Spring 1999 - Factor Nine News

From The Coalition for Hemophilia B

Factor Nine Products

The other important aspect of these products is viral safety. Unless they are treated to inactivate or remove viruses, all products purified from plasma will transmit viral infections. All blood and plasma donations are tested for viruses, but because no test is 100% accurate there are almost always viruses in the plasma used for purification of therapeutic products. All plasma products currently licensed in the U.S. have at least one viral inactivation or removal method in their production process. Many use more than one method either to eliminate viruses that are not affected by the first method, or as a back-up to ensure a higher degree of safety. Several manufacturers have also been able to prove that their purification process removes viruses from the product. The result is that today’s plasma-derived products have an excellent safety record.

The newest product is Coagulation Factor IX (recombinant) which was licensed in 1997. One of its major features is that it is produced in a completely plasma-free system and thus should be totally free of human viruses. In addition, the production process still contains steps to remove any viruses that might accidentally contaminate the product. Recombinant factor IX is produced in hamster cells that are grown in large 660 gallon fermentation vessels. The hamster cells have been modified by genetic engineering to contain the gene for human factor IX. Under the right conditions the cells produce human factor IX and secrete it into the broth in which the cells are being grown. This is like the fermentation process for making beer except that instead of yeast cells producing alcohol it employs hamster cells producing factor IX. The production process then purifies the factor IX from the fermentation broth. The final product is essentially 100% pure factor IX.

Coagulation Factor IX (recombinant) has been shown to be safe and effective in clinical studies and in two years of use in patients. One difference between recombinant and plasma-derived factor IX is that the recombinant product has a lower recovery. The recovery of a product is the ratio of the amount of product that shows up in the bloodstream compared to the amount injected. The reason for the difference is currently being investigated, but the practical outcome is that about 20% more recombinant factor IX must be given to a patient to achieve the same clinical benefit.

Significant improvements in safety and purity of factor IX concentrates have occurred. However, these have not been without a price. The cost to the patient for the more highly purified products has increased to a point where many hemophiliacs are struggling to afford the newer products. Although many physicians recommend recombinant products because of their theoretical freedom from viral infectivity, the less-expensive plasma-derived Coagulation Factor IX products have an excellent safety record and may be a viable alternative for many patients. Because of the problem of potentially-fatal thrombosis, the even less-expensive Factor IX Complex should probably be considered a product of last resort, although large numbers of patients were treated with it for years without incident. The final solution to the cost problem may lie with the second generation recombinant products currently being developed that would produce proteins such as factor IX in huge quantities at low cost in the milk of transgenic animals.

This has been a brief overview of the products available in the U.S. today. Because of the varied characteristics of both products and patients, the patient should work closely with his physician to select the best product for his needs and circumstances.

Novo 7 Approved by FDA

On March 25, 1999 the FDA approved Novo 7 manufactured by Novo Nordisk.

Good News Indeed!!

Hepatitis C

Hepatitis is an ever growing threat to the health of our society, though it is only beginning to get the attention it deserves. Compared to HIV which manifests itself as full blown AIDS in a relatively short period of time, hepatitis can take up to 20 or 30 years for it to show its full effects.

The U.S. News&World Report (6/22/98) said, and the American Liver Foundation concurs, that between 4-5 million Americans are currently diagnosed with hepatitis. New hepatitis C infections/year are between 28,000 to 180,000. The current mortality rate is 10,000/year and is projected to increase to 30,000/year over the next ten years. Hepatitis C health costs nationally are now $600 million/year and growing. 60% of those now infected are in their 30’s and 40’s, and some experts think as many as 60% of those now infected will experience liver failure. Hepatitis C has experienced a lack of research funding devoted to finding a cure for it, much as HIV was in the early ’80’s. It was only after a “critical mass” of people died, including a number of celebrities, capturing the public’s attention, that HIV received the research funding it deserved. Hopefully,this won’t have to be repeated with hepatitis in order for hepatitis to get the research funding it deserves.

Fortunately, there are some resources available for those infected with infected with hepatitis, such as a vaccine for hepatitis B, and interferon, and the combination of interferon with ribavirin,though at best the viral clearance rate for is interferon alone is 20% and with the combination is 40%. Schering is the main source worldwide for interferon-a and the combination therapy of interferon and ribavirin. Other pharmaceuticals, such as Amgen and Roche Laboratories make their own versions of interferon.

There are a number of national organizations that provide educational and research information, such as the Hepatitis Foundation International(HFI) 800 891-0707 and the American Liver Foundation(ALF) 800 465-4837. Both of these organizations have websites, e-mail addresses, newsletters (both on-line and off-line), and support groups located around the country. There are also on-line support groups such as thriveonline@aol.com and St. Johns University owner-hepv-L@maelstrom.stjohns.edu. A source for alterernative therapies, or complimentary meds is the Herb Research Foundation 303 449-2265. In Seattle Dr. Brad Lictenstein specilized in alternative medicine 206 285-4625.

In New York City, LOLA, or Latino Organization for Liver Awareness, 888 367-5652 provides support groups and doctor referrals, and is in touch with other liver support groups nationally.

The University of Pittsburgh Medical Center(UPMC) transplant clinic 412 648-3200 is one of less than a handful of transplant centers in the country willing to transplant those people coinfected with HIV/HCV(due to the success of the protease inhibitors to control HIV) and/or hepatitis C with auto-immune deficiency(AID) and has the staff with the expertise to handle these problems. Practically every transplant surgeon in the U.S. was either trained at UPMC or trained by someone who was trained at UPMC-this is where successful liver transplantation began. Despite what most transplant centers will tell you, coinfected patients can be and have been transplanted successfully.

If you don’t know if you have hepatitis, you owe it to yourself to find out! People should sign up for at least a couple of transplant center transplant candidate lists so they are not penalized by where they live. When considering a transplant center, questions that should be asked are:Where were the operating surgeons trained? How long has it been since they finished their fellowships during which they were trained? What is the transplant center’s mortality or outcomes rate? How many surgeons are on their staff? How many transplants does the center do per year? (If it is 10-12 or less, it’s a hobby, and the center’s expertise may be questionable.) What is the average waiting time to be transplanted?

At this point, interferon and ribavirin therapy and transplantion are the closest things there are to a cure for hepatitis C. Hopefully, as some hepatologists and immunlogists think, what has been learned about HIV therapy can be applied to finding a cure for hepatitis and within the next three to five years there will be drug therapies that will do for hepatitis what protease inhibitors have done for HIV.

Health Quest Travel - 724 935-3633 - This company has a computer listing of nationwide doctors who are specialists. If you are travelling and need a specialists call them and for a fee they will inform you of a specilist in your travel area and inform the doctor that you may need to seek their services.