Lou Gehrig disease:
Amyotrophic lateral sclerosis, or ALS,
is a disease of the nerve cells in the
brain and spinal cord that control
voluntary muscle movement.
The disease attacks the motor
neurons that carry the brain's impulses
to the muscles. While the brain
remains fully functional, the muscles
deteriorate to the point where the body
can no longer move or even breathe
on its own.
ALS is also known as Lou Gehrig's
KNOPP/BIOGEN'S NEW DRUG
SLOWING DOWN LOU GEHRIG
Biogen Idec Inc. agreed to develop
Knopp Neurosciences' experimental
treatment for amyotrophic lateral
sclerosis, also known as Lou Gehrig's
disease. The degenerative disease
attacks nerve cells that control the
body's voluntary muscles.
The drug, called dexpramipexole,
achieved its primary endpoint
evaluating safety and tolerability in a
midstage study conducted by Knopp.
The drug also helped in preserving
motor function and extending survival.
It has received orphan-drug
designation from the FDA and the
European Commission and a fast-
track designation from the FDA.
In the midstage study, 92 participants
received daily 50-milligram or 300-
milligram doses of dexpramipexole.
Results suggested a trend toward
slower disease progression and higher
survival benefit in subjects receiving
the higher dosage.
OLESOXIME DEVELOPED BY
FRENCH CO FOR LOU GEHRIG
Olesoxime is a mitochondrial pore
modulator, which promotes the
functioning and survival of neurons
and other cell types. It is active in
animal models of ALS. The 18-month
Phase III study of the compound
completed recruitment in the first
quarter. It is taking place at 15 centers
in France, Germany, Belgium, Spain
and the UK, and has the backing of a
€6 million European Union grant. The
primary endpoint is overall survival,
with secondary endpoints assessing
If the drug is approved it is expected to
command a high price, given the
serious nature of ALS and the lack of
any other treatment. There are
estimated to be 30,000 U.S. patients
and 45,000 European patients at any
Marseille, France-based Trophos'
512 patient study in the orphan
disease is due to report by the end of
Actelion Pharmaceuticals Ltd.
(Switzerland) is paying €10 million
(US$12.9 million) in cash for the option
to acquire Trophos SA for up to €195
million cash once it gets Phase III data
on olesoxime, a treatment for
amyotrophic lateral sclerosis (ALS), or
Lou Gehrig's disease.
STEM CELLS INJECTED INTO SPINE
OF LOU GEHRIG PATIENTS
Since January, a joint team of
researchers from the University of
Michigan and Emory University in
Atlanta has injected stem cells into the
spines of five patients with ALS. The
initial goal is to see if introducing those
cells will cause the patients any
"So far, there have been no problems
with sensation," said Dr. Eva Feldman,
director of the A. Alfred Taubman
Medical Research Institute on the
University of Michigan campus, where
the project was conceived. "There
have been no untoward side effects."
The first three patients received five
injections into one side of the lumbar
region of the spine, with each dose
carrying 50,000 stem cells. The next
two, as well as a third patient who will
be injected this month, received five
shots on each side of the spine.
TCA INJECTS CONCENTRATED
STEM CELL INTO LOU GEHRING
A small research company is
launching a ground breaking protocol
that the world will be watching. For the
first time ever, patients with Lou
Gehrig's disease could have a glimmer
TCA Cellular Therapy in Covington,
LA, US has the first Food and Drug
Administration approval to begin a trial
using adult stem cells to treat
amyotrophic lateral sclerosis, or ALS.
Cardiologist Dr. Gabriel Lasala is the
medical director of TCA.
ALS, or Lou Gehrig's disease, attacks
the nerve cells in the brain and spinal
cord that control voluntary muscle
movement. It has no cure.
In this trial stem cells are taken from a
patient's bone marrow, multiplied in the
lab for 21 days, and then are later
injected into the same patient's spinal
Lasala says a patient could see the
effects in six to eight weeks.
We should take note that TCA
multiplies (cultures) stem cells before
injecting them back into a patient. This
step is a crucial one. There are many
clinics all over the world that will
withdraw and isolate stem cells. Yet, if
these cells aren’t in a high
concentration, it seems unlikely that
you’ll get an effective dose. TCA
spends 3 weeks or so culturing a
patient’s cells until they have 30-40
million mesenchymal (multipotent)
adult stem cells. This work is
performed at their in-house GMP lab
and stored at Life Source Cryobanks
(which TCA has a controlling interest
in). Again, these are signs that TCA
knows what it’s doing.
Neuralstem, Inc. has started a Phase I
clinical trial, in which Neuralstem's
spinal cord stem cells are injected
directly into the gray matter of the
patient's spinal cord.
Neuralstem is able for the first time, to
produce neural stem cells of the
human brain and spinal cord in
commercial quantities, and the ability
to control the differentiation of these
cells into mature, physiologically
relevant human neurons and glia.