Tenth Cooley's Anemia Symposium

Tenth Cooley's Anemia Symposium

Sunday, October 18, 2015 - Thursday, October 22, 2015

Loews Chicago O'Hare Hotel

Presented By

Presented by the Cooley's Anemia Foundation and the New York Academy of Sciences

 

Since the Ninth Cooley's Anemia Symposium was held in 2009, all areas of thalassemia research have sustained major advances. The 2015 Symposium will bring together basic scientists, clinical investigators, and clinicians, for interactions and education. Areas of focus will include the biology of globin gene regulation and fetal hemoglobin induction; the evolving areas of stem cell transplant, gene therapy strategies, and gene editing; the biology of iron regulation and possible therapeutic interventions in the hepcidin regulatory system; and clinical issues in thalassemia treatment and imaging. Leading investigators, together with physicians involved in thalassemia care from around the world, will be featured speakers.  Interactive poster sessions will allow trainees involved in all of these research areas to present their work.

Since the inaugural meeting jointly presented by the Cooley's Anemia Foundation and the Academy in 1964, this series of symposia convened at 5–6 year intervals has been the preeminent forum for convening the thalassemia research community, the pharmaceutical industry, and funding agencies dedicated to fighting this blood disorder, and the 2015 meeting will carry on this proud tradition.

Registration Pricing

 

By 8/28/2015

After 8/28/2015

Onsite

Member

$490

$590

$650

Student/Postdoc Member

$300

$350

$400

Nonmember (Academia/Government)

$615

$715

$775

Nonmember (Corporate)

$850

$950

$1,050

Nonmember (Non-profit)

$615

$715

$775

Nonmember (Student/Postdoc/Fellow)

$325

$375

$425

Patient

$350

$350

$350

Day Rate

$350

$400

$425

Welcome Reception ONLY

$30

$30

$30

Farewell Luncheon ONLY

$18

$18

$18

 

Registration fee for the entire five-day symposium includes a complimentary, one-year individual membership to the New York Academy of Sciences. Complimentary memberships are provided to non-members only and cannot be used to renew or extend existing or expiring memberships. A welcome email will be sent upon registration, which will include your membership credentials.

Proceedings will be published as a volume of Annals of the New York Academy of Sciences. Registration fee for the entire five-day symposium includes one free copy of the symposium proceedings for this event.

Please note:

  • The Day Rate registration fee is available for participation on October 19, 20, or 21 only.
  • Patients are welcome to attend the entire five-day symposium for a flat registration fee of $350. For more information and to register, please contact Mary Woldegiorgis, Cooley's Anemia Foundation, at mwoldegiorgis@thalassemia.org or call 212.279.8090 Ext. 208.
  • Complimentary Membership and a free copy of the symposium proceedings are not included for Patient and Day Rate registration categories.

 

Image Credit: Wave of Hope, Wainwright Media Group.

Agenda

* Presentation titles and times are subject to change.


Day 1 — Sunday, October 18, 2015

5:00 PM

Registration
Location:
Artist Foyer

6:00 PM

Welcome Reception
Location
: Cassatt Ballroom

8:00 PM

Day 1 Adjourns

Day 2 — Monday, October 19, 2015

7:00 AM

Registration
Location
: Artist Foyer

7:00 AM

Breakfast
Location
: Cassatt Ballroom

8:40 AM

Welcome Remarks
Location
: Avedon Ballroom C/D

Brooke Grindlinger, PhD, The New York Academy of Sciences
Anthony J. Viola, BS, CPA, National President, Cooley’s Anemia Foundation
Ellis J. Neufeld, MD, PhD, Dana-Farber/Children’s Cancer and Blood Disorders Center; Harvard Medical School

9:15 AM

Joint Session
Science Keynote Address

Stuart H. Orkin, MD, Boston Children’s Hospital and Dana-Farber Cancer Institute

9:45 AM

Keynote Address Q & A

10:10 AM

Networking Coffee Break

Joint Session I: Gene Therapy

Location: Avedon Ballroom C/D
Session Chair: Alexis A. Thompson, MD, MPH, Ann and Robert H. Lurie Children’s Hospital of Chicago, Northwestern University Feinberg School of Medicine

10:30 AM

Gene Therapy for Beta-Thalassemia Major. The Memorial Sloan Kettering Experience
Farid Boulad, MD, Memorial Sloan Kettering Cancer Center

10:55 AM

Outcomes of Gene Therapy for β-Thalassemia Major via Transplantation of Autologous Hematopoietic Stem Cells Transduced ex vivo with a Lentiviral βA-T87Q-Globin Vector
Marina Cavazzana, MD, PhD, Hospital Necker, University Paris Descartes

11:20 AM

Toward a Gene Therapy Clinical Trial for the Treatment of Beta-Thalassemia Major at Cincinnati Children’s Hospital Medical Center
Punam Malik, MD, Cincinnati Children’s Hospital

11:45 AM

Update on Gene Therapy Clinical Trial for the Treatment of Beta-Thalassemia Major in Italy
Giuliana Ferrari, PhD, San Raffaele-Telethon Institute for Gene Therapy

12:10 PM

Networking Lunch
Location
: Cassatt Ballroom

Concurrent Session 2A: Gene Regulation 1

Location: Avedon Ballroom A/B
Session Chair: Stephen A. Liebhaber, MD, Perelman School of Medicine at the University of Pennsylvania

1:30 PM

Understanding the Regulation of α-globin Gene Expression and its Implication for the Treatment of β-Thalassemia
Douglas R. Higgs, MD, FRCP, FRS, University of Oxford, Weatherall Institute of Molecular Medicine

1:55 PM

Humanized Mouse Models of Cooley’s Anemia
Thomas M. Ryan, PhD, The University of Alabama at Birmingham

2:20 PM

Modulation of the Pathways that Control HbF Synthesis
Vijay Sankaran, MD, PhD, Boston Children’s Hospital and Dana-Farber Cancer Institute

2:45 PM

Long Range Enhancer Control of Beta-globin Genes
Ann Dean, PhD, National Institutes of Health

Concurrent Session 2B: Clinical Management 1

Location: Avedon Ballroom C/D
Session Chair: Elliott Vichinsky, MD, UCSF Benioff Children’s Hospital Oakland

1:30 PM

Nutrition for Patients with Thalassemia
Ellen B. Fung, PhD, RD, UCSF Benioff Children’s Hospital Oakland

2:00 PM

An ‘Aging’ Population of Adults with Transfusion-dependent Thalassemia (TDT)
John B. Porter, MD, University College London Hospitals

2:30 PM

Diagnostic Challenges: Carrier vs. Thalassemia Intermedia
Antonio Piga, MD, Torino University

3:10 PM

Comfort Break

Concurrent Session 3A: Gene Regulation 2

Location: Avedon Ballroom A/B
Session Chair: James Bieker, PhD, Mount Sinai School of Medicine

3:30 PM

Regulation of HbF Synthesis
Swee Lay Thein, MD, National Institutes of Health

4:00 PM

CRISPR/Cas-enhanced Gene Replacement for Sickle Cell Disease
Tim M. Townes, PhD, School of Medicine, The University of Alabama at Birmingham

4:30 PM

The LRF/ZBTB7A Transcription Factor Regulates BCL11A-independent Globin Switching
Takahiro Maeda, MD, PhD, Brigham and Women’s Hospital

Concurrent Session 3B: Clinical Management 2

Location: Avedon Ballroom C/D
Session Chair: Richard Ward, MD, Toronto General Hospital

3:30 PM

Diverse Hematological Phenotypes and Screening for β-Thalassemia Carriers
David H. K. Chui, MD, Boston University School of Medicine

3:55 PM

Cardiac Complications in Thalassemia
Dudley Pennell, MD, Imperial College London

4:20 PM

When to Transfuse in Patients with Non-transfusion–dependent Thalassemia (NTDT)
Vip Viprakasit, MD, DPhil, Faculty of Medicine Siriraj Hospital, Mahidol University

4:45 PM

Assessment and Treatment of Pain in Thalassemia
Ashutosh Lal, MD, UCSF Benioff Children’s Hospital Oakland

5:10 PM

Day 2 Adjourns

Day 3 — Tuesday, October 20, 2015

6:30 AM

Breakfast
Location
: Cassatt Ballroom

6:30 AM

Poster Session Set-up
Location
: Artist Foyer

Concurrent Breakfast Sessions

7:00 AM

SESSION A
Location
: Avedon Ballroom C/D

Studies of Fetal Hemoglobin Regulation in Cultured Human Erythroblasts
Jeffery L. Miller, MD, Molecular Medicine Branch, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health

New Insights of Ineffective Erythropoiesis in β-Thalassemia: Therapeutic Implications
Olivier Hermine, MD, PhD, Hospital Necker, University Paris Descartes

7:00 AM

SESSION B
Location
: Cassatt Ballroom A/B

Focus on Adherence: Facilitators and Barriers
Kathleen Durst, MA, LCSW, Cooley’s Anemia Foundation

Self-efficacy and Adherence with Iron Chelation Therapies: Motivational Interviewing Interventions
Marsha J. Treadwell, PhD, UCSF Benioff Children’s Hospital Oakland

8:00 AM

Comfort Break

Joint Session: Clinical Keynote

Location: Avedon Ballroom C/D

8:45 AM

Clinical Keynote Address: Clinical Features of Thalassemia Syndromes in 2015
Maria Domenica Cappellini, MD, FRCP, FACP, University of Milan, Ca Granda Foundation IRCCS

9:30 AM

Keynote Address Q & A

9:45 AM

Networking Coffee Break
Location
: Artist Foyer

Joint Session 4: Chelation

Location: Avedon Ballroom C/D
Session Chair: Alan R. Cohen, MD, The Children’s Hospital of Philadelphia

10:15 AM

How Do We Choose Target Iron Levels?
Thomas D. Coates, MD, Childrens Hospital Los Angeles, University of Southern California Keck School of Medicine

10:45 AM

Evolving Concepts in Safety, Efficacy, and Side Effects of Iron Chelators
Ellis J. Neufeld, MD, PhD, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center; Harvard Medical School

11:15 AM

Current Recommendations for Chelation Therapy
Janet L. Kwiatkowski, MD, MSCE, The Children’s Hospital of Philadelphia; Perelman School of Medicine, University of Pennsylvania

11:45 PM

Networking Lunch
Location
: Cassatt Ballroom

11:45 PM

Mentoring Lunch for Women, Underrepresented Minorities, and Early Career Investigators
Location: Cassatt Ballroom
Participants in the Mentoring Lunch are invited to join select Symposium speakers for an interactive, small group dialogue at tables marked “Reserved for Mentoring Lunch.”

Joint Session 5: Imaging

Location: Avedon Ballroom C/D
Session Chair: John C. Wood, MD, PhD, Children’s Hospital Los Angeles

1:20 PM

Imaging Assessment of Organs in Cooley’s Anemia with an Emphasis on MRI
David Dinan, MD, Nemours Children’s Hospital

1:50 PM

Evolving MRI Techniques and Application to Iron Detection
Scott B. Reeder, MD, PhD, University of Wisconsin School of Medicine and Public Health

2:20 PM

Expanding our Understanding of Organ Iron Overload — Evidence for Broader Use of MRI in Iron Detection
John C. Wood, MD, PhD, Children’s Hospital Los Angeles

2:50 PM

Networking Coffee Break
Location
: Artist Foyer

Joint Session 6: Iron Regulation/Metabolism

Location: Avedon Ballroom C/D
Session Chair: Mark D. Fleming, MD, DPhil, Boston Children’s Hospital

3:10 PM

Iron-related Pathways Controlling Hepcidin Expression
Jodie L. Babitt, MD, Massachusetts General Hospital

3:40 PM

Non-liver Regulation of Iron Metabolism: Kidney and Heart
Jonathan M. Barasch, MD, PhD, Columbia University

4:10 PM

Clinical Disorders of Iron Metabolism: New Insights into Diseases of Hepcidin Dysregulation
Mark D. Fleming, MD, DPhil, Boston Children’s Hospital

4:50 PM

Poster Session
Location
: Artist Foyer
Authors stationed at posters from 5:00 PM–6:00 PM

6:30 PM

Day 3 Adjourns (Poster Session posters removed)

Day 4 — Wednesday, October 21, 2015

7:00 AM

Breakfast
Location
: Cassatt Ballroom

7:00 AM

Poster Session Set-up
Location
: Artist Foyer

Joint Breakfast Session

Location: Avedon Ballroom C/D

7:30 AM

Special Clinical Focus: Morbidities in Non-transfusion–dependent Thalassemia (NTDT)
Ali Taher, MD, PhD, FRCP, American University of Beirut Medical Center

8:30 AM

Comfort Break

Joint Session 7: Clinical Management 3

Location: Avedon Ballroom C/D
Session Chair: Ashutosh Lal, MD, UCSF Benioff Children’s Hospital Oakland

9:00 AM

Iron Deficiency, Infection, Inflammation, and Hepcidin Regulation
Hal Drakesmith, PhD, Weatherall Institute of Molecular Medicine, University of Oxford

9:25 AM

Age of Transfused Blood and Blood-borne Infections
Eldad A. Hod, MD, Columbia University

9:50 AM

Management of Thalassemia-associated Osteoporosis
Andrea Giusti, MD, Galliera Hospital

10:15 AM

Hepatitis C Treatment Advances
Steven Flamm, MD, Northwestern Medical Center

10:40 AM

Nonmyeloablative Transplantation in Adults with Severe Sickle Cell Disease
John F. Tisdale, MD, National Heart, Lung, and Blood Institute

11:05 AM

Networking Coffee Break
Location
: Artist Foyer

Joint Session: Best of Oral Poster Presentations

Location: Avedon Ballroom C/D

11:30 AM

Clinical-scale Genome Editing of the Human BCL11A Erythroid Enhancer for Treatment of β-Thalassemia
Fyodor D. Urnov, PhD, Sangamo BioSciences

11:45 AM

Amelioration of Murine Sickle Cell Disease by Non-Ablative Conditioning and Gamma-globin Gene–corrected Bone Marrow Cells
Andrew Wilber, PhD, Southern Illinois University School of Medicine and Simmons Cancer Institute

12:00 PM

Rescue of Humanized Cooley’s Anemia Mice from Lethal Anemia by a Single Point Mutation in the ɣ-Globin Gene Promoter
Suean Fontenard, BS, University of Alabama at Birmingham

Patient Presentation

12:15 PM

Pioneering Gene Therapy – A Patient’s Perspective
Wanda Sihanath

12:30 PM

Networking Lunch
Location
: Cassatt Ballroom

12:30 PM

Poster Session
Location
: Artist Foyer

Authors stationed at posters from 12:30 PM–1:30 PM

Joint Session 8: Transplant

Location: Avedon Ballroom C/D
Session Chair: Mark Walters, MD, UCSF Benioff Children’s Hospital Oakland

1:30 PM

Management of Iron Overload Before, During, and After Successful Transplantation
Emanuele Angelucci, MD, Cancer Hospital “Armando Businco,” Cagliari

2:00 PM

Unrelated Donor Transplantation Using Reduced Intensity Conditioning in Children with Thalassemia Major
Shalini Shenoy, MD, St. Louis Children’s Hospital

2:30 PM

Comfort Break

Joint Session 9: End Organ Damage

Location: Avedon Ballroom C/D
Session Chair: Suthat Fucharoen, MD, Mahidol University Salaya Campus; Thalassemia Research Center, Thailand Institute of Science and Technology

3:00 PM

Pulmonary Hypertension in Thalassemia
Roberto F. Machado, MD, University of Illinois College of Medicine

3:25 PM

Renal Aspects of Thalassemia
Sunil Bhandari, MBChB, MRCP, FRCP, PhD, M Clin Ed, FHEA, Hull & East Yorkshire Hospitals NHS Trust & Hull York Medical School

3:50 PM

Hepatocellular Carcinoma in Thalassemia. An Update of the Italian Registry
Caterina Borgna-Pignatti, MD, University of Ferrara

4:15 PM

Iron and Cancer
Suzy V. Torti, PhD, University of Connecticut Health Center

4:40 PM

Day 4 Adjourns (Poster Session posters removed)

Day 5 — Thursday, October 22, 2015

8:00 AM

Breakfast
Location
: Cassatt Ballroom

Joint Session 10: Emerging Therapies

Location: Avedon Ballroom C/D
Session Chair: Jeffery L. Miller, MD, Molecular Medicine Branch, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health

9:00 AM

Luspatercept (ACE-536) Trial Update
Antonio Piga, MD, Torino University

9:25 AM

Using Murine Thalassemia Models to Evaluate Novel Therapies
Stefano Rivella, PhD, Children’s Hospital of Philadelphia

9:50 AM

Genome Editing Strategies to Induce HbF for the Beta-Hemoglobinopathies
Daniel E. Bauer, MD, PhD, Boston Children’s Hospital; Dana-Farber Cancer Institute; Harvard Medical School

10:15 AM

Hepcidin Regulators
Elizabeta Nemeth, PhD, University of California, Los Angeles

10:40 AM

Networking Coffee Break
Location
: Artist Foyer

Summary and Closing Remarks

Location: Avedon Ballroom C/D

11:00 AM

Symposium Summary
Arthur Nienhuis, MD, St. Jude Children’s Research Hospital

11:30 AM

The Bright Future and the Conundrum of Beta Thalassemia
David G. Nathan, MD, Dana-Farber Cancer Institute

11:50 AM

Concluding Remarks
Ellis J. Neufeld, MD, PhD, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center; Harvard Medical School

12:00 PM

Farewell Luncheon
Location
: Cassatt Ballroom

1:00 PM

Symposium Concludes

Speakers

Chair, Scientific Organizing Committee

Ellis Neufeld, MD, PhD

Boston Children’s Hospital/Harvard Medical School

Organizers

Emanuele Angelucci, MD

Cancer Hospital "Armando Businco" Cagliari

Maria Domenica Cappellini, MD

University of Milan

Gina Cioffi, JD

Cooley's Anemia Foundation

Suthat Fucharoen, MD

Mahidol University Salaya Campus
Thalassemia Research Center, Thailand Institute of Science and Technology

Janet L. Kwiatkowski, MD, MSCE

Children’s Hospital of Philadelphia
Perelman School of Medicine, University of Pennsylvania

Antonio Piga, MD

Torino University

Stefano Rivella, PhD

Children's Hospital of Philadelphia

Ali Taher, MD, PhD, FRCP

American University of Beirut Medical Center

Alexis Thompson, MD, MPH

Northwestern University Feinberg School of Medicine
Ann and Robert H. Lurie Children's Hospital of Chicago

Vip Viprakasit, MD, PhD

Faculty of Medicine Siriraj Hospital, Mahidol University

Speakers and Session Chairs

Emanuele Angelucci, MD

Cancer Hospital "Armando Businco," Cagliari

Jodie L. Babitt, MD

Massachusetts General Hospital

Jonathan M. Barasch, MD, PhD

Columbia University

Daniel E. Bauer, MD, PhD

Boston Children's Hospital
Dana-Farber Cancer Institute
Harvard Medical School

Sunil Bhandari, MBChB, MRCP, FRCP, PhD, M Clin Ed, FHEA

Hull and East Yorkshire Hospitals NHS Trust and Hull York Medical School

James Bieker, PhD

Mount Sinai School of Medicine

Farid Boulad, MD

Memorial Sloan-Kettering Cancer Center

Caterina Borgna-Pignatti, MD

University of Ferrara

Maria Domenica Cappellini, MD

University of Milan

Marina Cavazzana-Calvo, MD, PhD

Hospital Necker, University Paris Descartes

David H.K. Chui, MD

Boston University School of Medicine

Thomas D. Coates, MD

Childrens Hospital Los Angeles, University of Southern California Keck School of Medicine

Alan R. Cohen, MD

The Children's Hospital of Philadelphia

Ann Dean, PhD

National Institutes of Health

David Dinan, MD

Nemours Children’s Hospital

Alexander (Hal) Drakesmith, PhD

Radcliffe Department of Medicine, University of Oxford

Kathleen Durst, MA, LCSW

Cooley's Anemia Foundation

Giuliana Ferrari, PhD

San Raffaele-Telethon Institute for Gene Therapy

Steven Flamm, MD

Northwestern Medical Center

Mark D. Fleming, MD, DPhil

Boston Children's Hospital

Suthat Fucharoen, MD

Mahidol University

Ellen B. Fung, PhD, RD

UCSF Benioff Children's Hospital Oakland

Andrea Giusti, MD

Galliera Hospital

Brooke Grindlinger, PhD

The New York Academy of Sciences

Olivier Hermine, MD, PhD

Hospital Necker, University Paris Descartes

Doug Higgs, FRS

University of Oxford

Eldad Arie Hod, MD

Columbia University

Janet L. Kwiatkowski, MD, MSCE

The Children's Hospital of Philadelphia

Ashutosh Lal, MD

University of California San Francisco Benioff Children's Hospital Oakland

Stephen A. Liebhaber, MD

Perelman School of Medicine at the University of Pennsylvania

Roberto Machado, MD

University of Illinois College of Medicine

Takahiro Maeda, MD, PhD

Brigham and Women's Hospital

Punam Malik, MD

Cincinnati Children’s Hospital

Jeffery L. Miller, MD

National Institute of Diabetes and Digestive and Kidney Diseases

David G. Nathan, MD

Dana-Farber Cancer Institute

Elizabeth (Ella) Nemeth, MD

University of California, Los Angeles

Ellis Neufeld, MD, PhD

Boston Children's Hospital

Arthur Nienhuis, MD

St. Jude Children's Research Hospital

Stuart H. Orkin, MD

Boston Children's Hospital and Dana Farber Cancer Institute

Dudley Pennell, MD

Imperial College London

Antonio Piga, MD

Torino University

John Porter, MA, MD, FRCP, FRC(Path)

University College London Hospitals

Scott Reeder, MD, PhD

University of Wisconsin School of Medicine and Public Health

Stefano Rivella, PhD

Children's Hospital of Philadelphia

Thomas M. Ryan, PhD

The University of Alabama at Birmingham

Vijay Sankaran, MD, PhD

Boston Children's Hospital
Dana-Farber Cancer Institute

Shalini Shenoy, MD

St. Louis Children's Hospital

Ali Taher, MD, PhD, FRCP

American University of Beirut Medical Center

Swee Lay Thein, MD

National Institutes of Health (as of March 2015)

Alexis Thompson, MD, MPH

Robert H. Lurie Comprehensive Cancer Center, Northwestern University
Ann and Robert H. Lurie Children's Hospital of Chicago

John F. Tisdale, MD

National Heart, Lung, and Blood Institute

Suzy V. Torti, PhD

University of Connecticut Health Center

Tim M. Townes, PhD

School of Medicine, The University of Alabama at Birmingham

Marsha J. Treadwell, PhD

Children's Hospital Oakland

Elliot Vichinsky, MD

Children's Hospital Oakland Research Institute

Anthony J. Viola, CPA

Cooley's Anemia Foundation

Vip Viprakasit, MD, PhD

Faculty of Medicine Siriraj Hospital, Mahidol University

Mark Walters, MD

Children's Hospital and Research Center Oakland

Richard Ward, MD

Toronto General Hospital

John C. Wood, MD, PhD

Children's Hospital Los Angeles

Coordinator

Jenna O'Neil

Boston Children's Hospital

Sponsors

For sponsorship opportunities please contact:

Gina Cioffi, JD
National Executive Director
Cooley's Anemia Foundation
gcioffi@thalassemia.org
212.279.8090 Ext. 201

Brooke Grindlinger, PhD
Executive Director Scientific Programs
The New York Academy of Sciences
bgrindlinger@nyas.org
212.298.8625

Platinum Sponsor

Silver Sponsors

Bronze Sponsors

Isis Pharmaceuticals

Novartis

Academy Friends

Immucor

Pfizer

Sangamo BioSciences

Grant Support

Funding for this conference was made possible (in part) by 1R13HL129737-01 from the National Institutes of Health. The views expressed in written conference materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services; nor does mention of trade names, commercial practices, or organizations imply endorsement by the U.S. Government.

Promotional Partners

American Society for Blood & Marrow Transplantation

American Society of Hematology (ASH)

The American Society of Gene & Cell Therapy

Center for International Blood and Marrow Transplant Research (CIBMTR)

Cure Thalassemia

Elsevier Global Events

GTCBio

Nature Publishing Group

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