Montreal, Canada, January 10, 2012 — The World Federation of Hemophilia is pleased to announce the appointment of John Bournas as CEO/Executive Director. Mr Bournas brings over fifteen years of senior managerial experience in the healthcare and not-for-profit sectors and international experience as a diplomat. His areas of expertise include management, business development, healthcare advocacy and cultivating relationships with stakeholders of the highest level of government. Mr Bournas will start his new position on January 25.

“The WFH is delighted to welcome John Bournas to our team,” said WFH president Mark Skinner. “He brings a unique combination of skills and an international perspective that will be integral to achieving our strategic goals in the coming years.”

Prior to joining the WFH, Mr Bournas was senior director of international affairs with the American College of Cardiology Foundation (ACC), the largest international professional society for cardiologists. He was responsible for global strategic planning, international memberships, partnering with national healthcare societies, global advocacy, and for diversifying the organization’s revenue stream through engagement with its corporate partners. Before that, Mr Bournas was senior director-international with Cardinal Health in the U.S., a Fortune 19 company that specialized in improving the cost-effectiveness of health care.

Mr Bournas has also worked and travelled throughout the world developing relationships and bridging gaps between established and emerging countries. He worked as a diplomat in Chile, Australia, and Japan overseeing international trade and development projects. The son of Chilean and Greek parents, he was raised in Chile and the United States. He speaks Spanish, French, Portuguese, and some Japanese and Greek.

Mr Bournas holds a Master of Business Administration degree from Macquarie University in Australia, a Master of Arts in Political Science from Fordham University in the U.S., and a Bachelor of Arts in International Studies from Fairleigh Dickinson University in the U.S.

About the WFH
The World Federation of Hemophilia (WFH) is an international not for profit organization with member patient organizations in 118 countries and official recognition by the World Health Organization (WHO). It has been dedicated to improving and sustaining care for hemophilia and other inherited bleeding disorders worldwide since it was founded in 1963.

For more information, please contact:
Elizabeth Myles
Director of Communications
World Federation of Hemophilia
emyles@wfh.org
Tel: +1-514-875-7944

• The largest plan by enrollment in any of the three largest small group insurance products in the state’s small group market;
• Any of the largest three state employee health benefit plans by enrollment;
• Any of the largest three national FEHBP plan options by enrollment; or
• The largest insured commercial non-Medicaid Health Management Organization (HMO) operating in the state.

HHS announced this approach after seeking input from the Institute of Medicine (IOM), the public and the Department of Labor.  HHS intends to propose that if a benchmark is missing categories of benefits, the state must supplement the missing categories using the benefits from any other benchmark option.  Health insurance companies will have some flexibility to adjust benefits.

Since HHS has decided to let states decide EHBs, instead of creating a federal EHBs standard, many fear that states will continue the status quo or even have low EHBs standards because of costs concerns.  The federal government will subsidize coverage based on the essential health benefits, any coverage options above that standard states will have to pick up the cost.

It is important to evaluate how your state ranks regarding insurance coverage and benefits provided for patients with bleeding disorders.  Whether or not there are protections in place, continued advocacy is still necessary.  State advocacy of all stakeholders involved in this process: Legislators, Exchange Boards, Insurance Departments, Executive Branch, etc. will be crucial.

HHS is accepting public comments on the bulletin by Jan 31, 2012. The regulatory process for EHBs will likely continue into the year, guidance on calculating actuarial value, cost sharing and the provision of minimum value by employer-sponsored coverage will be released later, with more opportunities for public comment.

Read HHS press release here.

Read the Bulletin here.

BAXTER INITIATES PHASE I CLINICAL TRIAL OF LONGER-ACTING RECOMBINANT FVIII TREATMENT FOR HEMOPHILIA A
Investigational BAX 855 is based on ADVATE Factor VIII Molecule
DEERFIELD, Ill., January 5, 2012 – Baxter International Inc. today announced the dosing of the first patients in a Phase I clinical trial of its lead investigational candidate, BAX 855, a longer-acting (PEGylated) form of a full-length recombinant factor VIII (rFVIII) protein. BAX 855 is based on Baxter’s ADVATE [Antihemophilic Factor (Recombinant) Plasma/Albumin-Free Method] full-length rFVIII molecule and plasma/albumin-free (PAF) manufacturing process.
The Phase I trial is a prospective, open-label study that will assess the safety, tolerability and pharmacokinetics of BAX 855 in previously-treated patients aged 12 years or older with severe hemophilia A. When used for prophylaxis, Baxter’s ADVATE requires patients to infuse every two to three days to reduce the occurrence of bleeding episodes. This Phase I trial is the first step in assessing whether BAX 855 can be infused less frequently.
“This trial is designed to provide new insights about our investigational longer-acting FVIII molecule, BAX 855, with the ultimate goal of improving care for patients living with

hemophilia A,” said Prof. Hartmut J. Ehrlich, M.D., vice president of global research and development in Baxter’s BioScience business. “The Phase I results will serve as the foundation for advancing this important program through clinical development and determining whether BAX 855 can offer a treatment regimen requiring fewer infusions than ADVATE.”
BAX 855 employs Baxter’s proprietary full-length plasma/albumin-free recombinant protein platform that does not contain any human or animal-derived additives. BAX 855 leverages Nektar Therapeutics’ (NASDAQ: NKTR) proprietary PEGylation technology, which is designed to extend the duration of activity of proteins and larger molecules. Baxter and Nektar have a collaboration to develop PEGylated products designed to provide new long- acting therapies for hemophilia patients.
ADVATE was recently approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A. With the inclusion of prophylaxis in the adult patient population, ADVATE became the only antihemophilic factor approved in the United States for prophylactic use in both children and adults. This approval was supported by a Phase IV prophylaxis study sponsored by Baxter demonstrating that ADVATE for routine prophylaxis significantly reduced median annual bleed rates (ABR) in hemophilia A patients. In the study, patients experienced 44 bleeds (per patient per year) during on-demand treatment compared to one bleed (per patient per year) while on either of the prophylactic regimens evaluated, a 98 percent reduction in annual bleed rate (p<0.0001). Nearly half (42 percent) of patients experienced zero bleeding episodes during one year on

prophylactic therapy. Evaluable patients were those with at least 90 percent adherence to their prescribed prophylactic regimen.
About ADVATE
ADVATE was initially approved by the FDA in July 2003 for control and prevention of bleeding episodes in adults and children with hemophilia A.
ADVATE (derived from the complete FVIII gene) is a recombinant FVIII therapy that is processed without any blood-based additives. Because no blood-derived components are added at any stage of the manufacturing process, the potential risk of transmitting pathogens that may be carried in blood-based additives is eliminated. There have been no confirmed reports of transmission of HIV, HBV or HCV with rFVIII therapies.
ADVATE is approved in 53 countries worldwide including the United States, Canada, 27 countries in the European Union, Argentina, Australia, Brazil, Chile, Colombia, Croatia, Hong Kong, Iceland, Iraq, Japan, Macau, Malaysia, New Zealand, Norway, Panama, Puerto Rico, Serbia, Singapore, South Korea, Suriname, Switzerland, Taiwan, Uruguay and Venezuela. Since the initial approval of ADVATE, more than 10 billion international units have been distributed, and ADVATE is the number one chosen rFVIII worldwide.
ADVATE is an Antihemophilic Factor (Recombinant) indicated for: Control and prevention of bleeding episodes in adults and children (0-16 years) with hemophilia A Routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children (0-16 years) with hemophilia A Perioperative management in adults and children (0-16 years) with hemophilia A

ADVATE is not indicated for the treatment of von Willebrand disease
Important Risk Information for ADVATE
ADVATE is contraindicated in patients with known anaphylaxis to mouse or hamster protein or other constituents of the product.
Allergic-type hypersensitivity reactions, including anaphylaxis, are possible and have been reported with ADVATE. Symptoms have manifested as dizziness, paresthesia, rash, flushing, face swelling, urticaria, dyspnea, and pruritis. Discontinue use if hypersensitivity symptoms occur and administer appropriate emergency treatment.
Carefully monitor patients treated with AHF products for the development of factor VIII inhibitors by appropriate clinical observations and laboratory tests. Inhibitors have been reported following administration of ADVATE predominantly in previously-untreated patients (PUPs) and previously minimally-treated patients (MTPs).
If expected plasma FVIII levels are not attained, or if bleeding is not controlled with an expected dose, perform an assay that measures factor VIII inhibitor concentration.
The serious adverse drug reactions (ADRs) seen with ADVATE are hypersensitivity reactions and the development of high-titer inhibitors necessitating alternative treatments to Factor VIII. The most common ADRs observed in clinical trials (frequency ≥ 10 percent of patients) were pyrexia, headache, cough, nasopharyngitis, vomiting, arthralgia, and limb injury.
Please see full prescribing information at www.advate.com or click here.

About Hemophilia
Hemophilia is a rare genetic blood clotting disorder.1 People living with hemophilia do not have enough of, or are missing, one of the blood clotting proteins naturally found in blood.1 Two of the most common forms of hemophilia are A and B and primarily affect males.2 In people with hemophilia A, clotting factor VIII is not present in sufficient amounts or is absent.2 Without enough FVIII, people with hemophilia can experience spontaneous, uncontrolled internal bleeding that is painful, debilitating, damaging to joints and potentially fatal.2 People with hemophilia B (also called Christmas disease) do not have sufficient amounts of clotting factor IX.2 In about 30 percent of cases, there is no family history of hemophilia and the condition is the result of a spontaneous gene mutation.3 According to the World Federation of Hemophilia, more than 400,000 people in the world have hemophilia.1 All races and economic groups are affected equally.1
About Baxter International Inc.
Baxter International Inc., through its subsidiaries, develops, manufactures and markets products that save and sustain the lives of people with hemophilia, immune disorders, cancer, infectious diseases, kidney disease, trauma and other chronic and acute medical conditions. As a global, diversified healthcare company, Baxter applies a unique combination of expertise in medical devices, pharmaceuticals and biotechnology to create products that advance patient care worldwide.
This release includes forward-looking statements concerning a Phase I trial of the Company’s investigational compound BAX 855, including with respect to expectations related to clinical

BAXTER INITIATES PHASE I CLINICAL TRIAL OF LONGER-ACTING RECOMBINANT FVIII TREATMENT FOR HEMOPHILIA A –
outcomes. The statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: timely submission and approval of anticipated regulatory filings;
clinical results validating the use of BAX 855 to treat patients with severe hemophilia A; satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; and other risks identified in Baxter’s most recent filing on Form 10- K and other SEC filings, all of which are available on the company’s website. Baxter does not undertake to update its forward-looking statements.

ADVATE for routine prophylaxis reduced annual bleed events in hemophilia A patients from forty-four to one as compared to an on-demand regimen in a clinical study

Once every third day pharmacokinetic dosing option offers some patients
the opportunity for fewer infusions annually

DEERFIELD, Ill., December 16, 2011 – Baxter International Inc. today announced that the U.S. Food and Drug Administration (FDA) has approved ADVATE [Antihemophilic Factor (Recombinant) Plasma/Albumin Free Method] for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A.  ADVATE is the only antihemophilic factor approved in the United States for prophylactic use in both adults and children.

The approval is based on a Phase IV prophylaxis study sponsored by Baxter demonstrating that ADVATE for routine prophylaxis significantly reduced median annual bleed rates (ABR) in hemophilia A patients from 44 to one as compared to an on-demand regimen.  Forty-two percent of study patients experienced zero bleeds during one year on prophylaxis.  Of the two prophylactic regimens approved for use, the dosing schedule of every three days, a pharmacokinetic-driven regimen based on patient’s clinical response, offered some patients the option of fewer infusions over one year of treatment.

“Emerging data provide important information to help physicians optimize care for hemophilia patients of all ages by preventing unexpected bleeding events that can have a detrimental impact on the lives of patients,” said Leonard Valentino, M.D., Director, Rush Hemophilia and Thrombophilia Center and Section of Pediatric Hematology/Oncology, Rush University Medical Center, Chicago, and lead investigator of this study. “These data confirm the important clinical benefits of ADVATE when used as a prophylactic therapy to reduce bleeding episodes.”

The study findings demonstrated a statistically significant reduction in the median annual bleeding rate, with patients experiencing 44 bleeds (per patient per year) during on-demand treatment compared to one bleed (per patient per year) while on either of the prophylactic regimens evaluated, a 98 percent reduction in annual bleed rate (p<0.0001).  Nearly half (42 percent) of patients experienced no bleeding episodes while on one year of prophylactic treatment.  Evaluable patients had greater than or equal to 90 percent adherence to the prescribed prophylactic regimen.  While the trial was not powered to demonstrate equivalence in bleeding rate between the two prophylaxis arms, there was no statistically significant difference in bleeding frequency observed between the two prophylaxis regimens studied.

“This latest clinical milestone for ADVATE is an important step forward for people living with hemophilia A as we continue to research ways to advance care for this patient population,” said Bruce Ewenstein, M.D., Ph.D, vice president, clinical affairs, Baxter’s BioScience business. “This rigorous clinical study demonstrated that the number of bleeding episodes experienced each year could be reduced to as low as one event with prophylactic treatment.  Further, the pharmacokinetic-driven dosing regimen based on patient’s clinical response every third day, offers some patients the option of fewer infusions over one year of treatment than the current standard prophylaxis regimen.”

For the prophylaxis regimen to prevent or reduce frequency of bleeding episodes, ADVATE dosing of three to four times weekly (between 20 to 40 international units of factor VIII per kg body weight every other day) may be used.  Alternatively, an every third day dosing regimen targeted to maintain FVIII trough levels greater than or equal to one percent may be employed.  The serious adverse drug reactions (ADRs) seen with ADVATE are hypersensitivity reactions and the development of high-titer inhibitors necessitating alternative treatments to Factor VIII.  The most common ADRs observed in clinical trials (frequency ≥ 10% of patients) were pyrexia, headache, cough, nasopharyngitis, vomiting, arthralgia, limb injury.

Study Design
The approval is based on the ADVATE Phase IV study comparing two prophylactic regimens to on demand treatment of bleeding episodes in previously treated patients with severe or moderately severe hemophilia A.  The multi-center, open-label, prospective, randomized, controlled clinical trial evaluated the relative efficacy of ADVATE use in two prophylactic regimens (standard prophylaxis [20-40 international units/kg every 48 hours] and pharmacokinetic-driven prophylaxis [20-80 international units/kg every 72 hours, targeted to maintain FVIII trough levels at least 1 percent or higher]) compared to that of on-demand treatment in 53 previously treated patients with severe to moderately severe hemophilia A.  Initial treatment with six months of on-demand therapy was followed by 12 months of either prophylactic regimen.

All patients had a history of at least eight joint hemorrhages per year prior to entering the study.  Additionally, each patient evaluated was adherent to greater than 90 percent of the prescribed number of prophylactic infusions, and no patient in the study surpassed the upper boundary of 110 percent of the prescribed number of prophylactic infusions.

About ADVATE
ADVATE was initially approved by the FDA in July 2003 for control and prevention of bleeding episodes in adults and children with hemophilia A.  ADVATE (derived from the complete FVIII gene) is a recombinant FVIII therapy that is processed without any blood-based additives.  Because no blood-derived components are added at any stage of the manufacturing process, the potential risk of transmitting pathogens that may be carried in blood-based additives is eliminated.  There have been no confirmed reports of transmission of HIV, HBV or HCV with any rFVIII therapies.

ADVATE is approved in 52 countries worldwide including the United States, Canada, 27 countries in the European Union, Argentina, Australia, Brazil, Chile, Colombia, Croatia, Hong Kong, Iceland, Iraq, Japan, Macau, Malaysia, New Zealand, Norway, Puerto Rico, Serbia, Singapore, South Korea, Suriname, Switzerland, Taiwan, Uruguay and Venezuela. Since the initial approval of ADVATE eight years ago, nearly 10 billion international units have been distributed, and ADVATE is the number one chosen rFVIII worldwide.

ADVATE is an Antihemophilic Factor (Recombinant) indicated for:

• Control and prevention of bleeding episodes in adults and children (0-16 years) with Hemophilia A
• Perioperative management in adults and children (0-16 years) with hemophilia A
• Routine prophylaxis to prevent or reduce frequency of  bleeding episodes in adults and children (0-16 years) with hemophilia A
• ADVATE is not indicated for the treatment of von Willebrand disease

Detailed Important Risk Information for ADVATE
ADVATE is contraindicated in patients with known anaphylaxis to mouse or hamster protein or other constituents of the product.

Allergic-type hypersensitivity reactions, including anaphylaxis, are possible and have been reported with ADVATE.  Symptoms have manifested as dizziness, paresthesia, rash, flushing, face swelling, urticaria, dyspnea, and pruritus.  Discontinue use if hypersensitivity symptoms occur and administer appropriate emergency treatment.

Patients treated with AHF products should be monitored for the development of FVIII inhibitors.  Inhibitors have been reported following administration of ADVATE predominantly in previously untreated patients (PUPs) and previously minimally treated patients (MTPs).

If expected plasma FVIII levels are not attained, or if bleeding is not controlled with an expected dose, test for the presence of inhibitors.

The most serious adverse reactions seen with ADVATE are hypersensitivity reactions and the development of high-titer inhibitors necessitating alternative treatments to FVIII.

The most common adverse reactions observed in clinical trials (frequency less than 10% of patients) were pyrexia, headache, cough, nasopharyngitis, vomiting, arthralgia, and limb injury.

Please see full prescribing information at www.baxter.com/downloads/healthcare_professionals/products/ADVATE_PI.pdf.

About Hemophilia
Hemophilia is a rare genetic blood clotting disorder that primarily affects males.¹  People living with hemophilia do not have enough of, or are missing, one of the blood clotting proteins naturally found in blood.¹ Two of the most common forms of hemophilia are A and B.²  In people with hemophilia A, clotting factor VIII is not present in sufficient amounts or is absent.²  Without enough FVIII, people with hemophilia can experience spontaneous, uncontrolled internal bleeding that is painful, debilitating, damaging to joints and potentially fatal.²  People with hemophilia B (also called Christmas disease) do not have sufficient amounts of clotting factor IX.²  In about 30% of cases, there is no family history of hemophilia and the condition is the result of a spontaneous gene mutation.³  According to the World Federation of Hemophilia, more than 400,000 people in the world have hemophilia.¹ All races and economic groups are affected equally.¹

About Baxter International Inc.
Baxter International Inc., through its subsidiaries, develops, manufactures and markets products that save and sustain the lives of people with hemophilia, immune disorders, cancer, infectious diseases, kidney disease, trauma and other chronic and acute medical conditions. As a global, diversified healthcare company, Baxter applies a unique combination of expertise in medical devices, pharmaceuticals and biotechnology to create products that advance patient care worldwide.
1. What is Hemophilia? World Federation of Hemophilia. Accessed on: 29 June 2011. Available at: http://www.wfh.org/2/1/1_1_Hemophilia.htm .
2. Frequently Asked Questions About Hemophilia. World Federation of Hemophilia. Accessed on: 29 June 2011.  Available at: http://www.wfh.org/2/1/1_1_1_FAQ.htm#difference .
3. Hemophilia A. National Hemophilia Foundation. Accessed on: 29 June 2011. Available at: http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=180&contentid=45&rptname=bleeding .

Published: December 10, 2011

Medical researchers in Britain have successfully treated six patients suffering from the blood-clotting disease known as hemophilia B by injecting them with the correct form of a defective gene, a landmark achievement in the troubled field of gene therapy. Hemophilia B, which was carried by Queen Victoria and affected most of the royal houses of Europe, is the first well-known disease to appear treatable by gene therapy, a technique with a 20-year record of almost unbroken failure.

A virus carrying a replacement gene for blood clotting was used by University College London researchers to help six patients.
“I think this is a terrific advance for the field, said Dr. Ronald G. Crystal, a gene therapist at Weill Cornell Medical College. “After all the hype in the early 1990s, I think the field is really coming back now.
Gene therapy has had minor successes in very rare diseases but suffered a major setback in 1999 with the death of a patient in a clinical trial at the University of Pennsylvania. Another gene therapy trial treated an immune deficiency but caused cancer in some patients.
The general concept of gene therapy — replacing the defective gene in any genetic disease with the intact version — has long been alluring. But carrying it out in practice, usually by loading the replacement gene onto a virus that introduces it into human cells, has been a struggle.
The immune system is all too effective at killing the viruses before the genes can take effect.
The success with hemophilia B, reported online Saturday in The New England Journal of Medicine, embodies several minor improvements developed over many years by different groups of researchers.
The delivery virus, carrying a good version of the human gene for the clotting agent known as Factor IX, was prepared by researchers at St. Jude Children’s Research Hospital in Memphis. The patients had been recruited and treated with the virus in England by a team led by Dr. Amit C. Nathwani of University College London; researchers at the Children’s Hospital of Philadelphia monitored their immune reactions.
Hemophilia B is caused by a defect in the gene for Factor IX. Fatal if untreated, the disease occurs almost only in men because the Factor IX gene lies on the X chromosome, of which men have only a single copy.
Women who carry a defective gene on one X chromosome can compensate with the good copy on their other X chromosome, but they bequeath the defective copy to half their children. About one in 30,000 of newborn boys have the disease, with about 3,000 patients in the United States.
Dr. Nathwani and his team reported that they treated the patients by infusing the delivery virus into their veins. The virus homes in on the cells of the liver, and the gene it carries then churns out correct copies of Factor IX. A single injection enabled the patients to produce small amounts of Factor IX, enough that four of the six could stop the usual treatment, injections of Factor IX concentrate prepared from donated blood. The other two patients continued to need concentrate, but less frequently.
Treating a patient with concentrate costs $300,000 a year, with a possible lifetime cost of $20 million, but the single required injection of the new delivery virus costs just $30,000, Dr. Katherine P. Ponder of the Washington University School of Medicine in St. Louis notes in her commentary in The New England Journal of Medicine, calling the trial “a landmark study.
The patients have continued to produce their own Factor IX for up to 22 months, said Dr. Edward G. D. Tuddenham, director of the Hemophilia Center at the Royal Free Hospital in London. One patient, a geologist, had a good response at first, but his level of Factor IX has declined to 1 percent of normal, the level at which the disease kicks in.
“We attribute this to the fact that he had an inflammation, and although we treated it promptly, we should have been quicker off the mark, Dr. Tuddenham said.
The patient cannot be injected again with the same virus because his immune system is now primed to attack it. “He’s very philosophic about it, but he’s a scientist, and his motivation is to help the science, Dr. Tuddenham said.
Twenty more patients will be treated to assess the best dose of the virus, the goal being the highest dose that does not set off an immune system attack, Dr. Tuddenham said. “We are pretty close to the sweet spot, he said. If all goes well, a genetic treatment for hemophilia B “could be available for widespread use in a couple of years.
In a trial in 2006, a patient injected with a corrective gene produced his own Factor IX but only for 10 weeks. The designer of that treatment, Dr. Katherine A. High of Children’s Hospital of Philadelphia, said the new therapy had worked because the delivery virus had been made more efficient and because the research team had treated the patients with steroids to suppress immune system attacks on the virus.
“I think it’s incredibly exciting, and I say that even though these people are my competitors, she said. Dr. High is listed as a co-author of the report because her laboratory helped monitor the patients and provided proof for regulators that the virus would not insert its human gene into the patients’ sperm and make the change hereditary.
A serious problem with other delivery viruses is that they insert themselves randomly into chromosomes, sometimes disrupting a gene. The virus used by Dr. Nathwani’s team, known as adeno-associated virus-8, generally stays outside the chromosomes, so it should not present this problem. Still, patients will need to be monitored for liver cancer, a small possibility that has been observed in mice.
“I don’t think it’s a showstopper, but it’s a critical safety issue that has to be assessed, Dr. High said.
Patients have little or no immunity to the adeno-associated virus, which infects rhesus monkeys. The virus has a propensity for making liver cells its target, which is good for the therapy because these cells are the natural producers of Factor IX. However, liver cells do not live forever and slowly replenish themselves, possibly limiting how long the therapy will last.
About 80 percent of hemophilia cases are of the type known as hemophilia A, which is caused by defects in a different blood-clotting agent, Factor VIII. Researchers have focused on hemophilia B, in part, because the Factor IX gene is much smaller and easier to work with.
A version of this article appeared in print on December 11, 2011, on page A18 of the New York edition with the headline: In a Breakthrough for Gene Therapy, An Injection Works on Hemophilia B.

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To learn more about the Texas State Bleeding Disorders Advisory click here.

Anthony S. Fauci, M.D., Director, National Institute of Allergy and Infectious Diseases
Jack Whitescarver, Ph.D., Director, NIH Office of AIDS Research
Francis S. Collins, M.D., Ph.D., Director, NIH

Direct Link to NIH statement

This year, we commemorate World AIDS Day during the 30th year since the first reported cases of AIDS, a milestone that has led many to reflect on how far we have come since those dark days when HIV infection was almost always fatal. Remarkably, three decades of scientific progress in HIV/AIDS prevention and treatment have brought us to a time when we can begin to imagine an AIDS-free generation.

In those 30 years, the National Institutes of Health (NIH) has established and supports the world’s leading research program on AIDS. Beginning with a drug called zidovudine, or AZT, NIH scientists and grantees, in partnership with pharmaceutical companies, have developed more than 30 antiretroviral drugs for the treatment of HIV/AIDS and determined optimal combinations for their use. In parts of the world where HIV infection can be accurately diagnosed and where individuals have access to and can afford, tolerate and adhere to these medications, life expectancy can be dramatically increased.

Yet we know that to end the HIV/AIDS pandemic, we must not only treat HIV infection but also prevent new infections from occurring. No single HIV prevention modality will suffice. Rather, a combination of scientifically proven HIV prevention tools will be required to end the pandemic. In collaboration with our sister agencies of the Department of Health and Human Services, other governments, nongovernmental organizations and scientists around the world, the National Institutes of Health is leading the effort to develop the scientific basis for an HIV prevention toolkit robust enough to support the goal of realizing a generation without AIDS.

As a result of NIH-sponsored research, we have known for some time that the use of antiretroviral drugs during pregnancy can prevent HIV transmission from mother to child. More recently, medically supervised adult male circumcision was shown to decrease by more than half the risk of female-to-male sexual transmission in communities where men are not circumcised. We also have long known that correct and consistent condom use can prevent sexual transmission of the virus.

But an extraordinary burst of new scientific advances in HIV prevention during the past 18 months is fueling additional prevention research that could accelerate the pace toward our ultimate goal of ending the HIV/AIDS pandemic. For example, several clinical trials have shown that taking an antiretroviral pill as pre-exposure prophylaxis (PrEP) once a day could reduce the risk of HIV infection in certain HIV-negative populations. In addition, scientists are beginning to uncover the mechanisms behind the modest first success of a vaccine regimen against HIV infection.

Moreover, a carefully controlled clinical trial conducted this past summer by the NIH HIV Prevention Trials Network demonstrated that treating an HIV-infected person with antiretroviral drugs can dramatically reduce the likelihood that the individual will transmit HIV to his or her heterosexual partner. This study provided further evidence that HIV treatment is prevention and can be a critical component of the HIV prevention toolkit.

Mathematical models indicate that the use of parallel approaches to HIV prevention in a community could dramatically change the trajectory of the HIV/AIDS pandemic. Such approaches could include scaling up medically supervised adult male circumcision, consistent and proper use of condoms, the prevention of mother-to-child HIV transmission, and substantially increasing the number of HIV-infected individuals who receive antiretroviral therapy.

Despite these advances, critical challenges remain. Recent epidemiologic studies continue to show an increasing incidence of HIV co-infections such as tuberculosis and hepatitis C; HIV co-morbidities; AIDS-defining and non-AIDS defining malignancies; and complications associated with long-term HIV disease and its treatment. NIH will continue to support basic research to better understand the HIV disease process and to hasten the development of new strategies to prevent or control HIV infection and potentially find a cure.

With great anticipation, we plan to gather with our colleagues from around the world next summer at the XIX International AIDS Conference in Washington, D.C., to determine the next steps in research that will help translate the recent advances in HIV prevention into action toward ending the pandemic. NIH will eagerly participate in this international dialogue and will continue to support and promote the discovery, development and scientific validation of HIV treatment and prevention tools until we achieve a world without HIV/AIDS.

NIAID conducts and supports research — at NIH, throughout the United States, and worldwide — to study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses. News releases, fact sheets and other NIAID-related materials are available on the NIAID Web site at http://www.niaid.nih.gov.

The Office of the Director, the central office at NIH, is responsible for setting policy for NIH, which includes 27 Institutes and Centers. This involves planning, managing, and coordinating the programs and activities of all NIH components. The Office of the Director also includes program offices which are responsible for stimulating specific areas of research throughout NIH. Additional information is available at http://www.nih.gov/icd/od/.

The Office of AIDS Research, part of the Office of the Director, plans and coordinates the scientific, budgetary, legislative, and policy elements of the NIH AIDS research program. Additional information, including the trans-NIH strategic plan and budget, is available at http://www.oar.nih.gov/.

Information about federally-approved HIV prevention and treatment guidelines as well as access to HIV clinical trials can be found at http://www.aidsinfo.nih.gov/.

About the National Institutes of Health (NIH): NIH, the nation’s medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov.

NIH…Turning Discovery Into Health

Hemophilia Federation of America (HFA) observes and acknowledges this day in honor of our community members who contracted HIV and Hepatitis C after injecting contaminated anti-hemophilic clotting factor into their bodies prior to 1985. Approximately ten-thousand people with Hemophilia contracted HIV (and Hepatitis C) during this time and no less than eight thousand have died.

In honor of our community members who are deceased and those who still struggle with co-morbidities contracted from contaminated products, we offer our gratitude for your heroic diligence and sacrifice in ensuring a relatively safe blood supply for all.

Our Citizen Petition to the FDA in 2009 (with amendment), asked for recognition of the HCV crisis in our community and it requested the earliest possible access to advanced HCV therapies.  We have made substantial progress in moving regulatory policy toward appropriate urgency.

However, as advocates and patients, we are impatient for therapeutic improvements.

We therefore intend to find industry and clinicians who will support our use of advanced investigational agents in curative therapy protocols.
For those people with bleeding disorders and HCV who are less threatened, we will continue to lobby for early access to improved therapy via the clinical trial pathway.

Why this push?
The crisis is now.  Around 80% of those who received factor through the mid 1980s have chronic HCV.  HCV often advances slowly, but we were infected 30, 40, or 50 years ago. After decades of slow progression, HCV is now the leading cause of death in our community.

Problems with current medical therapies
Current therapies are inadequate.  The only currently approved medicines available for curative therapy of HCV are:
- Ribavirin (RBV) plus pegylated Interferon (p-IFN).  This combination is also known as Standard of Care (SOC) therapy, and,
- SOC plus a first generation protease, either telaprevir or boceprevir.

As to SOC, cure rates for our community are likely lower than average due to known factors including: age, co-infection, advanced disease, period of infection, genotype, etc.  This has been recognized by the NHF in MASAC bulletin 203.  We, People with Bleeding Disorders and HCV, recommend that people considering SOC should consult with their treater about clinic-specific cure rates for people with similar disease and co-infection status.  Further, we challenge the commonly held paradigm that failed therapy is benign.  Instead, we suspect that it frequently is harmful, particularly for those with advanced disease, based on anecdotal evidence and recently published papers generated by the NIH HALT-C program.

As to therapy with the first generation protease inhibitors, these require a concurrent long course of the SOC cocktail, plus they bring their own side effects including a high likelihood of viral resistance.  The NHF in MASAC bulletin 203 cautions that the new protease inhibitors are also anti-hemostatic and concludes that they cannot (yet) be recommended for our community.

Better treatment alternatives on the horizon
The newest investigational therapies are extremely promising.  The recent purchase by Gilead Pharmaceuticals of Pharmasset Pharmaceuticals is an indicator of how close we are to a better cure.  The sale valued Pharmasset at $11 billion based on investor expectations that an advanced investigational drug (Pharmasset’s PSI 7977) will be part of much improved therapy.  Many firms are developing their own agents, leading to a complex scene of optimistic claims and reports for investigational drugs.  We attach a report on the many drugs discussed at the American Association for the Study of Liver Disease conference meeting held in early November in San Francisco.

Possible Options
We think that the medical options for individuals with bleeding disorders and HCV are:
1. Continued hopeful waiting for far better HCV therapy, or
2. Taking a course of SOC or enhanced SOC therapy (not recomended where contraindicated), or
3. Aggressively seeking out access to better therapy (substantiated by fairly early safety and efficacy data).  This is particularly justified for people with bleeding disorders and HCV because we tend to have advanced disease (and thus a substantial potential for adverse medical events) and because we have lower- than-average likelihoods of good outcomes from currently approved therapy.

With whom we will work
- Regulators.  We have received substantial encouragement from FDA for this approach, but open doors don’t remain open forever.  We will continue to seek rapid action.
- Industry.  We have made preliminary contact with several drug companies which seem interested, perhaps because current FDA guidelines encourage them to provide compassionate access.  Certainly, successful therapy for ‘difficult to treat’ cases can only improve chances for early drug approval.
- Clinicians.  We hope some clinicians will be willing to step forward and work for curative protocols.
- Advocates.  We see increased crisis recognition and increased demand for urgency among bleeding disorder advocacy organizations.  We will solicit continuing outspoken support from HFA, NHF and COTT.   We will also continue to interact with other HCV advocacy organizations including the Hepatitis C Community Advisory Board (HCAB), and the Forum for HIV Collaborative Research, HCV Drug Advisory Group (HCV DrAG).  We will also continue to work with the Abigail Alliance for Better Access to Developmental Drugs.

Interested?
Obtaining drug access via participation in clinical trials, or via compassionate access protocols will be not be easy or without risk.  We will share our experience among the bleeding disorders community so that others can consider joining a trial or curative protocol. We are hopeful that our experience will help others determine a course of action best suited for their individual situation.

Mark Antell and Paul Brayshaw for People with Bleeding Disorders and HCV
write us at, accesshcvtherapy@gmail.com