Hematopathology

Donald S. Karcher, MD, FCAP, elected 38th President of the College of American Pathologists

Retrieved on: 
Saturday, October 7, 2023

Donald S. Karcher, MD, FCAP, is the newly elected President of the College of American Pathologists (CAP).

Key Points: 
  • Donald S. Karcher, MD, FCAP, is the newly elected President of the College of American Pathologists (CAP).
  • At a meeting of the membership on Saturday, October 7, 2023, Dr. Karcher was inaugurated as the organization’s 38th President.
  • “As pathologists we all share the same challenges, celebrate the same victories, and deal with the same defeats.
  • Another goal is to assure pathologists are increasingly visible to the healthcare system and to their patients.

CHOP Researchers Improve Fitness of Cells Used in Cell Transplants

Retrieved on: 
Wednesday, September 27, 2023

PHILADELPHIA, Sept. 27, 2023 /PRNewswire/ -- A readily available, inexpensive small molecule drug can improve the fitness of hematopoietic stem and progenitor cells (HSPCs) that are modified outside of the body, potentially improving the success of procedures like ex vivo gene therapy, according to a new study by researchers at Children's Hospital of Philadelphia (CHOP). The study, published in the journal Blood, showed that targeting components in the cells called extracellular vesicles (EVs) relieves the stress on cells when outside of the body, improving their performance when they are transplanted back inside.

Key Points: 
  • Hematopoietic stem cell transplantation (HSCT) involves the transfer of HSPCs from a donor to patients with both benign and malignant diseases.
  • However, maintaining the fitness of these cells outside of the body poses a challenge, and any loss of fitness introduces the risk that the cells will not properly restore the blood and immune cell system in patients.
  • One process that we now learn contributes to cell equilibrium is the secretion of EVs, which are important for delivering messages from cell to cell and maintaining proper cell function.
  • "Neutral sphingomyelinase blockade enhances hematopoietic stem cell fitness through an integrated stress response," Blood, online Sept. 12, 2023, DOI: 10.1182/blood.2023022147

Hemanext Partners with Sickle Cell 101 to Sponsor Sickle Cell Education

Retrieved on: 
Wednesday, July 28, 2021

and SAN FRANCISCO, July 28, 2021 /PRNewswire/ --Hemanext Inc.a privately held,US-based medical technology company dedicated to improving the quality, safety, efficacy, and cost of red blood cell (RBC) transfusions, has partnered with Sickle Cell 101 (SC101), a Silicon Valley-based non-profit, to sponsor and promote sickle cell disease (SCD) education.

Key Points: 
  • and SAN FRANCISCO, July 28, 2021 /PRNewswire/ --Hemanext Inc.a privately held,US-based medical technology company dedicated to improving the quality, safety, efficacy, and cost of red blood cell (RBC) transfusions, has partnered with Sickle Cell 101 (SC101), a Silicon Valley-based non-profit, to sponsor and promote sickle cell disease (SCD) education.
  • "It is a privilege to partner with Sickle Cell 101 and in support of their global mission of promoting patient awareness and education for sickle cell disease," said Alex Marichal, VP of Marketing at Hemanext.
  • "SC101's evidence-based, patient-friendly programs and communications enhance the lives of sickle cell patients, which is why Hemanext is committed to supporting this alliance."
  • SC101 is the largest global online platform dedicated solely to sickle cell disease and sickle cell trait research and education.

Hemanext Partners with Sickle Cell 101 to Sponsor Sickle Cell Education

Retrieved on: 
Wednesday, July 28, 2021

and SAN FRANCISCO, July 28, 2021 /PRNewswire/ --Hemanext Inc.a privately held,US-based medical technology company dedicated to improving the quality, safety, efficacy, and cost of red blood cell (RBC) transfusions, has partnered with Sickle Cell 101 (SC101), a Silicon Valley-based non-profit, to sponsor and promote sickle cell disease (SCD) education.

Key Points: 
  • and SAN FRANCISCO, July 28, 2021 /PRNewswire/ --Hemanext Inc.a privately held,US-based medical technology company dedicated to improving the quality, safety, efficacy, and cost of red blood cell (RBC) transfusions, has partnered with Sickle Cell 101 (SC101), a Silicon Valley-based non-profit, to sponsor and promote sickle cell disease (SCD) education.
  • "It is a privilege to partner with Sickle Cell 101 and in support of their global mission of promoting patient awareness and education for sickle cell disease," said Alex Marichal, VP of Marketing at Hemanext.
  • "SC101's evidence-based, patient-friendly programs and communications enhance the lives of sickle cell patients, which is why Hemanext is committed to supporting this alliance."
  • SC101 is the largest global online platform dedicated solely to sickle cell disease and sickle cell trait research and education.

GBT Provides Regulatory and Pipeline Updates in Sickle Cell Disease (SCD)

Retrieved on: 
Thursday, July 22, 2021

SOUTH SAN FRANCISCO, Calif., July 22, 2021 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced updates across several of its development programs in sickle cell disease (SCD). The company has submitted a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for Oxbryta® (voxelotor) for the treatment of SCD in children ages 4 to 11 years, together with a related separate New Drug Application (NDA) required to seek approval for a pediatric weight-based formulation of Oxbryta. Additionally, GBT initiated two global, randomized, placebo-controlled, pivotal Phase 3 clinical trials of inclacumab, a novel P-selectin inhibitor. The company also enrolled the first SCD patient in a Phase 1 study evaluating GBT021601 (GBT601), a next-generation hemoglobin S (HbS) polymerization inhibitor, in people with SCD.

Key Points: 
  • GBT requested priority review for the sNDA and NDA, which, if granted, could result in a six-month review process.
  • Our pipeline is the engine of our commitment to improving the lives of people living with this terrible disease.
  • We are excited to initiate enrollment in our two pivotal studies for inclacumab, in parallel with our Phase 1 study of GBT601 in people with sickle cell disease.
  • Founded in 2011, GBT is delivering on its goal to transform the treatment and care of sickle cell disease (SCD), a lifelong, devastating inherited blood disorder.

City of Atlanta and Gwinnet County Designate July 10-17 as "Cheek Week" To Help Raise Awareness of the Need for More Black and African American Donors on the Be The Match Registry

Retrieved on: 
Monday, July 12, 2021

Black patients are least likely to find a matching donor on the Be The Match Registry than any other community.

Key Points: 
  • Black patients are least likely to find a matching donor on the Be The Match Registry than any other community.
  • More Black donors are urgently needed to help cure patients."
  • Fourteen-year-old Joshua of Lawrenceville, Ga. had his first pain crisis from sickle cell disease when he was just 5 months old.
  • People can contribute to the cure as a member of the Be The Match Registry, financial contributor or volunteer.

GBT Announces New Employment Inducement Grants

Retrieved on: 
Friday, July 9, 2021

These awards were made under GBTs Amended and Restated 2017 Inducement Equity Plan (the Plan).

Key Points: 
  • These awards were made under GBTs Amended and Restated 2017 Inducement Equity Plan (the Plan).
  • The above-described awards were each granted as an inducement material to the employees entering into employment with the company in accordance with NASDAQ Listing Rule 5635(c)(4), and were granted pursuant to the terms of the Plan.
  • The Plan was adopted by GBTs board of directors in January 2017 and has been amended and restated from time to time.
  • Founded in 2011, GBT is delivering on its goal to transform the treatment and care of sickle cell disease (SCD), a lifelong, devastating inherited blood disorder.

Survey Finds People with Sickle Cell Disease Report Vastly Different Experiences with HCPs, Emergency Departments

Retrieved on: 
Thursday, July 1, 2021

The inaugural Sickle Cell Disease In America survey illuminates the perspectives and experiences of people living with sickle cell disease.

Key Points: 
  • The inaugural Sickle Cell Disease In America survey illuminates the perspectives and experiences of people living with sickle cell disease.
  • In fact, 92% of Sickle Cell Disease In America respondents said they see a hematologist for their sickle cell disease, while eight in 10 respondents said a hematologist is the primary HCP managing their condition.
  • As a result of these experiences, people with sickle cell disease might end up avoiding going to the emergency department, making access to care increasingly difficult.
  • The Sickle Cell Disease In America survey, which fielded from Oct. 19, 2020 to Feb. 26, 2021, included responses from 111 people living with sickle cell disease.

Agios Submits Marketing Authorisation Application to European Medicines Agency for Mitapivat for Treatment of Adults with Pyruvate Kinase Deficiency

Retrieved on: 
Monday, June 28, 2021

A full analysis of these data including patient-reported outcomes (PRO) was recently presented at theEuropean Hematology Association (EHA) Virtual Congress.

Key Points: 
  • A full analysis of these data including patient-reported outcomes (PRO) was recently presented at theEuropean Hematology Association (EHA) Virtual Congress.
  • An extension study for adults with PK deficiency previously enrolled in ACTIVATE or ACTIVATE-T is ongoing and designed to evaluate the long-term safety, tolerability and efficacy of treatment with mitapivat.
  • Pyruvate kinase (PK) deficiency is a rare, inherited disease that presents as chronic hemolytic anemia, which is the accelerated destruction of red blood cells.
  • The companys most advanced drug candidate is a first-in-class pyruvate kinase R (PKR) activator, mitapivat, that is currently being evaluated for the treatment of three distinct hemolytic anemias.

Networking California for Sickle Cell Care Initiative Launches "Neglected No More"

Retrieved on: 
Wednesday, June 23, 2021

"This program is only the start of addressing decades of neglect," said Jennifer Fields, an adult living with sickle cell disease and implementation strategist of the Sickle Cell Disease Foundation.

Key Points: 
  • "This program is only the start of addressing decades of neglect," said Jennifer Fields, an adult living with sickle cell disease and implementation strategist of the Sickle Cell Disease Foundation.
  • In late June, Governor Newsom, committed $15 million for adult sickle cell treatment programs statewide and granted funds to CIBD to create the NCSCC in partnership with the Sickle Cell Disease Foundation , the nation's oldest sickle cell disease nonprofit and patient support agency based in San Bernardino County.
  • The mission of the Sickle Cell Disease Foundation (SCDF) is to provide life-enhancing education, services, and programs for individuals living with sickle cell disease.
  • The Networking California for Sickle Cell Care Initiative (NCSCC) was developed through advocacy and stakeholder engagement supported by data.