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Hemophilia in the News!

February 8, 2012
What does NASA have to do with Hemophilia?

Check out this story on our page;
"The Latest".

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The Latest

This page is dedicated to bringing you the latest stories and news that impacts the Hemophilia and bleeding disorders community.

What does NASA have to do with Hemophilia?

As published by Gizmodo on February 8, 2012, NASA has something in the works that may benefit the bleeding disorders community. NASA has been looking into ways of providing healthcare to their astronauts while they are in space and away from the doctors and medicines the rest of us use. According to Gizmodo, Dr. David Loftus has invented a biocapsule that may be the solution to NASA's needs and a large step forward for people with hemophilia and many other chronic conditions.

The biocapsules are packed with cells that can release needed medications or proteins. Once specific events trigger the cells to begin production, the medication or protein would then cross through the porous biocapsule through diffusion where it can be absorbed by the body.

While it is probably not the most efficient method for factor replacement, it may be a new prospect for use with gene therapy. To read the full Gizmodo article, click here.

Study Shows Both Pros and Cons
to Fc Fusion and Factor IX!

January 19, 2012 - the Journal of the American Society of Hematology prints a study regarding a phase I trial of the Factor IX protein incorporating Fc Fusion technology. The study shows that the time that the Fc Fusion factor molecule lasts in a patient's body is extended by an incredible 3-4X that of the existing monoclonal factor molecules.

This paper also discusses the after effects that were noted with the new Fc Fusion product. While the study is only based on 14 individuals and it cannot be certain which after effects were directly related to the product, they will definitely be something to watch as more research is done.

To read the report yourself, the pros and the cons, follow this link and see what you think. Would possible headaches and other side effects be worth the dramatically extended half lives?

New Human Study With
Fc Fusion & Factor VIII!

A study published on January 5th, 2012 in The Journal of the American Society of Hematology reports that 16 Individuals with hemophilia participated is a study using the new Fc Fusion Technology.

The 16 people received varying doses of the recombinant clotting factor Advate and monitored in order to determine the rate at which the factor was depleted. Once the Advate was out of their systems, the same 16 individuals were then given the same doses of the rFVIIIFc (the recombinant Fc Fusion protein) as they had received of Advate.

While the peak factor levels reached for the rFVIIIFc were similar to those reached by the equivalent dose of Advate, the rFVIIIFc lasted significantly longer in the person's system than Advate. For the specifics on this study check it out here.

The FDA Gives Green Light
To Gene Therapy!

A new year brings a new realm of possibilities. On January 4, 2012 the company "Amsterdam Molecular Therapeutics" announced that they had been given approval by the FDA to conduct clinical trials on Hemophilia B patients in the United States. St. Jude's Children's Research Hospital in Memphis will be the Center point for the studies in the US.

These studies will be building on the earlier successes you've already read about right here on 'The Latest" page. More information on this announcement can be found here.

Follow Up!!! Hemophilia B, Treated With a Virus?

On December 11, 2011 The New York Times published a fantastic article with more updated information on the very story we broke over two months prior. To read this article in its entirety please click here. While their article is great the New England Journal of Medicine did publish an article about the study as well one day earlier. To read the New England Journal of Medicine's article, click here.

Tylenol® Dosing Information Changed

November 15, 2011: McNeil Consumer Healthcare (the makers of Tylenol®) releases new dosing instructions for Extra Strength Tylenol. Due to concerns of overdosing causing Liver damage the recommended dosing has been changed to limit the chances of liver damage. Since acetaminophen (the generic name for Tylenol) is used in many products it is easy to overdose without realizing it. To see the new recommended dosing, click here.

New Study on Immune Tolerance Therapy

November 13, 2011: According to a study published in the Journal of the American Society of Hematology high dose therapy for immune tolerance is shown to not only be more effective than low dose therapy, but it succeeds in much less time and without any noticeable increased chance of infection or other concerns. To view the article on the Journal's website, click here.

Non-sterile Alcohol Wipes

October 26, 2011: If you are a U&I customer, don't worry. we have never dispensed any PDI alcohol wipes. A recall was placed on alcohol wipes made by Pacific Disposables International (PDI) due to infections caused by non-sterile alcohol preparation pads. If you received alcohol wipes from other sources, please check to make sure Pacific Disposables International did not manufacture them. Look at the fine print as they are sold under various brand names.

To see the MASAC Advisory, click here.

New Factor Xa Research May Help Hemophilia A and B Patients with Inhibitors!

On October 23, 2011 Nature Biotechnology published a study completed by researchers at the Children's Hospital of Philedalphia on an engineered Factor Xa. The study will be appearing in the November 2011 issue. The genetically engineered Factor Xa should work as a bypassing agent and allow clotting even more efficiently than Factor VII does.

This could really enhance an inhibitors patient's treatment and prevent many of the prolonged bleeding episodes when the research comes to fruition. This may also lend itself to act as a treatment for people having rapid blood loss in other situations as well.

To read more on this subject you can check out; News-Medical or for the more scientifically minded, Nature Biotechnology. To access more than the summary on the Nature Biotechnology site, you will have to purchase the article if you do not have a subscription.

Factor IX Variants Can Bypass
Factor VIII & Treat Hemophilia A.

October 14, 2011: In an article released in the Journal of the American Society of Hematology, FIX variants can be used to bypass the lack of FVIII found in Hemophilia A mice. The variants were expressed using a non-viral gene transfer and resulted in normal clotting. This new avenue might open doors for people with inhibitors to factor VIII or even all Hemophilia A patients at some in the near future.

Read this article in the Journal of the American Society of Hematology.

Hemophilia B, Treated With a Virus?

In a recent meeting (October 7, 2011) at the National Institutes of Heallth hemophilia was a central part of the discussion about gene therapy. Six severe hemophiliacs with Hemophilia B were given a virus engineered to carry the genes for Factor IX production.

All six saw increases in Factor IX production ranging from 1% to 8% in increased levels. This provides new hope that some time in the future gene therapy may become reality for bleeding disorders.

We'll be trying to follow up on this story in the near future. More information is also available at the Science American Association for the Advancement of Science magazine's website.

Straight from the FDA!!!

On September 29, 2011, the Food and Drug Administration approved a135 mcg/0.5ml and 180 mcg/0.5 ml disposable autoinjector (DAI) to administer Pegasys (peginterferon alfa-2a), an antiviral indicated for treatment of Chronic Hepatitis C (CHC) by subcutaneous injection.

Pegasys continues to be available in a vial or prefilled syringe, and now also in a 135 mcg/0.5ml and 180 mcg/0.5 ml PEGASYS disposable autoinjector.

The updated label and Medication Guide can be found on the FDA web site by clicking here.

New Hope in Treating Hep C!!!

Two protease inhibitors, Boceprevir (aka Victrelis by Merck) and Telaprevir (aka Incivek and VX-950 by Vertex), were approved for use earlier this year. While these new medications must still be used along side Interferon and Ribaviren they have shown to be effective in treating most Type I HCV patients by helping them to respond quickly and effectively to the treatment. Use of the protease inhibitor has shown to lessen the time a person needs to be on treatment in most cases and dramatically increase the success rate.

These new medicines are only for people who have the Type I genotype of the Hepatitis C Virus. This is a huge breakthrough since Type I has been considered the most difficult to clear.

 

Have a suggestion for an update or news flash?

All suggestions welcome, please feel free to email them to us here.


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