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The following is a summary of Jeffrey Rothstein's talk at the "Ask the Experts" educational forum on November 30, 2004. A Plan for Treatment in ALS: Small Molecules,
Gene Therapy and Stem Cells Motor neurons may not be dying on their own. Their neighboring cells are likely contributing to the motor neuron injury and death; this complicates the task of discovering the cause of ALS and developing effective treatments. Models used to study the disease and learn more about therapeutic approaches include:
A number of potential therapeutic approaches are listed below. Research efforts are underway to develop all of these therapeutic approaches.
The delivery of small molecules and trophic (growth) factors, such as IGF-1 and GDNF, remains a challenge as the brain is surrounded by a protective barrier. This blood-brain barrier separates the blood and the brain and prevents access to the brain and motor neurons for many of these compounds. Dr. Rothstein described many studies in the laboratory involving stem cell research. He emphasized the many different sources of stem cells and he cautioned that, although very promising, there are many challenges scientists are working to overcome before stem cells can move into successful therapies in the laboratory. To overcome delivery issues for drugs, and encouraging them to pass the blood-brain barrier, technologies such as gene therapy are being developed for ALS. As a result of screening efforts using FDA-approved compounds and a partnership between The ALS Association and the National Institute of Neurological Diseases and Stroke (NINDS), Ceftriaxone will enter clinical trials. Originally prescribed for its antibiotic properties, this compound has shown an effect in model systems of ALS and is acting through properties other than its antibiotic function. Dr. Rothstein noted that patients should be aware of three potential outcomes when taking drugs or participating in a clinical trial. Each of these outcomes is possible. 1) The compound may slow disease. 2) The compound may do nothing. 3) The compound may speed up disease progression.
More highlights from the 15th Annual International ALS/MND Symposium
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