NCC and Pfizer Partner to Provide 10 Million Units of Hemophilia Medicines

source: PR Newswire

RALEIGH, N.C., Feb. 23 /PRNewswire-USNewswire/ — The National Cancer Coalition (NCC) is pleased to announce its recent collaboration with Pfizer, Inc to provide hemophilia medicines to underserved patients in the developing world.   To date, NCC has received more than 10 million international units of ReFacto® Antihemophilic Factor (Recombinant), a therapy used for the treatment and prevention of bleeding in people with hemophilia A, to assist patients who otherwise would have little opportunity for adequate treatment.    The value of this donation is more than $13 million, and it will help many hundreds of hemophilia patients around the world.

“Pfizer is dedicated to developing and delivering innovative hemophilia therapies and we are proud of our partnership with the National Cancer Coalition, which is helping to distribute Pfizer-donated factor product to patients in the global hemophilia community,” says Jean-Michel Halfon, President and General Manager, Emerging Markets, Pfizer. “We are committed to working with partner organizations like NCC to provide access to hemophilia medicines to patients who need them most.”

Pfizer’s donation will help to bring much-needed hemophilia therapy to patients who may be untreated or inadequately treated.  In addition, these donations help to support the development of the infrastructure which creates long term value beyond the donation itself.  This current donation to the National Cancer Coalition is part of the largest hemophilia donation that has ever been made in Pfizer’s history.

“Our organization is dedicated to providing the resources and tools needed in the treatment and prevention of cancer, hemophilia, and other serious diseases and illnesses throughout the world,” says Robert Landry, President of the National Cancer Coalition.  ”Product donations such as Pfizer’s are essential to addressing the needs of the patients and families that we serve globally.”   National Cancer Coalition, a non-governmental organization (NGO), is a preferred partner of Pfizer and the two organizations have collaborative projects in a number of other areas including cancer, depression, and arthritis.  

Hemophilia A is a rare, inherited blood-clotting disorder characterized by spontaneous hemorrhages or prolonged bleeding, typically into joints and soft tissue. People with hemophilia A are deficient in a key protein — factor VIII — that is vital in the clotting mechanism to prevent bleeding. Most patients with hemophilia A are dependent on factor VIII replacement therapy.

The National Cancer Coalition, a 501(c)(3) not-for-profit corporation, supports cancer relief, research, and educational programs throughout the world. The Coalition’s international medical assistance program, termed NCC Cares, has provided requested specialty pharmaceuticals, essential medicines, hospital supplies and medical equipment to public hospitals and local humanitarian organizations that help needy patients in over 50 developing countries around the world.  The NCC Provides program awards financial assistance for American cancer patients who would otherwise not be able to complete their treatments.  For additional information, visit their website at: www.nationalcancercoalition.org

Iran’s First Transgenic Goats Produced in Royan Institute

Iran and Middle East’s first transgenic goats which can produce recombinant proteins for treatment of Hemophilia were born on January 9, 2010 at Royan Institute.

Source: Royan Institute

During a news conference on Saturday, Jan. 30th, Hamid Gourabi, the head of Royan Institute said: Following 3 years of research on transgenic animals, the institute has succeeded in producing of two transgenic goats containing coagulation factor IX in their milk which is an important drug using in treatment of hemophilia patients

Transgenic animal is defined as one that has undergone a modification in its genome. The main purpose of producing transgenic animals is to produce animals that contain the gene for secretion of some proteins in their milk which can be used in treatment of human diseases.

Transgenic goats, cows, sheeps and pigs are already produced in USA, France, UK, Japan, Denmark, Canada, Scotland, Netherlands and China to extract tissue plasminogen activator (tPA), α-Antitrypsin, coagulation factor IIX, fibrinogen, α-Lactalbumin, human serum albumin, collagen type I/II and monoclonal antibodies from their milk.
Gourabi notified that producing drugs in transgenic livestock milk is an economical and cost benefit method since the expenses are much less than producing the same drugs in laboratory by tissue culture methods.

Scientists who were cooperating in this project explained the procedure:
Hemophilia is an X-linked disorder causing by deficiency of coagulation factor IX, which is commonly produced in liver. In this project we tried to produce this protein by transferring the related gene from human liver cells to the goat embryonic fibroblasts. The embryos were then transferred to the recipient goats. Different tests such as PCR have confirmed that the goats born from these embryos contain the gene in their cells and can secrete the protein in their milk.

Upon the completion of Royan Institute’s project on transgenic animals, Iran is expected to take an effective step for mass production of factor IX and other thrombolytics so as to increase its affordability for patients.
In 2006, Royan Institute produced the Iran and Middle East’s first cloned lamb named Royana, and then in 2009, Hanna, the first cloned goat was born. Bonyana and Tamina were also first cloned calves produced in Royan Institute in 2009 and were died due to an infectious disease in a couple of days.

Royan TGF91 and Royan TGF92, the two transgenic goats were also named “Shangoul” and “Mangoul”, names of the two leading characters of an Iranian traditional children story. They are now in a good health condition.

Hemophilia Video – Inhibitor

Found this video on Vimeo.  Great work by the dad at telling the story with his son.  Son has Hemophilia and has an inhibitor.  Great work!

To see the video please follow this link, MyLife with Hemophilia.

For more about the Director, click here.  Thank you, Mark and Brandston for the insight your family brings to the community.  Our hope is they can produce more.

Book – “The Immortal Life of Henrietta Lacks,” by science writer Rebecca Skloot

Why we are all indebted to a little-known black woman who died in the 1950s.

Taken from: Deborah L. Shelton is a health reporter for the Chicago Tribune.
                           Copyright © 2010, Chicago Tribune

Virtually all of us have benefited in some way from her immense contributions to science and medicine. Research using Lacks’ cells was essential to developing the polio vaccine, in-vitro fertilization, cloning and gene mapping. They aided scientists to better understand the workings of cancer and countless viruses and were used to develop drugs for herpes, leukemia, influenza, hemophilia and Parkinson’s disease.

Doctors at Johns Hopkins Hospital in Baltimore didn’t get informed consent to research tissue they sliced from her cervix as she battled a vicious form of cervical cancer. Treated at Hopkins because it was the only hospital for miles that treated black patients, she died an excruciating death in a segregated ward at the tragically young age of 31.

She left behind five young children. To this day, her body lays buried in an unmarked grave. 

Lacks’ husband and children did not know about the existence of her cells for more than 20 years after her death. Despite the fortune made by biotech companies from her cells, members of her family didn’t see a penny. Today, many of her descendants cannot afford health insurance.

For more of the story, click here.
For information on the book,  click here.

fVIII protein for blood cloting is produced by blood vessels in the lungs

Imperial College London News Release
Wednesday 10 February 2010

A key protein that causes the blood to clot is produced by blood vessels in the lungs and not just the liver, according to new research published today in the journal PLoS One, led by scientists at Imperial College London.

The findings may ultimately help scientists to develop better treatments for conditions where the blood’s ability to clot is impaired, including deep vein thrombosis, where dangerous blood clots form inside the body, and haemophilia A, where the blood cannot clot sufficiently well.

It has long been known that an agent called factor VIII plays a key role in enabling the blood to clot. Too much factor VIII puts people at risk of excessive clots. Low levels of factor VIII cause bleeding in people with haemophilia A and factor VIII replacement is used to treat this.

Prior to recent research it had been believed that most factor VIII was produced by the liver. Therapeutic options for patients with haemophilia have included replacing liver cells, for example through transplantation.

Dr Claire Shovlin , the lead author of the study from the National Heart and Lung Institute at Imperial College London, said: “Our study suggests that the blood vessels in the lung are playing a crucial role in altering how blood clots form in the body. There are a huge number of these blood vessels – they cover an area equivalent in size to a squash court, effectively 20 times the surface area of all other blood vessels combined.

“This means it’s really important for us to understand exactly how the behaviour of the lung blood vessels might be affecting diseases where blood clotting is a factor. Further research on how the lungs modify the clotting potential of the blood flowing through them could open up new avenues for treatments,” added Dr Shovlin.

In 2006, researchers from the University of Leuven in Belgium published a study in the journal Blood looking at donated lung tissue. The researchers passed fluid through the tissue and found that in three of the four lung samples studied, the levels of factor VIII increased in the fluid after passing through the lungs. They also provided evidence that some lung blood vessels could produce factor VIII in cell culture.

The researchers behind today’s study have confirmed that the blood vessels in the lungs should be an important site for regulating the formation of clots, both in the lungs themselves and also, potentially, in the rest of the body. They used different microscopic and biochemical techniques to look at samples of lung tissue and blood samples in minute detail.

Tissue and blood samples for the work leading up to this study were donated by patients from Imperial College Healthcare NHS Trust, with tissue processed through the Hammersmith Hospital Tissue Bank.

The team found that factor VIII could be seen in the lung tissue and was present on the surface of blood vessel cells in the lungs. They also found that factor VIII localises in the lungs with a protein called von Willebrand factor, which protects factor VIII from degradation. The work also showed that how the factor VIII gene may be decoded is much more complicated than previously thought. This could potentially alter how scientists approach the treatment of the many conditions where blood clotting is a factor.

The researchers hope that ultimately new drugs can be developed that target production of factor VIII in the lungs, to improve the body’s ability to ensure that the blood clots properly. By targeting the lungs, the researchers think it may be possible to make more effective drugs that produce fewer side effects than current treatments.

Original article: “Endothelial Cell Processing and Alternatively Spliced Transcripts of factor VIII: Potential Implications for Coagulation Cascades and Pulmonary Hypertension”, PLoS One, Wednesday 10 February 2010

Lead author: Dr Claire Shovlin, Imperial College London (for full list of authors please see paper)

For further information please contact:

Laura Gallagher
Research Media Relations Manager
Imperial College London
e-mail: l.gallagher@imperial.ac.uk
Telephone: +44 (0)207 594 8432 or ext. 48432
Out of hours duty Press Officer: +44 (0)7803 886 248