HULL PRENOSI NAJNOVIJE VIJESTI ZAKLADE ZA ISTRAŽIVANJA MIJELOFROLIFERATIVNIH NEOPLAZMI za veljaču 2022. godine

Zaklada za istraživanje mijeloproliferativnih neoplazmi je zaklada pacijenata za pacijente, a pomaže oboljelima od policitemije vere, mijelofibroze i esencijalne trombocitemije u poboljšanju kvalitete njihovog života, i to naporima u smjeru prevencije i liječenja ovih bolesti. 

Veljača je mjesec, na čijem kraju se obilježava Svjetski dan rijetkih bolesti, među kojima su i ove mijeloproliferativne neoplazme. Za oboljele od navedenih neoplazmi, ovo je vrlo važno, navode istraživači ove zaklade, jer mnogi oboljeli ostaju godinama nedijagnosticirani ili pogrešno dijagnosticirani. Navode i slogan: Mijeloproliferativne neoplazme su rijetke. Istraživanja o njima ne bi trebala biti.

U nastavku pogledajte i ostale vijesti iz biltena za veljaču.

February 2022
Under the Microscope
MPN Leaders Added to Scientific Advisory Board
 
 
An extraordinary group of MPN thought leaders help us strategically lean into the future through their participation on our Scientific Advisory Board. With their broad expertise and visionary view of MPN research priorities and gaps, they offer invaluable perspective to our work and mission.
 
We gratefully welcome these dedicated MPN researchers to our growing Scientific Advisory Board:
  • Saar Gill, MD, PhD, University of Pennsylvania
  • Catriona Jamieson, MD, PhD, University of California, San Diego
  • Ann Mullally, MD, Brigham and Women’s Hospital
  • Jyoti Nangalia, MBBS, PhD, Wellcome Sanger Institute
  • Radek Skoda, MD, PhD, University of Basel
 
IMPACT Spotlight
 
From a 2021 interview with Ann Mullally, MD, Brigham & Women’s Hospital/Dana Farber Cancer Institute
 
Q. What is your history with the MPNRF?
Dr. Mullally: I’ve received for my laboratory a total of five grants from the MPN Research Foundation over approximately 10 years. The most recent grant is a collaborative grant with Dr. Rebekka Schneider, looking at biomarkers in MPN. Initially, a lot of those projects focused on the genes that we know are the key causative genes of MPN. And in particular, we focused on JAK2 and Calreticulin mutations.
 
Q. How has your MPN research progressed?
Dr. Mullally: Over more recent years, we’ve increasingly been looking at the mutations that co-occur with JAK2 or Calr, and are associated with risk of progression of MPN, progression to myelofibrosis or even leukemia. What we’ve been also doing is integrating genetics with basic clinical and lab parameters, and now also with bone marrow pathology, and trying to identify things in the bone marrow that could be detectable early in the course of the disease by standard techniques, which could be used as predictors of progression, or biomarkers of MPN.
 
Q. Where is your laboratory work heading?
Dr. Mullally: Ultimately, what we’re most interested in is what we call actionable biomarkers, that not just are predictive of risk of progression, but biomarkers where we could intervene and block whatever pathway is activated to prevent progression or even prevent the development of MPN.
 
View this video interview and others in our IMPACT@21 report.
Rare Disease Day is February 28th
MPNs are rare. Research into them shouldn’t be.
 
Watch for an exciting challenge to build awareness and raise funds for MPN research. Rare Disease Day aims to raise global awareness and generate change for the millions of people worldwide living with a rare disease. For MPNs, this is still critically important, as many people living with ET, PV and MF continue to go undiagnosed or misdiagnosed for years. 
Clinical Trial Highlights: Currently Recruiting
 
LIMBER 304 (parsaclisib plus ruxolitinib) 
Company: Incyte
 
This is a phase 3 study of PI3 kinase inhibitor for MF patients who are on a stable dose of ruxolitinib and need an improved response.          
 
 
 
For a full list of currently recruiting MPN trials, view this list on our website.  
Fedratinib combination for patients with IDH mutation
Sponsor: University of Chicago
 
For MPN patients with an IDH mutation, this phase 1 study has two arms. Ivosidenib will be given initially to IDH1 participants, followed by a combination of ivosidenib with fedratinib. For
IDH2 patients, enasidenib monotherapy will be followed by combination with fedratinib. 
 

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