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  • Hemophilia Gene Therapy Breakthroughs | HemAware
    like this We have a good chance to not encounter some really difficult biological roadblocks says Calos In an experiment on mice with hemophilia B that were injected with factor IX 20 of their liver cells made the missing factor protein These are definitely therapeutic levels Calos reports Since Calos has effectively demonstrated that her nonviral methodology works in small animals her lab is running experiments on pigs to see if results are similar Soon the experiments will be conducted on monkeys the final model before trying the process on humans Although the DNA embeds itself safely into the genome the physical act of injecting DNA into the liver vein is being assessed for safety There is concern that the method could potentially encourage cancerous growth since the cells around the injection site become agitated and respond by dividing rapidly Although tumor growth as a result of hydrodynamic delivery has never been observed in the mice models Calos team will closely monitor the process in larger animals Stem Cells Another version of gene therapy now in development uses stem cells Rather than injecting viral vectors into the body this method first plugs the gene into hematopoietic stem cells those that have differentiated enough to become bone marrow cells which can then morph into various blood cell types Next researchers insert the cells into the body like a miniature version of organ transplantation We ve got a two pronged approach modifying the factor VIII to optimize its expression properties and at the same time combining it with a technology that has been proven to work clinically to cure a genetic disease says Christopher Doering PhD of Emory University School of Medicine in Atlanta Doering s lab found that a genetically modified version of a pig factor VIII gene is much more effective at secreting the protein than its human counterpart His research team plugged this gene into mouse stem cells transplanted them into mice and found consistently high levels of factor VIII in the blood A year later the mice were still producing their own factor VIII at normal levels With this type of transplantation the immune system must first be weakened Otherwise it will do its job of recognizing the new cells as foreign invaders that need to be destroyed Of course altering the immune system even temporarily has its risks Doering s lab continues working to find ways to transplant the modified stem cells with the least amount of immunosuppression Bioengineered Clotting Factors As many scientists focus on future therapies for hemophilia others are perfecting what already works therapeutic injections of the missing clotting factor The aim of this research is to make clotting factors with longer half lives decreasing the frequency of injections Therapeutic factor VIII is no longer derived mainly from transfused blood Instead scientists use cultured cells to make copies of the protein This is achieved by introducing the gene that codes for factor VIII into well characterized animal cells cultured in the laboratory Within one

    Original URL path: http://www.hemaware.org/story/hemophilia-gene-therapy-breakthroughs (2016-02-18)
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  • Send page by email | HemAware
    Profiles Health Wellness Fitness Nutrition Taking Charge Pain Management Parenting Family Parenting Family Hemaware Jr Women Women s Bleeding Disorders Carriers Watchdog Insurance Blood Safety Advocacy Community HemAware Blogs HemAware Videos HemAware Photos Chapters in Action Global Spotlight Community Voices En Español Need info Ask NHF Contact HANDI NHF s resource center for additional information on bleeding disorders Home Glossary Site Map Donate Advertise Privacy Policy Subscribe Contact Us Life

    Original URL path: http://www.hemaware.org/printmail/996 (2016-02-18)
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  • Progress Made for My Life, Our Future Genotyping Initiative
    low cost The five My Life Our Future IRB approved pilot sites are Bleeding and Clotting Disorders Institute Peoria IL Center for Comprehensive Care Diagnosis of Inherited Blood Disorders Orange CA Indiana Hemophilia Thrombosis Center Indianapolis IN The Pennsylvania State University and The Milton S Hershey Medical Center Hershey PA Puget Sound Blood Center Seattle WA Six additional pilot sites are currently in the process of IRB review as the

    Original URL path: http://www.hemaware.org/print/996 (2016-02-18)
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  • Wave of Progress
    nationally in 2013 Additionally participants have the option to contribute their data and samples to a central research repository housed at PSBC All data and samples are coded so confidentiality is protected Once 5 000 people contribute to this research bank scientists and researchers at academic institutions or companies can apply for access to study the samples and data potentially leading to improvements in treatment and care NHF s Medical and Scientific Advisory Council MASAC recommends that individuals strongly consider participating in this aspect of the program to further scientific understanding of hemophilia Volunteering to give just a small blood sample for this initiative not only provides you and your family with additional information but could also change the lives of future generations says Marion A Koerper MD vice chair of MASAC and medical advisor for NHF Each sample collected through My Life Our Future brings us one step closer to developing a cure for hemophilia Turning research into reality AzmanJaka Thinkstock Once the research repository opens applications to access the samples and data will be carefully reviewed by an independent multidisciplinary committee to determine their feasibility and scientific integrity All partners are committed to this independent review process and no one will have preferential access ATHN will provide oversight for this review committee which will be composed of at least one hematologist nurse consumer blood disorders scientist ethicist and geneticist The value of the research repository was discussed in depth during the ATHN Data Summit held in November 2014 in Chicago Attendees noted that there are many unanswered questions about hemophilia that warrant explanation including what causes a specific genetic mutation how the mutations relate to one another and why the physical manifestation of these mutations such as bleeding severity varies so greatly from person to person Many of these mysteries may be solved through research projects using data and samples from the MLOF repository Like other disorders the only way that progress in the treatment of hemophilia can be achieved is by thoughtful scientific review of data and judicious use of samples from affected people says Michael D Tarantino MD medical director of the Bleeding Clotting Disorders Institute in Peoria Illinois Only then can scientists develop life changing therapies Expanding MLOF to family members and carriers In addition the partners will be planning and piloting the next phase of the program offering genetic testing to potential carriers of hemophilia Carriers possess the gene for the disorder and therefore have a 50 chance of conceiving either a son with hemophilia or a daughter who is also a carrier In the case of hemophilia women are the carriers To identify carriers it s best to first determine the specific genetic mutation in an affected member of the family The potential carrier can then use this information to see if she has the same mutation The majority of our patients with hemophilia have participated in My Life Our Future and are very interested in having the female members of their family

    Original URL path: http://www.hemaware.org/print/1255 (2016-02-18)
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  • Wave of Progress | HemAware
    nurse at the Hemophilia Treatment Center of Central Pennsylvania at Penn State Milton S Hershey Medical Center Identifying a woman s carrier status can not only improve her health management but can also aid in family planning possibly improving outcomes during childbirth and delivery MLOF is preparing to launch a pilot carrier expansion in select HTCs across the country in 2015 A brighter future Though the prospect of what s to come is exciting the discoveries already made as a result of MLOF and the enrollment of more than 2 000 participants nearly half of the program s goal are impressive My Life Our Future is a true example of the hemophilia patient community s desire to drive progress and join together to make a difference says Val D Bias NHF s CEO I m confident that we re creating a brighter future for generations to come To be a voice for progress and learn how you can get involved in My Life Our Future visit MyLifeOurFuture org Together we can make knowledge hereditary Learn More Visit MyLifeOurFuture org for more information about My Life Our Future including how to find a participating hemophilia treatment center near you and to sign up to receive updates on the program Watch NHF CEO Val D Bias provide an overview of My Life Our Future at NHF s 66th Annual Meeting in Washington DC in September 2014 youtube com watch v lHI1Jvj6AV4 Read about the launch of My Life Our Future in the Spring 2013 issue of HemAware hemaware org story genotyping progress Read MASAC Recommendation 214 MASAC Recommendations on the NHF Genotyping Project for Persons with Hemophilia bit ly MASAC214 Contact HANDI NHF s information resource center Email handi hemophilia org Phone 1 800 42 HANDI Online hemophilia org Community Resources HANDI NHFs Information Resource Center Information for this article was provided by the National Hemophilia Foundation the American Thrombosis and Hemostasis Network the Puget Sound Blood Center and Biogen Idec Voices for Progress HemAware conducted the following Q A with two MLOF participants Kenny Stocker of Seattle and Guy Law of Erie Pennsylvania Vichly44 Thinkstock Q What is your hemophilia diagnosis and does anyone else in your family have the disorder Guy Law GL I have severe hemophilia A and am now partially blind because of the disorder Though I have no siblings or children with the disorder my mother and aunt are carriers My cousin has and grandfather had hemophilia as well As a result I am really close to my cousin It was helpful to grow up with someone who s about my age and I could relate to in ways I couldn t with most people Kenny Stocker KS I have severe hemophilia A which I inherited from my biological mother I am the first in my family to have the disorder I have 17 year old twins a boy and a girl neither of whom has hemophilia Q How did you learn about MLOF GL I learned

    Original URL path: http://www.hemaware.org/story/wave-progress?quicktabs_story_page_tabs=0 (2016-02-18)
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  • FDA Approves Kogenate for Hemophilia Prophylaxis
    factor VIII treatment that the FDA has approved for routine prophylaxis It is not however the only product used prophylactically Administering Kogenate FS to children with hemophilia A on a daily basis before a bleeding event occurs will reduce bleeding into joints and help prevent joint damage a major cause of disability in hemophiliacs says Jesse Goodman MD MPH director FDA s Center for Biologics Evaluation and Research Kogenate FS was first licensed for use during surgeries to prevent bleeding episodes It was also used in an off label capacity that is in a way other than its label indicated to treat people with hemophilia FDA s approval of Kogenate FS for routine prophylaxis treatment eases prescription issues Now physicians can prescribe it for young patients for on label use with clinical data to support it The new approval also makes it far more likely that the treatment will now be covered by a patient s insurance Many insurance companies will not pay for a drug if it is being used off label on the grounds that its use is experimental This can be the case even in instances where the off label use has been thoroughly studied Patients and their families have to foot the bill if their doctor recommends these kinds of treatments The approval came after the Joint Outcome Study headed by Marilyn Manco Johnson MD a multicenter US trial that included 65 boys with severe hemophilia A who were younger than 30 months at the time they entered the study Of the boys 32 were given routine prophylaxis with Kogenate while 33 were given therapy based on bleeding episodes The study published in The New England Journal of Medicine in 2007 was conducted during a 10 year period Special attention was given to the index joints

    Original URL path: http://www.hemaware.org/print/254 (2016-02-18)
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  • (Re)Search & Destroy
    means that testing is proactive An initial blood sample is used to measure baseline data and can then be compared with samples gathered at the patient s annual comprehensive treatment center visit The samples are all routinely tested for hepatitis A B and C and HIV some of each sample is saved to investigate other possible blood safety threats in the future For example stored specimens were tested for West Nile virus to make sure that it was not being transmitted in factor products If even a handful of patients suddenly test positive for an indicator that had previously been negative an investigation can be initiated immediately says Soucie Inhibitor Pilot Study Contrary to what one might expect though virulent viruses are not the most pressing threat to the bleeding disorders community Inhibitor s are The biggest blood safety issue acknowledged in the world for the hemophilia population is inhibitors says Soucie He observes that inhibitors would not develop at all if the underlying bleeding disorder were not treated with factor products So something about the way we re treating them what we re treating them with has to enter into it The impetus for the CDC s Inhibitor Pilot Study was a meeting with the US Food and Drug Administration FDA in 2005 Officials told the CDC that studies of new products typically involved so few patients that the FDA could not determine the causal relationship between inhibitor development and the product used When one or two people developed inhibitors did it mean that the product was worse for all patients Nobody could answer that question says Soucie The inhibitor study is being conducted at nine adult and four pediatric hemophilia treatment centers HTCs around the country So far more than 500 patients have enrolled A challenging aspect of the study is that patients are required to keep detailed treatment records What we re doing is collecting not only information on the product what it is how much they re using and how often but also what they re using it for says Soucie Is it for prophylaxis To treat a bleed Before surgery All these things have been identified as potential risk factors for developing an inhibitor And so has the product itself By taking a blood sample when a patient switches to a new product the CDC hopes to be at the forefront of prevention We ll be able to judge whether there were any changes says Miller If we do that in large enough numbers we ll pick up new occurrences of inhibitors much more rapidly than by simply collecting data The advantages of one lab overseeing all the testing are manifold The CDC can standardize the way blood is drawn and prepared It can test samples when patients first enroll a year later when they return for their annual comprehensive visit and again when they change factor products In addition it has the resources to collect data prospectively before patients develop inhibitors and compare it

    Original URL path: http://www.hemaware.org/print/189 (2016-02-18)
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  • Help Your Joints with Pilates
    1 safe to 3 dangerous It is in the safe to moderate risk category along with bicycling and spinning But keep in mind as with any new activity it is important to discuss Pilates with your comprehensive healthcare team Pilates Basics Pilates is named for Joseph Pilates its German born inventor While interned in England as a prisoner of war during World War I he developed exercises for other detainees that could be done on a mat or bed He opened a studio in New York City in the 1920s teaching dancers how to build their core strength Today a number of Pilates methods are taught including the popular Winsor and Stott Pilates The basic foundation of Pilates is focused movements that concentrate on strengthening the core part of the body which includes the powerhouse the abdomen lower back and buttocks The hips and shoulders are also important areas of focus Building a strong core enables people to do activities that involve the extremities without putting as much stress on the joints explains Sherry Herman Hilker PT a physical therapist in the University of Michigan Hemophilia and Coagulation Disorders Program at the C S Mott Children s Hospital in Ann Arbor Michigan If core muscles are strong you are stabilizing your pelvis and spine so that when you are doing things with the arms and legs you are operating from a strong base she notes Like other Eastern based exercises Pilates strongly emphasizes correct breathing Proper form is also crucial such as having what is known as a neutral spine along with correct pelvic rib scapular shoulder blade and head placement Pilates can be done on a mat or on an apparatus such as the Reformer where one lies down and uses attached springs and straps to perform resistance exercises Workouts may also incorporate balls foam rollers and resistance bands But experts say that first timers should begin with mat exercises before transitioning to the more complex equipment Shoes are not generally worn in Pilates and it is best to wear loose fitting clothing for unrestricted movement One can benefit from taking Pilates once a week but three one hour sessions a week are ideal Getting Started Although Pilates exercises are low impact injury is possible This is because Pilates is multifaceted involving correct movement and breathing simultaneously The exercises are more about the mind body connection and the concentration on form says Patty Connors PT OMPT a physical therapist at the University of Michigan Spine Program in Ann Arbor who is also a certified instructor of Stott Pilates When form is off injuries can occur For example she says If you are doing a back exercise with your legs in the air and your spine is pivoting and your legs are out of control you can injure yourself Most Pilates exercises can be modified to place less pressure or strain on specific joints and muscles For example since the most vulnerable area to bleeding is the hip flexor it

    Original URL path: http://www.hemaware.org/print/162 (2016-02-18)
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