Pacritinib Can Fill Unmet Need in Myelofibrosis

The investigational agent pacritinib (CTI BioPharma) holds promise as a treatment option for patients with myelofibrosis and baseline thrombocytopenia, and previous concerns that led to a hold on clinical trials have now been dispelled.

In a pivotal trial, PERSIST-2, pacritinib was significantly more effective than the best available therapy, including ruxolitinib (Jakafi, Incyte), in reducing splenomegaly and trended toward a reduction in total symptom score in patients with myelofibrosis and thrombocytopenia.

The study was published online March 8 in JAMA Oncology. Ruxolitinib, which was approved in 2011 and the first drug ever approved for myelofibrosis, is not safe for patients with low platelet counts.

“The label on ruxolitinib states it is for patients with platelet counts above 50,000, so it isn’t a viable option for those with thrombocytopenia,” said lead author, John Mascarenhas, MD, associate professor of medicine, hematology and medical oncology, at the Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai in New York.

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View Dr. Mascarenhas’ presentation at MPN Advocacy’s San Antonio Patient Program

Recruiting: New Phase 2 Clinical Trial for MF Patients on Ruxolitinib

People with myelofibrosis who are currently receiving ruxolitinib therapy may be eligible for a new phase II clinical trial led by Dr. Ellen Ritchie.

Navitoclax is an investigational agent that inhibits a family of BCL proteins. These proteins block some of the enzymes that keep cancer cells from dying and by inhibiting these proteins, navitoclax may cause the cancer cells to die.  Ruxolitinib is approved by the FDA for the treatment of myelofibrosis. Ruxolitinib blocks a protein called Janus-associated kinases (JAK) which may help keep abnormal blood cells or cancer cells from growing.

The purpose of this study is to evaluate the addition of navitoclax to ruxolitinib in patients who have been receiving ruxolitinib alone. Ruxolitinib treatment alone has not been fully controlling disease which is evident by an enlarged spleen. Preclinical data suggest that the combination of navitoclax with ruxolitinib are synergistic and that navitoclax may help to overcome disease resistance to ruxolitinib.

The study will help to determine the effect of the combination of navitoclax plus ruxolitinib on your cancer. The study will evaluate how the efficacy and safety of the drug.

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View MF Clinical Trials



Study Finds Nutritional Changes Can Improve Symptom Burden

Patients with MPNs have increased inflammatory cytokines, which contribute to symptoms such as fatigue, night sweats and bone pain, as well as low cholesterol and weight loss related to nutritional deficiencies.
BY Katie Kosko
PUBLISHED March 05, 2018
DIET AND SUPPLEMENT USE can help alleviate some of the symptom burden experienced by patients with myeloproliferative neoplasms (MPNs), according to a study conducted by Mayo Clinic researchers.

Patients with MPNs have increased inflammatory cytokines, which contribute to symptoms such as fatigue, night sweats and bone pain, as well as low cholesterol and weight loss related to nutritional deficiencies.

To examine the habits, needs and preferences among this population, researchers used an internet-based survey through the Mayo Clinic Survey Research Center and promoted it on many MPN-focused websites and communities in February 2017. The survey included data on demographics, disease characteristics, nutritional habits, supplement use and symptom burden using the Myeloproliferative Neoplasm Symptom Assessment Form and Total Symptom Score.

The 1,329 patients who responded to the survey represented 37 countries and had the following types of disease: myelofibrosis, 24 percent; polycythemia vera, 37 percent; and essential thrombocythemia, 38 percent. Of those patients, 1,131 consented to taking the survey. The average total symptom score was 31.

Survey results showed that 34 percent of patients endorsed using diet to help control their symptoms or disease, 23 percent had food allergies or intolerances and 31 percent followed a specific or restricted diet.

Nearly all patients who responded (96 percent) said they would be willing to eat only certain foods if it helped to control symptom burden, stabilize their disease or reduce the risk of it worsening (98 percent). Over-the-counter supplements were used by 72 percent of patients, more often by women (74 percent) than men (66 percent).

Supplement users also were more likely to be older, have lower self-reported body mass index and engage in at least 30 minutes of physical activity.

Lower symptom score was associated with alcohol intake, baked foods, dairy and pasta; higher score, with fast food, premade snacks, soda and refined sugar. Symptom burden was significantly lower among patients using amino acid supplements and N-acetylcysteine, but significantly higher in those taking Bach flower remedies

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Qiagen JAK2 Test Gets FDA Clearance for Additional Blood Cancer Types

NEW YORK (GenomeWeb) – Qiagen said after the close of the market on Tuesday that it received US Food and Drug Administration clearance for its Ipsogen JAK2 RGQ PCR Kit for additional use in the diagnosis of all myeloproliferative neoplasms (MPNs).

In March 2017 the FDA cleared the assay as a qualitative in vitro diagnostics test for the detection of the JAK2 V617F/G1849T allele in genomic DNA extracted from EDTA whole blood to aid in the diagnosis of the blood cancer polycythemia vera.

The new FDA clearance now covers two additional types of MPNs: essential thrombocythemia and primary myelofibrosis.

The assay runs on Qiagen’s Rotor-Gene Q MDx instrument, a component of the modular QiaSymphony family of laboratory automation products.

Qiagen said that it is the exclusive worldwide licensee of intellectual property covering the detection of the JAK2 V617F mutation for diagnostic purposes, and that it recently reached a settlement with an unspecified molecular diagnostics industry supplier in Europe, which agreed to stop selling its own JAK2 V617F test kit.

“We are eager to expand the use of our Ipsogen JAK2 assay, which is already available in Europe and other markets, for use in a wider range of patients in the US,” Thierry Bernard, senior vice president and head of Qiagen’s molecular diagnostics business area, said in a statement. The assay “makes it easier for hematologists and oncologists to follow recommended diagnostic testing algorithms and international guidelines for their patients suspected of having MPNs.”

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Learn more about MPN Clinical Trials

New Data Proves Role of Thrombosis in Patients with MPNs

New research published in Annals of Internal Medicine shows that the rate of arterial and venous thrombosis was significant in patients with myeloproliferative neoplasms (MPNs).

MPNs include a variety of blood disorders, like myelofibrosis, polycythemia vera, and essential thrombocytosis, and patients with them have been reported to be at increased risk for thrombotic events. Pharmacologic treatment can stabilize the blood counts, but they generally provide only partial symptom improvement. Until recently, no population-based study has estimated the excess risk with matched control participants.

Malin Hultcrantz, M.D., PhD and colleagues, in an effort to address the gap in knowledge, evaluated data from 9,429 patients – 46% of whom were male – diagnosed with MPNs who reported to the Swedish Cancer Register between 1987 and 2009, and matched them to 35,820 control participants and compared their rates of arterial and venous thrombosis. Median age of study participants was 72 years, and a mong them, 3,001 had polycythemia vera, 3,462 had essential thrombocythemia, 1,488 had PMF, and 1,478 had MPN unclassifiable.

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Incyte Reports Four Year Phase 3 Data Analysis Shows Durability of Response of Jakafi (ruxolitinib) in Patients with PV

Incyte Corporation (NASDAQ: INCY) today announced new 208-week (4-year) follow-up data from the ongoing, global, multi-center, open-label Phase 3 RESPONSE study of Jakafi® (ruxolitinib) comparing the efficacy and safety of Jakafi with best available therapy (BAT) in patients with polycythemia vera (PV) who are resistant to or intolerant of hydroxyurea (HU). The pre-planned data analysis showed a durable primary response to Jakafi in patients with PV who are resistant to or intolerant of HU and the overall safety profile for Jakafi remained consistent with previously reported 80-week RESPONSE data.1 The results were shared in an oral presentation today at the 59th American Society of Hematology (ASH) Annual Meeting 2017 in Atlanta, Georgia.

“With 30 months of additional follow-up, the four-year RESPONSE data analysis presented today at ASH further reinforces the potential of Jakafi as a long-term option for patients with PV,” said Peg Squier, M.D., Ph.D., Head of U.S. Medical Affairs at Incyte. “Given the few treatment options available to treat this chronic and progressive blood cancer, these long-term safety and efficacy data are meaningful to patients with uncontrolled PV.”

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Impact Biomedicines Closes First Tranche of Oberland Capital Financing Following FDA Meeting

Management team expands to accelerate fedratinib global manufacturing and business operations

SAN DIEGO — December 1, 2017— Impact Biomedicines (“Impact”) today announced that it has achieved the first milestone in the Company’s previously disclosed $90 million financing with Oberland Capital, triggering the closing on the first tranche of $20 million. Impact also announced the expansion of its management team to include Randy Adams as Senior Vice President of Commercial Operations and Jeff Barker as Senior Vice President of Global Technical Operations.

“The first tranche of this financing follows a positive meeting with the U.S. Food and Drug Administration (FDA) bringing this much needed potential treatment option for myelofibrosis closer to patients in need,” said John Hood, Ph.D., Chief Executive Officer of Impact Biomedicines. “With Oberland’s financial support, we have made some important investments to ensure that we are well-staffed and prepared for U.S. commercialization.”

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ASH: 1663 Germline ERBB2 Variants Associate with MPNs

Background: The myeloproliferative neoplasms (MPN) and myelodysplastic syndrome (MDS) are usually sporadic diseases, however, familial cases are well-described. Approximately 10% of MPN cases display familial clustering, and there is a 5-7 fold increased risk of developing an MPN among first degree relatives of MPN patients. While familial MDS appears to be less common, identification of multiple predisposition genes has led to the incorporation of this entity into the most recent WHO classification of myeloid neoplasms. Identification of predisposing germline mutations in the myeloid malignancies has led to a better understanding of the pathophysiology of these diseases and improved clinical care. However, many familial MDS and MPN cases exist in which the inherited genetic lesion is unknown. Our recent investigation of a multigenerational family with inherited MDS/AML marked by erythroid hyperplasia identified a missense variant in ERBB3that co-segregated with the disease phenotype (1). In this study, we investigated a kindred with an autosomal dominant familial cancer syndrome characterized by both MPN and solid tumor malignancies.
Program: Oral and Poster Abstracts
Session: 635. Myeloproliferative Syndromes: Basic Science: Poster I
Saturday, December 9, 2017, 5:30 PM-7:30 PM
Bldg A, Lvl 1, Hall A2 (Georgia World Congress Center)

Evan M Braunstein, MD, PhD1, Donna Marie Williams, PhD1*, Igor Makhlin, MD1, Aparna Pallavajjala2*, Christopher D Gocke, MD2*, Nara Sobreira, MD3*, Kala Visvanathan, MD4*, Linda Resar, MD5, Jerry L. Spivak, MD1 and Alison R. Moliterno, MD1

1Division of Hematology, Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, MD
2Department of Pathology, Johns Hopkins School of Medicine, Baltimore, MD
3Department Pediatrics, Johns Hopkins School of Medicine, Baltimore, MD
4Medical Oncology and Epidemiology, Johns Hopkins University, Baltimore, MD
5Division of Hematology, Department of Medicine, Johns Hopkins University SOM, Baltimore, MD

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Secondary Myelofibrosis Recruiting Phase 1 Trials for DB00493 (Cefotaxime)

This pilot clinical trial studies the side effects of combination chemotherapy, total body irradiation, and donor blood stem cell transplant in treating patients with secondary myelofibrosis. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high energy x-rays to kill cancer cells and shrink tumors. Giving combination chemotherapy and total body irradiation before a donor blood stem cell transplant helps to stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the patient they may help the patient’s bone marrow make stem cells, red blood cells, white blood cells, and platelets.

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MPN Advocacy and Education International Attends a Workshop Hosted by the FDA

By Ann Brazeau, CEO MPN Advocacy & Education International

MPN Advocacy & Education International was invited to attend a very important and timely meeting with the FDA last month. The invitation was extended to us and a few other MPN focused organizations. This meeting launched a unique beginning for those of us representing the MPN Community.

The FDA’s newly formed Oncology Center of Excellence and the Office of Patient Outcomes are committed to engaging MPN patients and advocates to better respond to the need for quality treatment options.

Among the many topics presented by their hematologists, researchers and cancer patient liaison, was an in depth explanation on drug approval processes and how valuable and critical the patient’s voice is to their mission.

After a drug is approved, companies are required to send frequent updates on any and all reported adverse side effects from their drug. Physicians also report on these events and patients can do so by contacting the FDA directly through Med Watch Consumer Voluntary Reporting. Go to Patient Reported Outcomes are extremely important and valuable to the Drug Approval Process and after a drug is approved.

We also learned the complexities surrounding drug “holds” and what happens and why when that hold begins and when it is released. They stressed the importance of ongoing communication between BioPharma and the FDA and encourage regular conversations and frequent updates with comprehensive data.

As the MPN Community pursues the use of Interferon and hopes for accessibility to Interferon as a viable option for treatment, we learned that company(ies) producing Interferon would have to apply for approval specifically for MPNs. It could be a daunting process but there may be avenues we can utilize through patient advocacy efforts.

Although we did not share all of our concerns at this meeting, we look forward to subsequent meetings where we hope to tackle the issues of endpoints, pricing, fast-tracking, and other challenges surrounding MPN drug approval. We were very pleased to have had this opportunity.