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PDSA E-News: April 26, 2017




Rigel Submits New Drug Application (NDA) for Fostamatinib for Treatment of Chronic ITP

RIGLLOGOExciting news for patients with ITP! Biotechnology company Rigel Pharmaceuticals announced April 17, 2017, that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for its drug fostamatinib in patients who have chronic and persistent immune thrombocytopenia (ITP). Rigel’s president and chief executive officer said, “This NDA submission in support of fostamatinib in ITP is a major milestone in bringing new treatment options to patients suffering from this disease.” He added, “We look forward to working closely with the FDA as they review the submission over the coming months.”

Fostamatinib is an oral (pill) investigational drug that inhibits oral spleen kinase (SYK), which is a key player in the immune process that leads to the platelet destruction which occurs in ITP. While other ITP therapies modulate the immune system to stop destruction or to stimulate production of platelets, fostamatinib addresses underlying autoimmune causes of ITP that interfere with platelet production.

Rigel’s NDA is supported by data from their Phase 3 clinical program for fostamatinib in ITP. The program was composed of three studies: two randomized placebo-controlled studies and an open-label extension study. A total of 163 ITP patients were evaluated and included in the NDA submission. Across all indications, fostamatinib has been evaluated in more than 4,600 patients. Data from all studies, including preclinical and drug manufacturing data, were included in the NDA submission. The company expects to receive notification of FDA’s acceptance of the NDA in June 2017. Previously FDA had granted Orphan Drug designation to fostamatinib for ITP treatment.

Press Release, Rigel Pharmaceuticals, “Rigel Submits New Drug Application to FDA for Fostamatinib in Chronic ITP.” April 17, 2017.

New ITP Therapy Management Guide Published by British Journal of Haematology

BJH logoThanks to medical innovations and new research discoveries, treatment plans for ITP patients have evolved remarkably the last 15 years. Still, many treatment decisions remain for physicians, with minimal breakthroughs in discovering a diagnostic test or biomarkers to help guide therapy. Comparative studies analyzing treatment efficacy exist, but are limited. Dr. Nichola Cooper, a leading ITP researcher and a hematologist at Hammersmith Hospital in the UK, recently published her recommendations on management decisions in the British Journal of Haematology.

Dr. Cooper echoes other prominent ITP experts, and explains that the platelet count alone is not as important as it once was in evaluating treatment options; adults with persistent counts above 30,000 should be managed with observation. Hematologists should consider overall bleeding symptoms when deciding on therapy for a patient so they do not receive unnecessary treatment. She notes lessened need for heavy steroid use and splenectomy in ITP patients, citing the increase in novel treatments like Anti-D, rituximab, and TPO agents for treatment of ITP.

In addition to bleeding symptoms, treatment decisions should be based on the patient’s clinical characteristics such as age, activity, physiological impact of platelets, and having other diseases. Therapy choice is also based on the length of ITP diagnosis; newly diagnosed (0-3 months) patients should receive steroids or IVIg and persistent ITP (<1 year) patients can consider mycophenolate mofetil, rituximab, or TPO agents. Chronic ITP patients can receive a combination of these treatments, but more research is needed as toxicity of long-term treatment is unknown.

Questions remain about variations and progression of ITP, which makes treatment decisions based on only platelet response difficult to predict. Looking forward, Dr. Cooper recommends research focusing on “how can we identify which patients need treatment, given that most remain asymptomatic despite thrombocytopenia; and, if treatment is needed, which treatment options should be used?”

Cooper, Nichola. “State of the Art - How I Manage Immune Thrombocytopenia.” British Journal of Haematology, 10 March 2017.

New Lung Function Discovered: Making Blood Cells

Blood CellsScientists have made an astonishing find – lungs play a role in producing blood! Until now, producing blood cells was believed done only by the bone marrow. However, studies of mice at the University of California San Francisco found that more than half of a mouse’s platelets are produced in the lungs. Platelets are components of the blood required for clotting to stop bleeding. In another surprise, researchers found a formerly unknown pool of blood stem cells capable of restoring blood production when stem cells in bone marrow are depleted. The findings show that lungs have function beyond breathing and respiration, and could benefit understanding of human diseases in which patients suffer from low platelet counts, or thrombocytopenia.

This study was possible because of refinement of a technique called two-photon intravital imaging, developed by Dr. Mark Looney (pulmonologist and UCSF professor) and Dr. Matthew Krummel (UCSF professor of pathology). The new imaging technique allows researchers to carry out the delicate task of observing individual cell behavior inside the tiny blood vessels of living mouse lungs.

The research team was studying interactions between the immune system and circulating platelets in the lungs of mice engineered to have platelets that emitted bright green light (fluorescence). During observation they noticed large numbers of megakaryocytes (platelet-producing cells) in the mouse lungs. A large population of platelets was living in the mouse lung tissue! In fact, megakaryocytes were producing more than 10 million platelets per hour inside the mouse lung tissue. This suggested that more than half of the mouse’s platelet production was occurring in the lung tissue and not all in bone marrow, as previously believed. Their findings also uncovered previously overlooked megakaryocyte progenitor cells and blood stem cells –estimated at 1 million per mouse lung. Additional research used lung transplants to study platelet movement between mouse lungs and bone marrow and it showed that blood stem cells and progenitors easily traveled back and forth.

The researchers conclude, “To our knowledge this is the first description of blood progenitors resident in the lung, and it raises a lot of questions with clinical relevance for the millions of people who suffer from thrombocytopenia.” They said their study suggests researchers who proposed treating platelet diseases with platelets produced from engineering megakaryocytes should look to the lungs as a platelet production resource. The findings have clinical relevance for future studies of platelet genesis and megakaryocyte function in lung inflammation, bleeding and thrombotic disorders, and other inflammatory conditions.

Emma Lefranҫais, Guadalupe Ortiz-Muñoz, Axelle Caudrillier, et al., “The Lung is a Site of Platelet Biogenesis and a Reservoir for Hematopoietic Progenitors.” Nature, 2017; DOI: 10.1038/nature21706.


New Test Developed to Rapidly Identify Harmful Bacteria in Blood Samples

Bag of donated bloodResearchers at the University of California San Diego have invented a diagnostic tool that identifies harmful bacteria in a sample of blood in only four hours with 99% accuracy. The current procedure for detecting bacteria that cause diseases such as listeriosis, pneumonia, meningitis, bacteremia, and sinus infections takes several days to be cultured and looks for only one bacterium at a time. The new test exposes all bacteria in a sample and can identify even low levels of pathogens.

Using this new procedure, bacterium can be detected based on their specific DNA sequences. DNA from a sample is isolated and multiplied 20,000 times. The sample is heated so that bonds in the DNA break. Using a specialized microscope and unique dye, lab technicians can observe the rate at which the DNA comes apart. The dye causes the DNA to fluoresce (emit light), from which the technician can create an algorithmic melting curve that matches reference data for specific pathogens. The algorithm can currently pinpoint the presence of 37 distinct bacteria in a variety of different conditions.

In the future, the engineers of the diagnostic tool want to include the capability to detect fungal and viral pathogens as well as genes for antibiotic resistance in blood samples. The new test is projected to be available on a wide scale to physicians in the next five years. Both the efficiency and efficacy of this new test is sure to significantly improve diagnostic procedures on blood samples, streamlining the process for physicians, technicians, and patients alike.

University of California - San Diego. "Method to Identify Bacteria in Blood Samples Works in Hours Instead of Days: Researchers Melt DNA at Unprecedented Scale." ScienceDaily. 8 February 2017.


Eating More Fruits and Vegetables Brings Rapid Improvements in Psychological Well-being

fruits and vegetablesWe know that fruits and vegetables are part of a healthy diet but now it turns out their benefits go beyond just physical health. New research indicates that increasing the consumption of fruits and vegetables may improve psychological well-being in just two weeks. Researchers at the University of Otago in New Zealand found that when young adults were given extra fruits and vegetables each day for 14 days they ate more produce and also experienced increased vitality and motivation.

Researchers enrolled 171 students (ages 18 to 25) in the study and divided them into three groups for two weeks. One group continued their normal eating pattern. One group was given two additional servings of fresh fruit and vegetables (carrots, kiwi, applies, and oranges) each day. A third group received prepaid vouchers and received text messages reminding them to eat more fruit and vegetables. Participants all received psychological assessments at the beginning and end of the study to evaluate vitality, mood, motivation, symptoms of depression, and anxiety.

The findings showed that participants in the first group, who received extra fruits and vegetables each day, consumed most of the produce they received over the two-week study. This group also showed the most improvement in psychological well-being. Positive changes were especially noted in motivation and vitality. The other two groups had no improvement in psychological well-being over the 2-week period. Also there were no improvements in symptoms of anxiety and depression in any of the groups. Most past research linking depression to diet has been over a much longer time period than the short 2-week period of this study.

The team concluded that increased intake of fruits and vegetables by personal delivery of the food to participants led to rapid psychological well-being benefits. They stated, “Providing young adults with high-quality FV [fruits and vegetables], not texting them reminders to eat more FV and giving them a voucher, resulted in improvements to their psychological well-being over a 2-week period.” Their findings offer initial validation of a causal relationship between eating fruits and vegetables and well-being, but they suggest larger studies are needed.

Honor Whiteman, “Eating More Fruits, Vegetables Boosts Psychological Well-being in Just 2 Weeks.” Medical News Today, Feb. 10, 2017.




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