May 16, 2020

Rare Disease Neurofibromatosis

Date and time

May 15, 2020 ~ May 15, 2020

Collaborations That Transform: Rare Disease Neurofibromatosis

View the full program recording HERE.

In honor of World Neurofibromatosis (NF) Awareness Month, the WuXi AppTec community is proud to join the millions around the world working to enhance our understanding of this rare disease. To this end, on May 15 WuXi AppTec – in close collaboration with the Children’s Tumor Foundation and other vital partners – convened a multi-track webinar series on neurofibromatosis, rare disease research awareness, industry collaborations and innovations. On the day of the premier, both tracks drew over 700 Zoom registrants from 22 countries and regions. Additionally, the 2 tracks were broadcast via international web platforms which brought a viewership of more than 1.3 million.

WuXi AppTec’s focus on NF awareness stems from our broader aspiration to work collaboratively to solve industry-wide problems and bring transformational medicines and treatments to patients, including those with rare diseases like neurofibromatosis. Scroll down for event details and follow us on social media to stay up-to-date on our “Collaborations that Transform” webinar series.

Agenda

10:00 AM - 10:05 AM Welcome
Hui Cai, VP and Head of Content, WuXi AppTec
10:05 AM - 11:00 AM Track One: Perspectives from Clinicians and Patients
This session highlights the important roles patients and healthcare professionals play in biomedical advances, and together with researchers, drug developers, and advocacy groups, collaborate to benefit neurofibromatosis and rare disease patients worldwide.

Bruce Korf, Associate Dean for Genomic Medicine, UAB School of Medicine; Chief Genomics Officer, UAB Medicine
Renie Moss, NF1 patient advocate and family member
Jaishri Blakeley, The Marjorie Bloomberg Tiven Professor of Neurofibromatosis, Johns Hopkins University School of Medicine; Director, the Neurofibromatosis Therapeutic Acceleration Program
McKinnon Galloway, NF2 patient
Gareth Evans, Professor of Medical Genetics and Cancer Epidemiology, The University of Manchester
Dale Berg, Schwannomatosis patient
Hosted by Luke Timmerman, Founder and Editor, Timmerman Report
11:00 AM - 12:00 PM Track Two: Non-Traditional Research & Development: The Making of the Breakthrough Therapy KOSELUGO® (Selumetinib) for NF1 and the Promise of Brigatinib to Treat NF2
KOSELUGO® (selumetinib), a MEK inhibitor from AstraZeneca and MSD (Merck in the US), was approved by the U.S. FDA in April 2020 as the first NF1 drug to treat children with plexiform neurofibromas, while Brigatinib is under clinical evaluation for the treatment of NF2 through a mechanism that is distinct from ALK activity. In both cases, these drugs were discovered through collaborative, non-traditional conduits.

Scott Plotkin, Neuro-Oncologist; Executive Director, Pappas Center for Neuro-Oncology, Massachusetts General Hospital; Professor of Neurology, Harvard Medical School
Annette Bakker, President, Children’s Tumor Foundation
George Kirk, Koselugo Global Product Leader, AstraZeneca
Brigitte Widemann, Chief, Pediatric Oncology Branch, NCI, NIH; Head, Pharmacology and Experimental Therapeutics Section
Mike Humphries, US Medical Affairs Scientific Director for Lung Cancer, Takeda Oncology
Moderated by Richard Soll, Head of Boston Office and Senior Advisor of Strategic Initiatives, WuXi AppTec

Speakers

Hui Cai
Forum Chair, VP and Head of Content Division, WuXi AppTec
Bruce Korf
Associate Dean for Genomic Medicine, UAB School of Medicine; Chief Genomics Officer, UAB Medicine
Renie Moss
NF1 patient advocate and family member
Jaishri Blakeley
The Marjorie Bloomberg Tiven Professor of Neurofibromatosis, Johns Hopkins University School of Medicine; Director, the Neurofibromatosis Therapeutic Acceleration Program
McKinnon Galloway
NF2 patient
Gareth Evans
Professor of Medical Genetics and Cancer Epidemiology, The University of Manchester
Dale Berg
Schwannomatosis patient
Luke Timmerman
Founder and Editor, Timmerman Report
Scott Plotkin
Neuro-Oncologist Executive Director, Pappas Center for Neuro-Oncology, Massachusetts General Hospital Professor of Neurology, Harvard Medical School
Annette Bakker
President, Children’s Tumor Foundation
George Kirk
Koselugo Global Product Leader, AstraZeneca
Brigitte Widemann
Chief, Pediatric Oncology Branch, NCI, NIH; Head, Pharmacology and Experimental Therapeutics Section
Mike Humphries
US Medical Affairs Scientific Director for Lung Cancer, Takeda Oncology
Rich Soll
Head of Boston Office and Senior Advisor of Strategic Initiatives, WuXi AppTec