May 24, 2020
From the SCNIR: Message for patients with neutropenia and their families-
The staff and physicians for the Severe Chronic Neutropenia International Registry (SCNIR) continue to work very hard on your behalf to understand neutropenia and to find best ways for its diagnosis and treatment. Our principal offices are in Seattle WA and Hannover Germany. The Registry depends upon voluntary participation of hundreds of patients, families, nurses, physicians, and other healthcare providers in more than 60 countries, held together for more than 25 years by a shared commitment to improving the lives of patients with neutropenia through clinical trials and basic research.
New Website: We continue to use the vital information that you and your physicians provide to give guidance on treatments and outcomes. Our reports are regularly published in medical journals. Our website https://www.scnir-neutropenia.uw.edu/ has a current list of publications. The website also provides access to our Patient Handbook, recommendations for treatments, and forms for enrollment and follow-up. The European SCNIR office also maintains similar information and resources: https://severe-chronic-neutropenia.org/
G-CSF: The Registry provides valuable information on the effectiveness and long-term outcomes for patients treated with G-CSF. Your annual reports and communications to us about how well you are doing and adverse events are extremely valuable. Please keep them coming. The long-term follow-up information is really important. We have several recent published reports listed on the website on the effectiveness and safety of Neupogen for severe chronic neutropenia.
Home monitoring: We are working with Athelas (Mountiain View CA) on development of a device for home monitoring of blood neutrophil counts. The Athelas One is now FDA cleared for point-of-care indication for monitoring WBC and neutrophil levels in a finger stick drop of blood. If you have used this device, please share your experience with us.
Transplant: In increasingly patients with congenital neutropenia are treated with hematopoietic stem cell transplantation. Overall this is a complex and expensive procedure, but results are steadily improving. We are currently summarizing all of our information on patients with transplants. We will be asking transplant patients and their families for more information; your help will be greatly appreciated by many other patients, families and physicians.
Gene editing: We are very busy searching for new and more specific treatments for chronic neutropenia. At the University of Washington, Harvard, Tuebingen, Germany and numerous other sites researchers are investigating ways to correct the genetic cause for blood diseases including chronic neutropenia. The work is exciting and challenging. At the University of Washington we have a new NIH grant to support this work and we are also working with a company Emendo on gene editing as an approach to treatment of neutropenia. We may ask you to help us with blood or bone marrow samples to advance this work.
WHIM syndrome: We are just completed a phase 2 study of a very specific oral therapy for the rare disease called WHIMs syndrome. The study was sponsored by the Boston biotechnology firm X4Pharma. Patients with WHIM syndrome have very low white blood cell counts and are extremely susceptible to having chronic severe warts on their hands and feet. The results of this preliminary study have been presented at several meetings. The drug increases neutrophil and lymphocyte counts and appears to reduce infections and warts. An international phase 3 control trial has just begun. If successful, this will be the first specific therapy for neutropenia available as an oral drug. If you or your doctor know of anyone with this condition, we will be pleased to tell them more about this exciting Phase 3 study.
ELANE neutropenia. We are at an advanced stage of developing a specific drug for this type of neutropenia. Beginning a clinical trial depends upon are gaining access to the best specific drug we have studied, a drug retired from development by Merck several years ago. They are helping us and we hope to have more information about beginning a trial soon.
Idiopathic and congenital neutropenia: We have a preliminary trial underway to see if the drug from X4Pharma that releases neutrophils from the bone marrow may work in other conditions. If it is effective it will be a major step forward and our efforts to develop oral therapies for neutropenia.
NIH Support: The NIH through its National Institute for Allergy and Infectious Diseases (NIAID) has supported the basic operations of the SCNIR for the last 15 years. We have in application pending for renewal of this critical support, but our current funding ended as of April 2020. We believe our renewal application is very strong, but research funding is very competitive. The grant renewal is critical for continuation of the work of the SCNIR.
Gifts : Many of you have been very generous in supporting the SCNIR. Right now because of a gap in our in NIH support, your gifts are particularly meaningful. Utilizing your previous gifts we have sufficient funds to continue of our current activities until the NIH application is reviewed and score in June 2020. Thank you very much.
NNN Summer 2020 Update Meeting: We have a Zoom meeting set for Friday, June 12, 2020 to catch up with you and answer questions. We look forward to talking with you.
David C. Dale MD for the SCNIR