[BillO]

To: The ALS Community Date: January 6, 2009 Subject: Robert Packard Center ALS News Network STUDIES INCH PROTECTIVE FACTOR VEGF FORWARD ON THERAPY’S PATHhttp://www.alscenter.org/news/briefs/090106.cfm These last few years, VEGF-A, a natural molecule in the body, hassurfaced regularly, linked with ALS in scientific journals and, morerecently, in newspapers. Vascular Endothelial Growth Factor-A encouragesblood vessels to grow out like so many roots of a plant. It's a keymolecule in wound healing. But VEGF-A also affects neurons, and that may be where the ALS tiecomes in. Increasingly, scientists are eyeing that trait in the VEGFfamily tree as they hunt for new therapeutic paths for theneurodegenerative disease. And recent advances suggest it's worthwhile. Nearly a decade ago, studies showed that mice short on VEGF-A losetheir motor neurons in a way that closely resembles ALS. Likewise, ALSanimal models engineered for higher VEGF-A levels, either by tweakingthe VEGF-A gene or by delivering the molecule directly to the nervoussystem, lived much longer. Much of that animal model work, as well as more recent research inhumans by Belgian scientists Peter Carmeliet and Wim Robberecht at theUniversity of Leuven stands out in the field. (Both are Packard Centerresearchers, with Carmeliet and Robberecht on the Packard scientificadvisory board and Robberecht a Packard grantee.) In 2004, a genetic study of ALS patients and healthy controls in Europeby Robberecht and Carmeliet's group with was the first to show that,like mice, some groups of humans with the disease appear to have lowerlevels of VEGF-A. That pushed studies of the molecule in a moretherapy-oriented direction. Even so, the worldwide scientific community was abuzz a year later atthe Belgians' report that VEGF-A introduced into the central nervoussystem via a brain route extended life significantly in ALS rats.Subsequently, their collaborations with Neuronova, a Swedish biotechcompany, showed that VEGF-A administered to monkeys in the same wayappeared safe in doses equivalent to those that helped the ALS rats. An important next step, a Phase I clinical trial of the agent - arequisite safety trial - is just underway as part of Robberecht'scollaboration with Neuronova, at the University Hospital in Leuven. Meanwhile, the group continues to explore how VEGF functions in thebody. Their study last October in The Journal of Neuroscience centers onVEGF-B, yet another member of the molecular family. Like VEGF-A, the Bversion also plays a key role in guarding neurons from internal assault.Unlike the A form, however, VEGF-B apparently has nothing to do withblood vessel sprouting, Carmeliet says. And that may make it even moreattractive to explore as ALS therapy. At this point, VEGF-B has been purified in small quantities forlaboratory studies, but doesn't yet exist in a refined form human trialswould require. "We're looking into that aspect, however," saysCarmeliet, "as well as continuing to explore the biology underlyingVEGF's ability to protect neurons." ___________________________________ About The Robert Packard Center for ALS Research at Johns Hopkinswww.alscenter.org Located in Baltimore, the Robert Packard Center for ALS Research atJohns Hopkins is a worldwide collaboration of scientists aimed atdeveloping therapies and a cure for amyotrophic lateral sclerosis (ALS),also known as Lou Gehrig's disease. The Center is the only institution of its kind dedicated solely to thedisease. Its research is meant to translate rapidly from the lab benchto the clinic, largely by eliminating time spent waiting for grants andlowering institutional barriers to sharing scientific results. Scientists and clinician members of the Packard Center have moved drugsreliably and rapidly from preclinical experiments to human trials.Direct or indirect links to international biotech or pharmaceuticalcompanies bring the infrastructure and experience needed to makepromising drugs into therapies. Packard scientists are the first to propose and test a combinationapproach to drug therapy, a tactic that has worked for AIDS, cancer andother diseases. ALS is a progressive, disabling neuromuscular disease that causescomplete paralysis and loss of function - including the ability to eat,speak and breathe. ALS progresses quickly and is not curable. Mostpatients die within five years of diagnosis. _________________________________________Rebecca BergerResearch Program CoordinatorRobert Packard Center for ALS Research at Johns Hopkins5801 Smith Avenue | McAuley Suite 110Baltimore, MD 21209410.735.7678 direct410.735.7680 faxrberger6@jhmi.edu www.alscenter.org www.fiesta5K.org _________________________________________Rebecca BergerResearch Program CoordinatorRobert Packard Center for ALS Research at Johns Hopkins5801 Smith Avenue | McAuley Suite 110Baltimore, MD 21209410.735.7678 direct410.735.7680 faxrberger6@jhmi.eduwww.alscenter.orgwww.fiesta5K. org

[russ conley]

I have a question regarding this and other growth factors that maybe you can help me understand. My question is might they also cause disease to grow, like cancer? How do you just make specific cells to grow? I was denied one trial already because of the fact I had cancer of the thyroid and had it removed. They said any history with cancer disqualified me.