Why is Amorfix developing a blood test for prions?

There is no ante-mortem (blood) test for prion disease such as mad cow disease (BSE) or its human equivalent vCJD.  The only way to diagnose these diseases is by an after-death examination of the brain.  Since the diseases are infectious, a diagnostic test that determines if an animal or a person has the disease is needed.

What is a Prion (pronounced, Pree-on)?

The nature of the infectious agent that causes BSE and vCJD and other diseases in other animals is a modified form of a normal cell protein known as a prion.  A prion is not a bacterium, parasite, or virus, and thus treatments usually used for treating or preventing bacterial infections (e.g. antibiotics) or viral infections are not effective against prions.   A prion is an aggregate (polymer) of a specific misfolded protein which is normally present in the body.  Nobody knows what causes the normal protein to misfold.  There are no cures or preventions for prion diseases.

What is the connection between BSE and the human form vCJD?

There is strong epidemiologic and laboratory evidence suggesting that new variant CJD (vCJD) and BSE are caused by the same infectious agent. For instance, all cases of confirmed vCJD have occurred in people who have lived in geographic areas which have had BSE cases. The majority of cases of vCJD have occurred in the United Kingdom, which has had the largest number of cases of BSE in cattle. A study published in 1996 showed that prion proteins obtained from 10 vCJD patients and BSE-infected animals had molecular characteristics that were similar to each other but distinct from prion proteins obtained from patients with classical CJD (Nature 1996; 383:685-90).

In contrast to the classic form of CJD, the new variant or variant form (vCJD) in the United Kingdom and France affects younger persons (average age at onset: 26 years), and has different clinical features from CJD. People with vCJD begin with serious psychiatric problems or problems with their senses (ears, eyes or smell). This first set of symptoms is followed weeks or months later by poor muscle coordination, muscle spasms, and mental confusion. On average people with vCJD die approximately 13 months after their symptoms begin. When patients' brains are examined by autopsy, there are clear changes in brain tissue structure, including many "spongiform," or open spongy-looking areas, abnormal clumps of prion protein called plaques, and other areas with less prominent accumulations of abnormal prion protein.  It is thought that the consumption of BSE infected beef is the source of this new disease.

Why do we need a test for prion diseases?

A test is needed now to eliminate BSE infected cows from all herds to prevent the spread of the disease and to make the food supply safe.  It is also needed to screen blood for transfusion so the disease is not passed to others and cause an epidemic.

How will Amorfix develop a test for prions?

Amorfix was founded on the breakthrough knowledge discovered by Drs. Neil Cashman and Marty Lehto that the prions can be detected in blood and other tissues provided the normal protein can be masked.  We call this epitope protection.  The problem with detecting prions using standard immuno-techniques used to other infectious agents is the presence of large amounts of normal protein.  Thus, it is a needle (prions) in the haystack (normal prion protein) problem.  Essentially, Amorfix has found a way to burn down the haystack and find the needle.  We must optimize the test to be as sensitive as possible so even small amounts of prions in blood will be detected.

Who will use the test?

The test will be used by blood transfusion services just like they use tests for HIV-AIDS, hepatitis and other diseases that can be spread by blood.  It will be used by cattle ranchers to screen their herds and be sure any new cattle obtained are BSE free.  It will be used by many companies who use bovine source materials to manufacture products such as cosmetics, pharmaceuticals and medical devices.

What is the commercial potential for Amorfix products?

There are over 100 million blood donations made each year worldwide.  Each donation is tested for a dozen properties including blood type and infectious agents.  This testing currently costs $50 per unit of blood.  The addition of a test for prions will increase the cost of testing a unit of blood by a small amount and represents a potential market of $500 million to $1 billion in revenue.

The current rapid test for BSE post-mortem costs an average of $75 for each animal tested.  Japan tests all its cows for BSE while most other countries test only a small fraction of animals; those with symptoms of neurological problems.  If a simple blood test were developed, all animals could be tested and the disease eliminated in a few years.   Amorfix’s pricing strategy would be designed to encourage ante-mortem testing of all animals.  There are estimated 1.5 billion cows in the world.