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Local Girl Hoping to Raise Money for Neutropenia Through Special Event

Hoping to help support a better treatment for Neutropenia, ten-year-old Kara Loader, who was diagnosed with the disorder, is hosting “Bowling for Neutrophils” on March 15 in St. Clairsville.  Ariana-Leilani also suffers from Neutroepenia. Neutropenia attacks and kills neutrophils which are the part of white blood cells that fight infections in the body. Kara and her mother, Tammy, decided on a bowling event because they say pretty much anyone can bowl and almost every community has a bowling alley.

Besides bowling plenty of strikes, Kara hopes to accomplish another thing next Saturday. “I would like to get at least a thousand dollars for neutropenia because they’re trying to come up with a pill so I don’t have to take a shot anymore,” said Kara. “That’d be really nice.”

Teams of 5 to 7 people can sign up before hand and raise funds for the event. Their goal is for each team to raise at least 100 dollars. Individual bowlers are welcome too.

Prizes will be given away for the best team name, and for whoever raises the most money. Bowling for Neutrophils will be held Saturday night, March 15, at 5:30 p.m. at St. Clair Lanes, St. Clairsville, Ohio.

For more information, call 740-680-3080.

Learn more: Fund Raising for Neutropenia

Amgen’s Neulasta One of the Top Selling Drugs

Medscape News published a list of the top selling drugs in the United States based on a list produced by IMS Health.   Amgen’s Neulasta, a long acting form of neupogen for the treatment of neutropenia (the treatment denied but needed by Ariana-Leilani), made tenth place at $3.6 billion in annual sales.   Read more at Medscape

Reducing GCSF Increases Incidence of Neutropenia

Hematopoietic growth factors

Hematopoietic growth factors (Photo credit: Wikipedia)

A study involving 167 patients was published in the December 1 issue of the Journal of Clinical Oncology and reported that when the use of prophylactic granulocyte-colony stimulating factor (G-CSF) to reduce febrile neutropenia (FN) was decreased in patients receiving chemotherapy for breast cancer, it led to a more than 5-fold increase in FN episodes and an early end to the clinical trial.

In an accompanying study, Dr. Aarts’s research team evaluated the cost effectiveness of pegfilgrastim prophylaxis for FN. They conclude that skipping G-CSF after the first 2 chemo cycles saved an average of €3491 per patient. They estimate that nationwide, this would translate into a saving of €14.3 million each year.

However, this resulted in an unexpected and substantial increase in FN incidence. The researchers calculate that the incremental cost-effectiveness ratio for preventing an FN episode by giving G-CSF with every chemotherapy cycle, compared with only for the first 2 cycles, worked out to €13,112 per patient.

“When one is willing to pay at least €13,122 per patient with FN episodes prevented, 6 cycles of G-CSF prophylaxis is considered a cost-effective strategy,” they conclude.

Read More: Medscape

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

European approval opens the door for Grastofil

Neutrophile segmented Granulocyte

Neutrophile segmented Granulocyte (Photo credit: Wikipedia)

STADA Arzneimittel AG has strengthened its activities in the biosimilars area and in-licensed a filgrastim product from the largest Canadian owned pharmaceutical company Apotex Inc. The biosimilar, marketed under the name Grastofil, received approval from the European Commission for the treatment of Neutropenia in adults. The condition results in an abnormally low number of neutrophil granulocytes, a type of white blood cell that is essential for a functional immune system. STADA shall assume sales of the biosimilar for nearly all EU countries. Sales are planned to commence in 2014.

Grastofil will be available in pre-filled syringes with two different active ingredient concentrations for intravenous or subcutaneous injection. “The effectiveness, tolerability and quality of Grastofil are comparable to the original product within the approved area of application,” explains Mack. “This was proven in comprehensive, comparative studies with the original product and has now been confirmed with the approval of the European Commission. With Grastofil, we will be able to offer an inexpensive alternative therapy in the future that doctors, patients and even the public health care system will benefit from.”

Neutropenia can arise as a result of cytotoxic chemotherapy, for example. Grastofil promotes the generation of neutrophil granulocytes and their release from the bone marrow and, therefore, can be used to support chemotherapy for malignant diseases in order to reduce the risk of neutropenia-induced infection as well as to support the antibiotic treatment of this type of infection. The approval also comprises the application to mobilize peripheral blood stem cells.

A biosimilar is a drug with a protein produced by biotechnological process as an active pharmaceutical ingredient and developed in comparison to an original product which is already on the market, and that is so similar to this original product that the biosimilar has proven therapeutic equivalence.

Ariana-Leilani suffers from Severe Chronic Neutropenia — where her neutrophil levels are at life threatening low levels making her prone to fatal infection.   She needs medicine to control her condition.  Please sign her petition:

GoPetition for ariana-leilani

Related articles

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.

ORPHAN DRUG AND NORD SAVED THEIR SON’S LIFE

The Millers’ three-year-old son, Evan, exhibited frequent symptoms of distress.  “People kept telling us he was just a cranky, colicky baby,” says Amanda.  At 12 weeks of age, Evan abruptly stopped taking any food and screamed when anyone touched his abdomen.  In the emergency room, it was determined that Evan had fluid in his abdomen and low blood sugar.  His blood wasn’t clotting and his kidneys and spleen were enlarged.  Most alarming, there were signs of liver failure.

The Children’s Hospital of Philadelphia diagnosed the condition as  tyrosinemia type 1, which is a rare metabolic disease.

The orphan drug Orfadin® was approved by FDA in 2002.  However, because Jon is self-employed as an auto mechanic, the family did not have insurance that would cover the cost of the drug.   Jon connected online with another parent who told him about NORD and its Patient Assistance Programs, which help patients obtain needed medications.  While other organizations and companies provide PAPs for medications today, NORD was the first to do so.

NORD partners with Rare Disease Therapeutics on a PAP for Orfadin®.  When Jon Miller called to inquire about that program, he spoke to Michelle Mortara, a patient assistance specialist.  NORD, the specialty pharmacy, and the company providing the drug.

Within one day, the medication arrived at the hospital, and little Evan and his parents were soon on their way home.  Today, Evan attends pre-school and “does all the things three-year-olds like to do,” his mother says.

Have Ariana-leilani have the same benefit of a cure.  Please Sign the petition to help save Ariana-Leilani.

GoPetition for ariana-leilani

Read More:

Read More: http://rarediseases.org/about/millers-story

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