Blood

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.

iECURE Secures Clearance from Australian Therapeutic Goods Administration for its Clinical Trial Application for the OTC-HOPE Phase 1/2 Study of ECUR-506

Retrieved on: 
Wednesday, December 13, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Research, Safety, Nerve, CTN, Gene editing, Ornithine transcarbamylase deficiency, Pediatrics, Gene, University, Coma, Mouse, Liver, Clinical trial, Disease, Risk, TGA, Patient, GTP, Nervous system, Pharmacokinetics, Child, Blood, Primate, Trial of the century, Ammonia, Death, Metabolic disorder, Neurology, Quality of life, OTC, Mount Sinai Hospital (Manhattan), Pharmaceutical industry, Dietary supplement, Medicine

“The TGA’s clearance to begin clinical trials for the OTC-HOPE study represents major milestones for the OTC deficiency community and for the iECURE team,” said Joe Truitt, Chief Executive Officer of iECURE.

Key Points: 
  • “The TGA’s clearance to begin clinical trials for the OTC-HOPE study represents major milestones for the OTC deficiency community and for the iECURE team,” said Joe Truitt, Chief Executive Officer of iECURE.
  • “ECUR-506 has the potential to restore the function of the OTC enzyme in infants who lack this critical component of the urea cycle.
  • We are hopeful that our approach will be a better option for these children.”
    The OTC-HOPE study is a Phase 1/2 first-in-human study in baby boys with genetically confirmed neonatal-onset OTC deficiency.
  • Currently available medical therapies do not correct the disease and do not eliminate the risk of life-threatening symptoms or crises.

City of Hope presents pivotal clinical trial data at American Society of Hematology (ASH) conference on revumenib, a potential new targeted therapy for high-risk subtypes of acute leukemias

Retrieved on: 
Tuesday, December 12, 2023
Oncology, Health, Clinical Trials, Research, Pharmaceutical, Science, Eye, Food, City, Volleyball, Postcholecystectomy syndrome, ASH, Disease, Haematopoietic system, Clinical trial, Patient, Multimedia, Bakersfield College, Viral, Division, FDA, CRH, KMT2A, Hematology, Blood, ORR, Nausea, Cancer, Survival, Chronic pain, AML, Family, ALL, Bone marrow, Travel, Acute leukemia, Pharmaceutical industry, Leukemia, Hope

The data was presented as a late-breaking abstrac t at ASH and in a press briefing.

Key Points: 
  • The data was presented as a late-breaking abstrac t at ASH and in a press briefing.
  • View the full release here: https://www.businesswire.com/news/home/20231212429407/en/
    City of Hope's Ibrahim Aldoss, M.D., presented the research at ASH conference.
  • Tatum Demontmorency , 19, participated in the clinical trial at City of Hope after she was diagnosed with leukemia in 2021.
  • If approved for use by the FDA, the product would be the first therapy to target what’s known as the menin-KMT2A interaction.

Precision BioSciences Announces Approval of First Clinical Trial Application of ARCUS Gene Insertion Program by Partner iECURE

Retrieved on: 
Wednesday, December 13, 2023
Research, Infectious Diseases, Genetics, Clinical Trials, Biotechnology, Health, Pharmaceutical, General Health, Science, Precision BioSciences, Nerve, Gene editing, Ornithine transcarbamylase deficiency, HDR, Gene, Clinical trial, Coma, IND, Liver, Disease, Risk, Patient, Cell, ESGCT, Blood, Hepatocyte, Primate, CRISPR, Ammonia, Death, Neurology, Congress, CTA, OTC, Precision, Metabolic disorder, Vaccine

“The acceptance of iECURE’s Clinical Trial Application marks an important milestone for patients with OTC deficiency and for both the iECURE and Precision BioSciences teams.

Key Points: 
  • “The acceptance of iECURE’s Clinical Trial Application marks an important milestone for patients with OTC deficiency and for both the iECURE and Precision BioSciences teams.
  • This is the first ARCUS in vivo gene editing program to progress into the clinic,” said Michael Amoroso, Chief Executive Officer of Precision Biosciences.
  • A 5% threshold of enzymatic activity has the potential for clinical benefit as previously identified in Annals of Clinical and Translational Neurology .
  • “ECUR-506 is the first of several partnered and wholly owned gene editing programs leveraging ARCUS in differentiated indications and we look forward to initial clinical data from this program.”

New Post Hoc Analysis from MAVORIC Trial Sheds Light on the Burden of Cutaneous T-Cell Lymphoma on Health-Related Quality of Life

Retrieved on: 
Monday, December 11, 2023
Oncology, Health, General Health, Clinical Trials, Research, Science, Pharmaceutical, Well, Mycosis fungoides, Organ, Neuropsychological test, CTCL, Cutaneous T-cell lymphoma, Central Statistics Office (India), Disease, Skin, Eastern Cooperative Oncology Group, Modified, Patient, Itch, Bivariate, Lymph, KKI, Neoplasm, Blood, ECOG, Cancer, ANOVA, Erythroderma, Female, Quality of life, Children's Oncology Group, Nursing, Medical imaging

Understanding how symptoms impact an individual physically, emotionally as well as in their daily life is critical,” said Susan Thornton, Chief Executive Officer, Cutaneous Lymphoma Foundation and one of the study authors.

Key Points: 
  • Understanding how symptoms impact an individual physically, emotionally as well as in their daily life is critical,” said Susan Thornton, Chief Executive Officer, Cutaneous Lymphoma Foundation and one of the study authors.
  • Bivariate analysis (t-tests and ANOVA) was used to identify demographic and medical history variables that had a relationship with HRQL.
  • LASSO (least absolute shrinkage and selection operator) regression analysis was used to identify factors that may drive poor HRQL.
  • In multivariate analysis, worse HRQL was associated with being younger, female, moderate or severe pruritus and impaired function (as measured by ECOG PS).

WHOOP Announces Its “Year In Review” Celebrating That 2023 Was the Year Human Performance Became a Lifestyle

Retrieved on: 
Tuesday, December 12, 2023
Software, General Health, Hardware, Lifestyle, Biometrics, Consumer Electronics, Consumer, Apps, Applications, Technology, Health Technology, Fitness & Nutrition, Wearables, Mobile Technology, Health, Meal, Melatonin, Best Buy, Longevity, Eating, Creatine, Football, Zedd, Amazon (company), Multimedia, RHR, Oxytocin, HRV, Endorphins, Meditation, Blood, Relaxation, Hygiene, Muscle soreness, Fever, Sunlight, Habit, Pharmaceutical industry, Liquor, Vaccine, Hotel, Sleep, Recovery

View the full release here: https://www.businesswire.com/news/home/20231211786571/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20231211786571/en/
    WHOOP announces its “Year In Review” celebrating that 2023 was the year human performance became a lifestyle.
  • WHOOP data shows that even the smallest behavioral changes can have a big impact on the body and mind.
  • “‘Year in Review’ is one of our favorite features to launch each year,” said Will Ahmed, Founder and CEO of WHOOP.
  • Stage performance also increased in 2023, with 19% more WHOOP members logging it as a Strain activity year-over-year.

Corsair Pharma Announces Appointment of George W. Mahaffey as Chief Executive Officer

Retrieved on: 
Monday, December 11, 2023
Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, Medical Devices, Other Health, Safety, Rash, Liver, Patient, Pulmonary hypertension, FDA, Orthostatic hypertension, Corsair, Blood, Pain, PAH, Skin, Physician, Quality of life, Pharma, Pharmaceutical industry

Corsair Pharma, Inc., a private biopharmaceutical company developing a transdermal treprostinil prodrug, today announced the appointment of George W. Mahaffey as Chief Executive Officer.

Key Points: 
  • Corsair Pharma, Inc., a private biopharmaceutical company developing a transdermal treprostinil prodrug, today announced the appointment of George W. Mahaffey as Chief Executive Officer.
  • Mr. Mahaffey brings more than 35 years of pharmaceutical, biotech, device, and diagnostics experience, including having launched Ventavis®, the first FDA-approved inhaled prostacyclin product indicated for pulmonary arterial hypertension (PAH).
  • The Corsair transdermal patch is intended to provide continuous and consistent blood levels of the prostacyclin drug treprostinil comparable to an infusion pump.
  • PAH patients need new options for the delivery of this foundational drug class,” said Mr. Mahaffey.

Geron Announces IMerge Phase 3 Presentations at ASH Highlighting Significant Durability of Transfusion Independence and Breadth of Effect Across MDS Subgroups with Imetelstat in Lower Risk MDS

Retrieved on: 
Monday, December 11, 2023
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The results showed that imetelstat consistently had higher RBC-TI response rates than placebo across these different risk subgroups.

Key Points: 
  • The results showed that imetelstat consistently had higher RBC-TI response rates than placebo across these different risk subgroups.
  • Overall, durable 24-week and 1-year RBC-TI responses were observed with imetelstat in all lower- and higher-risk subgroups.
  • This analysis suggests clinical benefit of imetelstat across different molecularly defined subgroups and independent of the underlying molecular mutation pattern.
  • This analysis indicates that achievement of RBC-TI was associated with improved survival, suggesting that transfusion dependence is a modifiable predictor of clinical outcomes in lower risk MDS.

New Study Finds Significant Correlation Between Masimo ORi™ and Arterial Partial Pressure of Oxygen During One-Lung Ventilation

Retrieved on: 
Monday, December 11, 2023
Biotechnology, Surgery, Health, Pharmaceutical, Health Technology, Odds ratio, Samsung Medical Center, De novo, Blood, OLV, Sungkyunkwan University, Hypoxemia, Risk, MASI, Patient, Multimedia, Journal, FDA, Hyperoxia, Oxygen, Blood gas tension, Perioperative, Ori, Oxygen saturation (medicine), Receiver operating characteristic, Medical device, Medical imaging, Anesthesia, ROC

ORi is trended continuously with SpO2 as a unit-less index between 0.00 and 1.00 to extend the visibility of the patient oxygenation beyond SpO2 under supplemental oxygen.

Key Points: 
  • ORi is trended continuously with SpO2 as a unit-less index between 0.00 and 1.00 to extend the visibility of the patient oxygenation beyond SpO2 under supplemental oxygen.
  • During anesthesia, ORi was monitored using Masimo RD rainbow SET® Pulse CO-Oximetry sensors, and blood gas analysis was performed 15 minutes after OLV was initiated.
  • Of the 11 potential predictors for PaO2
  • The researchers concluded, “ORi values during one-lung ventilation were significantly correlated with PaO2 measured simultaneously.

Bristol Myers Squibb Presents New Data at ASH 2023 Demonstrating Clinical Benefit Across B-cell Malignancies with Breyanzi as a Second-Line Treatment in High-Risk Follicular Lymphoma and in Relapsed or Refractory Chronic Lymphocytic Leukemia

Retrieved on: 
Monday, December 11, 2023
Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, Genetics, Clinical Trials, City, ASH, CRS, Pros, Patient, Probability, Conference, Bristol Myers Squibb, Prognosis, ORR, Quality of life, Disclosure, Safety, Food, SLL, Jungian cognitive functions, DOR, Trial of the century, Blood, Lymphoma, Society, Bone marrow, Neuropsychological test, Haematopoietic system, BMY, Pain, Journal of Clinical Oncology, NYSE, BTK, MCL, Clinical trial, Fatigue, Follicular lymphoma, PRO, Chronic lymphocytic leukemia, BMS, NHL, CLL, HIV disease progression rates, Pharmaceutical industry, Veterinary medicine, Lisocabtagene maraleucel, Hematology

TRANSCEND FL is the largest clinical trial to date to evaluate a CAR T cell therapy in patients with relapsed or refractory indolent non-Hodgkin lymphoma, including FL.

Key Points: 
  • TRANSCEND FL is the largest clinical trial to date to evaluate a CAR T cell therapy in patients with relapsed or refractory indolent non-Hodgkin lymphoma, including FL.
  • Additionally, the company presented long-term data from the TRANSCEND CLL 004 study evaluating Breyanzi in patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic leukemia (SLL) with 24-month follow-up ( Oral Presentation #330 ).
  • Bristol Myers Squibb (BMS) has the broadest ongoing cell therapy development program in CD19-positive malignancies with Breyanzi, which also includes trials investigating its use in patients with relapsed/refractory mantle cell lymphoma (MCL).
  • In the TRANSCEND FL clinical trial, 130 patients with relapsed or refractory follicular lymphoma (FL) were enrolled and treated with Breyanzi in the second-line and third-line plus settings.