Featured Clinician Scientist: Dr. Sabrina Paganoni – Investigating Novel ALS Therapies

 

Today, we are pleased to feature ALS researcher Dr. Sabrina Paganoni from Massachusetts General Hospital and Spaulding Rehab Hospital. She is this year’s recipient of the Clinician Scientist Development Award in ALS Research given in partnership with the American Academy of Neurology (AAN). The award gives her the freedom to pursue multiple ALS clinical studies, including an exciting new trial testing inosine, and to continue to attend to people living with ALS in the clinic. Get to know this dedicated ALS researcher both in and out of the hospital.

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Sheila Essey Award Winner Profile: Dr. John Ravits Offers a Continuum of Care

Dr. John Ravits, Professor of Clinical Neurosciences and Head of the ALS Translational Research Program at the University of California, San Diego (UCSD) is a physician-scientist at the forefront of ALS thought and research of sporadic and familial ALS. Yesterday, at the 69th Annual American Academy of Neurology (AAN) Meeting in Boston, he was presented the prestigious 2017 Sheila Essey Award by Dick Essey, founder of the award named in honor of his wife Sheila who battled with ALS for ten years and died from the disease in 2004.

Over the course of his career, Dr. Ravits has emerged from the clinics where he cared for thousands of patients to formulate specific ideas about onset and progression of ALS and to take these ideas to create idealized models. His basic science research interests and contributions to science are far reaching from understanding the onset and progression of ALS in the body to exploring ALS neuropathology to functional genomics in motor neurons and more. Dr. Ravits also played an integral part in antisense oligonucleotide (ASO) therapy development for the most common genetic cause of ALS, the repeat expansion of the C9orf72 gene.

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Care Connection – Easing Stress on ALS Families

Families living with ALS are faced with a whole host of everyday challenges that can become a burden over time. The ALS Association created the Care Connection program to provide support to meet families’ needs to ultimately alleviate stress. Learn more about this extraordinary program and all the help it delivers.

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Featured Scientist: Dr. Nicholas Olney – Exploring new techniques for early detection

Today, we are happy to feature Dr. Nicholas Olney, this year’s recipient of the prestigious Clinical Research Training Fellowship in ALS Research Award given in partnership with the American Academy of Neurology (AAN). Dr. Olney is currently working on an ALS biomarker project aimed at developing clinical markers of disease progression, a major unmet need in ALS, at the University of California at San Francisco (UCSF) School of Medicine under the mentorship of Drs. Howard Rosen, Cathy Lomen-Hoerth and Bruce Miller.

Receiving this award is particularly poignant for Dr. Olney. His father, Richard K. Olney, M.D., was an internationally respected neurologist and researcher, who founded the ALS Treatment and Research Center at the University of California, San Francisco (UCSF), an ALS Association Certified Center of Excellence and served on the Board of Directors for The ALS Association Golden West Chapter. Dr. Olney was himself diagnosed with ALS in 2004, the same disease he helped his patients fight for over 30 years, and lost his battle in 2012. That same year, The AAN and The Association launched the Clinician Scientist Development Award and named the award in his honor.

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New C9orf72 Biomarker Shows Promise

Last month, Dr. Leonard Petrucelli at Mayo Clinic Jacksonville in Florida and colleagues reported discovering a new ALS biomarker that specifically detects a protein made by the C9orf72 expansion, the most common inherited cause of ALS. Their results are crucial to complement an upcoming clinical trial testing antisense drugs targeting the C9orf72 expansion. The ALS Association supported this study, including funding to bright, young scientists that significantly contributed to this project –  past and current Milton Safenowitz Postdoctoral Fellows, Drs. Tania Gendron, Marka van Blitterswijk, Veronique Belzil, Mercedes Prudencio from the Mayo Clinic Jacksonville and Clinical Research Fellow, Dr. Lindsey Hayes from Johns Hopkins University. The paper with Drs. Tania Gendron, Jeannie Chew, Jeannette Stankowski and Lindsey Hayes as co-first authors, along with 78 contributing researchers, was featured on the Science Translational Medicine front cover of the March 29th issue, which is a great scientific honor.

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PREVIEW: 2017 National ALS Advocacy Conference – Register Now

More than 500 people have already registered for the 2017 National ALS Advocacy Conference and time is running out to sign up. This conference is an annual opportunity for our advocates – people living with ALS, their families, friends, doctors and researchers – to share the ALS story and let Members of Congress know the true nature of the disease and why it is important to take action immediately.

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Misfolded SOD1 is not a primary component of sporadic ALS

Today, we welcome a guest scientist blogger, Dr. Sandrine Da Cruz from University of California San Diego (UCSD).  She, along with her colleagues, just published an important paper that looks into how SOD1 misfolding, the second most common inherited cause of ALS, impacts sporadic ALS (SALS). A huge percentage of ALS cases – approximately 90% are sporadic – meaning we do not know the cause. It is extremely important to understand more about this population of people living with ALS. The ALS Association is proud to support Dr. Da Cruz’s work. She is a past awardee of our Milton Safenowitz Postdoctoral Fellowship and is now an Assistant Investigator the Ludwig Institute at UCSD.

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