Archive for Research

The New England Journal of Medicine – Article on JAK Inhibition Study

The New England Journal of Medicine – 1 March 2012

JAK Inhibition with Ruxolitinib versus Best Available Therapy for Myelofibrosis

Abstract from the journal regarding the study undertaken by leading MPD physicians into the treatment options for myelofibrosis, evaluating the efficacy and safety of Ruxolitinib compared with the best available therapy, in patients with myelofibrosis.

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Incyte press release – Ruxolitinib approved for patients with MF

Incyte have announced that the FDA (U.S. Food and Drug Administration) have granted marketing approval for  Jakafi™ (ruxolitinib) for the treatment of patients with intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythaemia vera MF and post-essential thrombocythaemia MF. Patients with intermediate and high-risk MF represent 80 to 90 percent of MF patients. Jakafi (JAK-ah-fye) is the first and only product to be approved by the FDA for MF, and the first in a new class of drugs, known as JAK inhibitors, to be approved for any indication. Jakafi is an oral JAK1 and JAK2 inhibitor.

You can read the full press release here.

Incyte is a Wilmington, Delaware-based drug discovery and development company focused on developing proprietary, orally available drugs for serious unmet medical needs in oncology and inflammation.  For more information visit the Incyte website.

New Incyte press release

The company Incyte, working under a strategic alliance with Novartis, has several new drugs currently under trial for the treatment of myelofibrosis.  Novartis has issued a press release describing how the trial is going. The data from the Comfort II study shows that people being treated with the JAK2 inhibitor drug INC424 under trial are showing “marked clinical improvement”. Their spleen size after 48 weeks of treatment was reduced, compared with the best available therapy currently being used to treat their illness.

Low-dose aspirin – benefits in ET?

Does low-dose aspirin offer benefits for people with “low-risk” essential thrombocythaemia (ET)? Researchers in Spain have written an article suggesting that low-dose aspirin may particularly benefit people with ET if they have the JAK2V617F mutation or cardiovascular risk factors. Dr Claire Harrison of Guy’s and St Thomas’ Hospital (UK) and Dr Tiziano Barbui of the Division of Hematology, Ospedali Riuniti di Bergamo in Bergamo, Italy offer commentary on this article about aspirin and MPDs.

New article discusses ET diagnosis and treatment

In the 1^st February issue of the journal Blood you will find an article from our colleagues in Cambridge entitled “How I treat ET”. The article tackles key issues including gaps in our knowledge, the differences between ET and PV or PMF and also discusses real cases. Link here to read the article in full: http://bloodjournal.hematologylibrary.org/cgi/content/full/117/5/1472

MPD Voice funds research

MPD Voice began funding research towards a cure in 2008. The UK trial of the drug vorinistat recently opened in the UK with our support. Jo McAllister is research coordinator for the trial and the cost of her role is funded by MPD Voice.
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MPDs in families

In very rare cases, myeloproliferative disorders can run in families. This is exceedingly rare, so it is generally not something most people with MPDs need to feel concerned about.

Researchers at Harvard University and Brigham and Women’s Hospital in the US are conducting research on genetic links in MPDs. Ashkenazi Jews have a higher incidence of MPD, indicating a particular genetic predisposition in this population. Using recently developed DNA sequencing technologies the researchers conducting the study propose to identify the gene or gene(s) that underlie the genetic predisposition to MPD in Jewish individuals.

If you are a Jewish MPD patient and have at least one living first-degree relative (i.e., parent, child, or sibling) who also has MPD (Polycythemia Vera, Essential Thrombocythemia or Myelofibrosis) and are potentially interested in participating in this study, please contact Dr. Ann Mullally at amullally@partners.org in the laboratory of Dr. Benjamin Ebert (Brigham and Women’s Hospital, Harvard Medical School, Boston) for further details.

If you other questions about MPDs in your family please email us at info@mpdvoice.org.uk for more information.

MPD origins: JAK2 may just be middle step

Five years ago we made several discoveries crucial to our under- standing of myeloproliferative diseases. We discovered three mutations associated with these disorders: the JAK2 V617F mutation and two other mutations called c-MPL and JAK2 exon 12. In these last five years, what have we found? We’ve learned that these mutations may well be a middle step in the development of MPDs – they may not be the complete cause. In 2010 we’re conducting ongoing research into myeloproliferative disorders, which includes attempting to identify what exactly causes these diseases.

We’re looking at whether additional mutations (for instance the recently discovered TET2) or predispositions (such as haplotype 46/1) may play a role in why people develop MPDs. We’re also testing new agents – including JAK2 inhibitors (INCB-018424, SB1518, CEP701, TG101348), and HDAC inhibitors (LBH589) – which are all having some benefit in terms of reducing the size of the spleen and improving symptoms in patients with myelofibrosis.

We are in an unprecedented era of MPD research. We’re looking into the causes of MPDs and their impact on the patients who have these disorders. And finally, we’re studying many interesting new agents that will hopefully have an impact on or even cure these diseases. —Dr Ruben Mesa, the Mayo Clinic, US