Thank You Letter
On behalf of all of us on the ALS research team at UMass Medical School, our heartfelt thanks for your continued, extraordinary support of our ALS research projects. Your generosity has facilitated some of the most innovative work we do, and has accelerated our efforts to develop human clinical trials in ALS.
The last year has seen continued progress in several fronts. Perhaps most importantly, we have made additional progress in moving our program to silence the SOD1 ALS gene toward testing in humans. This has entailed moving our studies from mice to not only non-human primates but also, in conjunction with collaborators at Tufts Medical School, dogs with a form of SOD1-mediated ALS. With these projects well underway, we are hopeful that the next year will see us make the transition to human trials of SOD1 gene silencing. Discussions of this approach are underway with the FDA.
In parallel studies, we are also aggressively pursuing three different strategies to turn off the ALS gene known at C9orf72. Mutations in this gene are the most common known cause of ALS, and also frequently cause fronto-temporal dementia. Our three approaches are all promising in cells in culture; one has been tested in a C9orf72 mouse model and also looks promising in vivo. If the mouse experiments continue to look encouraging, we will build on our experience with the SOD1 program above to move the C9orf72 studies into larger animals as a necessary first step toward a human trial.
In other investigations, we have begun studies of a new mouse model based on an ALS gene called profilin-1 (PFN1) which we identified and which we have now put into mice. Like the SOD1 mice, the PFN1 mice develop an adult onset motor neuron disorder which very much resembles ALS. We now have the ability to test novel therapies in these SOD1 and PFN1 ALS mouse models . Our contention is that any therapy that works in two different genetic forms of ALS may well be a therapy that works in sporadic (non-familial) ALS. We are also mindful of ways in which we can translate progress in familial ALS to non-familial forms of this disease.
Another project that has substantial momentum has involved developing a possible biomarker for ALS. Both in understanding the basic biology of ALS and in designing trials, it would be extremely helpful to have a biochemical substance or physiological activity that reflects the activity of the underlying ALS disease process. This is a bit like blood tests for heart attacks that powerfully correlate with the extent of the heart damage. With a good ALS biomarker, it should be possible to accelerate the diagnosis of the disease, begin trials earlier, and perform trials more rapidly using the biomarker as a study outcome. The marker we are studying looks very promising in our mouse models. The issue for us now is undertaking the steps required to move this to human testing. This is another way in which the support of the Timlin Race is so helfpul.
Finally, I note that there are many active ALS research programs at UMass. As he has for many years, Dr. John Landers continues to study new ALS genes. Dr. Fen-Biao Gao has made exceptional progress in decoding ALS using fruit flies. Dr. Larry Hayward is making models of ALS in cells in culture and in mice, focusing on the FUS gene. Dr. Daryl Bosco is making excellent progress in determining how selected proteins misfold and cause motor neuron toxicity. Many other investigators at UMass have been pivotal in ALS studies, including those in the Gene Therapy Center (Drs. Chris Mueller, Miguel Sena-Esteves, Guangping Gao) and in the RNA Therapy Institute (including Drs. Anastasia Khvorova and Victor Ambros).
Let me again tell you how grateful we are for your support of our ALS investigations. It is not an exaggeration to say that the generous funding raised by the Timlin Race has profoundly benefited ALS research and brought the whole field closer to a cure for ALS. We look forward to seeing you on race day!
Robert H. Brown, Jr., D.Phil., M.D.
Chair, Department of Neurology
UMass Medical School
55 Lake Avenue, North
Worcester, MA 01655
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