I spoke with Tierney Saccavino at Acorda Therapeutics this morning. She told
me that she probably won't have any information on Phase III trials until late
fall. I expressed disappointment in the delay. She countered that it is
because they are so convinced that 4-AP will help so many people that it is
important that Phase III not be rushed into. It's purpose is to get FDA
approval, so everything must be in place. Phase II has been evaluated.
Apparently there is a very small, local Phase IIB going on to further define
dosage guidelines. When those evaluations are completed, then work on
defining Phase III criteria, guidelines, etc. will be started, which she felt
would be sometime in the late fall. Tierney also said that Acorda's webpage
has been under construction and that she hoped that they might have it up and
running with current information on ongoing projects as soon as Tuesday.
www.acorda.com. That will be nice!
Below is the reposting of the letter. Call for your information package if
you are interested. : ] I did!
Subj: 4apDate: 12/28/98 12:10:53 PM Eastern Standard Time
From: rcohen(AT)acorda.com (Ron Cohen, M.D.)Reply-to: rcohen(AT)acorda.com
To: Magtastic(AT)aol.comDear Dennis:
4-AP is a "potassium channel blocker" whose effect is to increase the
ability of damaged axons to conduct impulses. In people with nervous
system injury, such as SCI, the surviving axons frequently lose part of
their myelin sheath--the insulation around the axon that permits it to
conduct impulses without "short circuiting." When the myelin sheath is
damaged, the axon can no longer conduct past the site of the injury and
the nerve does not function. 4-AP appears to restore the ability of
such nerves to conduct.
In previous human studies totaling about 200 patients with chronic
spinal cord injury (SCI),
about a third have shown some clinical benefit. Depending on the
individual, these benefits appear to include reduction of central pain
and/or spasticity, restoration of sensory function, increase in stamina
and possibly some increase in strength, and in some cases, improvements
in bowel, bladder and sexual function. Most of those benefiting had
incomplete (versus complete) lesions. Several hundred patients with
multiple sclerosis have also been studied in clinical trials over the
past several years.These
trials indicate that 4-AP may also restore some function in MS,
particularly as regards increased stamina and reduced fatigue.
Acorda recently completed a clinical trial of fampridine (the generic
name of 4-AP) in 60 subjects with incomplete chronic SCI. The results
were consistent with those mentioned above. We used an oral tablet
developed by Elan Corporation, Acorda's corporate partner; this tablet
is a sophisticated, sustained release formulation which has been
thoroughly tested and quality-controlled. Our goal is to obtain FDA
approval for fampridine so that we can make it available to everyone who
may benefit. To do this, we must conclusively demonstrate that the drug
is safe and effective, which we hope to do through a series of clinicaltrials.
We expect to begin additional trials using the Elan formulation for both
the SCI and MS indications, in the first half of 1999.
Volunteer subjects for these trials will be selected
by each center according to the inclusion criteria specified in thetrial
design. The trial design will be determined over the next couple ofmonths.
With regard to obtaining 4-AP now, to our knowledge there is no
FDA-approved source of medicinal 4-AP. Some people have obtained 4-AP
from certain pharmacies around the country with a doctor's
prescription. These pharmacies compound the drug into capsules
in-house. We have no information about the nature of such formulations
or their quality control. This may be an important consideration, since
4-AP has potential side effects, including epileptic-type seizures, and
must be administered by experts and with caution. You might want to
check various 4-AP chat groups on the internet or America Online to
obtain other information. You also may want to join a free e-mail list
server for SCI by writing to: <scipin-l(AT)health.state.ny.us>
Meanwhile, if you would like more information about Acorda and its
therapy development programs for SCI, please contact Tierney Saccavino
at tsaccavino(AT)acorda.com and she will be glad to send you aninformationkit.
Sincerely,Rn Cohen, M.D.President and CEOAcorda Therapeutics, Inc.
15 Skyline DriveHawthorne, NY 10532Tel: (914) 347-4300Fax: (914) 347-4560
E-mail: rcohen(AT)acorda.com