Due to the ever-evolving impact of the coronavirus pandemic, FARF staff will be working remotely for the next few weeks. While we are equipped with the necessary technology to continue meeting our mission and providing service, this will create minimal coverage in our office, so the best way to reach us is to email info@fanconi.org, or call and leave a voicemail 541-687-4658, and we’ll get back to you as soon as we’re able. Thank you!

MARCH 11:

The Fanconi Anemia Research Fund (FARF) is closely monitoring the updates regarding COVID-19, a respiratory illness caused by a new coronavirus, as well as the potential implications on the FA community. The severity of the illness and how many people may be affected is unknown at this time. We have a particular concern for those in the FA community who are immunocompromised. There is currently no vaccine to protect against COVID-19, and at this point, the best way to prevent infection is to avoid exposure to the virus that causes it. Stopping the transmission of the virus through everyday practices is the best way to keep healthy.

Because of the concern for transmission of the disease, we are preparing for the possibility that this virus may impact our two significant gatherings: the Annual Family Meeting at Camp Sunshine (Maine, June) and the Scientific Symposium held in conjunction with our Meeting for Adults with FA, (Texas, September). While it is too early to predict how these events may be affected, we want you to be aware that we are mindful of the risks associated with gathering at this time. We are in contact with Camp Sunshine regarding this matter. We will also be carefully monitoring the appropriate health authority recommendations for protecting against exposure provided by the Center for Disease Control and Prevention (CDC) and the World Health Organization (WHO).

The National Organization for Rare Diseases (NORD) recommends that you speak with your doctor to see how they may be able to advise you if you should become exposed to the virus. Finally, should you or a loved one need to travel, review the CDC’s travel notices to ensure you aren’t putting yourself or others at risk.

We will continue to monitor and follow guidance from the CDC and other agencies and communicate with the FA community directly as the situation develops.

Mark Quinlan
Executive Director
Fanconi Anemia Research Fund

Here are some resources with other information:

World Health Organization:

https://www.who.int/emergencies/diseases/novel-coronavirus-2019

CDC Centers for Disease Control and Prevention

https://www.cdc.gov/coronavirus/2019-ncov/about/index.html

The National Organization of Rare Diseases:

https://rarediseases.org/nords-peter-l-saltonstall-on-coronavirus-prevention-and-risk-for-the-rare-community/

National Institutes of Health

https://www.nih.gov/health-information/coronavirus

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Recent studies from the Kiem lab have examined the role of endothelial cells in iPSC-derived HSC specification and expansion. We have also used endothelial cell support to expand adult marrow HSCs and used novel small molecules for expansion. We have demonstrated successful ex vivo expansion of human cord blood cells when cultured in the presence of specific small molecules. We also have 4 active clinical gene therapy studies. One study aims at treating patients with glioblastoma and we have now treated 7 patients and have shown in our most recent manuscript improved survival in high-risk patients with glioblastoma. One clinical study involves the correction of the genetic defect in blood and marrow stem cells from patients with Fanconi anemia and we have now treated 2 patients. Two clinical studies involve gene therapy approaches for patients with HIV and lymphoma undergoing either primary chemotherapy or requiring an autologous HSC transplantation. The Kiem lab is also part of multiple collaborations to improve the efficiency by which HSCs can be cultured and transduced, while closely monitoring the gene modified clones through cutting-edge high-throughput retroviral integration site analysis.

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The current research in our laboratory focuses on studies to:

Understand basic hematopoietic stem cell (HSC) and transplantation biology and clonal composition of hematopoiesis after transplantation, especially the clonality of gene-modified HSCs
Develop and evaluate novel virus-based gene therapy technology and nuclease technology including megaTals, zinc finger nucleases and CRISPR/Cas technology to edit hematopoietic cells with the goal to improve the treatment for genetic and infectious diseases and cancer.
Develop novel ways to derive HSCs from induced pluripotent stem cells (iPSCs) and to expand HSCs to facilitate gene therapy and stem cell transplantation
Develop clinical gene therapy protocols for genetic and acquired diseases, including cancer. Current target diseases include Fanconi anemia, severe combined immunodeficiency, hemoglobinopathies, glioblastoma, and HIV
Develop less toxic hematopoietic cell transplantation protocols especially for patients with nonmalignant diseases

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