VCU School of Medicine

AskBio Announces First Patient Dosed in Phase 1 / Phase 2 Trial of AB-1003 Gene Therapy for Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)

Retrieved on: 
Donnerstag, August 3, 2023

Gene therapy developed to restore FKRP enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9.

Key Points: 
  • Gene therapy developed to restore FKRP enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9.
  • "Hearing that the first patient has been dosed in this study evaluating AB-1003 is an exciting moment for the limb-girdle muscular dystrophy community and individuals living with this debilitating disease," said Kelly Brazzo, Co-Founder & CEO, CureLGMD2i.
  • "Given the current lack of disease modifying treatments for LGMD, many in the community know of the potential of gene therapy.
  • The initiation of this trial offers hope that patients with this condition may, in the future, have a significantly improved quality of life."

New Study Uncovers Unprecedented Links Between Liver and Heart Health and the Importance of Doctors' Prioritizing Liver Health to Promote Heart Health

Retrieved on: 
Montag, Juni 26, 2023

Highlighting the relationship between liver disease, arrhythmia and heart failure may change this, experts hope.

Key Points: 
  • Highlighting the relationship between liver disease, arrhythmia and heart failure may change this, experts hope.
  • And those with clinically significant liver disease are 38% more likely to be hospitalized for a major adverse cardiovascular event.
  • “This study is making us re-consider the impact liver disease has on the human body and its relationship with heart-related conditions.
  • This study tells them: if you want to look after your heart, start with your liver.”

Validation of a Pragmatic Clinical Risk-Based Classification of NASH - New Supporting Real World Evidence from TARGET-NASH

Retrieved on: 
Donnerstag, März 30, 2023

"Validation of a Clinical Risk-Based Classification System in a Large Nonalcoholic Fatty Liver Disease Real-world Cohort," was published in the peer-reviewed publication Clinical Gastroenterology and Hepatology .

Key Points: 
  • "Validation of a Clinical Risk-Based Classification System in a Large Nonalcoholic Fatty Liver Disease Real-world Cohort," was published in the peer-reviewed publication Clinical Gastroenterology and Hepatology .
  • More than 20% of the US population is affected by NAFLD, which represents a major and growing public health concern with significant health economic implications.
  • While there are no approved curative therapies to date, there are multiple new therapies currently under review with the FDA.
  • Identifying patient populations in the real world who will need these upcoming treatments will be critical when these novel treatments become available.

PaxMedica Holds Meeting of Scientific Advisory Board

Retrieved on: 
Montag, März 20, 2023

TARRYTOWN, NY, March 20, 2023 (GLOBE NEWSWIRE) -- via NewMediaWire – PaxMedica, Inc. (Nasdaq: PXMD), a clinical stage biopharmaceutical company focusing on the development of novel anti-purinergic drug therapies (“APT”) for the treatment of disorders with intractable neurologic symptoms, today announced that it held a Scientific Advisory Board meeting on March 10-12, 2023.  A number of new members have been added to the board, which consists of many key opinion leaders in autism spectrum disorder (ASD) in the United States including:

Key Points: 
  • Board Includes Several Key Opinion Leaders on Autism Spectrum Disorder in U.S.
    TARRYTOWN, NY, March 20, 2023 (GLOBE NEWSWIRE) -- via NewMediaWire – PaxMedica, Inc. (Nasdaq: PXMD), a clinical stage biopharmaceutical company focusing on the development of novel anti-purinergic drug therapies (“APT”) for the treatment of disorders with intractable neurologic symptoms, today announced that it held a Scientific Advisory Board meeting on March 10-12, 2023.
  • We continue to be very excited about studying a product for the potential treatment of the core symptoms of autism spectrum disorder.”
    Dr. Robert Naviaux added, “At the scientific advisory board meeting this weekend, experts from many different areas of medicine came together to share their thoughts on how suramin may work in ASD and how it might work in several other complex disorders.
  • What emerged was a clear vision for the next steps needed to test suramin in autism.
  • This is expected to be done in a series of international, multicenter, clinical trials that are projected to begin in 2024.”

Aspiration Partners with Trees for the Future to Promote Reforestation and Sustainable Agriculture in Kenya

Retrieved on: 
Mittwoch, Februar 15, 2023

LOS ANGELES, Feb. 15, 2023 /PRNewswire/ -- Aspiration, a high-impact climate finance company, has announced a $21 million investment in carbon projects through Trees for the Future (TREES), a non-profit organization training farmers in sustainable agroforestry practices across sub-Saharan Africa. This partnership will enable TREES to provide necessary support and training to 15,000 farmers in and around Lake Victoria Watershed in western Kenya to plant 87 million trees, effectively creating 15,000 hectares of regenerative agroforestry land. The restoration effort will generate 4.13 million Verified Carbon Units (VCUs) and help farmers adapt to the impacts of climate.

Key Points: 
  • The restoration effort will generate 4.13 million Verified Carbon Units (VCUs) and help farmers adapt to the impacts of climate.
  • Aspiration will provide a portion of the profits from the sale of the VCUs back to TREES and the farmers involved in the projects.
  • "We are proud to partner with Trees for the Future, a proven leader in promoting reforestation and sustainable agriculture."
  • Since its founding in 1989, TREES has planted over 300 million trees with farming communities around the world.

INmune Bio, Inc., Virginia Commonwealth University, and Barrow Neurological Institute Receive Department of Defense Funding to Study the Role of Traumatic Brain Injury in the Development of Alzheimer’s Disease

Retrieved on: 
Donnerstag, Dezember 22, 2022

Boca Raton, Florida, Dec. 22, 2022 (GLOBE NEWSWIRE) --  INmune Bio, Inc. (NASDAQ: INMB) (the “Company”), a clinical-stage immunology company focused on developing treatments that harness the patient’s innate immune system to fight disease in concert with Virgina Commonwealth University (VCU) and Barrow Neurological Institute have received a grant from the Department of Defense (DoD). The grant is titled: Novel solTNF inhibitor improves outcomes in a mouse model of TBI-induced AD. The funding covers a collaboration that studies the impact of traumatic brain injury (TBI) in the development of Alzheimer’s disease (AD) related pathology. These DoD funded studies are a collaboration between Dr. Kirsty Dixon, Associate Professor of Surgery and director of Surgery’s Neurotrauma Repair Laboratory at Virginia Commonwealth University, and Dr. Elliott Mufson, Professor of Neurobiology and the director of the Alzheimer’s disease research laboratory at Barrow Neurological Institute and INmune Bio.

Key Points: 
  • The grant is titled: Novel solTNF inhibitor improves outcomes in a mouse model of TBI-induced AD.
  • The funding covers a collaboration that studies the impact of traumatic brain injury (TBI) in the development of Alzheimer’s disease (AD) related pathology.
  • “This is a great example of collaboration for innovation,” said RJ Tesi MD, CEO of INmune.
  • "This study is an important step toward helping us better understand the relationship between these disorders and to develop effective therapies."

Nonalcoholic Fatty Liver Disease (NAFLD) Research from Target RWE's Real-World Study Presented at International Liver Congress 2022

Retrieved on: 
Freitag, Juni 24, 2022

DURHAM, N.C., June 24, 2022 /PRNewswire/ -- New TARGET-NASH data shows longitudinal trajectories of alanine aminotransferase (ALT) remained comparatively stable among patients with nonalcoholic fatty liver disease (NAFLD) in the latest real-world study by Target RWE .

Key Points: 
  • DURHAM, N.C., June 24, 2022 /PRNewswire/ -- New TARGET-NASH data shows longitudinal trajectories of alanine aminotransferase (ALT) remained comparatively stable among patients with nonalcoholic fatty liver disease (NAFLD) in the latest real-world study by Target RWE .
  • The TARGET-NASH real world study analyzed 3,600+ adult patients with NAFLD in the U.S. with 3 or more ALT measures.
  • "Researchers have not been able to keep pace with the rapidly growing disease burden of NAFLD that is increasing concern from a public health standpoint.
  • Dr. Sanyal is also co-chair of the TARGET-NASH steering committee and is a co-author of the study.

National Comprehensive Cancer Network Honors Oncology Leaders Promoting Progress in Cancer Care

Retrieved on: 
Montag, Mai 23, 2022

PLYMOUTH MEETING, Pa., May 23, 2022 /PRNewswire/ -- The National Comprehensive Cancer Network® (NCCN®) today announced the recipients of 2022 awards for both internal and external champions advancing NCCN's mission and helping all people with cancer to live better lives. The annual awards include the Rodger Winn Award for expert judgment and commitment to excellence in service of the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®), the Partners in Cancer Care Award for people outside of NCCN Member Institutions who are instrumental in helping NCCN achieve its mission, and the Pat Daulerio Employee of the Year Award honoring dedicated staff members who particularly exemplify the core values of NCCN.

Key Points: 
  • PLYMOUTH MEETING, Pa., May 23, 2022 /PRNewswire/ -- The National Comprehensive Cancer Network ( NCCN ) today announced the recipients of 2022 awards for both internal and external champions advancing NCCN's mission and helping all people with cancer to live better lives.
  • The two co-chaired the Elevating Cancer Equity Working Group, which was convened by NCCN, the American Cancer Society Cancer Action Network (ACS CAN), and the National Minority Quality Forum (NMQF) to identify actionable strategies for overcoming inequity in oncology.
  • "Their visionary leadership has contributed to a sea change in the national conversation and spurred action to address racial disparities in cancer care.
  • The National Comprehensive Cancer Network ( NCCN ) is a not-for-profit alliance of leading cancer centers devoted to patient care, research, and education.

Black patients more likely to be excluded from pancreatic cancer clinical trials

Retrieved on: 
Dienstag, März 29, 2022

Looking at pancreatic cancer patients treated between 2010 and 2019, the researchers found that 42% of Black patients were ineligible for clinical trials compared to 33% of white patients.

Key Points: 
  • Looking at pancreatic cancer patients treated between 2010 and 2019, the researchers found that 42% of Black patients were ineligible for clinical trials compared to 33% of white patients.
  • Historically, Black, Asian or Pacific Islander, American Indian or Alaskan Native and Hispanic patients have been underrepresented in pancreatic cancer clinical trials.
  • Black patients tend to be diagnosed with pancreatic cancer at a younger age than white patients and therefore are less likely to be covered by Medicare.
  • Although this study looked at pancreatic cancer patients, the researchers believe their findings are likely applicable to clinical trials for other cancer types as they reflect common barriers that are not specific to pancreatic cancer.

Southeastern cancer centers fight racial inequities in lung cancer

Retrieved on: 
Dienstag, März 15, 2022

Community navigators within the FQHCs and nurse and financial navigators within the cancer centers will guide patients through lung cancer screening by providing education and support.

Key Points: 
  • Community navigators within the FQHCs and nurse and financial navigators within the cancer centers will guide patients through lung cancer screening by providing education and support.
  • Lung cancer screening has the potential to reduce lung cancer mortality and close the disparities gap, yet initial reports indicate low uptake of screening, said Louise Henderson, Ph.D. , the teams principal investigator and program leader of cancer epidemiology at UNC Lineberger.
  • MUSC Hollings Cancer Center is a National Cancer Institute-designated cancer center and the largest academic-based cancer research program in South Carolina.
  • It is one of only 52 National Cancer Institute-designated comprehensive cancer centers and the only public comprehensive cancer center serving the state and people of North Carolina.