Supported Research
Cure HHT: Driving Impact and Innovation in HHT Research & Treatment
The Impact of Our Work
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Funds Granted to Cure HHT$0
Cure HHT's Investments to HHT Researchers$0
Investment in Clinical Trials$0
Funds Secured Through PartnershipsResearch Spotlight
Antibodies: Path to a Cure
Developing Agonist Antibodies to Restore Signaling
Dr. William Sellers, Director of the Cancer Program at the Broad Institute, aims to find and test antibodies which will bind together parts of the HHT signaling pathway that are not working properly due to HHT-causing mutations.
Innovative Research Focus
There are medical therapies to repurpose drugs currently being studied and trialed in HHT patients, but no therapies exist that are directly based on our understanding of the gene mutation causing HHT. Dr. Sellers and his lab aim to develop a cell therapy to restore signaling in HHT.
Key Objectives of the Study:
- Discover the best agonist antibody candidates: Dr. Seller’s team will identify the best antibody candidates that can bind to two parts of the HHT signaling pathway simultaneously, bringing them together and allowing them to interact.
- Test candidates in lab: Best antibody candidates will be tested extensively in the lab to ensure they are specific to their targets and function properly.
- Test candidates in animal models of HHT: Restoring the HHT signaling pathway will be tested in animal models of HHT where Dr. Sellers and his team will be able to see if they can prevent AVMs from forming or reverse the formation of them altogether.
Impact: There are currently no therapies approved to treat HHT. Those in trial are repurposed drugs targeting downstream parts of the HHT signaling pathway, but this proposal targets the area where the HHT mutations occur, offering a chance at prevention of AVM formation and restoration of the BMP9/10 signaling cascade.
For more information on Dr. Sellers research and his lab, click the button below.
This study was generously funded by Terry and Juli Thompson.
Breakthrough: AVMs on Chips
From Lab to Treatment: AVMs on Chips
Christopher Hughes is transforming HHT research with his "AVMs on chips" method. The "AVMs on chips" method involves creating miniature, lab-grown models of AVMs on microchips. These microchips are engineered to mimic the complex environment of human tissues, allowing researchers to study AVMs in a controlled and precise manner.
By replicating blood vessel abnormalities seen in HHT right in the lab, this groundbreaking approach revolutionizes the way we study and treat the disease.
- Summary: The model creates the small and large blood vessel malformations typical of HHT on a chip in the lab for efficient drug screening and monitoring.
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Impact: Screening with these lab-generated AVMs, showed that the drug pazopanib can block lesion formation. This is critical information to validate in the laboratory, as we are currently trialing pazopanib in a Phase II/III clinical trial!
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Critical Insights from the Huges Lab: By manipulating the Alk1 gene, researchers identified a crucial window during vessel formation where this gene prevents malformations. The study found that malformations can contain both normal and mutant cells, providing new insights into disease mechanisms.
Funded by a $4.4 million NIH grant, Hughes's work showcases the power of innovative science coupled with collaborative efforts. His research is paving the way for future gene therapy advancements, marking a significant leap forward in the battle against HHT.
For more details on Dr. Hughes' study and its impact on HHT, click the button below.
Improving Quality of Life
Improving Quality of Life for HHT Patients
Dr. Clifford Weiss from Johns Hopkins University is leading an important study to better understand and assess the Health-Related Quality of Life (HRQOL) for HHT patients.
Supported by a $50,000 grant from Cure HHT, this cross-sectional survey study seeks to understand the multifaceted impact of HHT on patients' lives.
Vision and Goals for the Future of HHT
- Comparative Analysis: Assessing how HRQOL, fatigue, and depression/anxiety among HHT patients compare to the general US population and other chronic illness groups.
- How it all connects: Investigating how physical, mental, and symptom-specific QOL metrics are connected.
- Demographic & Clinical Factors: Exploring how age, gender, race, genetic mutations, and symptom severity impact HRQOL and symptom severity.
Impact on HHT Patients
This study is a significant step forward for the HHT community. By creating a reliable HRQOL tool, clinicians can better understand and address the comprehensive needs of HHT patients.
This means more personalized and effective treatments, and increased awareness about the impact of HHT on patients' overall wellbeing.
To explore the impact of Dr. Weiss's study, click the button below.
Advances in Drug Repurposing
Drug Repurposing Breakthrough
Dr. Edda Spiekerkoetter and her team at the Stanford HHT Center of Excellence received a $2.8 million NIH/NHLBI grant to study the molecular and cellular events behind pulmonary AVMs.
Addressing a Critical Need
There is an urgent need for therapies that target the root causes of HHT and Pulmonary Arterial Hypertension (PAH). Despite advancements, current treatments primarily alleviate symptoms rather than address the underlying mechanisms of these conditions.
Fast-Tracking Treatments: Dr. Spiekerkoetter's groundbreaking research leverages the potential of existing drugs to treat HHT and PAH. By repurposing medications such as FK506 (tacrolimus) and tyrosine kinase inhibitors, her work aims to quickly transition from laboratory studies to clinical trials.
This approach offers a faster, more cost-effective way to bring new treatments to patients, enhancing their quality of life significantly sooner than traditional drug development methods.
Addressing the Root Cause: Repurposing already-tested drugs speeds up the availability of new treatments. Dr. Spiekerkoetter’s research not only targets symptoms but also aims to address the root causes of HHT and PAH, potentially reversing disease progression.
For more detailed information on Dr. Spiekerkoetter’s research, click the button below.
Decoding Genetic Variations
Decoding Genetic Variations
Dr. Anthony Anzell from the University of Pittsburgh has received a $20,000 research grant from Uplifting Athletes' Young Investigator Draft for his promising work on genetic variations associated with HHT.
Innovative Research Focus
Dr. Anzell’s current research focuses on understanding the ACVRL1 gene mutation in HHT. His study employs a "mosaic" context, investigating how normal and abnormal cells interact within AVMs.
Key Objectives of the Study:
- Understanding AVMs: Analyze AVMs at a cellular level to study interactions between normal and abnormal cells, using primary endothelial cells and zebrafish embryos to study AVM formation mechanisms
- Mosaic-Style Culture System: Creating a detailed and accurate model of the disease with both normal and abnormal cells together to better understand how it affects the body.
- From Understanding to Intervention: Testing proteins to rescue abnormal cells, restoring cellular function and develop effective treatments.
Impact and Future Directions
There are currently no FDA-approved therapies for HHT, and the mechanism of AVM formation is not fully understood. Dr. Anzell’s research aims to fill these gaps, potentially leading to new, effective therapies for HHT patients.
For more information on Dr. Anzell’s research and his journey, click the button below.
Research Spotlight
Click on a topic below to uncover our latest research discoveries!
Developing Agonist Antibodies to Restore Signaling
Dr. William Sellers, Director of the Cancer Program at the Broad Institute, aims to find and test antibodies which will bind together parts of the HHT signaling pathway that are not working properly due to HHT-causing mutations.
Innovative Research Focus
There are medical therapies to repurpose drugs currently being studied and trialed in HHT patients, but no therapies exist that are directly based on our understanding of the gene mutation causing HHT. Dr. Sellers and his lab aim to develop a cell therapy to restore signaling in HHT.
Key Objectives of the Study:
Discover the best agonist antibody candidates: Dr. Seller’s team will identify the best antibody candidates that can bind to two parts of the HHT signaling pathway simultaneously, bringing them together and allowing them to interact.
Test candidates in lab: Best antibody candidates will be tested extensively in the lab to ensure they are specific to their targets and function properly.
Test candidates in animal models of HHT: Restoring the HHT signaling pathway will be tested in animal models of HHT where Dr. Sellers and his team will be able to see if they can prevent AVMs from forming or reverse the formation of them altogether.
Impact: There are currently no therapies approved to treat HHT. Those in trial are repurposed drugs targeting downstream parts of the HHT signaling pathway, but this proposal targets the area where the HHT mutations occur, offering a chance at prevention of AVM formation and restoration of the BMP9/10 signaling cascade.
For more information on Dr. Sellers research and his lab, click the button below.
This study was generously funded by Terry and Juli Thompson.
From Lab to Treatment: AVMs on Chips
Christopher Hughes is transforming HHT research with his "AVMs on chips" method.
This groundbreaking approach involves creating tiny models of blood vessel abnormalities seen in HHT right in the lab.
Breakthroughs and Insights:
Summary: The model creates the small and large blood vessel malformations typical of HHT on a chip in the lab for efficient drug screening and monitoring.
Funded by a $4.4 million NIH grant, Hughes's work showcases the power of innovative science coupled with collaborative efforts. His research is paving the way for future gene therapy advancements, marking a significant leap forward in the battle against HHT.
For more details on Dr. Hughes' study and its impact on HHT, click the button below.
Improving Quality of Life for HHT Patients
Dr. Clifford Weiss from Johns Hopkins University is leading an important study to better understand and assess the Health-Related Quality of Life (HRQOL) for HHT patients.
Supported by a $50,000 grant from Cure HHT, this cross-sectional survey study seeks to understand the multifaceted impact of HHT on patients' lives.
Vision and Goals for the Future of HHT
Comparative Analysis: Assessing how HRQOL, fatigue, and depression/anxiety among HHT patients compare to the general US population and other chronic illness groups.
How it all connects: Investigating how physical, mental, and symptom-specific QOL metrics are connected.
Demographic & Clinical Factors: Exploring how age, gender, race, genetic mutations, and symptom severity impact HRQOL and symptom severity.
Impact on HHT Patients
This study is a significant step forward for the HHT community. By creating a reliable HRQOL tool, clinicians can better understand and address the comprehensive needs of HHT patients.
This means more personalized and effective treatments, and increased awareness about the impact of HHT on patients' overall wellbeing.
To explore the impact of Dr. Weiss's study, click the button below.
Drug Repurposing Breakthrough
Dr. Edda Spiekerkoetter and her team at the Stanford HHT Center of Excellence received a $2.8 million NIH/NHLBI grant to study the molecular and cellular events behind pulmonary AVMs.
Addressing a Critical Need
There is an urgent need for therapies that target the root causes of HHT and Pulmonary Arterial Hypertension (PAH). Despite advancements, current treatments primarily alleviate symptoms rather than address the underlying mechanisms of these conditions.
Fast-Tracking Treatments: Dr. Spiekerkoetter's groundbreaking research leverages the potential of existing drugs to treat HHT and PAH. By repurposing medications such as FK506 (tacrolimus) and tyrosine kinase inhibitors, her work aims to quickly transition from laboratory studies to clinical trials. This approach offers a faster, more cost-effective way to bring new treatments to patients, enhancing their quality of life significantly sooner than traditional drug development methods.
Addressing the Root Cause: Repurposing already-tested drugs speeds up the availability of new treatments. Dr. Spiekerkoetter’s research not only targets symptoms but also aims to address the root causes of HHT and PAH, potentially reversing disease progression.
For more detailed information on Dr. Spiekerkoetter’s research, click the button below.
Decoding Genetic Variations
Dr. Anthony Anzell from the University of Pittsburgh has received a $20,000 research grant from Uplifting Athletes' Young Investigator Draft for his promising work on genetic variations associated with HHT.
Innovative Research Focus
Dr. Anzell’s current research focuses on understanding the ACVRL1 gene mutation in HHT. His study employs a "mosaic" context, investigating how normal and abnormal cells interact within AVMs.
Key Objectives of the Study:
Understanding AVMs: Studying AVMs at a cellular level and interactions between normal and abnormal cells.
Mosaic-Style Culture System: Creating a detailed and accurate model of the disease with both normal and abnormal cells together to better understand how it affects the body.
From Understanding to Intervention: Using proteins to rescue abnormal cells, restoring cellular function and develop effective treatments.
Impact and Future Directions
There are currently no FDA-approved therapies for HHT, and the mechanism of AVM formation is not fully understood. Dr. Anzell’s research aims to fill these gaps, potentially leading to new, effective therapies for HHT patients.
For more information on Dr. Anzell’s research and his journey, click the button below.
Advancing HHT Research
We are the catalyst for driving advancements in HHT research.
Through strategic grants, direct funding, and building industry partnerships, we have significantly grown the amount of research taking place within our disease over the years and have empowered groundbreaking discoveries that ultimately improve the lives of patients.
Our efforts to over the years grow the research community focused on HHT and advance our understanding of the underlying mechanisms of this disease, have allowed us to reach a point where yesterday’s dreams are becoming today’s accomplishments.
Advancing HHT Research
We are the catalyst for driving advancements in HHT research.
Through strategic grants, direct funding, and building industry partnerships, we have significantly grown the amount of research taking place within our disease over the years and have empowered groundbreaking discoveries that ultimately improve the lives of patients.
Our efforts to over the years grow the research community focused on HHT and advance our understanding of the underlying mechanisms of this disease, have allowed us to reach a point where yesterday’s dreams are becoming today’s accomplishments.
Our Mission
To be at the forefront of accelerating progress in understanding, treating, and finding a cure for HHT.
We aim to:
Recipients of the Young Investigator Awards at the 2022 14th International HHT Scientific Conference, Portugal.
Recipients of the Young Investigator Awards at the 2022 14th International HHT Scientific Conference, Portugal.
Our Mission
To be at the forefront of accelerating progress in understanding, treating, and finding a cure for HHT.
We aim to:
Cure HHT Research Network Hub
The Cure HHT Research Network Hub is a pivotal initiative funded by Cure HHT, fostering community and collaboration to accelerate discoveries by building a robust repository of knowledge.
This platform connects HHT researchers and clinicians worldwide, providing access to research publications, real-time news and announcements, continuing medical education, and biobank samples. The hub supports innovative studies and sharing insights to discover effective treatments and, ultimately, a cure for HHT.
*For researchers and clinicians only.
Cure HHT Research Network Hub
The Cure HHT Research Network Hub is a pivotal initiative funded by Cure HHT, fostering community and collaboration to accelerate discoveries by building a robust repository of knowledge.
This platform connects HHT researchers and clinicians worldwide, providing access to research publications, real-time news and announcements, continuing medical education, and biobank samples. The hub supports innovative studies and sharing insights to discover effective treatments and, ultimately, a cure for HHT.
*For researchers and clinicians only.
Powering HHT Research Through Partnerships
Cure HHT's research initiatives are significantly enhanced by our the partnerships we have formed with leading academic institutions, government agencies, foundations, and industry leaders. Each collaboration brings unique strengths to our mission:
Foundations and Non-Profits, including the Chan Zuckerberg Initiative and Argosy Foundation, boost our research funding, allowing us to pursue ambitious projects.
Academic Partnerships drive innovation and discovery in HHT research, enriching the projects we fund.
Government Agencies like the NIH, DoD, and CDC provide essential support, expanding the scope and impact of our research.
Industry Collaborations with companies like Diagonal Therapeutics, Medtronic, Nasaclip, and more introduce cutting-edge technology and treatments into our research efforts.
These strategic alliances not only diversify our research funding but also ensure that our supported projects have a broader impact, accelerating progress toward understanding, treating, and ultimately curing HHT.
Supporting Research Across All Fronts
Cure HHT advances research across various areas.
Click on the categories below to explore the full list of funding. Each category represents a step forward in our mission to transform lives and bring hope to those affected by HHT.
Research Grants
Basic Science Research Grants
Our grants program is the lifeline for initiating and continuing groundbreaking studies in HHT.
Cure HHT's research program fosters the development of lasting cures for the underlying mechanisms and causes of HHT. To spur development of new ideas leading to novel drug therapies, Cure HHT funds basic science research grants to help learn more about the mechanisms of pathology in HHT. Cure HHT Basic Science Research Grants.
| Researcher | Institution / Country | Grant Type | Year | HHT Funding | Other Funding |
|---|---|---|---|---|---|
| Beth Roman, PhD | University of Pittsburgh (USA) | Basic Science | 2021 | --- | $2,000,000 (DOD) |
| Jennifer Fang, PhD | University of California, Irvine (USA) | Basic Science | 2020 | --- | $314,000 (DOD) |
| Stryder Meadows, PhD | Tulane University (USA) | Basic Science | 2018 | --- | $1,819,500 (NIH) |
| Christopher Hughes, PhD | University of California, Irvine (USA) | Basic Science | 2017 | --- | $1,923,000 (NCATS) |
| Stryder Meadows, PhD | Tulane University (USA) | Basic Science | 2016 | --- | $1,785,199 (DOD) |
| Marie Faughnan, MD; Douglas Marchuk, PhD; Philippe Marambaud, PhD; S. Paul Oh, PhD; Beth Roman, PhD | University of Toronto (Canada); Duke University (USA); Feinstein Institute for Medical Research (USA); Barrow Neurological Institute (USA); University of Pittsburgh (USA) | Basic Science and Clinical Science | 2016 | --- | $10,000,000 (DOD) |
| Michaela Aldred, PhD | Lerner Research Institute, Cleveland Clinic (USA) | Discovery Award | 2015 | --- | $2,600,000 (DOD) |
| Rong Wang, PhD | University of California, San Francisco (USA) | Investigator Initiative Award | 2015 | --- | $1,500,000 (DOD) |
| S. Paul Oh, PhD | University of Florida (USA) | Basic Science | 2015 | $50,000 | $1,500,000 (NIH / NHLBI) |
| Beth Roman, PhD | University of Pittsburgh (USA) | Genetics | 2011 | $45,000 | $2,000,000 (NIH) |
| Rosemary Akhurst, PhD | University of California, San Francisco (USA) | Basic Science | 2008 | $50,000 | $1,800,000 (NIH / NHLBI) |
| Christopher Hughes, PhD | University of California, Irvine (USA) | Basic Science | 2008 | $50,000 | --- |
| S. Paul Oh, PhD | University of Florida (USA) | Basic Science | 2008 | $50,000 | $1,000,000 (NIH) |
| Michelle Letarte, PhD | The Hospital for Sick Children (Canada) | Basic Science | 2006 | $50,000 | --- |
| Douglas Marchuk, PhD | Duke University (USA) | Basic Science | 2004 | $50,000 | --- |
| Mary Porteous, MD | The University of Edinburgh (Scotland) | Genetics | 2004 | $5,000 | --- |
Clinical Science Research Grant Funding
Cure HHT has been instrumental in significant advances in HHT treatment, and will continue to act as a catalyst for improved care for HHT patients.
Clinical science research determines the safety and effectiveness of medications, devices, diagnostic products and treatments intended for human use. To facilitate the translation of basic science research into clinical trials and new treatments for HHT, Cure HHT collaborates with industry and academia to test the efficacy of therapeutic drugs, medical devices, and treatment protocols. Cure HHT Clinical Science Research Grants.
| Researcher | Institution / Country | Grant Type | HHT Funding | Other Funding |
|---|---|---|---|---|
| Clifford Weiss, MD | Johns Hopkins University (USA) | Clinical Science (2022) | $49,926 | --- |
| Steven Hetts, MD | University of California, San Francisco (USA) | Clinical Science (2022) | --- | $2,584,000 (DOD) |
| Hanny Al-Samkari, MD | Massachusetts General Hospital (USA) | Clinical Science (2020) | $40,000 | $1,000,000 (NIH) |
| Edda Spiekerkoetter, MD | Stanford University (USA) | Clinical Science (2019) | --- | $1,800,000 (DOD) |
| Marie Faughnan, MD; Douglas Marchuk, PhD; Philippe Marambaud, PhD; S. Paul Oh, PhD; Beth Roman, PhD | University of Toronto (Canada); Duke University (USA); Feinstein Institute for Medical Research (USA); Barrow Neurological Institute (USA); University of Pittsburgh (USA) | Basic Science and Clinical Science (2016) | --- | $10,000,000 (DOD) |
| Amelia Clark, MD | Stanford University (USA) | Clinical Science (2014) | $10,000 | --- |
| Kristen Anton, MS | BioInformatics Service Center, Dartmouth College (USA) | Therapeutic Outcomes (2013) | $165,000 | --- |
| Jonathan Zaroff, MD | Kaiser Foundation (USA) | Prevalance / Undiagnosed (2013) | $30,000 | --- |
| Jeffrey Hoag, MD | Drexel University (USA) | Treatment of Disease (2007) | $50,000 | --- |
| Christine Mummery, PhD | Leiden University Medical Center (Netherlands) | Treatment of Disease (2006) | $50,000 | --- |
| Carmelo Bernabeu, PhD | Centro de Investigaciones Biologica (Spain) | Treatment of Disease (2005) | $50,000 | $500,000 (European Research Agency) |
| Karen Swanson, DO | Mayo Clinic (USA) | Treatment of Disease (2004) | $50,000 | $1,000,000 (FDA Orphan Drug Grant) |
| Stanley Shapshay, MD | Albany Medical Center (USA) | Treatment of Disease (2004) | $5,000 | --- |
| Jamie McDonald, MS, CGC | ARUP Laboratories (USA) | Genetics / DNA Patent Release (2003) | $10,000 | --- |
| Arupa Ganguly, PhD | University of Pennsylvania Genetic Diagnostic Laboratory (USA) | Genetics / DNA Patent Release (2003) | $10,000 | --- |
| Kirk Vandezande | HHT Solutions, Inc. (Canada) | Genetics / DNA Patent Release (2003) | $10,000 | --- |
Clinical Trial Grants
Cure HHT Clinical Trials
Clinical trials drive HHT treatment advancements, testing new therapies to improve patient lives.
The HHT community is in desperate need of effective and safe treatments to improve health and quality of life. Clinical trials test the safety and efficacy of potential drug therapies and treatments as well as new applications for FDA approved drugs and/or treatments.
| Researcher | Clinical Trial Description | Study Year | HHT Funding | Other Funding |
|---|---|---|---|---|
| Randomized Trial for Pazopanib in HHT-Related Bleeding | A Phase II/III randomized, placebo-controlled, double-blind study to evaluate the effects of up to 24 weeks of low dose oral pazopanib on hereditary hemorrhagic telangiectasia (HHT) related epistaxis and anemia. | 2022-2025 | $877,824 (FDA) | |
| Randomized Trial for Pazopanib in HHT-Related Bleeding | A Phase II/III randomized, placebo-controlled, double-blind study to evaluate the effects of up to 24 weeks of low dose oral pazopanib on hereditary hemorrhagic telangiectasia (HHT) related epistaxis and anemia. | 2021-2024 | $350,000 | $5,240,964 (DOD) |
| Pomalidomide for the Treatment of Bleeding in Hereditary Hemorrhagic Telangiectasia | A Phase II randomized, placebo-controlled double-blind study of pomalidomide in patients with hereditary hemorrhagic telangiectasia (HHT) with moderate to severe epistaxis who have anemia and/or require parenteral iron infusions or blood transfusions. Treatment with oral pomalidomide or matching placebo for 24 weeks. | 2019-2023 | --- | $3,638,312 (NIH) |
| North American Study of Epistaxis (NOSE) | Multi-center clinical trial designed to test the efficacy of three nasal spray agents as compared to a placebo in reducing nose bleed frequency, duration and increasing quality of life. | 2010 - 2014 | $180,000 | --- |
Young Investigator Awards
Cure HHT Young Investigator Awards
Fostering the next generation of researchers, these awards support innovative research and career development for early-stage investigators.
The Cure HHT Young Scholar Research Program supports scientists* and clinicians* who show exceptional ability and promise for conducting HHT basic science, clinical science or translational science research. The objective of this program is to foster creative HHT research; enhance early career development of outstanding young investigators; and increase opportunities for the young investigator to recognize our mission to FIND, TREAT and CURE HHT. Cure HHT Young Investigator Awards.
*Residents, Postdoctoral/Clinical Fellows, PhD Candidates or Early Stage Investigator who are within 3 years of completing their terminal research degree or within 3 years of completing their medical residency/sub-specialty fellowship
| Researcher | Institution / Country | Grant Type | Year | Grant Type |
|---|---|---|---|---|
| HHT International Scientific Conference | Young Investigator Travel Awards and Presentation Awards | --- | 2022 | $42,000 |
| HHT International Scientific Conference | Young Investigator Travel Awards and Presentation Awards | --- | 2019 | $20,250 |
| HHT International Scientific Conference | Young Investigator Travel Awards and Presentation Awards | --- | 2017 | $15,500 |
| Veronique Vorselaars, PhD | St. Antonius Hospital (Netherlands) | --- | 2017 | $1,200 |
| HHT International Scientific Conference | Young Investigator Presentation Awards | --- | 2003 - 2015 | $14,000 |
| Hongyu Tian, PhD | Duke University (USA) | Basic Science | 2015 | $30,000 |
| Wan Zhu, PhD | University of California, San Francisco (USA) | Basic Science | 2015 | $30,000 |
| Simon Tual-Chalot, PhD | Newcastle University (UK) | Basic Science | 2015 | $30,000 |
| Whitney Wooderchak-Donahue, PhD | University of Utah / ARUP Laboratories (USA) | Genetics | 2015 | $30,000 |
| Chadwick Davis, PhD | Recursion Pharmaceuticals (USA) | Drug Therapy | 2015 | $30,000 |
| Pinar Bayrak-Toydemir, MD, PhD | University of Utah / ARUP Laboratories (USA) | Genetics | 2008 | $20,000 |
| Sebastian Velthius, MD, PhD | St. Antonius Hospital (Netherlands) | --- | 2014 | $2,000 |
| Sabine Bailly, MD | CEA-Grenoble (France) | NIH Travel Award | 2006 | $1,000 |
| Terri DiMaio, PhD | University of Wisconsin (USA) | NIH Travel Award | 2006 | $1,000 |
| Africa Fernandez, PhD | Centro de Investigaciones Biologicas (Spain) | NIH Travel Award | 2006 | $1,000 |
| Eric Finkelstein, PhD | Syracuse University (USA) | NIH Travel Award | 2006 | $1,000 |
| Tassilo Forg, PhD | University of Applied Sciences (Germany) | NIH Travel Award | 2006 | $1,000 |
| Arupa Ganguly, PhD | University of Pennsylvania (USA) | NIH Travel Award | 2006 | $1,000 |
| Fatima Govani, PhD | University of Oxford (UK) | NIH Travel Award | 2006 | $1,000 |
| Jose Miguel Lopez-Novoa, PhD | Universidad de Salamanca (Spain) | NIH Travel Award | 2006 | $1,000 |
| Mourad Toporsian, PhD | McGill University (Canada) | NIH Travel Award | 2006 | $1,000 |
| Liana Zucco | University College London (UK) | NIH Travel Award | 2006 | $1,000 |
Robert I. White Awards
Robert I. White Awards
Recognizing the dedication and compassion of early-career clinicians, honoring those who embody Dr. White's unwavering commitment to advancing care and research in the field.
This award was established in recognition of Dr. Robert I. White Jr.’s outstanding contribution to HHT care and to mentoring other clinicians to develop expertise in HHT care and research. This award is given annually to a clinician (physician or health professional), who is within the first years of work involving HHT and who embodies Dr. White’s qualities as a compassionate and dedicated clinician, devoted to improving HHT care.
| Researcher | Institution / Country | Award Year | HHT Funding |
|---|---|---|---|
| Hanny Al-Samkari, MD | Massachusetts General Hospital (USA) | 2022 | $,2500 |
| Vivek Iyer, MD, MPH | Mayo Clinic (USA) | 2019 | $2,500 |
| Steven Hetts, MD | University of California, San Francisco (USA) | 2017 | $2,500 |
| Marcelo Serra, MD | Hospital Italiano de Buenos Aires (Argentina) | 2015 | $2,500 |
| Justin McWilliams, MD | University of California, Los Angeles (USA) | 2011 | $2,500 |
| Takeo Nishida, PhD | Yale University (USA) | 2010 | $2,500 |
| Els Marie De Gussem, MD | Grace Hospital (Canada) | 2009 | $2,500 |
| Sophie Dupuis-Girod, MD | CHU de Lyon (France) | 2008 | $2,500 |
| Roberto Zarrabeitia, MD | Hospital Sierrallana (Spain) | 2007 | $2,500 |
| Pinar Bayrak-Toydemir, MD, PhD | University of Utah / ARUP Laboratories (USA) | 2006 | $2,500 |
| Gaetan Lesca, MD | CHU de Lyon (France) | 2005 | $2,500 |
| Tom Letteboer, MD, PhD | University Medical Centre Utrecht (Netherlands) | 2004 | $2,500 |
Research Conference Awards
Research Conference Awards
Cure HHT connects and unites the HHT community worldwide through events and gatherings, igniting knowledge and collective action.
Cure HHT has long acted as a catalyst by making personal connections within the relevant communities and hosting scientific and medical conferences. To date, Cure HHT has partnered with NAVBO, NIH, CDC, American Academy of Otolaryngology and many other organizations to educate physicians and scientists about HHT.
| Event | Institution / Country | Award Year | HHT Funding |
|---|---|---|---|
| HHT International Scientific Conference | Conference Host (Portugal) | 2022 | TBD |
| Christopher McMahon Memorial International HHT Guidelines Conference | Conference Host (Canada) | 2019 | $175,000 |
| HHT International Scientific Conference | Conference Host (Puerto Rico) | 2019 | $27,000 |
| HHT International Scientific Conference | Conference Host (Croatia) | 2017 | $61,500 |
| North American Vascular Biology Organization (NAVBO) | Sponsor - HHT Symposia | 2016 | $5,000 |
| HHT International Scientific Conference | Conference Host (USA) | 2015 | $33,000 |
| HHT International Scientific Conference | Sponsor - Speaker Travel Awards | 2001 - 2013 | $70,500 |
| North American Vascular Biology Organization (NAVBO) | Sponsor - HHT Symposia | 2014 | $10,000 |
| North American Vascular Biology Organization (NAVBO) | Sponsor - HHT Genetics Research Presentation | 2009 | $10,000 |
| National Institutes of Health (NIH) | Sponsor - Brain AVM Presentation | 2008 | $2,000 |
| North American Vascular Biology Organization (NAVBO) | Sponsor - HHT Symposia | 2007 | $2,000 |
| HHT Clinical Guidelines Conference | Sponsor - Expert Travel Awards | 2006 | $60,000 |
| National Institutes of Health | Sponsor - Physician Training | 2006 | $5,000 |
Research Grants
Basic Science Research Grants
Our grants program is the lifeline for initiating and continuing groundbreaking studies in HHT.
Cure HHT's research program fosters the development of lasting cures for the underlying mechanisms and causes of HHT. To spur development of new ideas leading to novel drug therapies, Cure HHT funds basic science research grants to help learn more about the mechanisms of pathology in HHT. Cure HHT Basic Science Research Grants.
| Researcher | Institution / Country | Grant Type | Year | HHT Funding | Other Funding |
|---|---|---|---|---|---|
| Beth Roman, PhD | University of Pittsburgh (USA) | Basic Science | 2021 | --- | $2,000,000 (DOD) |
| Jennifer Fang, PhD | University of California, Irvine (USA) | Basic Science | 2020 | --- | $314,000 (DOD) |
| Stryder Meadows, PhD | Tulane University (USA) | Basic Science | 2018 | --- | $1,819,500 (NIH) |
| Christopher Hughes, PhD | University of California, Irvine (USA) | Basic Science | 2017 | --- | $1,923,000 (NCATS) |
| Stryder Meadows, PhD | Tulane University (USA) | Basic Science | 2016 | --- | $1,785,199 (DOD) |
| Marie Faughnan, MD; Douglas Marchuk, PhD; Philippe Marambaud, PhD; S. Paul Oh, PhD; Beth Roman, PhD | University of Toronto (Canada); Duke University (USA); Feinstein Institute for Medical Research (USA); Barrow Neurological Institute (USA); University of Pittsburgh (USA) | Basic Science and Clinical Science | 2016 | --- | $10,000,000 (DOD) |
| Michaela Aldred, PhD | Lerner Research Institute, Cleveland Clinic (USA) | Discovery Award | 2015 | --- | $2,600,000 (DOD) |
| Rong Wang, PhD | University of California, San Francisco (USA) | Investigator Initiative Award | 2015 | --- | $1,500,000 (DOD) |
| S. Paul Oh, PhD | University of Florida (USA) | Basic Science | 2015 | $50,000 | $1,500,000 (NIH / NHLBI) |
| Beth Roman, PhD | University of Pittsburgh (USA) | Genetics | 2011 | $45,000 | $2,000,000 (NIH) |
| Rosemary Akhurst, PhD | University of California, San Francisco (USA) | Basic Science | 2008 | $50,000 | $1,800,000 (NIH / NHLBI) |
| Christopher Hughes, PhD | University of California, Irvine (USA) | Basic Science | 2008 | $50,000 | --- |
| S. Paul Oh, PhD | University of Florida (USA) | Basic Science | 2008 | $50,000 | $1,000,000 (NIH) |
| Michelle Letarte, PhD | The Hospital for Sick Children (Canada) | Basic Science | 2006 | $50,000 | --- |
| Douglas Marchuk, PhD | Duke University (USA) | Basic Science | 2004 | $50,000 | --- |
| Mary Porteous, MD | The University of Edinburgh (Scotland) | Genetics | 2004 | $5,000 | --- |
Clinical Science Research Grant Funding
Cure HHT has been instrumental in significant advances in HHT treatment, and will continue to act as a catalyst for improved care for HHT patients.
Clinical science research determines the safety and effectiveness of medications, devices, diagnostic products and treatments intended for human use. To facilitate the translation of basic science research into clinical trials and new treatments for HHT, Cure HHT collaborates with industry and academia to test the efficacy of therapeutic drugs, medical devices, and treatment protocols. Cure HHT Clinical Science Research Grants.
| Researcher | Institution / Country | Grant Type | HHT Funding | Other Funding |
|---|---|---|---|---|
| Clifford Weiss, MD | Johns Hopkins University (USA) | Clinical Science (2022) | $49,926 | --- |
| Steven Hetts, MD | University of California, San Francisco (USA) | Clinical Science (2022) | --- | $2,584,000 (DOD) |
| Hanny Al-Samkari, MD | Massachusetts General Hospital (USA) | Clinical Science (2020) | $40,000 | $1,000,000 (NIH) |
| Edda Spiekerkoetter, MD | Stanford University (USA) | Clinical Science (2019) | --- | $1,800,000 (DOD) |
| Marie Faughnan, MD; Douglas Marchuk, PhD; Philippe Marambaud, PhD; S. Paul Oh, PhD; Beth Roman, PhD | University of Toronto (Canada); Duke University (USA); Feinstein Institute for Medical Research (USA); Barrow Neurological Institute (USA); University of Pittsburgh (USA) | Basic Science and Clinical Science (2016) | --- | $10,000,000 (DOD) |
| Amelia Clark, MD | Stanford University (USA) | Clinical Science (2014) | $10,000 | --- |
| Kristen Anton, MS | BioInformatics Service Center, Dartmouth College (USA) | Therapeutic Outcomes (2013) | $165,000 | --- |
| Jonathan Zaroff, MD | Kaiser Foundation (USA) | Prevalance / Undiagnosed (2013) | $30,000 | --- |
| Jeffrey Hoag, MD | Drexel University (USA) | Treatment of Disease (2007) | $50,000 | --- |
| Christine Mummery, PhD | Leiden University Medical Center (Netherlands) | Treatment of Disease (2006) | $50,000 | --- |
| Carmelo Bernabeu, PhD | Centro de Investigaciones Biologica (Spain) | Treatment of Disease (2005) | $50,000 | $500,000 (European Research Agency) |
| Karen Swanson, DO | Mayo Clinic (USA) | Treatment of Disease (2004) | $50,000 | $1,000,000 (FDA Orphan Drug Grant) |
| Stanley Shapshay, MD | Albany Medical Center (USA) | Treatment of Disease (2004) | $5,000 | --- |
| Jamie McDonald, MS, CGC | ARUP Laboratories (USA) | Genetics / DNA Patent Release (2003) | $10,000 | --- |
| Arupa Ganguly, PhD | University of Pennsylvania Genetic Diagnostic Laboratory (USA) | Genetics / DNA Patent Release (2003) | $10,000 | --- |
| Kirk Vandezande | HHT Solutions, Inc. (Canada) | Genetics / DNA Patent Release (2003) | $10,000 | --- |
Clinical Trial Grants
Cure HHT Clinical Trials
Clinical trials drive HHT treatment advancements, testing new therapies to improve patient lives.
The HHT community is in desperate need of effective and safe treatments to improve health and quality of life. Clinical trials test the safety and efficacy of potential drug therapies and treatments as well as new applications for FDA approved drugs and/or treatments.
| Researcher | Clinical Trial Description | Study Year | HHT Funding | Other Funding |
|---|---|---|---|---|
| Randomized Trial for Pazopanib in HHT-Related Bleeding | A Phase II/III randomized, placebo-controlled, double-blind study to evaluate the effects of up to 24 weeks of low dose oral pazopanib on hereditary hemorrhagic telangiectasia (HHT) related epistaxis and anemia. | 2022-2025 | $877,824 (FDA) | |
| Randomized Trial for Pazopanib in HHT-Related Bleeding | A Phase II/III randomized, placebo-controlled, double-blind study to evaluate the effects of up to 24 weeks of low dose oral pazopanib on hereditary hemorrhagic telangiectasia (HHT) related epistaxis and anemia. | 2021-2024 | $350,000 | $5,240,964 (DOD) |
| Pomalidomide for the Treatment of Bleeding in Hereditary Hemorrhagic Telangiectasia | A Phase II randomized, placebo-controlled double-blind study of pomalidomide in patients with hereditary hemorrhagic telangiectasia (HHT) with moderate to severe epistaxis who have anemia and/or require parenteral iron infusions or blood transfusions. Treatment with oral pomalidomide or matching placebo for 24 weeks. | 2019-2023 | --- | $3,638,312 (NIH) |
| North American Study of Epistaxis (NOSE) | Multi-center clinical trial designed to test the efficacy of three nasal spray agents as compared to a placebo in reducing nose bleed frequency, duration and increasing quality of life. | 2010 - 2014 | $180,000 | --- |
Young Investigator Awards
Cure HHT Young Investigator Awards
Fostering the next generation of researchers, these awards support innovative research and career development for early-stage investigators.
The Cure HHT Young Scholar Research Program supports scientists* and clinicians* who show exceptional ability and promise for conducting HHT basic science, clinical science or translational science research. The objective of this program is to foster creative HHT research; enhance early career development of outstanding young investigators; and increase opportunities for the young investigator to recognize our mission to FIND, TREAT and CURE HHT. Cure HHT Young Investigator Awards.
*Residents, Postdoctoral/Clinical Fellows, PhD Candidates or Early Stage Investigator who are within 3 years of completing their terminal research degree or within 3 years of completing their medical residency/sub-specialty fellowship
| Researcher | Institution / Country | Grant Type | Year | Grant Type |
|---|---|---|---|---|
| HHT International Scientific Conference | Young Investigator Travel Awards and Presentation Awards | --- | 2022 | $42,000 |
| HHT International Scientific Conference | Young Investigator Travel Awards and Presentation Awards | --- | 2019 | $20,250 |
| HHT International Scientific Conference | Young Investigator Travel Awards and Presentation Awards | --- | 2017 | $15,500 |
| Veronique Vorselaars, PhD | St. Antonius Hospital (Netherlands) | --- | 2017 | $1,200 |
| HHT International Scientific Conference | Young Investigator Presentation Awards | --- | 2003 - 2015 | $14,000 |
| Hongyu Tian, PhD | Duke University (USA) | Basic Science | 2015 | $30,000 |
| Wan Zhu, PhD | University of California, San Francisco (USA) | Basic Science | 2015 | $30,000 |
| Simon Tual-Chalot, PhD | Newcastle University (UK) | Basic Science | 2015 | $30,000 |
| Whitney Wooderchak-Donahue, PhD | University of Utah / ARUP Laboratories (USA) | Genetics | 2015 | $30,000 |
| Chadwick Davis, PhD | Recursion Pharmaceuticals (USA) | Drug Therapy | 2015 | $30,000 |
| Pinar Bayrak-Toydemir, MD, PhD | University of Utah / ARUP Laboratories (USA) | Genetics | 2008 | $20,000 |
| Sebastian Velthius, MD, PhD | St. Antonius Hospital (Netherlands) | --- | 2014 | $2,000 |
| Sabine Bailly, MD | CEA-Grenoble (France) | NIH Travel Award | 2006 | $1,000 |
| Terri DiMaio, PhD | University of Wisconsin (USA) | NIH Travel Award | 2006 | $1,000 |
| Africa Fernandez, PhD | Centro de Investigaciones Biologicas (Spain) | NIH Travel Award | 2006 | $1,000 |
| Eric Finkelstein, PhD | Syracuse University (USA) | NIH Travel Award | 2006 | $1,000 |
| Tassilo Forg, PhD | University of Applied Sciences (Germany) | NIH Travel Award | 2006 | $1,000 |
| Arupa Ganguly, PhD | University of Pennsylvania (USA) | NIH Travel Award | 2006 | $1,000 |
| Fatima Govani, PhD | University of Oxford (UK) | NIH Travel Award | 2006 | $1,000 |
| Jose Miguel Lopez-Novoa, PhD | Universidad de Salamanca (Spain) | NIH Travel Award | 2006 | $1,000 |
| Mourad Toporsian, PhD | McGill University (Canada) | NIH Travel Award | 2006 | $1,000 |
| Liana Zucco | University College London (UK) | NIH Travel Award | 2006 | $1,000 |
Robert I. White Awards
Robert I. White Awards
Recognizing the dedication and compassion of early-career clinicians, honoring those who embody Dr. White's unwavering commitment to advancing care and research in the field.
This award was established in recognition of Dr. Robert I. White Jr.’s outstanding contribution to HHT care and to mentoring other clinicians to develop expertise in HHT care and research. This award is given annually to a clinician (physician or health professional), who is within the first years of work involving HHT and who embodies Dr. White’s qualities as a compassionate and dedicated clinician, devoted to improving HHT care.
| Researcher | Institution / Country | Award Year | HHT Funding |
|---|---|---|---|
| Hanny Al-Samkari, MD | Massachusetts General Hospital (USA) | 2022 | $,2500 |
| Vivek Iyer, MD, MPH | Mayo Clinic (USA) | 2019 | $2,500 |
| Steven Hetts, MD | University of California, San Francisco (USA) | 2017 | $2,500 |
| Marcelo Serra, MD | Hospital Italiano de Buenos Aires (Argentina) | 2015 | $2,500 |
| Justin McWilliams, MD | University of California, Los Angeles (USA) | 2011 | $2,500 |
| Takeo Nishida, PhD | Yale University (USA) | 2010 | $2,500 |
| Els Marie De Gussem, MD | Grace Hospital (Canada) | 2009 | $2,500 |
| Sophie Dupuis-Girod, MD | CHU de Lyon (France) | 2008 | $2,500 |
| Roberto Zarrabeitia, MD | Hospital Sierrallana (Spain) | 2007 | $2,500 |
| Pinar Bayrak-Toydemir, MD, PhD | University of Utah / ARUP Laboratories (USA) | 2006 | $2,500 |
| Gaetan Lesca, MD | CHU de Lyon (France) | 2005 | $2,500 |
| Tom Letteboer, MD, PhD | University Medical Centre Utrecht (Netherlands) | 2004 | $2,500 |
Research Conference Awards
Research Conference Awards
Cure HHT connects and unites the HHT community worldwide through events and gatherings, igniting knowledge and collective action.
Cure HHT has long acted as a catalyst by making personal connections within the relevant communities and hosting scientific and medical conferences. To date, Cure HHT has partnered with NAVBO, NIH, CDC, American Academy of Otolaryngology and many other organizations to educate physicians and scientists about HHT.
| Event | Institution / Country | Award Year | HHT Funding |
|---|---|---|---|
| HHT International Scientific Conference | Conference Host (Portugal) | 2022 | TBD |
| Christopher McMahon Memorial International HHT Guidelines Conference | Conference Host (Canada) | 2019 | $175,000 |
| HHT International Scientific Conference | Conference Host (Puerto Rico) | 2019 | $27,000 |
| HHT International Scientific Conference | Conference Host (Croatia) | 2017 | $61,500 |
| North American Vascular Biology Organization (NAVBO) | Sponsor - HHT Symposia | 2016 | $5,000 |
| HHT International Scientific Conference | Conference Host (USA) | 2015 | $33,000 |
| HHT International Scientific Conference | Sponsor - Speaker Travel Awards | 2001 - 2013 | $70,500 |
| North American Vascular Biology Organization (NAVBO) | Sponsor - HHT Symposia | 2014 | $10,000 |
| North American Vascular Biology Organization (NAVBO) | Sponsor - HHT Genetics Research Presentation | 2009 | $10,000 |
| National Institutes of Health (NIH) | Sponsor - Brain AVM Presentation | 2008 | $2,000 |
| North American Vascular Biology Organization (NAVBO) | Sponsor - HHT Symposia | 2007 | $2,000 |
| HHT Clinical Guidelines Conference | Sponsor - Expert Travel Awards | 2006 | $60,000 |
| National Institutes of Health | Sponsor - Physician Training | 2006 | $5,000 |
Acknowledgments
We extend our heartfelt thanks to the broad community that supports HHT research. Your contributions, from groundbreaking scientific discoveries to generous donations, are the driving force behind our progress.
Each study funded, every piece of new knowledge gained, and the continuous improvement in patient care wouldn't be possible without your unwavering support. Thank you for being an integral part of this journey toward understanding, treating, and ultimately curing HHT.
Calling All Researchers
If you are a HHT researcher, clinician or fellow, learn more about our awards and grants designed to help you further your research and career in the HHT field.
How to Get Involved - information for researchers interested in applying for funding and for patients looking to participate in clinical trials.