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MPNRF ADVANCING RESEARCH
First US Clinical Trial for MPN Vaccine
The idea that a vaccine might prevent or treat an MPN seemed like a pipe dream just a few years ago. Today, MPNRF proudly funds the first US clinical trial to test a vaccine that could reverse the effects of a mutation associated with MPNs, specifically the calreticulin (CALR) mutation. 

Through a 2022 Thrive Initiative award, Marina Kremyanskaya MD, PhD, and Cansu Cimen Bozkus, PhD, both from the Icahn School of Medicine at Mount Sinai in New York City, aim to harness the human immune system to target mutant CALR MPNs. The project is a phase 1 trial, representing initial testing for the safety and efficacy of a specific vaccine. MPNRF's 2015 MPN Challenge award supported research leading to this clinical study.  

Mutations in the CALR gene are believed to be the genetic driver in 30-35% of people living with essential thrombocythemia (ET) and myelofibrosis (MF). All CALR mutations lead to an altered protein with a unique and identical ending. This altered protein can be recognized as foreign by the immune system and is therefore an ideal immunotherapy target, since it is not found on normal tissues. 
Marina Kremyanskaya MD, PhD
Cansu Cimen Bozkus, PhD
  • Preclinical studies showed that mutated CALR can induce immune responses but the response in MPN patients was not as robust. 
  • This research team has developed a vaccine that targets mutated CALR with the goal of enhancing the immune response in MPN patients. 
  • It has the potential to eliminate the mutant malignant cells, leading to a reduction in tumor (disease) burden. 
RESOURCES TO SHARE
Patient Bill of Rights
Beyond their laser focus on a patient's diagnosis and treatment, clinicians can be instrumental in educating patients so they better understand the complex information presented to them and their right to ask questions and make choices.

Clinicians: Please share this important document Patient Bill of Rights - MPN Research Foundation with your medical practice team members and other appropriate patient-facing colleagues. Encourage patients to read it and share it with friends and family members.  
 
Patients: Bring a copy of this document to your doctor's office. Ask if they can make a copy of it (or share a link to it) to make it available as a resource to other MPN patients. You can also make it available in a hospital's medical library that has patient access, send it to other patients you know, or share it online or with your support group. 
STUDY
Chronic Sleep Disturbances Study from Mays Cancer Center
A Study Request Shared from Mays Cancer Center
Mays Cancer Center is seeking blood cancer patients, ages 18 and up and currently undergoing active treatment, to participate in a National Institutes of Health-funded research study lasting for 20 weeks total. The purpose of the study is to test an app-based wellness intervention for chronic sleep disturbances among blood cancer patients. We will be asking eligible and consented study participants to use a smartphone-based wellness app for 10 minutes per day for a total of 8 weeks. Additionally, we will ask that study participants complete in-person blood draws and online surveys at 3 different time points over the course of the 20-week study (baseline, after 8 weeks, and after 20 weeks). Finally, we will also ask that study participants wear an Oura Ring daily and complete a brief daily sleep diary for 8 weeks and then again for 1 more week during week 20. All study participants that finish the 20-week study will receive $75 in compensation. If you are interested in learning more about, or joining this study, please contact us by phone (210-450-8253) or email at [email protected], or visit this link to complete the eligibility survey. You will need to meet a range of eligibility criteria to be considered eligible to participate in this study. Participation is voluntary.
WEBINAR
Can Digital Tools Help Manage MPN Symptoms and Health?
Register for PEN Webinar
The great majority of patients living with an MPN are affected by chronic fatigue. Can digital health alleviate this and other symptoms? What exactly is mobile app intervention, and how can it help you? Register to join this free patient-moderated webinar to learn about: 
  
  • Technological interventions in MPN symptom management 
  • Telemedicine limitations and the importance of connecting with an MPN specialist 
  • How your MPN care could change with artificial intelligence, biosensors, and telepathology 
  
Thank You
A grateful thank you to everyone who donated to MPN Research Foundation via AmazonSmiles this past year. While this opportunity is no longer available, you can invest in MPN research anytime, including in honor or in memory of someone. Watch for a special giving opportunity during MPN May.
Clinical Trial Highlights
PXS-5505
Company: PharmAxis
 
Phase 1/2 trial for intermediate or high-risk MF patients who are not currently taking a JAK inhibitor. PXS-5505 is a pan-LOX inhibitor that has shown reduction in fibrosis.  
PACIFICA (Pacritinib)
Company: CTI BioPharma Corp  
 
For MF patients with very low platelets (<50,000) who have had no or limited exposure to a JAK2 inhibitor. Pacritinib was approved by the FDA for the treatment of MF in 2022. This trial is focused on gathering additional data on the safety and efficacy of the drug in patients outside of the US. Long-term survival data will also be captured. 
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