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An Enhanced Factor VIII Protein Accelerates Blood Clotting

OnJuly 20, 2005 researchers at the University of Rochester Medical Center in New York announced a new approach to making factor VIII protein more potent and more efficient at blood clotting. The study’s findings, which may have major implications for the future of hemophilia treatment, was published in the July 26th edition of Biochemistry. Dr. Philip Fay, a professor in the department of biochemistry and biophysics, and Dr. Hironao Wakabayashi a research assistant professor at the university are credited with the innovation, which is now awaiting patent approval.

By redesigning factor VIII, a key blood protein involved in forming clots, researchers were able to find a way to double its potency. Speaking on behalf of the research team, Dr. Fay said, "We set out to improve upon nature by developing gain-of-function factor VIII proteins that are superior to the factor VIII protein found in healthy individuals. These more potent forms are not likely to occur naturally since they would theoretically result in excessive clotting, blocked arteries and heart attacks in otherwise healthy people. In patients with hemophilia, however, enhanced clotting is desirable.”

Hemophilia A is characterized by the deficiency or absence of Factor VIII protein in plasma. While there are different levels of severity, the majority of patients have a severe form of the disorder. If treatments for hemophilia could be made more potent, then patients with severe hemophilia would, in theory, no longer need as many weekly infusions and presumably have fewer bleeds and complications.

As Dr. Fay stated, "a more potent form of factor VIII, one that could treat effectively with a lower dose, would reduce the cost and potentially, avert immune reactions.” He added that if the innovation reaches the market, the treatment could ultimately reduce the cost of hemophilia treatments. "It will be cheaper for pharmaceuticals to make because it's about how much (clotting) activity you can get per unit of protein."

One of the long-standing concerns over manipulation of the factor VIII protein in treatment has been the fear of triggering an immune response or inhibitor in patients. Reportedly, the new factor VIII developed by Fay and Wakabayashi can "get under the immune surveillance system" and still be more efficient at stimulating the process of blood clotting.

For about 20 years now, researchers at the University of Rochester Medical Center have been studying the structure of factor VIII. In earlier research, Fay's team had identified a single amino acid with the potential to alter the performance of entire protein. By swapping out one amino acid with another, they were able to double the ability of factor VIII to bind with factor IX. Results were measured by introducing each form of factor VIII into hemophilic blood plasma and recording the time it took to cause clotting.

Wakabayashi called their discovery “a pleasant surprise”, adding that "this finding was sort of accidental." In the future, the team will target additional binding sites with the goal of making changes that further increase factor VIII potency. If issued, the University of Rochester will own the patent to this procedure. The next step will be to conduct clinical trials to affirm the technology's benefits.

Source: University of Rochester Medical Center press release dated July 20, 2005

Factor VIII is a “co-factor” of Factor IX, i.e. it forms a “complex” with Factor IX thus enabling the clotting process to proceed
i.e. Without provoking formation of inhibitors

New Clinical Trial to Test Tarvacin®

Peregrine Pharmaceuticals, Inc., a biotechnology company based in Tustin, CA, has received U.S. Food and Drug Administration (FDA) approval to begin a clinical trial using its product Tarvacin® to treat patients infected with chronic hepatitis C (HCV). Under an investigational new drug (IND) authorization, the study will assess the effect of a single intravenous infusion of Tarvacin® in as many as 32 HCV patients. The trial subjects will be patients who were either unresponsive to standard pegylated interferon plus ribavirin treatment or who relapsed after being treated with the combination therapy.

Peregrine hopes that Tarvacin®, an antiphospholipid therapy that targets exposed cellular components of cancer and viral cells, will become a treatment alternative for the approximately 50% of HCV patients who do not achieve therapeutic response to pegylated interferon plus ribavirin. “We are excited to begin exploring another clinical indication for Tarvacin®. We anticipate this study will accrue patients rapidly due to the prevalence of chronic hepatitis C infections in the U.S. and the need for new therapies to treat this disease,” said Joseph Shan, senior director of clinical and regulatory affairs for Peregrine.

The World Health Organization estimates that 2.7 million people in the U.S. and 170 million people worldwide are currently living with chronic HCV.

Source: Immunotherapy Weekly, June 29, 2005

USDA Reports First Case of Mad Cow Disease Originating in U.S.

On June 24, 2005 the U.S. Department of Agriculture (USDA) confirmed the second case of mad cow disease (Bovine Spongiform Encephalopathy or BSE) in the United States. The previous case was in December 2003, in a cow imported from Canada. This new occurrence is unprecedented in that it is the first apparent case of BSE in a U.S.-born cow. People who eat BSE-contaminated meat are potentially at risk of developing the human form of the disease known as variant Creutzfeldt-Jakob disease or vCJD. However, since the meat of this cow did not enter the food supply, there was no risk of a person contracting vCJD.

Mad cow in animals and vCJD in humans, are prion-related diseases, which result in very serious neurological symptoms with no available treatment. Theoretically, if a person was to become infected with vCJD there exists the possibility of them donating blood and bringing the disease into the blood supply.

The USDA disclosed more information on the infected animal. It was a 12 year old Brahman cross-breeding cow from a ranch in Southeast Texas . It was initially labeled a “downer” because it could not walk on its own and, per standard practice was deemed unfit for human consumption. Months of delays caused by conflicting test results then ensued. Discrepancies were not reconciled until intervention by the USDA’s Inspector General’s office prompted further action. Testing done by England ’s Weybridge Laboratory finally confirmed the case.

According to the USDA’s Chief Veterinarian John Clifford the strain of the disease in this cow was similar to one found in France. It is possible that this infection originated from BSE-contaminated feed imported from that country. Officials also revealed that in all likelihood, this particular animal, born four years prior to the 1997 ban on cow parts in cattle feed, was infected through contaminated feed.

In response, the USDA has analyzed the chain of events that led to this infection and has taken steps to improve BSE-testing protocol. Officials are also screening the Texas cow’s herd of origin and tracing the feed supply in question.

The National Veterinary Services Laboratory in Ames , IA is conducting rapid-screening tests on the 67 cows that make up the original herd. USDA spokesperson Jim Rogers announced that officials have already tested 29 cows, all of which tested negative for BSE. The remaining 38 cattle are currently being screened for the disease. As per the new protocol, if results from rapid tests are inconclusive, thereby indicating the potential presence of mad cow disease, then the animal in question will be incinerated. Brain samples, taken after the cows are killed for initial rapid-screening, are then tested using the immunohistochemistry (IHC) and Western blot tests. These advanced tests are designed to detect the abnormal prion proteins responsible for mad cow disease in animals. The USDA will apply this standard of screening and follow-up testing in the hopes of preventing BSE transmission to humans.

The National Hemophilia Foundation will continue to monitor this situation and will report any significant developments.

Sources: Associated Press, June 24, 2005, AberdeenAmerican News, June 25, 2005, HoustonChronicle, July 1, 2005, America’s Blood Centers (ABC) Newsletter, July 1, 2005, The HoustonChronicle, July 10, 2005

To read USDA Chief Veterinarian John Clifford’s statement, “Regarding the Epidemiological Investigation into the Recently Confirmed BSE Case,” go to www.aphis.usda.gov/lpa/issues/bse/bse_statement6-29-05.docx

WHO confirms hemophilia treatment products as 'essential medicines'

הועבר באדיבות חיים וייסמן

The WHO Expert Committee on the Selection and Use of Essential Medicines has decided to retain plasma-derived factor VIII and factor IX concentrates on its Model List of Essential Medicines. The move followed a 15-month WFH campaign involving doctors, medical organizations, patient associations, industry representatives, and other WHO departments. "The WHO Expert Committee decision will save lives by continuing to make safe and effective treatment available," says WFH President Mark Skinner. "The Essential Medicines list is important, because governments refer to WHO recommendations when making decisions on health spending." WHO's list is a guideline for governments that want to ensure they have an inventory of medicines needed by every health system. The list had long included hemophilia treatment products, but in 2003, members of the Essential Medicines committee decided to review the public health relevance of factor concentrates. The WFH and its supporters pressed strongly for retention of hemophilia treatment products on the list and sent a total of 19 submissions to the WHO committee. Skinner and WFH executive member, Professor Mammen Chandy were invited to Geneva to present the case for retaining the treatment products. The WFH submission argued that removing the products from the list would violate WHO's founding principle that access to life-saving medicines is a fundamental human right. "The retention of factor concentrates will support the efforts of the WFH and hemophilia organizations to lobby health departments for adequate treatment, " says Skinner. "The concentrates have enjoyed an excellent safety record for over 12 years. They also meet the list's other requirements, particularly in the area of public health relevance. One in 10,000 people is born with hemophilia and the number of people who need factor concentrates increases greatly with the addition of those with von Willebrand disease and other bleeding disorders." The WFH submission was prepared by Chandy, a leading Indian hematologist, who hopes health authorities see the WHO decision as endorsement of the "essential" nature of clotting factor concentrates for the care of people with bleeding disorders. "Unavailability of safe concentrates has resulted in bleeding, HIV/AIDS, and hepatitis C as chief causes of death for many people with hemophilia," he says. "Death and disability rates in developing countries will increase if governments do not make more safe products available or if viral-inactivated concentrates are replaced, for economic reasons, by products that have potential for viral infection. "Assessments of the cost of factor concentrates also need to consider the alternative "cost" of early death, life-long disability, and possible viral infection, as well as the burden on families and society by people who do not receive adequate care. The WFH sees provision of on-demand therapy as a more humane and economic proposition." The WFH wishes to thank all the individuals and organizations that provided submissions and support for this campaign. To view the 14th Model List of Essential Medicines, click http://www.who.int/medicines/organization/par/edl/eml.shtml

Sheba saves life of Palestinian hemophiliac

Judy Siegel-Itzkovich, THE JERUSALEM POST Jul. 20, 2005

A 14-year-old Palestinian hemophiliac, who nearly bled to death after injections and orthopedic surgery at Makassed Hospital in east Jerusalem on Saturday, was transferred to Sheba Hospital at Tel Hashomer, where NIS 120,000 worth of special medications were given that stopped repeated hemorrhages.

Prof. Uri Martinovich, head of the National Hemophilia Center at Sheba, told The Jerusalem Post that Makassed doctors should not have given him the treatments. He said the boy had antibodies to the conventional and less-expensive hemophilia drug Clotting Factor 8, and thus it could not have stopped the bleeding. Makassed had no immediate comment.

The urgent transfer of the boy, Deeb Harfoush of Harbata east of Ramallah, was approved on an emergency basis by Health Ministry deputy director-general Dr. Yitzhak Berlovich, who heads its medical branch.

"We did not discuss payment," he said. "We knew his life was in danger, and we gave the green light to save him. Now that he is stable and the bleeding has stopped, we can discuss payment."

Berlovich added that as Makassed is in east Jerusalem, the ministry supervises its functioning. "First we will ask Makassed for a copy of the boy's medical file and explanations in writing. Then, if necessary, we will consult with an orthopedics expert and then decide if we will send an investigator to look into the case," he said.

Martinovich and the boy's father, unemployed electrician Ayid Harfoush, said that four years ago, the boy underwent circumcision at Ramallah Hospital, after which he began to bleed. When the bleeding did not stop with Factor 8, he needed treatment in Israel with Factor 7, a highly expensive, genetically engineered treatment for the 15 percent of hemophiliacs who develop antibodies to Factor 8.

Novo Nordisk-Israel, which makes Factor 7, donated the units for his treatment.

Deeb was born with a severely deformed leg that is turned at a 90-degree angle from the normal one. Over the weekend, Makassed attempted Ilizarov treatment, in which metal screws are inserted to bones to gradually extend and straighten them, but injections into his healthy leg caused severe bleeding, as did the surgery on his abnormal leg.

The father told the Post that a few months after Deeb was born, they realized he was a hemophiliac. The couple, who are first cousins, have six children, and their youngest – 16 months old – also has hemophilia, an inherited condition.

The father complained that Deeb cried terribly at Makassed when his leg swelled and his bleeding was uncontrollable after treatment there.

Dr. Rostum Namari, head of orthopedics at Makassed, could not be reached for comment. Dr. Hitam Hassan, director-general of the hospital, said he was not familiar with the case and could not comment within a matter of hours.

"We will look at the medical records and see what was done. We will investigate as usual. I do not know who did the operation or treated him, he said.

Speaking excellent Hebrew, the father said he worked for years in Israel as an electrician but since the recent violence has not had a permit to work in Israel. "I do odd jobs in our village and nearby and earn NIS 20 a day."

He blamed Makassed for performing the surgery even though it was dangerous. "I told the doctors he was bleeding, but they said they knew what they were doing," he said.

Dalia Bassa, medical coordinator for the Civil Administration, learned of the case over the weekend and contacted Martinovich and Berlovich, who agreed to the transfer to Sheba.

"I don't know if Makassed doctors knew of Deeb's medical history that meant he could not be treated with Factor 8. They must have received his medical file in advance," she said. "Perhaps they just decided to go ahead with the operation despite knowing about his case."

Bassa added that Makassed should have declined to do the surgery and instead ask for permission to send him to Sheba.

"It may be that the Ilizarov operation is impossible to perform on somebody with the inability to take Factor 8," Bassa said.
Bassa said she received a Palestinian Authority commitment to cover any costs for treatment of the boy. "It is an open commitment, so the PA will have to cover the expenses," she said.

Martinovich said that research he and his colleagues have done made it possible to successful use Factor 7 not only on hemophiliacs, but also on trauma victims or women who bleed severely in childbirth.

Red Cross Tainted HCV/HIV Blood
Canadian Red Cross fined for tainted blood scandal
הועבר באדיבות חיים וייסמן

30 May 2005
Source: Reuters
By Rachelle Younglai

TORONTO, May 30 (Reuters) - The Canadian Red Cross Society was fined C$5,000 ($4,000) on Monday for its part in distributing tainted blood products that infected more than 15,000 Canadians with HIV and hepatitis C in what is dubbed the worst public health tragedy in the country.

The charity will provide C$1.5 million for a scholarship fund for students affected by the tragedy and for a project to improve health care practices.

"Red Cross deeply regrets not developing and adopting measures more quickly to reduce the risk of infection," Canadian Red Cross chief executive, Pierre Duplessis, said in a statement.

"We could have and should have done more and we accept responsibility for that. We are very sorry for the suffering that has caused and apologize to those who were infected and their families."

Around 1,000 Canadians were infected with HIV, the virus that causes AIDS, before the Red Cross started testing blood. Up to 20,000 people are estimated to have been infected with hepatitis C, a debilitating and often deadly liver disease.

The charity, which was stripped of its blood supply operations following the scandal, pleaded guilty in an Ontario superior court to violating the Food and Drugs Act for distributing the contaminated products.

As part of a plea agreement, the authorities withdrew six charges of common nuisance by endangering the public.

The settlement comes about 2 years after the police charged the Red Cross and others for failing to warn the public about the risks associated with unscreened blood, and for failing to test blood donated during the 1980s for hepatitis C or HIV.

"This is a historic day without a doubt. This is the first time that it made clear that at the heart of the tainted blood tragedy people broke the law, and by breaking the law people died," said 37-year old John Plater, a lawyer and member of the Canadian Hemophilia Society.

But Plater, who was infected with HIV and hepatitis C, was angry that there had still been no deal on compensation for the victims of the scandal.

"The most obscene thing in all of this, is here we are 20 years later, the Red Cross has finally been forced to accept their responsibility in this and we still don't have a federal government that is providing compensation to everybody," he said.

"No one is brought back to life and this is not over. It's only been because victims have constantly pressured and pushed and had to drag people involved to the table that we have gotten this far."

The Canadian Red Cross has since transferred its blood operations to a government-funded agency, and officials at the agency when decisions were made to distribute the blood are no longer there.

Former Red Cross Medical Director Roger Perrault has been charged with criminal negligence causing bodily harm and common nuisance by endangering the public.

The charges allege that Perrault and three other doctors knew enough during this period to warn blood recipients of the dangers and screen potential donors to reduce risks to the system. The trial is expected to continue later this year.

The case against Bridgewater, New Jersey-based Armour, which sold a blood clotting drug to the Red Cross, is also in the courts. The firm faces charges of criminal negligence causing bodily harm.

($1= $1.26 Canadian)

Israeli Researchers Study Expanded Use of Factor VII

הועבר באדיבות חיים וייסמן

Dr. Uri Martinowitz of Tel Aviv University and Dr. M. Michaelson of Rambam Medical Center, both in Israel, assembled a task force of medical professionals from several quarters in Israel, to create guidelines for the use of recombinant activated factor VII (rFVIIa) in trauma patients.

The task force, consisting of personnel from National Medical Associations, the Medical Corps of the Army, the Ministry of Health and the Israel National Trauma Advisory Board, formed their recommendations after studying the efficacy of rFVIIa treatment in 36 multi-trauma patients. This mode of treatment stemmed bleeding in 26 of the 36 patients, a 72% success rate.

Martinowitz and Michaelson acknowledged that further trials are necessary to establish official guidelines but also pointed to the study’s utility. “As a result of the lack of controlled trials, our guidelines should be considered as suggestive rather than conclusive. However, they provide a valuable tool for physicians using rFVIIa for the expanding off-label clinical uses.”

The study entitled, “Guidelines for the Use of Recombinant Activated Factor VII (RFVIIa) in Uncontrolled Bleeding: A Report by the Israeli Multidisciplinary rFVIIa Task Force,” was published in the April 2005 issue of the Journal of Thrombosis and Haemostasis.

Coagulation Factor VIIa (Recombinant) is currently approved by the U.S. Food and Drug Administration for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX.

Source: Blood Weekly, June 9, 2005

Bayer to Begin Trials for Enhanced Factor VIII Product

הועבר באדיבות חיים וייסמן

On June 29, 2005 Bayer Biological Products (BP) announced that it has been granted permission by the Food and Drug Administration (FDA) to begin Phase I clinical trials for a new formulation of its recombinant factor VIII product, Kogenate®.

This new formulation uses PEGylated liposome technology patented by the Dutch company Zilip-Pharma, to produce a longer-acting factor product. Prolonging Kogenate’s half-life would result in fewer infusions for hemophilia A patients. Zilip-Pharma has demonstrated success with this application of PEGylated liposomes through clinical trials in other countries.

The investigators for the Phase I trial are Jerry Powell, MD, from the University of California-Davis Medical Center in Sacramento and Diane Nugent, MD, from the Children’s Hospital of Orange County in California.

Dr. Nugent felt this could be an exciting development for the hemophilia community, with potential benefits to patients. “This is a treatment option that would result in less frequent dosing, say, once weekly or less, and represents a major advance in hemophilia care, significantly improving convenience for patients.”

Source: Bayer BP News Release dated June 29, 2005