Multiple Myeloma:

Multiple myeloma is a cancer of the
white blood cells known as plasma
cells. A type of B cell, plasma cells are
a crucial part of the immune system
responsible for the production of
antibodies in humans and other
vertebrates. They are produced in the
bone marrow and are transported
through the lymphatic system. Due to
the fundamental nature of the system
affected, multiple myeloma manifests
systemic symptoms that make it difficult
to diagnose. Myeloma is generally
thought to be incurable, but remissions
may be induced with steroids,
chemotherapy, thalidomide and stem
cell transplants.



FDA APPROVES VELCADE SHOT
In January 2012 the FDA  approved  a
new formulation of Takeda's multiple
myeloma drug Velcade that doctors
can administer as a shot under the
skin instead of as an intravenous
infusion.

The new subcutaneous version of
Velcade is both easier to administer
and may also reduce nerve pain and
numbness that is the most bothersome
side effect of the multiple myeloma
drug.

Takeda markets Velcade through its
Millennium Pharmaceuticals subsidiary.

The drug competes primarily against
Celgene's Revlimid although newer
multiple myeloma medicines, including
Onyx Pharmaceutical's carfilzomib, are
undergoing regulatory review.
Carfilzomib, in particular, is designed
to cause less nerve pain and
numbness, known as peripheral
neuropathy.

In a phase III study, 6% of multiple
myeloma patients treated with
injectable Velcade experienced severe
peripheral neuropathy compared to
16% of patients treated with the older
IV-infused Velcade.

Tumor response rates between both
forms of Velcade were similar,
although one-year survival rates were
slightly higher for the older version of
the drug compared to the newly
approved injectable.

Velcade was initially approved in 2003.
The drug recorded 2010 U.S. sales of
$593 million.




CARFILZOMIB: A NEW LAST DITCH
TREATMENT FOR MULTIPLE
MYELOMA
In December 2011 the FDA  granted a
standard, 10-month review time to
carfilzomib, raising questions about
whether the "benefit and risk" of the
drug are balanced given that Onyx is
seeking approval based on a single-
arm study.

FDA's approval decision date for
carfilzomib is July 27, 2012, although
the agency's comments suggest Onyx
may need to submit additional clinical
data on carfilzomib to get the drug
approved. Onyx is running a large
phase III study of carfilzomib with data
expected in the first half of 2013.

Carfilzomib is a new last-ditch
treatment for patients with multiple
myeloma, a cancer of the bone marrow.

Of the patients taking carfilzomib, 24%
responded to the drug, and their
response lasted a median of 7.4
months. What's most impressive is that
these are very sick patients, having
relapsed after a median of five prior
lines of therapy, including drugs such
as Johnson & Johnson  and Takeda's
Velcade and Celgene's  Revlimid.

Carfilzomib works by inhibiting the
proteasome, which is the essentially
the garbage-disposal unit of cells,
breaking down proteins that are no
longer needed. Gumming up the
garbage disposal can also hurt normal
cells, but somehow cancer cells cope
less well than healthy cells.

One proteasome inhibitor is already on
the market as a treatment for multiple
myeloma — Velcade, from Millennium
Pharmaceuticals, a division of Japan’s
Takeda Pharmaceutical.

Despite recent advances in treating
multiple myeloma, all patients
eventually relapse. The unmet medical
need remains great, as the outlook for
patients with relapsed and refractory
disease is grim. The single-agent
activity with durable disease control
and favorable tolerability observed in
this study indicate that carfilzomib has
the potential to alter the natural course
of this deadly disease.




REVLIMID TESTED FOR MULTIPLE
MYELOMA
In December 2011 Onyx's setback with
the FDA opens the door for Celgene,
which said it intends to seek U.S. and
European approval for its multiple
myeloma drug pomalidomide based on
data from a phase II study data.

Earlier Celgene  announced that data
from  a phase III, randomized, double-
blind study of continuous REVLIMID
(lenalidomide) for the treatment of
elderly patients with newly diagnosed
multiple myeloma show improvement in
progression-free survival (PFS), the
primary endpoint of the study. The
data were presented during the
European Haematology Association's
annual congress in Barcelona, Spain.

The study of 459 patients 65 years or
older evaluated patients receiving
lenalidomide in combination with
melphalan and prednisone, followed by
lenalidomide alone  

At the time of the second analysis, it
was estimated that 55% of all patients
receiving the medicine would remain
progression free after two years
compared to only 16% of patients not
receiving it.

REVLIMID is not approved as an initial
treatment for patients with multiple
myeloma.





APLIDIN TESTED FOR MULTIPLE
MYELOMA
PharmaMar SA  started a trial in
relapsed/refractory multiple myeloma
patients  with Aplidin (plitidepsin) in
combination with dexamethasone
versus dexamethasone alone.

This pivotal, international, multicenter
Phase III clinical trial, called ADMYRE,
will enroll 300 patients in 60 medical
centers across 20 countries (including
the U.S., Europe, Asia and South
America), with an estimated
recruitment period of 24 months. The
primary objective of ADMYRE is
Progression Free Survival (PFS).

Aplidin is an anti-tumor agent originally
isolated from the Mediterranean
tunicate Aplidium albicans and
currently obtained by chemical
synthesis.

OTHER DEVELOPMENTS
STEM CELL TREATMENT
Lisa Ray, Canadian-Indian actress was
cured of multiple myeloma with stem
cell treatment at Toronto's Princess
Margaret Hospital.