From the Founder, Dr. Eszter Hars
Dear SDS community,
I am delighted to announce that the SDS Alliance has initiated a project with The Jackson Laboratory (JAX) to develop a mouse model for SDS!
This is very exciting because for decades, we have wanted a cure for SDS and a mouse model is the key first step that will allow new therapy development to begin. Many new therapeutic approaches — such as gene therapy, antisense oligonucleotides, and small molecule drugs — are waiting to be applied to SDS. With a good mouse model, all these possibilities will open up! Wouldn’t it be wonderful if we could just take a pill to restore our bone marrow function? A medicine like that would be one of the many things that researchers could work on as soon as the mouse model is ready!
This project represents the first step of our strategy. We have recruited JAX — the leading mouse model developer — to create the mouse model for us. JAX has over 2300 employees, most of whom are PhD scientists, and $500 million of revenue a year. If you know any scientists working on therapy development — whether for cancer, diabetes, immunology, or blood disease — chances are they are using mouse models from JAX. I know that because I have used them in my own work!
The project will include two parts:
Create the mouse model (introduce SDS disease genes into mice using CRISPR and breed) for $150,000;
Characterize the model (for example, how does SDS present in mice compared to human SDS? Do the mice get pancreatic problems or neutropenia?) for another $150,000.
JAX has agreed to use their NIH grant funding to cover the model creation cost for us! Essentially, JAX is creating the mouse model for free; all we have to do is characterize it. I am very appreciative of JAX for helping our community. I am thrilled that the SDS Alliance is the first patient organization that JAX chose to support this way! They like the fact that we speak their scientific language — which they had never seen with other patient organizations — and they are convinced of our ability to take their mouse model to the next level — therapy development. Just like we envisioned in our strategy, we are leveraging the NIH grant through JAX!
The mouse model is the first step toward finding a cure. Are you ready to turn hope into action? To complete the mouse model, the SDS Alliance is launching a “Turn Hope into Action” fundraiser to raise $150,000. We will commission the best experts to characterize the model and make sure they deliver. Once developed, we will make the mouse model available to any researcher, anywhere in the world, who studies SDS. This mouse model will also enable us to attract therapy development experts, including biotech companies, to test and refine their new therapies for SDS. I look forward to a future where we no longer have to worry about bone marrow failure or leukemia. The future is in our hands!
Our strategy is working, but we can’t do it alone. We need your support! Together, let’s turn hope into action, and action into results!
Eszter Hars, Ph.D.
President and CEO