These have not been approved by the FDA
however they are in the experimental
stages for the Treatment of MS.
Cladribine: This drug is given orally for
4 - 5 consecutive days, ranging from once
every 28 days to twice yearly, depending
on the study regimen. This drug
interferes with the proliferation of a
specific class of T cells in the immune
system. The ongoing 2 year, Phase III
CLARITY extension study will assess the
safety and efficacy of oral cladribine in
RRMS as well as its effect on progession
of disability.
Fingolimod (FTY720): Oral medication
taken daily. Blocks T cells from leavng
lymph nodes, lowering their number in the
blood and tissues, may reduce damage to
nerves and enhance nerve repair. Adverse
events may include slowed heart rate,
increased blood pressure, airway
obstructions and infection.
BG00012 (BG 12; fumarate; fumaric acid
esther): Oral medication taken daily.
This drug is an immunomodulator with
anti-inflammatory properties; may
potentially have neuroprotective effects.
This drug is being studies in RRMS. This
drug was reported to be safe and
tolerable.
Laquinimod: Oral medication taken daily.
Laquinimod is being studied in RRMS and is
an immunomodulator. The drug was well
tolerated.
Teriflunomide: Oral medication taken
daily. This drug is an immunomodulator,
affecting the division of T cells.
Treatment was well tolerated.
Statins: Statins are oral medications
most commonly prescribed to lower
cholesterol. Their anti-inflammatory
properties make them of interest for
possible use in MS. Atorvastatin
(Lipitor) and Simvastatin (Vytorin,
Zorcor) are among the statins presently
being studies as potential treatments for
RRMS. An initial pilot trial of
Simvastatin in an uncontrolled study
showed it was safe and reduced MRI
activity at six months. The effects of
statins combined with interferons are
controversial. In one study, combination
of Rebif with Lipitor increased MRI and
clinical disease activity, suggesting that
statins may block the therapeutic effects
of interferons. However, two other
studies did not support this, and more
data are needed.
The following 3 drugs are "Experimental
Monoclonal Antibody Medications"
Campath (alemtuzumab): Administered once
yearly by intravenous infusion over 3 - 5
consecutive days. The drug is approved
for the treatment of B-cell leukemia and
targets T cells, B cells and macrophages.
Side efforts include reduction in blood
clotting, thyroid disorders, infusion
reactions and infections. Patients need
to be monitored closely due to risk of
significant toxicities.
Rituxan (rituximab): Administered via
intravenous infusion. Binds to molecule
(CD20) on the surface of B cells and
deplets them from the circulation for an
average of nine months. Used in lymphoma,
rheumatoid arthritis and lupus. Serious
adverse events have been reports in
Rituxan treated patients and must be
closely monitored.
Zenapax (daclizumab): Administered via
intravenous infusion every 4 weeks; also
studied in subcutaneous injections. This
drug is used to prevent renal (kidney)
transplant rejection. It is a genetically
engineered antibody against interlukin 2,
a substance necessary for the growth of T
cells, limiting their growth and reducing
inflammation. Drug is wel tolerated, has
been reported to improve or stablize 60%
of patients; and reduce the number of
active lesions in both RRMS and SPMS
patients.
Other therapies being studied for MS:
MBP8298: Administered intravenously every
6 months. This peptide is a synthetic
fragment of myelin basic protein (MBP).
It replicates the site on the MBP molecule
that is believed to be a target of attack
by cells of the immune system, in 65 to
75% of all people with MS. It is believed
to induce or restore immunologic tolerance
to attack. Safe and tolerable.
Tovaxin: T cell vaccine given via
subcutaneous injection every 4 weeks. T
cells are removed frm a small amount of
the patients blood, inactivated, then
injected back into the patient; the immune
system is stimulated to recognize and
elminate the inactivated cells as well as
active cells. Tolerable and safe.
Vitamin D3: There appears to be an
inverse relationship between vitamin D3
status and the probability of developing
MS, most likely due to its
immunoregulatory effects. Vitamin D3
supplementation therefore appears to be a
possible therapy in MS. Safe and
tolerable.
Tetracycline Antibiotics: Have been shown
to have immunomodulatory and
neuroprotective activities. They appear
to decrease the passage of leukocytes
across the blood brain barrier.
BHT-3009: This is a DNA facdcine to
myelin basic protein (MBP); it contains
the gene for MBP and is administered by
intramuscular injection. This therapy is
designed to cause immune tolerance, by
reprogramming the immune system to
modulate the response of the antigen
specific immune cells involved with MS and
reducing the attack against MBP in the
myelin sheath. Safe and well tolerated.
Approved Drugs for MS:
Avonex (interferon beta-1a): Taken via
weekly intramuscular injections; dosage is
30 mcg. RRMS and individuals with
clinically isolated syndrome (CIS), a
single attack not yet dx'd as MS.
Betaseron (interferon beta - 1b):
Administered by subcutaneous injectin
every other day, dose is 250 mcg. Aproved
for RRMS and individuals with CIS.
Rebif (interferon beta - 1a):
Administered by subcutaneous injection 3
times weekly; dosage is 22 or 44 mcg.
Approved for RRMS
Copaxone (glatiramer acetate - GA): Given
through daily subcutaneous injections;
dosage is 20 mg. Approved for RRMS.
Novantrone (mitoxantrone): Given via
intravenous infusion once every 3 months
for a maximum of 2 - 3 years. The total
does that can be taken is limited to avoid
the risk of damage to the heart. Careful
monitoring both during and after treatment
is necessary. Dose varies according to an
individuals weight. It is approved for
SPMS, PRMS or worsening RRMS and for
people who are not responding favorably to
standard therapies.
Tysabri (natalizumab): Administered via
intravenous infusion every 4 weeks. Dose
is 300 mg. It was approved for RRMS.
Following a suspension of the drug after 2
patients developed Progressive Multifocal
Leukoencephalopathy (PML), an often fatal
viral infection of the brain, Tysabri was
re-released in 2006. All patients now
receive the drug through safety monitoring
programs such as the TOUCH Prescribing
Program and registered infusion centers
and pharmacies...2 new cases of PML were
recently reported. Nearly 31,800 patients
are on the drug worldwide. In early 2008
the FDA mandated a label change to include
the possibility of serious liver injury
associated wth the drugs use.
