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Nose Knows
This year we began work that puts us in touch with tissues from living patients. We've desperately needed that, both to find markers for ALS that will let us diagnose it earlier and, more immediately, to have something "real" for testing therapies. A tiny, painlessly retrieved bit of neural tissue from the lining of a patient's nose—yes, it exists there—should let us tell if a new drug hits its proper cell target. We have the sampling down; now its usefulness is being tried. PET Project
PET-scan the nervous system to see if an ALS therapy is working? The idea's not far-fetched. This spring, two Center grantees reported progress on an agent to penetrate brain and spinal cord so as to flag telltale glutamate transporters there. The latter are in short supply in a number of ALS patients. A successful PET scan technique could tell which patients would respond best to a therapy that increases transporters. It could also be used to show if the therapy is working. ExCellerate It's like having a diamond in your pocket. This year, Center Director Jeff Rothstein unveiled ExCellerate, an endeavor to push therapeutic leads. The program was sparked by Center advances in using stem cells as disease models and insights into ALS molecular pathology. But experience in higher-throughput gene and drug screening and the promise in pilot therapeutic stem cell and gene-silencing studies, for example, play a part.
It's also born of a huge frustration with the status quo. "We're fast," says Rothstein, "but still not fast enough." ExCellerate advocates an industry-based approach to translational research. Center scientists would offer funding, direction and expertise to mold drug discovery professionals from pharma and biotech companies into a high-performance team with multimillion-dollar facilities. The incentive for them is more than basic funding: It's the dawning that ALS and more common neurodegenerative diseases like Parkinson's share pathways—that and having access to the Packard Center's expertise and clinical trial machinery. The diamond's still in the pocket, though, because of expense. It'll take some $30 million to let it shine. In the Pipeline Packard research has contributed to the existence of these potential therapies now in various stages in the clinical pipeline:
A small number of ALS patients—about 5 percent—find themselves with a thinking disorder, frontotemporal dementia (FTD). Likewise, though most people with FTD are ill just with the dementia, some have signs of ALS-like motor neuron symptoms. What's the tie between the two diseases? Is there an overlapping pathway that might provide clues to therapy for both? This year, psychiatrist Chaidi Onyike became a Center collaborator, adding his expertise as head of the Johns Hopkins FTD clinic to this new research direction. The Center keeps close ties with the nearby ALS Multidisciplinary Clinic at Johns Hopkins as a partner in research. Our scientist-clinicians are also quick to point patients to its care, knowing staff help them rise to their maximum capabilities. Kelly Showalter's work is typical. The occupational therapist commandeers the newest technology—such as a glance-activated computer—into patient service. And she fine-tunes its use through clinical studies. |
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