The stats:

Name: Agnieszka Czechowicz MD, PhD

Institution: Stanford University; Lucile Packard Children’s Hospital

Area of expertise: Pediatric Stem Cell Transplant and Regenerative Medicine

My work:

I am a translational physician-scientist. I care for patients at Stanford Children’s and specifically focus on treating bone marrow failure patients with stem cell therapies. In addition to helping diagnose, monitor and transplant patients with stem cells from various types of donors, I have also helped design and treat Fanconi Anemia-Type A patients with promising but exploratory gene therapies.

I also oversee a basic science research lab that is aimed primarily at understanding how blood-forming stem cells work and how they interact with their environment with a goal to then alter these interactions to improve the function of the blood and immune system. We have been using these findings to try to improve bone marrow transplantation through development of new antibodies and alternative methods that can eliminate chemotherapy and irradiation from transplant protocols. We are further studying how these antibodies work in FA and how they can be combined with gene therapy, as well as exploring alternative genemodification approaches that potentially could be used in a variety of ways for FA patients.

What motivates me to work on FA:

I have long been passionate about developing better treatments for FA, as I see this as an unfortunate condition that really should be curable! Ever since I was a medical student and cared for FA patients in Minnesota and India, I set my heart on this goal. First and foremost I aspire to eliminate the use of known DNA-damaging agents including chemotherapy and irradiation from treatment protocols, but ultimately, I hope to play a role in broadly changing care for FA patients in many ways. Additionally, I am motivated to work on FA as I think there is a lot that we can learn from this illness that we can then use to help in the treatment of many more patients with other diverse conditions from various genetic diseases from cancers to aging.

When I’m not in the lab, you could find me:

In our clinic or on our inpatient ward taking care of patients! I am also a big advocate of encouraging various broader collaborations, especially with industry, and can often be found in environments where we are brainstorming how to develop better and more innovative therapies. I also LOVE traveling and experiencing how different people enjoy life, so sometimes you can find me globe-trotting and working from afar.

Something I want FA families to know:

No one has a perfect life and while FA is an unfortunate condition, we are very fortunate to have the FA family to help navigate the complexities of this disease! If there is anything I can do to help, I am always available. I truly care. Especially if you have any questions about gene-therapy or reduced conditioning protocols, please let me know. We have FA trials that I would love to talk about!