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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

ABC’s Robin Roberts Urges You to Be a Bone Marrow Donor (Day 5 of 19 Days of Activism)

English: Logo of National Marrow Donor Program

“In June of 2012, ABC’s Good Morning America anchor, Robin Roberts announced that she had been diagnosed with myelodysplastic syndrome (MDS) and her best treatment option was a bone marrow transplant. Fortunately her sister Sally-Ann was a perfect match and became her marrow donor. More than 70% of patients will not have a match in their family. They depend on the Be The Match Registry® to find that match — someone like you.

As a Be The Match Ambassador of Hope, Robin is asking others to join the Be The Match Registry as a potential marrow donor. Together we can help eliminate blood cancer.”   Click here for more information

Also, sign the petition to save 10 year old Ariana-Leilani, who needs life saving medicine to cause her bone marrow to produce enough neutrophils:

GoPetition for ariana-leilani

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

Talia Joy Castellano Dead: Inspirational YouTube Child Star Dies After 6-Year Battle With Cancer

We regret to announce that Talia Joy Castellano lost her battle to cancer yesterday.  “The bubbly girl who wanted to do something about childhood cancer.”   #‎prayfortalia Talia is 13, a little older than Ariana-Leilani.   They both became sick at about the same age.  In September, Ellen DeGeneres invited the Florida native to appear on her show and revealed that Talia had been chosen to be an honorary CoverGirl.

As Talia says, there should be so many other people helping people.   There is still time to help Ariana-Leilani who suffers from untreated Severe Chronic Neutropenia in Washington DC.   Please sign her petition today:

GoPetition

Inspirational 10 Year Old, “Live Like Bella,” Loses 6 year Fight to Cancer

Live Like Bella 10 Year Old Loses Fight to Cancer

Live Like Bella 10 Year Old Loses Fight to Cancer

Bella Rodriguez-Torres died after a 6-year battle with rhabdomyosarcoma, a cancerous tumor of the muscles that are attached to bones.   When she was first diagnosed stage IV cancer at 4 years old, doctors only gave Bella four months to live. She surprisingly recovered, and started to walk on her own two feet and the tumors soon disappeared. However, the cancer returned throughout her life, necessitating numerous rounds of chemotherapy, radiation and surgery. She still didn’t give up.

Supporters of Bella began to “Live Llke Bella,” a motto for pursuing everything that life has to offer.
“Live Like Bella means to eat something and enjoy it, savor it, go play with gusto, even the simple things. Do them and enjoy them. Learn something new, get out of your comfort zone and enjoy life,” Ralph explained.

LeBron James adopted the motto. He and his teammate Dwyane Wade wrote the slogan on their shoes before the Miami Heat’s game against the Indiana Pacers on Tuesday.

Live like Bella, please sign 10 year old Ariana-Leilani’s Petition to get needed medicine and medical care for her life-threatening Severe Chronic Neutropenia:

GoPetition