I spent a lot of time reviewing newly released research studies on amyloidosis (amy) at the American Society of Clinical Oncology (ASCO) meetings in Chicago last week.

I am certainly not an expert on amy. I do know a number of fellow multiple myeloma patients who have been diagnosed with this disease/disorder.

Is amy cancer? Some say yes—some no. Let’s review the facts together. According to Mayo Clinic’s Website:

Amyloidosis is a disease that occurs when substances called amyloid proteins build up in your organs. Amyloid is an abnormal protein usually produced by cells in your bone marrow that can be deposited in any tissue or organ. Amyloidosis can affect different organs in different people, and there are many types of amyloid. Amyloidosis frequently affects the heart, kidneys, liver, spleen, nervous system and gastrointestinal tract…

Therapies include melphalan (Alkeran), a chemotherapy agent also used to treat certain types of cancer, and dexamethasone, a corticosteroid used for its anti-inflammatory effects. Researchers are investigating other types of chemotherapy regimens for amyloidosis. Several medications that are used in the treatment of multiple myeloma are being tested to see if they’re effective in treating amyloidosis. These include bortezomib (Velcade), thalidomide (Thalomid), and a thalidomide derivative called lenalidomide (Revlimid). These drugs may be used alone or in combination with dexamethasone.

These are all drugs used to treat multiple myeloma. 15% of multiple myeloma patients will also be diagnosed with amy.

Like multiple myeloma, there is currently no cure for amy. And like myeloma, once the disease progresses, a stem cell transplant is part of the standard of care. The 4 year survival rate for patients with primary systemic amyloidosis who undergo peripheral blood stem cell transplantation is 81% ((Blood. 2004;103:3960-3963, The American Society of Hematology) That is similar to the survival rate for multiple myeloma patients.

However, treating amy as agressively as multiple myeloma with different chemotherapy combinations is just starting to become common. This is a very serious disease—but researchers are making progress. I could tangibly see this at ASCO.

One study in particular caught my attention. Like multiple myeloma, most of the ongoing studies deal with patients who have relapsed. This particular international study, Weekly and twice-weekly bortezomib in AL amyloidosis: Results of a Phase 2 study, is no exception. But the results were exceptional.

88% of patients responded to bortezimib (Velcade). Preliminary results of another ongoing study “is also showing similar effectiveness early-on for newly diagnosed patients,” according to one of the researchers. Early results from another ongoing study where a combination of melphalan, dexamethasone and bortesomib are used is even more promising—94% of participating patients are responding so far, with a 63% complete response rate—even in patients where organs are involved.
A new drug, panobinostat, is also shows some early promise—both against amy and multiple myeloma.

I can only imagine what it would be like to have not only one fatal disease but two, both with similar life expectancies of under ten years. Specialists treating friends I know with both disorders have been concentrating on the multiple myeloma first. Apparently the “life expectancy clock” for people with amy doesn’t kick-in until a person’s organs are involved and amy progresses.

I would like to take this opportunity to share my positive message of hope for amyloidosis patients everywhere. Sounds like there are going to be more effective options available for you soon!

Feel good and keep smiling! Pat

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