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Girl With Rare Blood Disorder Works to Inspire Others

Kara Loader is just like any sixth grade girl. She’s been playing the violin for seven years, loves softball, and says she’s really good at school. She’s a huge Taylor Swift fan, and she has a lot of friends. But something sets Kara apart from her friends and classmates, though it’s nothing you’d be able to see on the outside.

11-year-old Kara has been battling severe congenital neutropenia since birth. Neutropenia is a rare blood disorder, meaning her body doesn’t make enough neutrophils in her white blood cells. So when Kara gets sick, or even gets a cut, her body may not fight off the infection as quickly. 

Kara has to be extra cautious and carry a medical kit with her at school, and when she gets really sick, Kara visits a hematology and oncology specialist at the Cleveland Clinic. She takes a shot of neupogen every day, which helps her build her neutrophils. However, she has to watch how much she takes, because the drug increases the risk of developing leukemia.

Kara has become a bit of a celebrity of her own in the neutropenia community. She said her video inspired a woman in her 30’s to talk about the disorder for the first time .”She always thought that it wasn’t okay, like, it wasn’t normal to tell people that you actually have it. And she said that my video actually gave her the courage to tell it to other people,” Kara said.

Kara said she has one message for anyone else battling neutropenia: “I would tell them everything’s going to be okay.” Kara and her family remain in contact with families they have encountered in the hospitals and at a yearly conference for neutropenia.

Ariana-Leilani also has suffered from neutropenia.   Sign her petition to assure she gets proper treatment and protection from abuse.

Read more: Kara Loader

 

Novartis Now Has Version of Amgen Neupogen Drug

Sandoz announced that its version of Amgen’s NEUPOGEN proved to be highly similar to the original in preventing severe neutropenia in individuals with breast cancer being treated with neoadjuvant myelosuppressive chemotherapy. Neutropenia is a condition where the body has an excessively low number of white blood cells.

The U.S. Food and Drug Administration had accepted Sandoz’s filgrastim application in July, making the company the first to have an accepted filing under the Biologics Price Competition and Innovation Act of 2009.

According to Professor Kimberly Blackwell from the Duke University School of Medicine, biosimilars have the potential to play important roles in widening access to top-quality biologics in the U.S. Since biosimilars are more affordable than the originals they are copying, savings will be generated which can then be used to finance other medical needs.

The biosimilar that Sandoz intends to distribute in the U.S. is already available in over 40 other countries. By the end of the decade, it is expected that biosimilars will account for around a quarter of the $100-billion sales generated by off-patent biological drugs.

Read More: Tech Times

Informative Presentations and Photos from the 2014 National Neutropenia Network Family Conference

The 2014 Neutropenia Family Conference was the most informative and well attended.

Copies of the informative presentations from the 2014 Conference on neutropenia are now available, including those by Dr. David Dale, Dr. Boxer, Dr. Bonilla, and Dr. james Connelly:

 

Read More: National Neutropenia Network Family Conference 2014

 

 

Congratulations to Ariana-Leilani on Graduating from Elementary School

Congratulations to Ariana-Leilani from her mother Dr. Ariel King.   Although Ariana-Leilani has made it this far, she still needs your help in urging that she get the free medicine and an second medical opinion she needs to finally rid herself of her Severe Chronic Neutropenia.

Sign her petition today:http://www.ariana-leilani.com

Neutropenia Family Conference 2014 July 11- 13th Ann Arbor, MI

IMG_0464Participants in the 2014 Neutropenia Family Conference will have a chance to speak with the experts, hear presentations covering the latest treatment and research, and to participate in programs geared toward managing life with neutropenia in it. It is a weekend of learning and fun for the children. There will be many opportunities for participants to get to know each other in a comfortable and nurturing setting.

This year sessions will start on Friday July 11th at 1:00 PM with a choice of workshops and an opening session for all.  The popular Friday Meet and Greet Reception has not gone away. As in the past families and presenters will have a chance to mingle and get to know each other while enjoying a filling assortment of appetizers.

Private one-on-one sessions with the doctors will be offered towards the end of the conference on Saturday afternoon instead of before the opening session on Friday.This should make them more productive than ever as patients will have the benefit of knowledge gained at workshops.

This year with the first ever Friday Kids Kamp parents will have the opportunity to attend the educational sessions on Friday afternoon from 1:00 to 5:00 knowing their children are having fun in a safe environment with their peers. Children will be welcomed back to Kids Kamp on Saturday from 8:30 to 4:30 where another day of learning and fun activities will be presented by the Child Life staff from the University of Michigan.

Kids Kamp is now divided into two age groups. One for ages 3-5 and one for ages 6 and older.

On Saturday evening there will be a family dinner where participants can relax and enjoy time with new friends.

The 2014 Neutropenia Family Conference comes to a close on Sunday, July 13 after breakfast and a final session.

Friday, July 11, 2014 at 1:00 PM Sunday, July 13, 2014 at 11:00 AM (EDT), Ann Arbor, MI

To learn more and/or to register go to Neutropenia Family Conference. www.neutropenianet.org.

 

Teva UK Limited Launches A New Long-Acting G-CSF

Teva UK Limited has today launched Lonquex® (lipegfilgrastim) in the UK.  Lonquex® is indicated for the reduction of the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes).

Lonquex® is a new long-acting recombinant granulocyte colony-stimulating factor (G-CSF) with the active ingredient lipegfilgrastim – a novel glycoPEGylated (PEG; polyethylene glycol) filgrastim molecule.  Lonquex® is intended as a once per cycle fixed dose, subcutaneous injection for neutrophil support in cancer patients receiving myelosuppresive chemotherapy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes).

The UK launch is part of a Europe-wide market launch of the medicine, and it has already been launched in several European markets, including Germany, the Netherlands and Ireland.

Read more: PR Newswire

How Plerixa Fights Neutropenia

English: Reference ranges for white blood cell...

Ariana-Leilani suffer from Severe Chronic Neutroepnai.   Neutropenia is a condition characterized by the lack of a type of white blood cells, also known as neutrophils, in one’s blood circulation. Patients with neutropenia who receive plerixafor become prone to oral, skin, genital infections and in worst cases, a fatal whole-body infection.

Previously, it was known that Plerixafor increases the concentration of these white blood cells in the blood by inhibiting a protein called CXCR4. This inhibition prevents neutrophils in the blood stream from returning to the bone marrow, which is the primary compartment where the white blood cells are stored and released. It is therefore commonly accepted that the efficacy of the drug arises only from the release of these white blood cells from the bone marrow.

However, scientists at SIgN found that the inhibition of CXCR4 by the drug actually plays a dual role – it increases the neutrophil count in the blood through their release from the lungs, while simultaneously promoting their retention in the blood stream. Results of the study were published in the Journal of Experimental Medicine (JEM).

Discovery of this additional mode of action not only provides a deeper understanding on the drug’s mechanism; it also contributes to a more effective use of the drug, which may help to reduce the risk of bacterial infections in neutropenic patients.

“We have identified the precise mechanisms of plerixafor treatment, which has important implications on its usage. We can understand through this study the effectiveness or limitations of the drug on certain patients and thereafter craft new clinical approaches to better treat them. Our study forms a conceptual framework to establish improved therapeutic strategies for neutropenia,” said team leader Dr. Ng Lai Guan from SIgN.

The article can be found at: Devi S et al. (2013) Neutrophil mobilization via plerixafor-mediated CXCR4 inhibition arises from lung demargination and blockade of neutrophil homing to the bone marrow.

Sign the petition to make sure that Ariana-Leilani gets the life saving medicine to fight her severe chronic neutropenia:

GoPetition for ariana-leilani

The US Must Respect Human Rights, Stop Spying on Its Allies and Sign the UN Convention on the Rights of the Child

Official photographic portrait of US President...

Official photographic portrait of US President Barack Obama (born 4 August 1961; assumed office 20 January 2009) (Photo credit: Wikipedia)

The recent revelations of US spying on allied leaders is part of a greater public relations problem of the US: appearing to exempt itself from the rules that apply to other countries.

The moral authority of the US is at an all time low, and the diminished view is, for the most part, the fault of the US itself.   Its failure to ratify the UN Convention on the Rights of the Child (UNCRC) exemplifies the problem and is having detrimental life threatening effect on children in the US.

The US helped draft the UN CRC and even signed it when it was originally adopted.  However, after 20 years the US is the only developed country that has not ratified it.    The failure of the US to provide life saving medicine to dual citizen, German-American Ariana-Leilani after six years of suffering is a direct consequence of the US failure to respect the international rights of children living within its borders.

It is time for the US to respect 10 year old Ariana-Leilani’s rights and for the US to ratify the UN Convention on the Rights of the Child.  It is time for the US to join the rest of the world.

Join the thousands of others who have signed the petition urging President Obama to immediately take action to save Ariana-Leilani and to ratify the UNCRC.

GoPetition for ariana-leilani

Amgen To Buy Roche Rights to Neutropenia Drugs from Roche In 100 Markets

AMGen Corporate Logo, 1983

AMGen Corporate Logo, 1983 (Photo credit: Wikipedia)

Amgen is acquiring Roche‘s rights to the neutropenia drugs filgrastim and pegfilgrastim in approximately 100 markets outside the United States, effective January 1, 2014, for an undisclosed price.

Filgrastim and pegfilgrastim are marketed by Amgen in the United States and Europe under the trade names Neupogen® and Neulasta®, respectively. Since 1989, Roche has held the rights to filgrastim and pegfilgrastim under license from Kirin-Amgen, a joint venture between Amgen and Japan’s Kirin Holdings, in Eastern Europe, Latin America, Asia, the Middle East, and Africa.

In those regions of the world, filgrastim and pegfilgrastim generated a combined approximately $200 million in sales last year. Roche had not disclosed sales for either drug last year or earlier this year.

The drugs are designed to reduce the risk of infection in patients receiving chemotherapy by boosting white blood cell counts.

Japan’s Kyowa Hakko Kirin will continue to retain product rights and to market filgrastim and pegfilgrastim in parts of Asia, including China and Japan.

Alternative to Amgen’s Neupogen Ready to Launch

עברית: פועלת במפעל "אסיא"

Filgrastim stimulates the production of neutrophils, a type of white blood cells important in the body’s fight against infection. One treatment option for individuals with certain types of cancers is myelosuppressive chemotherapy which targets rapidly dividing tumor cells. Since rapidly dividing normal cells, such as bone marrow precursor neutrophils are also vulnerable to the cytotoxic effects of myelosuppressive chemotherapy, lower numbers of neutrophils are produced, a condition called neutropenia, which increases the risk of severe infection. Amgen launched filgrastim, under the Neupogen brand, in 1991 to decrease the incidence of infection, as manifested by febrile neutropenia in patients with nonmyeloid malignancies receiving myelosuppressive anticancer drugs associated with a clinically significant incidence of febrile neutropenia.

Dr. Karl Welte is the researcher who discovered neupogen (GCSF).   Dr. Welte has been a major advocate for Ariana-Leilani to receive life saving GCSF to save her life.

Since Amgen’s principal European patent related to filgrastim was due to expire in August 2006, the company launched a long acting formulation, a pegylated filgrastim (pegfilgrastim), under the brand name Neulasta in 2002. The move, part of Amgen’s product lifecycle management strategy, resulted in Neulasta replacing Neupogen in major treatment centers. Amgen’s US patent for Neupogen expires in December 2013. Although Neulasta’s patent does not expire until December 2015, the launch of generic competition could adversely affect sales of both brands.

Teva is a serious player in the international biosimilars market. Teva also has two long acting filgrastim formulations in late stage development.  In August 2012, the FDA approved Teva‘s (TEVA) Neutroval (tbofilgrastim). The approval, which was based on a full Biologic License Application (BLA) rather than under the FDA’s new biosimilar approval pathway, would allow Teva to compete directly with Amgen‘s (AMGN) US filgrastim franchise, worth $4.2 billion at the end of 2012. However, due to a court ruling related to Amgen’s filgrastim patents, Teva may not start selling its product in the US until the 10th of November 2013, in just six weeks’ time.  Read More: Seeking Alpha