CINC-NEWSLETTER FEBRUARY 2024
I hope you all had a chance to “share your colours” or those of your patients yesterday on Rare Disease Day.
With the recently created RareKids-CAN: Pediatric Rare Disease Clinical Trials and Treatment Network and MICYRN securing a $20 million CIHR grant for it, the work of CORD, ImmUnity Canada, and other organizations, and federal government announcements throughout last year on supports for research and treatments for rare diseases, plus all the work that you all do on our field, the future seems brighter and we should all jump on this momentum for the benefit of our patients.
SURVEY on behalf of PIDTC
- Survey for clinicians caring for patients with Combined Immunodeficiencies
- To facilitate development of a study population focused on patients with combined immunodeficiencies, we are surveying clinical immunologists that serve patients in this space. Our objective is to determine the approximate number of study participants in specific categories that are available at your institution/in your practice.
We’re specifically seeking patients with proximal TCR signaling defects, which do NOT overlap with SCID as defined in Dvorak et al JACI 2023 or PIDTC protocol 6907, as well as canonical and non-canonical NF-kB signaling defects. The data collected in this survey will allow us to build a U54 grant proposal focused on how the genetics of these defects relate to peripheral blood/tissue immune profiles, clinical presentation, and treatment outcomes. Please note, specific gene defects are listed in the survey.
While the intent of this survey is to gather preliminary data for grant submission and protocol development, we anticipate that data collected both from this survey and protocol implementation will also support development of independent manuscripts. Given budgetary constraints and limitations of the U54 proposal structure, we may not be able to include in the application all responding centers. However, we will be happy to offer co-authorship to all investigators who contribute patients, clinical data, and/or sample collections.
- To facilitate development of a study population focused on patients with combined immunodeficiencies, we are surveying clinical immunologists that serve patients in this space. Our objective is to determine the approximate number of study participants in specific categories that are available at your institution/in your practice.
- On behalf of the PIDTC Combined Immunodeficiencies Interest Group: Drs. Elena Hsieh, Jennifer Heimall, Ottavia Delmonte, Shan Chandrakasan, Luigi Notarangelo, Andrew Snow, Bertrand Boisson, Sara Johnson, Eyal Grunebaum.
- https://ucdenver.co1.qualtrics.com/jfe/form/SV_0xHzO9GEDomIk18
NEWS
- The Celebration of Life Memorial service for Vicki Modell that was held in NYC a couple of weeks ago is now available to watch through this link : Vicki Modell Memorial – Celebration of Life . The speeches starting at around minute 18 are worth watching/listening too (Jordan Orange, Vanessa Tenembaum, Fred Modell, a patient, and Charlotte Cunningham-Rundles).
- Jeffrey Modell Foundation 12th cycle of the Translational Research Program is now open. A letter of intent must be submitted by March 13th and invited investigators will have to submit full applications by July 8th. Click here for more details.
- CBS announcements:
- Hizentra pre-filled syringes: https://www.blood.ca/sites/default/files/2024-02/CL_2024-09.pdf