Past generous supporters have enabled us to fund eight APBD research grants through the annual Million Dollar Bike Ride for rare disease research. You can help us reach our goal of funding two more research grants in 2021.
This year, your donation will be matched by the Orphan Disease Center at the University of Pennsylvania and again by generous community supporters so that every dollar you donate generates $3 in research.
Don’t miss this extraordinary matching opportunity to help us accelerate the path to clinical trials.
Dr. Angela Genge Discusses Biomarker Work In Related Fields
22:33
2020 APBD Biomarkers Workshop - Dr. Angela Genge Discusses Biomarker Work In Related Fields
Dr. Angela Genge, McGill University, Montreal, provided a thorough review of the efforts being made in the ALS community to develop biorepositories which will serve as a resource for scientists seeking to develop biomarkers and therapeutics. A biorepository is simply a library of biological specimens, images, and cells collected from ALS patients and then made available to research scientists. Following years of failure to develop effective therapeutics for ALS, Dr. Genge and other researchers realized that they simply had to do better. They learned that drug development for ALS means finding answers quickly, long before research enters phase III clinical trials – which ultimately, Dr. Genge notes is a long, drawn out process.
The ALS community has established and continues to develop a number of these ‘biobanks’. One such example is CALSNIC, a long-term, or longitudinal, study which focuses on clinical data collection such as cognitive testing results and imaging studies. Other initiatives include ANSWER ALS which collects stem cells for scientists and industry to use for studies on new potentially therapeutic molecules. Another major initiative, CAPTURE ALS, seeks to bring together the resources of all ALS clinics in Canada. Dr. Genge urges clinicians and laboratories to come together to form the necessary partnerships to establish biorepositories, clinical data and imaging resources. Dr. Genge says that these projects will allow scientists with important ideas to have a resource to develop their ideas for treatments.
For all of these initiatives, Dr. Genge emphasizes the importance of maintaining ‘open science’. This means that at McGill no claim will be made for intellectual property rights on any specimen. Further, all specimens will be shared openly with scientists and industry. Dr. Genge has found that because of their interest, industry sometimes offers a financial partnership in order to use these resources. Also, to encourage industry partnerships, McGill allows all intellectual property issues to ultimately remain with industry.
Dr. Genge notes that any institution can be a source for developing a program like CAPTURE and once established, it will serve as a catalyst for drug development.
Dr. Gülin Öz Discusses Biomarker Work In Related Fields
16:09
2020 APBD Biomarkers Workshop - Dr. Gülin Öz Discusses Biomarker Work In Related Fields
Dr. Gülin Öz , Professor of Radiology at the University of Minnesota Medical School spoke of the potential for using magnetic resonance imaging scans (MRS) to uncover many of the chemical secrets of the brain. In reference to APBD Dr. Oz addressed the issue of applying this technology to monitor progress in APBD clinical trials. At present there is not yet a way to measure the accumulation of polyglucosan bodies in the brain using MRS, however, in discussion, she noted that at least theoretically we may one day be able to do so. During the presentation Dr. Oz further reviewed many applications of MRS technology for measuring brain health and function, all of which may one day be applied as markers for APBD studies.
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