to NextGen Conference 2017
Friday 13th of October: 9 AM
to
Sunday 15th of October: 12 PM
Mason & Rook Kimpton Hotel
1430 RHODE ISLAND AVENUE NW
WASHINGTON, DC 20005
Map & Driving directions are here.
Quick Links
- Goals
- Conference Schedule & Reception
- Conference Participants
- Conference Organizers
- Scientific Program Advisors
- Conference Supporters
Goals
The primary goal of this conference is to accelerate drug development for Systemic JIA, MAS, and related diseases like NLRC4 mutation by bringing together scientists, patients and pharmaceutical companies to discuss drugs coming down the pipeline for SJIA/MAS or already in clinical trials for related diseases.
A second focus for 2017 is Lung Complications in SJIA. Lung issues are not textbook complications of SJIA, but in recent years there has been an increase in the number of children diagnosed with Interstitial Lung Disease, Pulmonary Alveolar Proteinosis and / or Lung infections. Such cases can have a high mortality rate (Kimura et al., 2012). Our goal at this conference is to develop some shared understanding of how to treat these cases.
Participants will include
- Scientists focused on finding treatments for SJIA/ MAS / Stills Disease and related disease like NLRC4 mutation
- Pulmonary & other experts who can speak to Lung Disease in SJIA
- Parents of children with SJIA / MAS
- Scientists from pharmaceutical companies
- Young upcoming scientists
Conference Schedule & Reception
SJIA Therapies: Sessions will cover both current and upcoming therapies such as IL1, IL6, IL18BP & IL17, Mesenchymal Stem Cells, Thalidomide, Less Toxic Steroids such as Vamorolone and Jak Inhibitors such as Tofacitinib.
It will also cover MAS Therapies such as Anakinra, Interferson Gamma Blocker & Etoposide.
Sessions on Lung Complications on SJIA will cover review of such cases from Cincinnati & Stanford, including hearing from clinicians treating such cases and some parents. It will also cover Lung Disease in other Rheumatic diseases such as Rheumatoid Arthritis and Myositis.
Discussion on treating such cases will cover immunosuppression, any change in usage of biologics, prophylaxysis for lung infections, therapies for Lung Clearance such as those used in Cystic Fibrosis.
SJIA Foundation Cocktail Reception: There will be a networking reception with drinks & food and on evening of Friday, October 13th at the Mason & Rook Hotel. It will include not just attendees of the NextGen Conference but also anyone local interested in all Autoinflammatory Diseases. This could include local physicians and other local experts in such diseases, patients & parents, representatives from FDA and other Rare Disease Organization. The invitation for the reception is here: https://nextgen_reception.eventbrite.com
Conference Participants:
Physicians
- Ed Behrens: Profile | Publications
- Fabrizio De Benedetti: Profile | Publications
- Claudia Bracaglia: Profile | Publications
- Scott Canna: Profile | Publications
- Randy Cron: Profile | Publications
- Sonye Danoff: Profile | Publications
- Gail Deutsch: Profile | Publications
- Dirk Foell: Profile | Publications
- Alexei Grom: Profile | Publications
- Annacarin Horne: Profile | Publications
- Christof Kessel: Profile | Publications
- Raju Khubachandani (Remote): Profile | Publications
- Michael Matthay (Remote): Profile | Publications
- Betsy Mellins: Profile | Publications
- Peter Nigrovic: Profile | Publications
- Michael Ombrello: Profile | Publications
- Sampath Prahlad: Profile | Publications
- Giusi Prencipe: Profile | Publications
- Suhas Radhakrishna: Profile | Publications
- Mona Riskalla: Profile | Publications
- Vivian Saper: Profile | Publications
- Joost Swart: Profile | Publications
- Grant Schulert: Profile | Publications
- Nora Singer: Profile | Publications
- Chris Towe: Profile | Publications
- Bas Vastert: Profile | Publications
- Lehn Weaver: Profile | Publications
- Jason Woods: Profile | Publications
Patient Families
- Rashmi Sinha
- Jon Boutelle
- Leah Nicholson Bush
- Kari Cupp
- Frank Giamie
- Sandra Giamie
- Samantha Lyn Spears
- Alex Spears
- Lucian Papaleo Peixoto
- Sheila Taylor
- Mindy Nowak
- Dr. Jeff Jellin
- Julie Allott
Pharma
- Kathleen Lomax, Novartis
- Cristina de Min, Novimmune
- Shephard Mpofu, Novartis
- Suzanne Crowley, Sobi
- Christina Ström Möller, Sobi
- Claudia Pena Ross, Sanofi
- Andrew Sleight, AB2Bio
- Gene Wang, Novartis
FDA
- Larissa Lapteva, FDA
- Beth Fritsch, FDA
Others
- James Valentine, Hyman Phelps & McNamara
- Karen Durrant, Autoinflammatory Alliance
- Jen Horonjeff, Savvy Coop
- Jason Borbour, Simpatica
Video & Presentation
| Day 1 | |
|---|---|
| Laurie Conklin | NextGen Steroids: Vamorolone |
| Raju Khubchandani | ThalidomideÊ-ÊtheÊ'Soap'ÊContinues |
| Alexei Grom (on behalf of Hermine Brunner) | Jak Inhibitors: Safety of Tofacitinib |
| James Valentine | Lunch talk: Bringing the Patient Voice to Drug Development: Opportunities for parents, investigators and drug companies |
| Cem Gabay | A Phase II, dose escalating clinical trial with Tadekinig alfa in Still's disease |
| Randy Cron | A Randomized and Double-Blinded Clinical Trial of Recombinant Human Interleukin-1 Receptor Antagonist to Treat MAS in Children and Adults |
| Fabrizio DeBenedetti | Interferon Gamma Blocker in MAS |
| AnnaCarin Horne | Our experience of Etoposide in children with MAS-HLH |
| Ed Behrens | IL-33 controls pathogenic IFNg in HLH |
| Nora Singer | Cell based therapies: anticipated challenges in sJIA trial design and US regulatory burden |
| Micheal Ombrello & Peter Nigrovic | Innate vs Adaptive Immune System in SJIA and what it means for Therapies |
| Beth Fritsch | How We Involve Patients and Advocates |
| Day 2 | |
| Christopher Kessel | IL-17 producing gdT cells as kick-starters of inflammation in SJIA |
| Vivian Saper & Beth Mellins | Lung Disease in sJIA: 59 Cases |
| Grant Schulert & Chris Towe | The Spectrum of sJIA Associated Lung Disease |
| Scott Canna | Cases followed at NIH |
| Gail Deutsch | The Pathology of Lung Disease in sJIA |
| Sonye Danoff | Lung Disease in Rheumatoid Arthritis & Dermatomyositis (Sonye Danoff) |
| Raphaela Goldbach-Mansky | Insights about Lung Disease from SAVI (Raphaela Goldbach-Mansky) |
Conference Organizers:
Rashmi Sinha & Jon Boutelle, founders: SJIA Foundation
About the SJIA Foundation: The conference is organized by the Systemic JIA Foundation as part of their efforts is to find a cure for Systemic JIA. The foundation takes a startup approach to finding new treatments – organizing conferences, conducting surveys, making seed grants, creating web-based registries, working with researchers and parents to accelerate the search for a cure. This conference is inspired by drug-discovery conferences by other rare disease foundations which have successfully accelerated drug development by bringing together patients, researchers, pharma and regulators.
Rashmi Sinha is a scientist, entrepreneur and the mother of a child with SJIA. She has a PhD in Cognitive Neuropsychology and has run web companies in the past.
Jon Boutelle is a technologist, entrepreneur and father of a child with SJIA. He has a degree in Computer Science, has worked in Silicon Valley and was founder & CTO of SlideShare.
Jon & Rashmi’s single-minded focus is to find new treatments for refractory cases and ultimately a cure for SJIA.
Scientific Program Advisors:
Dr.Alexei Grom, Dr. Betsy Mellins, Dr. Fabrizio DeBenedetti
Dr. Alexi Grom is Professor at Cincinnati Childrens Hospital. He is Research Director, Division of Rheumatology. Profile page | Publications on Pubmed
Dr. Betsy Mellins is Professor of Pediatrics at Lucille Packard Children’s Hospital at Stanford. Her Stanford profile | Publications on pubmed
Dr. Fabrizio DeBenedetti is Head of Rheumatology, Pediatrics at Ospedale Pediatrico Bambino Gesù. Profile on ResearchGate | Publications on pubmed
Conference Supporters:
Contact US:
All questions about the conference should be directed to Rashmi Sinha: email.
3584 Outlook Avenue, Cincinnati OH, 45208