October 2024

Taylore and her fiancé, D.J., are showing just how much of an impact individuals can make when they decide to take action.

This RUNX1-FPD patient and her partner have turned their desire to raise awareness about RUNX1-FPD and support RRP into reality by organizing a fundraiser through D.J.’s workplace, Redwood Logistics.

The Redwood Logistics "Cash Us If You Can" fundraising team.

The team preparing for a pop-up fundraiser at Redwood.

This Chicago-area company holds an annual fundraiser called the Redwood Games, where employees form teams and compete to raise funds for each team’s chosen nonprofit.

“We knew we wanted to do what we could to give back to RRP, as they continue to do so much for patients and families. So we decided to nominate and lobby for RRP to be chosen as our team’s Redwood Games beneficiary this year,” Taylore explained.

D.J.’s team was initially unfamiliar with RUNX1-FPD, but after setting up a call where Taylore explained the disease and shared her story, the team quickly embraced the cause. The fundraiser has been a tremendous success, raising over $10,000 so far, with Redwood matching employee donations to bring the total to more than $21,000.

“The support has been incredible,” Taylore said. “We’re so grateful to everyone who’s contributed, and there is still time for anyone to donate!” With a final deadline of November 14, donations can be made through this GiveButter link.

Taylore and D.J. encourage others to consider their own networks and to reach out to RRP for support when planning fundraisers. “We hope this shows how easy it is to raise funds for something you’re truly passionate about,” Taylore said.

“Even something as small as a pop-up shop works. We set up a booth at Redwood and our team members donated items like plants, raffle tickets and baked goods for us to sell. This helped us generate $700 in donations,” D.J. added.

We at RRP are deeply grateful to both of them, their team members and all of the generous donors for helping us advance in the fight to prevent blood cancer.

Growing up, Taylore noticed that she and her father shared a tendency to bruise and bleed easily.

Over time, they realized other family members also had these symptoms—from her aunt and uncle to a few cousins and her grandmother. The cause remained unknown until last June, when Taylore’s father was diagnosed with MDS.

Doctors quickly identified a potential link between the family’s history of blood cancer and their bruising and bleeding issues. Genetic testing confirmed that Taylore, her father, and several relatives had a RUNX1 deletion.

Thankfully, her father’s MDS was caught early, and he received a stem cell transplant in September 2023. “He’s doing amazing now!” she shared, adding that he recently returned from a well-deserved trip to Italy.

For Taylore, RRP has been “a lifeline as I navigate my new diagnosis and contemplate what this means for me,” including her hopes of starting a family. She and her fiancé, D.J., knew each other from high school, but their paths rarely crossed until after graduation. They reconnected through mutual friends at a New Year’s party, hit it off immediately and the rest is history. With their wedding coming up next year, they continue to look at life through an optimistic lens and are excited to tackle the future together.

Dr. Chitra Hosing from MD Anderson Cancer Center shared insights on this clinical trial sponsored by RRP at a recent webinar. Click on the button below to watch the recording: 

The trial aims to investigate the safety and feasibility of collecting and banking blood stem cells from RUNX1-FPD patients, preparing for potential future advancements in gene editing that could one day help improve patient health. 

This study is also now open at Children’s Hospital of Philadelphia (CHOP) led by Dr. Tim Olson. Like the study at MD Anderson, only adult patients (18 years or older) are eligible for now. If you are interested in learning more please contact Amanda Eggen via email.

This ongoing grant partnership between RRP and Alex’s Lemonade Stand Foundation supports early-career scientists focused on inherited hematologic malignancy predisposition disorders. Funding is awarded to projects designed to prevent the progression of pre-malignancy to hematologic malignancy in RUNX1-FPD patients.

Click the button below for more details on applying:

We are excited to announce that enrollment* for the updated RUNX1 Patient Data Hub (RUNX1 PDH) opens Friday, November 1st!

This secure platform allows patients worldwide to contribute their information, and was built to support RRP’s mission of improving the quality of life and preventing cancer in RUNX1-FPD patients via three main methods:

• Research Advancement: Helping inform and design future studies aimed at treating RUNX1-FPD and preventing cancer transformation.
• Patient Care: Supporting optimal patient care by generating frequent reports that summarize RUNX1-FPD patient experiences, which patients can then share directly with their healthcare teams.
• Advocacy & Education: Enhancing our understanding of patient challenges to better inform advocacy and educational initiatives.

We are committed to making clinical trial participation as accessible as possible for patients. In response to valuable feedback shared during clinical trials webinars, emails and direct conversations with both patients and study leaders, the study teams are now implementing adjustments. 

These include reducing the number of required tests and exploring options for home-based testing and sample submissions, making it easier for participants to remain close to home.

You can find the updated imatinib study summary here. 

Look out for updates on the sirolimus and stem cell harvesting and banking studies soon!

Additionally, if you are in need of travel support to be able to participate in the sirolimus and/or stem cell harvesting and banking studies, please reach out to Amanda Eggen via email at aeggen@runx1-fpd.org. RRP can help. 

We Look Forward to Seeing You at the Final Coffee Chat of 2024!

Please join your fellow RUNX1 Patients and Family Members to discuss concerns and questions that are on your mind.

We particularly hope those who are new to the community will join and introduce yourselves, and learn from those who have been managing life with RUNX1 for some time. Click the button below to register today!

Meeting the Challenge of Rare Disease in the Family:
25 Years of Lessons Learned

We were delighted to hear from Dr. Al Freedman, a licensed psychologist with over 20 years of experience in psychotherapy, who shared invaluable lessons from his journey as a father to his son, Jack, who lived with Spinal Muscular Atrophy for 26 years. Some of the lessons he shared included:

• A rare disease diagnosis is a trauma.
• Hope breeds hope.
• Anything is possible.
• Scientific progress brings new hope…and new challenges.
• A rare disease may also bring rare gifts (perspective, resilience, creativity, compassion, connection and love).

Dr. Freedman also answered specific audience questions from his perspective as a psychologist. If you are a patient or family member who missed the event and would like to review the recording, please reach out to Amanda Eggen: aeggen@runx1-fpd.org.

NIH RUNX1 Natural History Study: 2024 Patient Update and Q&A

Dr. David Young, Medical Director for the NIH's RUNX1 Natural History Study, recently gave an update on the study to date, including overall information on participation:

• There are 206 confirmed RUNX1 patients enrolled;179 have visited the clinical center at least once (and many more than once!).
• Of these, 70 individuals enrolled in the study have had blood cancer, with AML as the most common followed by MDS.
• Overall, 65% of families involved in the study have a blood cancer history.

Some additional key points he shared:

• Bone marrow biopsy remains the gold standard and the only way to conclusively catch blood cancer.
• Talk to your doctor if you see any of the following at home:
  • New or worsening bruising or bleeding
  • Fatigue or exercise intolerance
  • Frequent infections
  • Pallor or jaundice (yellow skin/eyes)
  • Abdominal distention
  • Unintentional weight loss or gain
  • Recurrent fevers
• If you’re considering pregnancy, there have been many healthy deliveries among the enrolled patients. However, given the bleeding concern for individuals with RUNX1-FPD, it is important to begin planning with your health care team early for the best possible outcome.
• The team has been developing more resources for patients. You can find them here: NIH RUNX1-FPD Clinical Research Study.

Though many have enrolled, the study learns more with every new patient and family. If you would like to be connected to the team to discuss enrolling, please reach out to Amanda Eggen: aeggen@runx1-fpd.org.

2024 RUNX1 Scientific Conference Research Takeaways

Friday, November 22nd

11 a.m. PST  •  1 p.m. CST  •  2 p.m. EST

Join RRP’s Executive Director, Dr. Katrin Ericson, and Scientific Advisory Board Chair, Dr. Nancy Speck, as they break down the key research findings presented by scientists at the 8th Annual RUNX1 Scientific Conference, held in Princeton, New Jersey. This webinar is designed for a general audience—no scientific expertise required.

NIH RUNX1 Natural History Study: 2024 Update

Dr. Paul Liu, Principal Investigator of the NIH RUNX1 Natural History Study, recently presented a comprehensive update on the clinical and genomic data collected and analyzed over the past four years.

This session was held in advance of the 8th Annual RUNX1 Scientific Conference to provide RRP's scientific community with a dedicated hour to review and discuss these findings, laying the groundwork for deeper discussions at the conference.

If you would like to view a recording of this presentation, please please click here to email us your request.

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Please support blood cancer prevention research and help find treatments for RUNX1-FPD patients.

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