The Food and Drug Administration granted marketing clearance Friday for Hizentra, a treatment for chronic inflammatory demyelinating polyneuropathy (CIDP) developed by CSL Behring. Lisa Butler, executive director of the GBS/CIDP Foundation International, said “The approval of Hizentra offers patients who were once burdened by traveling to the infusion center or hospital the flexibility to self-administer their treatment at a time, place, and on a schedule that’s convenient for them.”
The GBS|CIDP Walk & Roll program is a great opportunity to raise awareness, build a local support network, and get involved with GBS|CIDP community. This year we are pleased to announce that funds raised through the Walk & Roll program will be used to support research studies focused on the treatment and diagnosis for GBS|CIDP and variants of the condition such as MMN. Funds raised through our walk program will help to support our research initiatives including:
- Quicker Diagnosis – Developing methods to more rapidly diagnose GBS, CIDP and its variants, such as MMN.
- Better Treatments – Developing more effective treatments to limit the disease progression, prevent complications and reduce long-term adversities.
- Genetics – Identifying mechanisms involved in the pathogenesis of family disorders.
Interested in attending a GBS|CIDP Walk & Roll? Here is the 2018 schedule (more dates to come). Interested in starting a walk in a city near you? See the packet for more information and details on hosting a walk. Please feel free to contact Walk Coordinator, Jessica McManus at the Foundation, with any questions.
(#1) Title of the project: Enhance Peripheral Nerve Repair by Modulating Macrophage Subsets
Gang Zhang, M.D; Ph.D
Assistant Professor of Neurology
University of Texas, Health Sciences Center at Houston
Intravenous immunoglobulin (IVIg) is now the first-line therapy for Guillain-Barré syndrome (GBS). However, there are many disadvantages including high cost, supply shortages, and multiple side effects that are usually associated with high dose and long infusion time of IVIg. Therefore, new therapeutic strategies that can limit the nerve injury during the acute phase of the disease and enhance repair during recovery period are highly desirable. It is in this context we propose a novel strategy of modulating macrophage polarization (promote M2 polarization) for treating GBS.
(#2) Title of the project: Probing the role of skin biopsy in CIDP nodo-paranodopathies
Raffaella Lombardi, B.S., Fondazione IRCCS Instituto Neurologico C. Besta, Milan, Italy
Jerome Devaux, Ph.D,
Diego Franciotta, M.D., Ph.D,
Giuseppe Lauria, M.D.1
IgG4 antibodies against some proteins at the node of Ranvier, anti-neurofascin 155 (Nfasc155) or anti-contactin 1 (CNTN1) have recently been identified in the serum of a subset of CIDP patients. Identification of specific changes in myelinated dermal skin nerves and correlation with clinical course and serological data are unknown. We aim addressing these issues to provide new biomarkers of disease activity and patient stratification.
(#3) The Ernest Hayden Award
Title of the project: Flavivirus and Arbovirus associated Guillain-Barré syndrome in South and South-East Asia.
Dr. Thirugnanam Umapathi
Department of Neurology, National Neuroscience Institute (NNI), Singapore,
Dr Say Saysavath, Mittaphab Hospital; Dr Somchit Vorachit, Setthathirath Hospital,Vientiane,
Laos. Dr Hoang Nghia, Vietnam 175 Hospital, Ho Chi Minh City, Vietnam,Dr Surat Tanprawate, Chiangmai University, Thailand, Dr Mohammad Wasay; Dr Sara Khan, Aga Khan University, Karachi, Pakistan, Dr Terrence Thomas, KKH Children’s Hospital, Singapore,Dr Hugh Willison, Glasgow University, UK Dr Bart Jacobs, Erasmus University, Rotterdam, Netherlands
Dr Ooi, Eng Eong, Duke-NUS, Singapore, Dr Lisa F.P. Ng, A*Star Singapore.
Lao-Oxford-Mahosot Hospital Wellcome Trust Research Unit’s (LOMWRU), Vientiane, Laos
This is a prospective, observational multi-center study of GBS in six S SEA countries. GBS patients and hospital, community controls will be tested for evidence of recent infection and serological response to previous/coinfections with flavi/arbovirus.
For information regarding Grants please contact Lori.Basiege@gbs-cidp.org.
In 2008 the GBS|CIDP Foundation awarded a $60,000 to Ingemar S. J. Merkies, MD, PHD Universiteit Maastricht(The Netherlands), Professor Pieter A. van Doorn (GMAB member) Erasmus MC (The Netherlands) and Richard A. Lewis, MD (GMAB member), Cedars Sinai, for their study in Peripheral Neuropathy Outcome Measures Standardisation (PeriNomS). On February 20, 2018 a paper was published with the results of this study entitled, Quality of Life in Inflammatory Neuropathies: the IN-QoL, in the Journal of Neurology, Neurosurgery and Psychiatry.
February 16, 2018
Hansa Medical today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to IdeS (INN: Imlifidase) for the treatment of Guillain-Barré syndrome (GBS).
Hansa Medical, a Swedish company, is developing a drug, IdeS, which inactivates IgG. This inactivation would be like an instant plasmapheresis treatment. In February 2017, preclinical data from Hansa Medical showed that in an animal model of GBS, inactivation of IgG by IdeS treatment significantly promoted the recovery and reduced the degeneration of peripheral nerves. These data suggest that treatment with IdeS could potentially become a novel therapeutic strategy for the treatment of GBS. Several members of our GMAB are working with Hansa Medical to develop a study in GBS
More information about the ODD has been posted on .