Pat Killingsworth Pat Killingsworth

Great news for our friends with amyloidosis!

Amyloidosis is a close cousin to multiple myeloma.

Often referred to as a disorder or disease, most hematologists consider it to be cancer.

Like multiple myeloma, amyloidosis effects the plasma cells in the bone marrow.  Here is what Mayo Clinic’s website has to say about it:

Amyloidosis us 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 different types of amyloid. Amyloidosis frequently affects the heart, kidneys, liver, spleen, nervous system and gastrointestinal tract.

I know several patients with “amy.”  Several of them also have multiple myeloma, which is not uncommon.

Amy patients tend to have the same type of prognosis as myeloma patients.  A lot seems to depend on how their internal organs are involved.

Like multiple myeloma, stem cell transplants are a standard of care for otherwise healthy amy patients.  Results seem to mirror those of myeloma patients who undergo the same procedure.

However, Revlimid and Velcade are often not as successful in patients with amy as those in multiple myeloma.

So hematologists have been experimenting a lot lately, trying a wide variety of myeloma therapies on amyloidosis patients.  There have been some successes and even more failures.

Following this very brief background, I wanted to share this excellent news from an article in yesterday’s Medscape Today.  Here are some important excerpts from the article:

From Medscape Medical News > Oncology

Best Responses Ever in Amyloidosis With Myeloma Drug Combo

By Zosia Chustecka

February 16, 2012 — A combination of drugs, one of which is the targeted agent bortezomib (Velcade), which is successful in the treatment of multiple myeloma, has shown striking activity in patients with light-chain amyloidosis.

Two unrelated groups of researchers have written brief reports on the use of the combination in amyloidosis; both were published online February 13 in Blood.

Joseph Mikhael, MD, MEd, FRCPC, FACP, from the Mayo Clinic Scottsdale, Arizona, and colleagues report results from a retrospective series of 17 patients with a median follow-up of 21 months. Christopher Venner, MD, from the National Amyloidosis Center at University College London, United Kingdom, and colleagues report results from 43 patients with a median follow-up of 14 months.

Both groups used bortezomib in combination with cyclophosphamide and dexamethasone, and report seeing rapid, deep, and durable responses, better than anything that has been seen previously with any other combination…

Some of the results, such as the 94% response rate and the 71% complete response rate, are unprecedented, Dr. Mikhael said in an interview. “Although I am cognizant that these are preliminary results from small studies, I am excited…. These results will have to be corroborated in larger studies, but this combination holds great promise.”

“In view of the rapidity and the depth of the responses, as well as the tolerability of the regimen, I think this combination is the best choice for patients,” he said…

Rare enthusiasm and strong words from a clinician!  But Dr. Mikhael isn’t alone.

Good Results From the United Kingdom

Similarly good results were reported in the British series of patients. Compared to what we have seen in the past with various treatment regimens, the results with the 3-drug combination of cyclophosphamide, bortezomib, and dexamethasone appear to be better, especially in terms of response rates, Dr. Venner reported.

“The response rates are higher and deeper… [and] there is a higher rate of complete responses,” he said. In the British series of 43 patients, the overall response rate was 81.54%, and 39.50% of those were complete responses. In responding patients, the time to response was 4.1 months…

…This is a “first hint that bortezomib-induced deep hematological responses may overcome prognostic features of stage 3 disease,” Dr. Venner and colleagues write…

Want more proof researchers may be on to something using this combination of Cytoxan, Velcade and dexamethasone?  Medscape Today staff interviewed an amy expert who was not involved with either trial, David Vesole, MD:

“These are very encouraging results in a rare plasma cell dyscrasia…”

“Response rates are almost double those observed with the ‘gold standard’ of melphalan and dexamethasone. These outcomes appear to be superior to doublet regimens of bortezomib and dexamethasone or lenalidomide and dexamethasone. Further, these results are comparable to some of the transplant data,” he said.

Note how something as simple as adding an additional drug, Cytoxan, to a standard mix of Velcade and dex can achieve such promising results.

This is an example of how using existing therapies already being used in a wide variety of cancers can be successful–and costs a lot less to achieve.

The same principle applies to multiple myeloma of course.  After all, cytoxan was originally used in breast cancer, leukemia and ovarian cancer patients–both as a single agent or in combination with other drugs–and now multiple myeloma.

I’m so excited about this!  The feeling I get is having amyloidosis is a lot like living with multiple myeloma, except patients feel even more alone and isolated.  There aren’t a lot of amy support groups out there.  It is even more rare than multiple myeloma.  And it tends to present itself in so many different ways it is hard to get a handle-on.

Multiple myeloma may negatively affect kidney function.  Amyloidosis can affect the heart, liver, kidneys and gastrointestinal tract.  That has got to be scary!

So if you have any friends or neighbors with amyloidosis, let them know about this awesome news.  Get them to ask their physicians to add cytoxan to their therapy mix.  Sounds like it should help a lot.

Want more information?  Here is a link back to the Medscape Today article.

And CLICK HERE to access a the Amyloidosis Foundation.

So great to end on such a positive note!  Feel good and keep smiling!  Pat