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Old 02-19-2017, 09:07 PM
Tupelo3 Tupelo3 is offline
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Tupelo3 Tupelo3 is offline
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Join Date: Mar 2013
Location: New Jersey
Posts: 832
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Quote:
Originally Posted by Debi Brooks View Post
Earlier this week, Sanofi Genzyme announced a clinical trial to test a drug designed for people with Parkinson’s disease (PD) who have a specific genetic mutation (GBA).

This study is a major step forward in PD research — it’s the first drug trial in which a person’s genetic status determines eligibility and is testing a potential therapy to slow or stop disease progression.

Read more on The Michael J. Fox Foundation’s blog: First Trial Begins Testing Drug in People with GBA Mutation | Parkinson's Disease

Best, Debi
I agree, Debi. This is a major advance as we move into more individualize drug development and research design. Pharmacogenomics is already being used quite successfully in cancer research and will likely become more useful in PD research. In fact, Nilotinib, the cancer drug we are all following closely, was developed for people with Philadelphia positive CML, a form of leukemia associated with a specific genetic mutation.

However, what I find to be incredibly frustrating is the length of time to complete the study. Five years to conduct a phase 2 trial is just ludicrous. Even if this drug eventually proves to be successful and comes to market, it will likely be 10 years from now, too late to do much good for most of us on this forum. The research system is broken and changes are needed throughout the process to speed up drug research. The whole process is inefficient and the rules and regulations are overwhelming. And, patients deserve some of the blame as the lack of willing volunteers is one of the major contributors to trial delays, which is inexcusable. It's time for all the stakeholders involved, including patients, researchers, drug companies, IRB's, funding organizations, the FDA, etc., to come together to improve and significantly speed up the process.

Gary
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