RUNX1 Research Program

Arlo and I live with RUNX1, a rare genetic disorder that carries a very high risk of us developing leukemia and other serious blood diseases. It is something we think about every single day.

What has given us hope is the RUNX1 research program and the clinical trials they have made possible. I have participated in three clinical trials and a history study so far, all with one goal: helping researchers find treatments, prevention strategies, and hopefully someday a cure for families like ours.

Now our son Arlo wants to participate too.

As a parent, it is incredibly emotional knowing your child carries the same risks you do. But we are deeply grateful that these trials exist and that there is finally real progress being made.

Participating in clinical trials comes with major costs, that are unfathomable for many people. We are asking for help so we can continue being part of this research and give Arlo the opportunity to participate as well.

We do not want leukemia to be our future. We want hope, answers, and a chance at prevention for our family and for others living with RUNX1.

Every donation means the world to us. Thank you for helping us continue this fight and support research that could change our lives!

Hi, I’m raising money for the RUNX1 Research Program because I believe in the power of prevention. RUNX1-FPD is a rare inherited disorder, like BRCA for breast cancer, that puts people at high risk for blood cancers. After nearly a decade in biotech, I’ve seen how hard it is to treat cancer once it’s full-blown. But what if we could stop it before it takes hold?

That’s what we’re doing at RRP. For the past 6.5 years, I’ve worked alongside incredible scientists and families to turn groundbreaking research into action. Today, I’m proud to say our work has led to the first-ever clinical trials for RUNX1-FPD and they are focused on cancer interception, therapies that target precancerous changes before it's too late.

Please help us accelerate this research, thank you!

Katrin

Help Us X-Out Blood Cancer for Me, Arlo & Families Like Ours

I’m raising money for the RUNX1 Research Foundation because this cause is deeply personal. My family has lived with the weight of the RUNX1-FPD genetic disorder—a rare inherited condition that drastically increases the risk of developing blood cancer. My mom passed away from it last November. Now, my son Arlo and I face the same difficult reality.

The average age of blood cancer onset for those with RUNX1 is right around my age. That’s a sobering thought. I’m currently enrolled in two clinical trials at MD Anderson, not just to try and help myself—but to push science forward so that Arlo and others like him have a better future.

For the first time, there are clinical trials specifically focused on RUNX1-FPD. One is testing a low dose of sirolimus to improve blood cell production by calming a cancer-linked pathway. Another is working to collect and preserve RUNX1 patient stem cells—laying the groundwork for gene-editing therapies in the future. PLEASE consider donating to help fund patients to be able to participate in these trials, including me and Arlo.

Research like this has long been supported by the NIH, but funding is dwindling fast under the current administration. If we want to continue this momentum—and give every RUNX1 family hope—we need people like you to step up now more than ever.

Please consider donating. You’ll be helping real families—including mine—fight for a future without blood cancer.

Thank you,

Jill

Hi, I’m Amanda Eggen, and I’m raising funds for the RUNX1 Research Program (RRP).

As of April 15, I’ve been honored to work with RRP for six years. I love our team—but what drives me even more is our mission: to change the future for individuals with RUNX1-FPD.

RUNX1-FPD is a rare, inherited blood disorder. For some, it causes frequent bruising, bleeding, and allergic symptoms. But the most concerning impact is the significantly increased risk—up to 30 times higher than average—of developing blood cancer. The constant uncertainty that patients and families live with is what keeps me pushing for answers.

At RRP, we’re working toward a bold goal: to prevent blood cancer before it starts. And we’re getting closer than I ever imagined possible.

Because RUNX1-FPD is rare, few pharmaceutical or biotech companies are willing to invest in clinical trials for this community. That’s why we’ve stepped in. We’re currently supporting three promising trials to give families real options—hope backed by science. We’re also working hard to raise funds to also cover out-of-pocket costs so more patients can participate.

To date, I’ve connected with more than 440 patients, and we estimate there are up to 18,000 people in the U.S. who have RUNX1-FPD but don’t yet know it.

If interested, you can read more about why I feel this effort is so important: https://www.runx1-fpd.org/runx1-pulse/new-clinical-trials-offer-hope-and-opportunity

With your help, we can support patients in participating in these trials and get closer to life without blood cancer.

Please consider donating—every bit helps.

Thank you for being part of this effort.

Hi, for those of you who don't know- I'm Leah.

Together with my partner Joseph, we are planning to climb Snowdon at sunrise on the 5th of June 2024 to raise funds for the RUNX1 Research program. We will also be joined on the climb by a handful of our friends!

We are also doing this fundraiser to raise awareness for the RUNX-1 FPD gene mutation, so please share!

*As this is an American charity, donations are taken at the dollar-equivalent to the pound donation.

Now, the backstory...

Back in 2019, I became unwell with treatment-resistant ITP (Immune thrombocytopenic purpura) - a rare, but usually manageable autoimmune / bleeding condition.

To better understand why my body resisted treatment, my doctors ordered genetic testing. In this, they found I have an even more rare genetic mutation called RUNX1-FPD.

Without being too scientific, this gene mutation has been linked to a 40% chance of developing Acute Myeloid Leukaemia (Cancer) and Myelodysplastic syndrome- as well as being linked to a whole host of autoimmune / bleeding / dietary disorders! Not very fun!

There is a small number of families who are known to have this mutation globally, with a smaller handful being within the UK. The research for the gene is currently conducted in the USA, under the non-profit charity.

We are hoping to raise funds and awareness in order to support their continued research into the RUNX1 gene mutation and cure, so please- if you can, donate!

Thank you!

Leah & Joseph

For your information: https://www.runx1-fpd.org/runx1-patients-and-family/#what-is-runx1fpd

Hi! On April 23rd, 2022, I'll be running the Garmin Marathon in Olathe, KS, in support of my brother, Regan. Regan was diagnosed with leukemia when he was 10 years old. 20 years later, his body is currently progressing towards developing cancer again. Thankfully, he has not crossed that threshold. After genetic testing was performed on one of his recent bone marrow biopsies, his team of doctors found an inherited mutation in the RUNX1 gene, which may be responsible for his recent illness. The following is taken from the RUNX1 Research Program website to provide some background on Regan's situation:

 

"RUNX1-familial platelet disorder (RUNX1-FPD) with predisposition to hematologic malignancies is a hereditary disorder due to being born with an error, called a mutation, in the gene RUNX1 ¹. The mutation is passed down from generation to generation, and patients are at a heightened risk for developing blood cancers. Most experience low platelet counts (thrombocytopenia) and low-functioning platelets."

 

"The link between germline RUNX1 mutations and blood cancer was first discovered in 1999.² Since then studies have estimated that the average lifetime risk of getting a blood cancer with mutations in the RUNX1 gene is close to 50%. It is important to note that there is a huge range in the incidence of cancer between families, with some being as low as 11% and others as high as 100%.¹ The predominant type of blood cancer RUNX1-FPD patients acquire is acute myeloid leukemia (AML), the second deadliest of all blood cancers.⁴ Other types of hematologic malignancies may also occur in patients, including myelodysplastic syndromes, T-cell acute lymphoblastic leukemia, and non-Hodgkin lymphoma.¹ Twenty-nine years is the average age of onset of a blood cancer for a person carrying a germline RUNX1 mutation,⁷ while the average age of onset in the general population is 68 years of age. Moreover, 50% of families with RUNX1-FPD have at least one or more children who progress to cancer. ³ ⁵ ⁶"

 

In mid-March, Regan will be undergoing a bone marrow stem cell transplant, receiving his donation from my sister, Jerilyn, a half-match. Out of 30 million + registered bone marrow donors, the only two matches for him were Jerilyn and my dad, Jerry. Regan has a long road of recovery ahead of him, as he'll need to receive a week of chemotherapy and radiation before the transplant, then follow an extremely strict regimen following his transplant to get his immune system up and running.

 

Regan, my mom Cindy and I all share a familial platelet disorder. Since his genetic testing showed that he inherited the gene, it's very likely that my mom and I also have this gene mutation. We learned about RUNX1 only after Regan's genetic testing came back. Thankfully, Regan's insurance is covering almost his entire treatment. The purpose of this fundraiser is to raise money to donate to the RUNX1 Research Program, and to spread awareness of this rare condition, bringing it to light for others that are affected by familial platelet disorders. Additionally, 100% of donations to the RUNX1 Research Program go directly towards research and education; all administrative/overhead fees are paid for by the co-founders.

 

I'll be starting a short blog with weekly updates on Regan's treatment, info on RUNX1, and updates on marathon training. I'll throw the link to that website here once it's been created.

 

If you can't donate directly to this fundraiser, there are still ways you can help others that may be in a situation similar to Regan's! First, donate blood if you can. There is a massive blood shortage nationwide, and transfusions have been vital in keeping Regan's blood counts stable while his bone marrow is out of commission. Second, register to be a bone marrow donor! You may end up saving someone's life, and it is a relatively easy procedure if you are called upon to donate. Links for both are below.

RUNX1 Research Program: https://www.runx1-fpd.org

Find blood donation events and locations near you: https://www.redcross.org/give-blood.html

Register to become a bone marrow donor: https://bethematch.org

 

Thank you for visiting this fundraising page and please spread to anyone and everyone!

We're joining in to help raise funds for RUNX1 Research Program because this fantastic organization has helped us so much in our journey and makes a difference in so many lives.

If you're able to give, we'd be so grateful for your support. Let's X-Out Blood Cancer together!

I’m fundraising for the X-Out Blood Cancer Campaign to support research into RUNX1 familial platelet disorder (RUNX1-FPD), a rare inherited blood disorder that increases an individual’s lifetime risk of developing blood cancer by approximately 20 to 50%.

While RUNX1-FPD is rare, the research currently underway could help scientists better understand how blood cancers develop and how they may one day be prevented for patients with this condition and beyond. Insights from rare diseases often lead to breakthroughs that benefit far more people.

This cause is also deeply personal to me. My dad passed away from angiosarcoma, a rare blood vessel cancer, and that experience shaped how I view the importance of research, early detection, and the impact on families facing uncertainty.

Researchers are making progress, but advances depend on sustained support, especially amid recent cuts to federal research funding.

If you’re able, I’d be incredibly grateful for your support. Every contribution helps move this work forward. Thank you!

Families with RUNX1-FPD face a significantly higher risk of developing blood cancer. For many, the uncertainty of what lies ahead is a constant reality.

Research is essential to changing that future.

Today, scientists are actively working to better understand how RUNX1 mutations lead to blood cancer and how it may be prevented. They are pursuing multiple approaches, from studying how inflammation may increase risk, to exploring ways to restore healthy blood development, to identifying high-risk cells before cancer develops.

But progress in rare disease research depends on community support.

Traditional funding is often limited, and advancing this work requires people coming together to make it possible.

You can help by making a donation, or even better, start your own fundraiser on this platform and invite others to be part of this effort.

Every gift helps move this work forward.

Join us in the X-Out Blood Cancer Campaign and help create change.