johnson.
  • Prior to that, she held various leadership roles as Medical Director or Associate Medical Director, as well as roles in Investigator Research and Medical Affairs at Celgene International.

Florida Cancer Specialists & Research Institute Welcomes Hematologist and Medical Oncologist Raphael Bosse, MD, PhD to Collier County

Retrieved on: 
Tuesday, September 12, 2023
University of Florida College of Medicine-Jacksonville, Florida College, Pediatric Hematology and Oncology, University, Research, Doctor of Philosophy, Documentation, Patient, Paul Walcker, Internal medicine, Fellows, FCS, Nursing, Hematology, MD

FORT MYERS, Fla., Sept. 12, 2023 /PRNewswire/ -- Florida Cancer Specialists & Research Institute, LLC (FCS) is pleased to announce that Board-certified hematologist and medical oncologist Raphael Bosse, MD, PhD has joined the statewide practice.

Key Points: 
  • FORT MYERS, Fla., Sept. 12, 2023 /PRNewswire/ -- Florida Cancer Specialists & Research Institute, LLC (FCS) is pleased to announce that Board-certified hematologist and medical oncologist Raphael Bosse, MD, PhD has joined the statewide practice.
  • He is providing care to patients at the Naples-Goodlette location , 1100 Goodlette Road N., Naples, Florida 34102.
  • Dr. Bosse received his medical degree and doctorate in Molecular Cell Biology & Hematology from the University of Florida College of Medicine.
  • "We are delighted to welcome Dr. Bosse to our team of cancer experts in Collier County," said FCS Chief Executive Officer Nathan H. Walcker .

Celebrating the Remarkable Impact of the Isabella Santos Foundation in the Battle Against Pediatric Cancer

Retrieved on: 
Wednesday, August 30, 2023
Levine Children's Hospital, Neuroblastoma, Survivor, Hair, Partnership, Hematology, Survival, Cancer, Hope, Rare, Research, BMT, Birth, Dream, ISF, Hospital, Memory, Pediatric Hematology and Oncology, Birth control, Pharmaceutical industry, Nursing, Oncology

CHARLOTTE, N.C., Aug. 30, 2023 /PRNewswire-PRWeb/ -- The Isabella Santos Foundation (ISF), a renowned non-profit foundation dedicated to funding pediatric cancer research and improving the lives of those affected by cancer, celebrates 16 years and more than $7 million raised. Through their relentless dedication, the foundation has left an indelible mark on the pediatric cancer community. It is a testament to the power of community spirit and the transformational impact of targeted philanthropy in the quest to combat cancer. As they look ahead, ISF is focused on expanding their reach and building a global rare pediatric cancer research and clinical trial program in Charlotte, furthering their mission to change outcomes and save lives.

Key Points: 
  • CHARLOTTE, N.C., Aug. 30, 2023 /PRNewswire-PRWeb/ -- The Isabella Santos Foundation (ISF), a renowned non-profit foundation dedicated to funding pediatric cancer research and improving the lives of those affected by cancer, celebrates 16 years and more than $7 million raised.
  • Through their relentless dedication, the foundation has left an indelible mark on the pediatric cancer community.
  • The Birth of a Legacy: The Isabella Santos Foundation
    Founded in 2007, the Isabella Santos Foundation was born from a mission to honor the memory of a brave little girl, Isabella Santos, who lost her life to neuroblastoma at just 7 years old.
  • Investment in Pediatric Cancer Research: $4,551,942
    Donations to National Pediatric Cancer Trials & Studies: $1,953,635
    One of ISF's most significant achievements is the launch of the Isabella Santos Rare & Solid Tumor Program in collaboration with Levine Cancer Institute.

Verismo Therapeutics Welcomes Dr. Mala Talekar as Vice President of Clinical Development

Retrieved on: 
Tuesday, July 11, 2023
Einstein Medical Center Philadelphia, GSK, University, Children's Hospital of Philadelphia, MBBS, Drug, Philadelphia, DABP, American Board of Pediatrics, DNB, Grant Medical College and Sir Jamshedjee Jeejeebhoy Group of Hospitals, Haematopoietic system, Penn, Verismo, Public key certificate, American Board of Commissioners for Foreign Missions, The Economist, Patient, Medical Center, American Society of Pediatric Hematology/Oncology, Hospital, Pediatric Hematology and Oncology, Therapy, POST, Diplomate of National Board, Pharmaceutical industry, Nursing, Pediatrics

PHILADELPHIA, July 11, 2023 /PRNewswire/ -- Verismo Therapeutics, a clinical-stage CAR-T company, Penn spinout, and pioneer of the novel KIR-CAR platform technology, today announced that Dr. Mala Talekar, MBBS, DABP, DNB has joined the company as Vice President of Clinical Development.

Key Points: 
  • PHILADELPHIA, July 11, 2023 /PRNewswire/ -- Verismo Therapeutics , a clinical-stage CAR-T company, Penn spinout, and pioneer of the novel KIR-CAR platform technology, today announced that Dr. Mala Talekar, MBBS, DABP, DNB has joined the company as Vice President of Clinical Development.
  • Mala is board certified in both General Pediatrics and Pediatric Hematology/Oncology with more than 20 years of experience in the field.
  • "I am excited to be a part of the Verismo team and to continue developing and delivering cutting-edge treatments to patients," said Mala.
  • Mala is Board Certified physician in both General Pediatrics and Pediatric Hematology-Oncology by the American Board of Pediatrics (DABP).

Autolus Therapeutics strengthens its Board with the appointment of Dr. Robert Iannone as a Non-Executive Director

Retrieved on: 
Tuesday, June 20, 2023
Yale University, Drug development, University, Senior, ALL, Acute lymphoblastic leukemia, Head, Merck & Co., University of Pennsylvania, Global production network, AstraZeneca, Bangladesh Technical Education Board, Assistant, Government, Research and development, Biomarker, CAR, Development, Johns Hopkins University, Patient, Fellow, Senior management, Knowledge, Jazz Pharmaceuticals, Merck, Pediatric Hematology and Oncology, Therapy, BLA, Annual general meeting, Pharmaceutical industry, Nursing, Pediatrics, Research

Dr. Iannone will serve as a Class I Director, with an initial term expiring at Autolus’ 2025 Annual General Meeting.

Key Points: 
  • Dr. Iannone will serve as a Class I Director, with an initial term expiring at Autolus’ 2025 Annual General Meeting.
  • Prior to then, Dr. Iannone served as Assistant Professor of Pediatrics at the University of Pennsylvania School of Medicine.
  • He currently serves on the Board of Directors of iTeos Therapeutics and previously served on the Board of Directors of Jounce Therapeutics (acquired by Concentra Biosciences in 2023 for $96.46 million).
  • He completed his Residency and Chief Residency in Pediatrics and a Fellowship in Pediatric Hematology-Oncology at Johns Hopkins University.

Nothing but net! Coach Dawn Staley warms children's hearts with award-winning My Special Aflac Duck®

Retrieved on: 
Friday, June 16, 2023
DNA, UVA Children's Hospital, UVA, Hematology, Cancer, Light, Duck, Athlete, Patient, Happiness, Sickle cell disease, System, Pediatric Hematology and Oncology, Female, Nursing, Vaccine, Aflac

COLUMBUS, Ga., June 16, 2023 /PRNewswire/ -- Trailblazing University of Virginia basketball legend and South Carolina women's basketball head coach Dawn Staley teamed up with Aflac Incorporated, a leading provider of supplemental health insurance in the U.S., to deliver My Special Aflac Ducks, a free-of-charge resource created for children ages 3 and up with cancer and sickle cell disease, to patients at UVA Children's in Charlottesville, Virginia. Coach Staley, Aflac U.S. President Virgil Miller and UVA women's basketball coach Amaka "Coach Mox" Agugua-Hamilton participated in a special event where 5 patients each received their own social robot as part of Aflac's 28-year, $168-plus million philanthropic commitment supporting children with cancer and blood disorders.

Key Points: 
  • My Special Aflac Duck uses medical play, lifelike movement and mimics emotion to help children cope with treatments.
  • It was great to be back in Charlottesville to deliver My Special Aflac Duck and bring joy to these patients."
  • The My Special Aflac Duck event is one example of how Aflac and Coach Staley are giving back.
  • We are thankful that Coach Staley, UVA's own Coach Mox and Aflac were here with us today to deliver this free-of-charge resource."

Positive Results From Pivotal Trials of exa-cel for Transfusion-Dependent Beta Thalassemia and Severe Sickle Cell Disease Presented at the 2023 Annual European Hematology Association (EHA) Congress

Retrieved on: 
Friday, June 9, 2023
Stem Cells, Biometrics, Biotechnology, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, CRISPR, Medical Affairs Bureau, Pediatrics, Hematology, Vertex, SCD, Quality of life, VOC, TI6, Congress, CRSP, Patient, Bambino, EHA, Sickle cell disease, Sapienza University of Rome, CRISPR Therapeutics, Therapy, TDT, Pediatric Hematology and Oncology, Safety, Vertex Pharmaceuticals, Pharmaceutical industry, Medical imaging

The results are being presented at the Annual European Hematology Association (EHA) Congress.

Key Points: 
  • The results are being presented at the Annual European Hematology Association (EHA) Congress.
  • The data shared are from 83 patients (48 with TDT and 35 with SCD) dosed with exa-cel with follow-up up to 43.7 months.
  • All patients treated with exa-cel demonstrated clinical benefit, and these data continue to demonstrate the potentially transformative profile of exa-cel.
  • This presentation will include updated pivotal trial data from patients treated with exa-cel in CLIMB-111 and CLIMB-121 and followed in CLIMB‑131.