>TIME reports on the latest treatment developments for AIDS, cancer,
>mental illness, autoimmune disorders, obesity, Alzheimer's, heart
>disease and Parkinson's

http://www.time.com/time/health/printout/0,8816,93959,00.html

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This is all very promising, but notice this "cure" is 5 years away from being
on the market.  This fact has prompted me to investigate the process of drug
evaluation and approval at the FDA.  I was patient representative on an FDA
advisory committee that review DBS last spring.  I have used this entry to begin
discussions with FDA officials on ways that patient groups can facilitate the
evaluation and testing that must be done to prove a medication is safe and
effective (i.e. worth the inevitable risks).  I have written executives at Amgen
(with no reply yet) and this week Rusty Glazer and I met with the Director of
the Neuropharmacological Products Division at FDA to explore ways to facilitate
the process.  The FDA staff were very open and positive about the desirability
of including patient input to the drug approval process.  Just what form that
input will take is not worked out yet, but we will be developing proposals in
the next few weeks.  I will keep the community informed about progress and once
the ideas are crystallized I would like to call on the community to support the
effort with petitions or other appropriate actions.

At this point I have two requests.  First, I would like to be in contact with
anyone who is a participant in the Amgen/Guildford Trial to be able to get a
reading on the the experience of patients with this new treatment and track the
actual potential of the medication.  If it lives up to our highest expectations,
there are mechanisms available to allow the drug to be available to patients in
great need while testing is still going on.  Please email me and ask people you
know in the trial to contact me.

Second, I would like to form an advisory committee to obtain reactions to more
detailed proposals as they are available.  Please contact me if you would like
to be considered for such a group.

Perry D Cohen
Washington DC

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Parkinson's Disease
Lubricating Gummed-Up Brains

 By Jeffrey Kluger

 It's hard to imagine asking a doctor to destroy a part of your brain.
 For the more than 1 million Americans suffering from Parkinson's
 disease, however, such a drastic measure — in which an electrode is
 used to kill tremor-causing neurons — may become necessary.

 The irreversible procedure is just one of many therapies, both surgical
 and pharmacological, that Parkinson's patients have tried over the
 years to control the tremors, rigidity and other symptoms that
 characterize the disease. All the treatments offer some relief, but
 none can remotely be called a cure. Now that may be changing. The
 further scientists peer into the human genome, the more they are
 uncovering the secrets of Parkinson's — and the more they are
 becoming convinced that next-generation drugs may at last be able to
 beat back the disease. What's more, the benefits may not be limited
 to Parkinson's but may also be used to treat Huntington's, Lou Gehrig's
 and even Alzheimer's disease — all of which have similar
 neurodegenerative roots and may respond to similar drugs. Says
 neurobiologist Michael Zigmond of the University of Pittsburgh: "A
 breakthrough in any of these diseases could have an impact on the
 others."

 The first place to look for causes of and treatments for Parkinson's
 disease is in the brains of patients. As the illness worsens, brain tissue
 becomes clogged with a protein muck that includes a substance called
 alpha-synuclein. No one knows exactly what alpha-synuclein does, but
 it's believed to play a role in the smooth transmission of nerve signals.
 When the substance clumps, it can't do the work it was designed to
 do, leading to neuron damage, loss of the neurotransmitter dopamine
 and eventually the familiar shakiness of such well-known Parkinson's
 sufferers as Janet Reno and Muhammad Ali.

 The hunt for a Parkinson's cure got a boost in 1997 when researchers
 discovered a handful of patients whose alpha-synuclein genes had
 mutated. This might seem like open-and-shut evidence that the cause
 of the illness had been found, except that the vast majority of
 Parkinson's patients, whose brains also grow gummed up, do not carry
 the mutation. Still, scientists believe that the bad gene is a powerful
 clue. "There appear to be more clumps in the brains of people with the
 mutant gene," says Zigmond. "Learning how the protein functions may
 help us develop drugs that target it."

 Another gene, newly dubbed the parkin gene, has also been getting a
 lot of attention. In 1998 Japanese researchers reported on studies
 conducted on five young patients with early-onset Parkinson's, all of
 whom carried a recessive gene that appeared to be associated with
 the disease. Though relatively rare, the parkin gene is much more
 common than the defective alpha-synuclein gene, and in places like
 China, with high rates of early-onset Parkinson's, it may play a role in
 a significant number of cases. Just last October, researchers at Duke
 University Medical Center went a step further, reporting their
 discovery of a previously unseen mutation on the parkin gene that
 appears to link it to late-onset forms of the disease as well. "Once we
 figure out how that gene functions," says neurology professor Ira
 Shoulson of the University of Rochester Medical Center, "we could
 perhaps disrupt it pharmacologically."

 Just as important as protecting healthy neurons is repairing or
 replacing nerve cells that have been damaged. The body produces a
 whole bath of trophic — or growth — factors that help cells develop.
 If the brains of Parkinson's patients could be fortified with additional
 trophic doses, many scientists believe, damaged neurons might be
 reawakened or repaired. While there is some thought in the medical
 community about engineering genes to churn out the substances, the
 pharmaceutical industry is taking a more direct approach.

 Currently, Guilford Pharmaceuticals in Baltimore, Md., and Amgen in
 Thousand Oaks, Calif., are collaborating on a synthetic neurotrophic
 compound that can be taken orally and then travels to the brain,
 where it bonds with proteins in dopamine neurons. The tricky part is
 that most trophic molecules are too big to move across the minuscule
 blood vessels in the brain, so Guilford and Amgen are working on a
 smaller one that can get where it needs to go. The progress so far is
 promising. "We're in Phase 2 human trials now," says Dr. Craig Smith,
 president of Guilford. "Although we don't have results yet, if the drug
 proves safe and extremely effective in trials, it could be on the market
 as early as 2005."

 Whether such an anti-Parkinson's potion is really so close at hand is
 unclear, but scientists are not waiting to find out. Other possible
 treatments under study include boosting antioxidants, which would
 protect brain cells from free radicals, highly reactive molecules that
 are by-products of oxidation; and blocking the body's production of
 compounds called excitatory amino acids, which can cause neuron
 damage. It's hard to say which, if any, of these treatments will
 succeed, but with science closing in from so many directions, it's
 possible that for the first time, Parkinson's disease may find itself on
 the run.
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