Charting the Course of NASH

Charting the Course of NASH

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International NASH Day was a success!

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The 6th International NASH Day was a resounding global success, with over 100 partners from 55 countries participating. Our hashtag #NASHDay reached over 10 million impressions and engaged with millions of unique social media accounts. Additionally, our media advisories and news releases reached thousands of journalists worldwide, potentially reaching billions of people. NASH Day 2023 received endorsements from 30 medical specialty societies across the globe, including research institutions, community organizations, and medical centers. Thank you to all our partners, endorsers, and sponsors who chose to Step Up For NASH and helped make the 6th Annual International NASH Day possible.


 

GLI Addresses Need for NAFLD/NASH Nomenclature Implementation Plan

GLI has initiated efforts to assist the healthcare community in planning for the implementation of the recent change in nomenclature for NAFLD, announced at the International Liver Congress. GLI will continue to use the terms NAFLD and NASH until September 5, 2023 at the earliest, when we will announce a comprehensive transition plan in consultation with stakeholders and experts in various fields, affecting program names, educational materials, media resources, websites, and more. Input and questions can be directed to NASH@globalliver.org.

Lived Experiences – Challenges on the Ground & Patient Perspectives

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In a thought-provoking presentation held at this year’s EASL Congress in Vienna, Austria, Jeff McIntyre, VP of Liver Health Programs at GLI, contributed valuable insights on the topic of steering blame away from patients and individuals towards systemic policies that reinforce and encourage unhealthy behaviors. Collaborating with fellow experts, Jeff shed light on the actions necessary to address this issue. By emphasizing the need to redirect responsibility from individuals to the underlying systemic factors, the panel explored crucial steps for creating a healthier society.

American Diabetes Association Releases a Guideline Update in NAFLD and Diabetes

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The American Diabetes Association (ADA) has published updated guidelines in the Standards of Care in Diabetes—2023, highlighting the detection and management recommendations for NAFLD/NASH in individuals with diabetes. The guidelines emphasize the significant impact of liver disease on people with type 2 diabetes, its association with serious complications, and the importance of early detection and appropriate management. These updates aim to provide healthcare professionals with the most current information to effectively address the connection between diabetes and liver disease.


 

Upcoming events:

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A3: The Learning Experience will be held in Washington DC from September 30 – October 2, 2023! 

If you are a liver patient, caregiver, family member, or clinician searching for an opportunity to become an effective advocate through interactive lessons on how to share your story, understand recent research & development, how to leverage social media and more.

✍🏼 Apply now


 

To learn more about the NASH Council or become a member, please visit https://globalliver.org/nash-council/ or reach out to NASHday@globalliver.org.

 

Noteworthy Progress in Patient Advocacy & Clinical Trials

Noteworthy Progress in Patient Advocacy & Clinical Trials

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Actively supporting advocacy efforts and engaging in novel clinical trials can provide patients with a sense of hope for potential therapies and assurance in the management of their condition. Read below about advancements made in June through clinical trials and events focused on inclusivity, equity, and advocacy for patients with rare liver disease.


GLI attends the PBC Foundation Summit, Edinburgh, Scotland

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GLI participated in the PBC Foundation Summit, which served as an opportunity to delve into various aspects of PBC, including its impact on patient’s lives, advancements in research and treatment, and the significance of raising awareness and providing support. A few takeaways: 

  • Early diagnosis and intervention are imperative;
  • Patients should be empowered through education and support systems;
  • Research funding must be increased;
  • Collaboration between healthcare professionals, researchers, and patient communities should be enhanced.

New Treatment Option for Cholestatic Pruritus in Alagille Syndrome Announced

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With a greater demand for pruritus relief, patients diagnosed with Alagille syndrome and PFIC in the US welcome a new medication that recently received FDA approval for the treatment of cholestatic pruritus. Developed by Ipsen, a member of GLI’s Pediatric and Rare Liver Disease Council,  Bylvay is now available by prescription. This news widens the array of treatment options, offering patients a chance to explore and determine which medication aligns best with their situation.

GLI Acknowledges the Achievements of Mirum and CANbridge in Pioneering a Global and Inclusive Clinical Trial Process for LIVMARLI

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GLI commends the contributions made by Mirum and its collaborators in spearheading a comprehensive clinical trial process that prioritizes global accessibility and inclusivity for LIVMARLI, a drug developed for the treatment of cholestatic pruritus associated with Alagille Syndrome and PFIC. The substantial level of participation observed in this clinical trial for biliary atresia reflects a strong desire to find a cure.

GLI Commends Wilson’s Disease Association for Advocating for Affordability of Trientine Hydrochloride, a Generic Drug for Wilson’s Disease

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Wilson’s Disease Association, a member of GLI’s Pediatric & Rare Diseases Council, in collaboration with the Mark Cuban Cost Plus Drug Company and Dr. Reddy’s Laboratories, has issued a formal statement emphasizing the significance of affordable pharmaceuticals for rare liver diseases. This statement specifically highlights the advancements and criticality of generic alternatives, such as trientine hydrochloride, as a substitute for Syprine. Cost-effective options will be made available for patients following this collaboration.


Upcoming Events

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A3: The Learning Experience will be held in Washington DC from September 30 – October 2, 2023! 

If you are a liver patient, caregiver, family member, or clinician searching for an opportunity to become an effective advocate through interactive lessons on how to share your story, understand recent research & development, how to leverage social media and more.

✍🏼Apply now


For more information about the Pediatric and Rare Liver Diseases Council or to learn more about joining, please visit https://globalliver.org/pediatric-rare-liver-diseases-council/ or email pedsrare@globalliver.org.

Discoveries in HCC and Biliary Tract Cancer

Discoveries in HCC and Biliary Tract Cancer

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It’s not only important to GLI to keep you updated; we value your input and updates. This is a reminder to Liver Cancers Council members and supporters to share news to be featured in our monthly newsletter or on our Liver Central website.


Innovating and Advancing Research in Liver Cancers: A Webinar

On June 27, GLI held a discussion of how the field is responding to the needs and challenges of those living with liver cancers. The Webinar focused on three topics: reducing disparities in clinical trials, liver cancers screening options and innovation, and liver cancers research updates. Sarah Manes presented the virtual event with Dr. Lionel Fonkua (Mayo Clinic), Dr. Neehar Parikh (University of Michigan), Dr. Amit Singal (UT Southwestern), Dr. Ahmed Kaseb (MD Anderson; NCI SPORE), Dr. Mark McNiven (Mayo Clinic; NCI Spore), and Dr. Bruno Sangro (Clínica Universidad de Navarra) as key speakers. Read more about the webinar or watch it today!

STAT2 Expression is Highest in African American and Asian Patients with HCC

STAT2 is a gene known to regulate the formation of cancer cells. The expression of STAT2 is typically higher in patients with hepatocellular carcinoma (HCC), but expression increases at greater rates among Asian and African American patients compared with Caucasian patients. These findings were from a study presented at the 5th Annual Regional Synergistic Partnership for Enhancing Equity in Cancer Health (SPEECH) Conference and Retreat in Philadelphia, Pennsylvania. It was also found that STAT2 expression is higher among female patients. The results were deemed significant, which emphasizes that there needs to be appropriate inclusion of African American and Asian patients in clinical trials. Read more about the study.

FDA Accepts Application for Merck’s KEYTRUDA (pembrolizumab) for Advanced Biliary Tract Cancer

KEYTRUDA is an anti-programmed death receptor-1 (PD-1) therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. This treatment in injection form, in addition to chemotherapy, has been approved by the FDA to treat advanced biliary tract cancer, or cancer in the gallbladder and bile ducts of the liver. The Phase 3 clinical trial showed a significant overall survival benefit in patients. Merck is currently evaluating the drug in gastrointestinal, hepatobiliary, esophageal, pancreatic, colorectal and gastric cancers. Read more about the treatment and the trial.

Applications of AI in Liver Transplants with HCC

Liver transplantation is a curative treatment option where it solves the chronic underlying liver disorder while removing the malignant lesion. Artificial intelligence (AI) has many applications in medicine that work with medical imaging, and the use of AI to aid evaluations in liver transplantation candidates with hepatocellular carcinoma (HCC) would help automate laborious tasks. Such uses for AI include a detection tool to monitor patients on the liver transplant list and new lesion imaging criteria based on pixel-level analysis. Read more.


Upcoming Events:

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A3: The Learning Experience will be held in Washington DC from September 30 – October 2, 2023! 

If you are a liver patient, caregiver, family member, or a clinician searching for an opportunity to become an effective advocate through interactive lessons on how to share your story, understand recent research & development, how to leverage social media, and more.

✍🏼Apply now


For more information about the Liver Cancers Council or to learn more about joining, please visit https://globalliver.org/liver-cancers-council/ or email cancer@globalliver.org.

GLI Leads Bipartisan Access to Innovation Strategy Across HHS, Congress, and the White House

GLI Leads Bipartisan Access to Innovation Strategy Across HHS, Congress, and the White House

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In June, we celebrated International NASH Day and spent the month pushing for policymakers to address regulatory and legislative hurdles facing patient access to high-quality care. We worked to raise awareness that liver health improves when people are able to access preventive services, screenings and diagnostic tests that allow for early detection of disease; when our organ donation and procurement processes operate in a manner that supports both donors and recipients; and when hurdles are not placed in front of access to high-quality treatment for conditions such as NASH, viral hepatitis and obesity. We have so much more to do to support liver health and appreciate your consistent engagement and support in those efforts!


 

Global Liver Institute Weighs in on Legal and Regulatory Priorities Impacting Access to Care and Innovation

GLI joined the HIV+Hepatitis Policy Institute, along with 24 other HIV and hepatitis organizations, in filing an amicus brief in support of the U.S. government in Braidwood Management v. Becerra, the challenge to the ACA’s preventive services coverage requirement before the U.S Court of Appeals for the Fifth Circuit.  The brief emphasizes the important role of testing for HIV and hepatitis to connect people to life-saving medications and, in the case of hepatitis C, curative treatment. People should be allowed to know if they have an infectious disease. Thankfully, the Fifth Circuit Court of Appeals granted a partial stay pending this appeal, protecting free preventive care for 150 million Americans as the case moves through the court.

GLI responded to the FDA Request for Input on the Voluntary Use of Patient Preference Information (PPI) in Regulatory Submissions. Patient perspectives are lacking in research and policy discussions, particularly related to the differences among patients that are under-represented in the data. We appreciate that the FDA is prioritizing efforts to address such challenges and promote innovations that meet patient needs. Learn more.


 

Global Liver Institute Supports Legislation to Bolster Access to High-Quality Care

GLI supports the Nancy Gardner Sewell Medicare Multi-Cancer Early Detection Screening Coverage Act (H.R. 2407) / Medicare Multi-Cancer Early Detection Screening Coverage Act (S. 2085), joining a letter from 500+ organizations calling for its passage. These multi-cancer early detection (MCED) tests utilize advances in genomic science and computing power to, through a simple blood draw, find cancer before it spreads throughout the body. This legislation will ensure that Medicare beneficiaries, and their health care providers, have access to these transformative new early detection tests.

GLI supports the Saving Access to Laboratory Services Act, known as SALSA (H.R. 2377  and S. 1000), which is legislation ensuring coverage of screening and diagnostic tests performed by clinical laboratories that inform some of life’s most important health care decisions. GLI joined 36 other patient groups on a letter urging Congress to protect access to essential laboratory tests. Reach out to your legislators today to cosponsor this important legislation and avoid payment cuts for about 800 tests set for January 2024.

GLI still needs your help on robust support for H.R. 2544 to modernize and reform the U.S. Organ Procurement and Transplantation Network (OPTN). This bipartisan legislation responds to the urgent need for reforming the OPTN by breaking up the national monopoly contract so that the Health Resources Services Administration (HRSA) can run an open, competitive process and thus fixing this broken system. The bill succeeded in being voted affirmatively out of the Energy and Commerce Committee and is waiting for a final vote by the full House of Representatives. Reach out to your legislators today to support the bill!

GLI still needs your help to convince Congress to advance the Living Donor Protection Act (H.R. 2923). This bipartisan legislation was introduced to protect living organ donors and remove barriers to greater donation, and with your help, has garnered co-sponsorship from over 100 Members of Congress. The bill addresses tactics to deny or limit coverage by insurers, family and medical leave for donors, and public awareness. Contact your Member of Congress through the American Association of Kidney Patients Action Center.

GLI supports the Hepatitis C Elimination Program. GLI joined over 120 stakeholder organizations on a letter to Congressional leadership urging them to leverage the roadmap provided by the White House plan to eliminate hepatitis C to develop legislation to provide the resources needed to save the lives of the almost 2.4 million Americans living with this virus. The White House, led by former NIH Director Dr. Francis Collins, has made eliminating hepatitis C a top policy priority. The next step is for Congress to develop the legislative text to advance the new program providing increased access to hepatitis C treatments.


Past Events

GLI joined the American Association of Gastroenterologists at a June 7, 2023 Obesity Day briefing for Congressional staff on improving access to obesity care and treatment, shedding light on the close connection between obesity and NASH. Representative Brad Wenstrup, DPM, R-OH, introduced the event with comments on how essential it is to treat obesity and to advance the Treat and Reduce Obesity Act (TROA), legislation that would expand Medicare coverage to include the screening and treatment of obesity provided by a wide range of health care specialists and would also cover FDA-approved medications for chronic weight management.


 

Upcoming Events 

A3 Save The Date

A3 will be held in Washington DC from September 30 – October 2, 2023! 

If you are a liver patient, caregiver, family member, or a clinician searching for an opportunity to become an effective advocate through interactive lessons on how to share your story, understand recent research & development, how to leverage social media, and more.

✍🏼Apply now: https://globalliver.org/advanced-advocacy-academy/apply/

GLI provided comments to FDA on the importance of patient preference information (PPI)

GLI provided comments to FDA on the importance of patient preference information (PPI)

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GLI provided comments to FDA on the importance of patient preference information (PPI)

 

Global Liver Institute provided comments to the U.S. Food and Drug Administration (FDA) on the importance of patient preference information (PPI). As described by the FDA, PPI captures the value patients place on features of medical devices. Well-designed and well-conducted PPI studies elicit which attributes are important to patients, how important they are, and what trade-offs patients are willing to make. 

GLI founder and CEO, Donna R. Cryer, JD stated, “When I founded Global Liver Institute, it was clear to me that patient perspectives were lacking in research and policy discussions, particularly related to the differences among patients that are under-represented in the data. We appreciate that the FDA is prioritizing efforts to address such challenges…We look forward to continuing to play a role in the development of revised guidance on PPI to ensure that the patient voice is genuinely reflected in the attributes identified and incorporated into clinical trials.”

Link to letter

 

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About Global Liver Institute

Global Liver Institute (GLI) was built to solve the problems that matter to liver patients, equipping advocates to improve the lives of individuals and families impacted by liver disease. GLI promotes innovation, encourages collaboration, and supports the scaling of optimal approaches to help eradicate liver diseases. GLI believes liver health must take its place on the global public health agenda commensurate with the prevalence and impact of liver illness. GLI is the only patient-created, patient-driven nonprofit organization tackling liver health and all liver disease holistically, operating globally. Follow GLI on Facebook, Instagram, LinkedIn, and YouTube.

GLI provided comments to FDA on the importance of patient preference information (PPI)

GLI to Address Need for NAFLD/NASH Nomenclature Implementation Plan

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GLI to Address Need for NAFLD/NASH Nomenclature Implementation Plan

 

Since the joint medical society announcement of the change in nomenclature for NAFLD at the recent International Liver Congress in Vienna, Austria, was accompanied by explanation of the process but no plan for implementation, Global Liver Institute, convener of the 80-member GLI NASH Council and International NASH Day, has begun to assist the hepatology and larger healthcare and health policy communities in thinking through the myriad elements that will need to be adjusted to accommodate this change and calculate the resources needed to ameliorate the impact.

GLI will continue to use the terms NAFLD and NASH, unless necessary for clarity, until at least September 5, 2023, at which time we will announce a coherent transition plan produced in consultation with our stakeholders and experts in brand management, global communications, policy, and business best practices.

Here are just a few of the items affected:

    • Program names
    • Patient/caregiver education material
    • Provider education materials
    • Media kits
    • Social media graphics and infographics
    • Educational videos
    • Translations of materials
    • Websites
    • Social media accounts
    • Logos
    • Legislative language
    • Medical coding
    • Insurance coverage and plan materials
    • Regulatory documents
    • Patient-reported outcomes instruments

We look forward to your input and questions. Please join the conversation at the next NASH Council Meeting – to join or direct your questions, please email nash@globalliver.org

GLI Leads Bipartisan Access to Innovation Strategy Across HHS, Congress, and the White House

GLI Continues to Advocate for Liver Patients with the Biden Administration, Congress and Federal Agencies

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While a divided Congress and the upcoming Presidential campaign year make for challenging times for policymakers, Global Liver Institute is committed to advocating for liver patients at all levels of regulatory and legislative decision making. With Congressional activity slowed, GLI is active at all levels of advocacy to educate regulators and legislators, impact federal agency actions, and support the liver patient voice for involvement and decision-making with all policymakers.


Policy Developments at Global Liver Institute

GLI Applauds Dr. Monica Bertagnolli’s Nomination as Director of the National Institutes of Health

Donna R. Cryer, founder and CEO of GLI, shared the following quote in response to Dr. Bertagnolli’s nomination: “All of us here at Global Liver Institute are grateful for President Biden nominating Dr. Monica Bertagnolli to be the next NIH director. We appreciate that Dr. Bertagnolli is a cancer survivor and will continue to drive forward the Cancer Moonshot goals to end cancer as we know it. With her background coming from rural America, she brings to the job a fundamental recognition of the challenges facing people who struggle to access health care. We share her concern that clinical trials need to be more diverse, a significant challenge for liver disease and cancers that disproportionately affect people of color. We are excited to work with Dr. Bertagnolli as she leads NIH with a focus on collaboration, as she has demonstrated in her leadership of the National Cancer Institute.” Read more.

GLI Encourages Robust Support for H.R. 2544 to Modernize and Reform the U.S. Organ Procurement and Transplantation Network

This bipartisan legislation responds to the urgent need for reforming the organ procurement transplantation network (OTPN) by breaking up the national monopoly contract so that the Health Resources Services Administration (HRSA) can run an open, competitive process and thus fixing this broken system. Reach out to your legislators today to support the bill!

GLI Provides Comments on the NIDDK Advisory Council’s Report of the Health Disparities and Health Equity Working Group

Global Liver Institute provided comment on the Report of the Health Disparities and Health Equity Working Group of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Advisory Council supporting NIDDK’s recommendations.

GLI Frustrated by FDA GIDAC Committee Failure to Recognize Serious, Unmet Need of Liver Patients in Rejection of Obeticholic Acid for Pre-Cirrhotic Fibrosis Due to NASH

Despite acknowledging that OCA met the endpoint established by the FDA of reducing fibrosis by at least one stage, the FDA’s Gastrointestinal Drugs Advisory Committee (GIDAC) voted against approval for treatment for NASH. Read more here.

GLI Finds New AHRQ Study to be a Call to Action for Inter-Agency Research Collaboration on Links Between Nutrition and Liver Disease and Liver Cancer

A systematic review from the Agency for Health Care Research and Quality (AHRQ) highlights what we have known to be true: there are few studies that look at the impact of nutritional interventions on cancer health outcomes, and those that do exist are prone to bias. Nutrition can be a significant factor leading to obesity and NASH, many of whom develop liver cancer. This review should be a call to action for more inter-agency research collaboration from the National Cancer Institute, the National Institutes for Diabetes and Digestive and Kidney Diseases, the Centers for Disease Control and Prevention, the Veterans Administration, and others to better understand the role of nutrition in liver diseases associated with liver cancer.

GLI Supports the Living Donor Protection Act (H.R. 2923)

Bipartisan legislation was introduced to protect living organ donors and remove barriers to greater donation. The bill addresses tactics to deny or limit coverage by insurers, family and medical leave for donors, and public awareness. Click here to contact your Member of Congress through the American Association of Kidney Patients Action Center.


Upcoming events


Further reading: Organ transplants

Growth in Patient and Medical Community Engagement in NASH Community Persists Despite Recent Regulatory Disappointment

Growth in Patient and Medical Community Engagement in NASH Community Persists Despite Recent Regulatory Disappointment

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Growth in Patient and Medical Community Engagement in NASH Community Persists Despite Recent Regulatory Disappointment

 Global Liver Institute Convenes Largest Number of Community Events around the World in the 6 Years of International NASH Day

(Washington, DC – June 8, 2023) – Today is International NASH Day, a time where healthcare experts and community members around the globe step up and raise awareness of the most common liver disease in the world – nonalcoholic fatty liver disease (NAFLD) – and it’s more severe form, nonalcoholic steatohepatitis (NASH).

Fatty liver disease is a condition in which excess fat builds up in the liver, which can lead to scarring and cirrhosis (permanent damage that impairs liver function). An estimated 25% of the global population, or about 2 billion people, are affected by NAFLD. 20% of those with NAFLD progress to NASH.

International NASH Day 2023 is endorsed by 30 medical specialty societies from around the world, and more than 50 global organizations, from research institutions to community organizations to medical centers and more, will be hosting events to inform and educate their communities.

“By releasing new, multilingual resources to help patients access NASH diagnostics and treatment, leading even more awareness and educational events worldwide, and continuing to foster opportunities for patients to step up and speak out, GLI and its partners are stepping up for those with or at risk of NASH this year,” shared Donna R. Cryer, JD, President and CEO of Global Liver Institute. “We have been heartened through collaboration with the broader patient advocacy community on health technology assessment (HTA) and regulatory processes. Together we have made certain that the patient voice is the clear, powerful force that it ought to be.”

While NASH can develop at any point in life, many of the factors that heighten an individual’s risk of developing NASH are well-known, including overweight or obesity, type 2 diabetes, and high blood pressure. 

Despite established risk factors, too often patients in high-risk groups are still not triaged, not screened, and not connected to the care they require. We are grateful that, just this year, several societies have updated their treatment guidelines for identifying and caring for NAFLD/NASH to reflect our best current understanding of the disease. We are grateful to the scientists who have expanded our understanding of – and ability to control – the disease. It is time for health systems to step up and invest effort in operationalizing these new guidelines.

We urge health system leaders to step up and examine the NASH patient pathway, identify where patients are lost, and bolster those processes. 

We encourage primary care and endocrinology providers to step up and learn which of their patients are at heightened risk of NAFLD/NASH – and to connect them to the screening they need.

We challenge the media to step up against the stigma and cover stories about NAFLD/NASH and liver health, especially when discussing obesity, type 2 diabetes, and other risk factors.

We implore regulators to step up for the livelihood of the millions of patients around the world whose lives are affected by NAFLD/NASH.

Clear, strong connections between primary care providers, specialists, and intervention support are all critical – and within reach – to help more patients find and stop the progression of NASH while they have the opportunity.

“It can be overwhelming how widespread the chronic, progressive disease of NAFLD and NASH has become. Those at risk should know they are supported and empowered to step up for a healthier liver,” encouraged Jeff McIntyre, VP of Liver Health Programs at Global Liver Institute. “Ask your doctor for a screening if you have a risk factor – and receive the treatment you need before the disease progresses. Individuals can make small changes with big impacts to support better liver health through better nutrition and incorporating more movement into their daily habits. Although fatty liver disease can progress, it can be controlled if caught early. Get screened now for a healthy liver.”

It’s time to #StepUpforNASH! 

Join the Movement

Get tested! Check if you are at risk for NAFLD/NASH and make a note to talk to your primary care provider at your next appointment.

Support the #NASHday social media campaign using hashtags #NASHday #StepUpforNASH. Please direct any additional questions to NASHday@globalliver.org.

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About Global Liver Institute 

Global Liver Institute (GLI) is a patient-driven 501(c)3 nonprofit organization headquartered in Washington, DC, with offices in the EU and UK, founded in the belief that liver health must take its place on the global public health agenda commensurate with the prevalence and impact of liver disease and the importance of liver health to well-being. GLI promotes innovation, encourages collaboration, and supports the scaling of optimal approaches to improve research, care, and policy. By bringing together more than 200 community-based, national, and international organizations across its councils, campaigns, and events, GLI equips advocates to identify and solve the problems that matter to liver patients. Follow GLI on Twitter, Facebook, Instagram, LinkedIn, and YouTube. GLI is the global host of International NASH Day.

International NASH Day and its logo are registered trademarks of Global Liver Institute.

Noteworthy Progress in Patient Advocacy & Clinical Trials

Efforts for Improved Treatment and Diagnosis of Rare Liver Diseases

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During May, significant strides were made towards prioritizing the diagnosis and treatment of rare liver diseases. This month, we are excited to share some innovative approaches to enhance the diagnostic and treatment processes of rare liver diseases, which have traditionally been overlooked and lacked appropriate medical interventions.


GLI Applauds Congress for Urging FDA to Review Rare Disease Therapy Procedures, A Major Step Towards Better Treatment Options

With numerous liver diseases lacking effective treatment options, GLI is encouraged to see the Congressional Rare Disease Caucus advocating for the FDA to prioritize rare diseases on their agenda. Specific initiatives that were mentioned include assessing inconsistencies in rare disease policies, reviewing the implementation of approval pathways, and providing increased clarity for employees to ensure continuity of in-process application reviews. We commend the Caucus’s work in promoting a more consistent and reliable approval process for these essential medications.

Significant Progress Made in Streamlining Diagnosis of Wilson’s Disease in China

Wilson’s disease is a rare genetic disorder that causes copper to build up in the body, leading to various neurological and liver-related symptoms. Due to its variable symptoms that can overlap with other conditions and the need for specialized tests to measure copper levels, diagnosis can be challenging. However, a recent study published in Liver Research suggests that a novel and comprehensive nomogram based on routine clinical indicators, which can be obtained through noninvasive methods, may improve the diagnosis of Wilson’s disease in the future. This streamlined diagnostic process, using six routine blood indicators, is expected to reduce the likelihood of missing or misdiagnosing the disease.

Upcoming Report of Ocaliva and bezafibrate as promising treatment option for Primary Biliary Cholangitis (PBC)

GLI is looking forward to the presentation of Phase II trial data and research by Ipsen, one of our council members, at EASL in June 2023. They will be sharing their findings on the utilization of Ocaliva in conjunction with bezafibrate for the treatment of PBC. This significant development, coupled with the FDA’s grant of orphan drug status, holds great promise for the future of PBC treatment.

GLI attends the BARE Inc. Symposium, Chicago, IL

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GLI joined patients, advocates, and healthcare providers in Chicago for BARE Inc’s first Biliary Atresia Symposium on May 5-6, 2023. Key focal points included emphasizing early diagnosis in pediatric liver diseases, highlighting breakthroughs in research and treatment, recognizing the importance of collaboration and multidisciplinary care, advocating for patient-centered approaches, and addressing challenges related to access to care and health equity. The overarching objective was to enhance the well-being of affected children and advance the global comprehension and management of rare liver diseases.


Upcoming Events


For more information about the Pediatric and Rare Liver Diseases Council or to learn more about joining, please visit https://globalliver.org/pediatric-rare-liver-diseases-council/ or email pedsrare@globalliver.org.

Discoveries in HCC and Biliary Tract Cancer

New Leadership & New Understanding in the Cancer World

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Globally, liver cancer is the third leading cause of cancer death. It’s not only important to GLI to keep you updated; we value your input and updates. This is a reminder to Liver Cancers Council members and supporters to share news to be featured in our monthly newsletter or on our Liver Central website.


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White House Plans to nominate Dr. Monica Bertagnolli for NIH director

The White House plans to nominate Dr. Monica M. Bertagnolli to be the next director of the National Institutes of Health (NIH). Bertagnolli is an oncologist who was the director of the National Cancer Institute (NCI) for the last six months as well as a cancer survivor herself. She is the first woman to run the NCI and will be the second woman to run NIH. Read more here.

Despite low health literacy in Hispanics, researchers find high adherence to cancer screenings

A study from the Regenstrief Institute and Indiana University on cancer health literacy revealed that most of the Hispanics surveyed were not aware of the age to begin cancer screenings. The report from Regenstrief Institute exposes the gaps in cancer health literacy among Hispanics in Indiana. There were a variety of factors that influenced health literacy, including income, age, and urban/rural residency. Despite low health literacy of guidelines, there was still a high adherence to cancer screenings – a reminder that a complete picture of the evidence is necessary to avoid misunderstanding the situation.

The Role of ATF4 in Liver Cancer

A study by scientists at University of California San Diego School of Medicine investigated the role of activating transcription factor 4 (ATF4), a protein that is a key mediator of the liver stress response. ATF4 was thought to be linked to advanced liver cancer, but the study found that the protein actually protects the liver from the death of liver cancer cells and the formation of tumors. Specifically, ATF4 protects against ferroptosis, an iron-dependent form of liver cell death. These results were unexpected and can now pave the way for new clinical strategies for liver cancer and liver disease prevention.

The LGBTQIA+ community continues to face cancer inequities

June is Pride Month. To bring awareness to the lack of liver cancer research and to highlight inequities that this population faces, GLI convened a health equity roundtable featuring professionals from the field to discuss issues, research needs, and next steps. Make sure to watch our LGBTQIA+ Health Equity roundtable here.


Upcoming Events:


For more information about the Liver Cancers Council or to learn more about joining, please visit https://globalliver.org/liver-cancers-council/ or email cancer@globalliver.org.