New Treatments

At Patient's Day last week, there was a lot of optimism regarding the significant number of new treatments that were in the clinical trial phase for treating both primary and secondary amyloidosis. A good number of the newer drugs are based on studies that are so new, they haven't been reported in a medical journal.

Most of these treatments aim to do so by reducing the rogue light chain levels in the body back down to normal levels. Some of the treatments are chemotherapy-based, known and used in other diseases such as multiple myeloma; others are working off of new theories, and deal with the protein cells directly.


Here's a slide from the presentation last week showing how scientist believe amyloids (and resulting fibrils) from in the body. You see those small arrows on the diagram? Those show the different processes which scientists are directly attacking to stop the process. As you can see, some are trying to stop amyloid fibrils from forming, while others are working directly with RNA to prevent an inbalance of light chains from ever forming in the first place.

For those who couldn't read the slide, here are some of the notable clinical trials in process:

Phase III

• MRA (Humanized anti-IL-6 receptor MAb)
• Genasense (Bcl-2 antisense)

Phase II

• Actimid (TM) (IMiD)
• CC1I006 (IMiD)
• Aplidin (R) (apoptosis inducer)
• Atlizimab (Humanized anti-IL-6 receptor MAb)
• AP23573 (anti-angiogenic)
• Bevacizumab (Humanized anti-VEGF receptor)
• Deptipeptide (rapamicin an.)
• Mylovenge (Idiotypic vaccine)
• Anakinra (IL-1R antagonist)
• 2-methoxyestradiol (antiangiogenic)
• RAD001 (mTOR inhibitor)
• SU5416 (antiangiogenic)
• Temodar (R) (chemotherapy)
• Arsenic trioxide (multiple act.)
• Vatalanib (antiangiogenic)
• ZD6474 (antiangiogenic)
• Tipifarnib (farnesyl transferase inhibitor)

Phase I/II

• AMG 162 (Ab anti-RANKL)
• Atiprimod (anto-angiogenic)
• Gallium maltolate (chemotherapy)
• GCS-100 (chemotherapy)
• Samarium153 EDTMP (Skeletal-targeting)
• GVAX(R) (myeloma vaccine)
• SU6568 (anti-angiogenic)
• GX 15-070 (apoptosis induc.)
• Alvocidib (chemotherapy)
• CP-571 (MoAb anti-IGF-1)

Oh my hands hurt from typing that in. Anyway, there was a lot of praise being given to bortezomib (Velcade), which is now being used on AL patients without multiple myeloma. It is in clinical trials internationally, and many patients are positively reacting to it. I don't have any more information other than that, but I thought that I should pass it on.