from the world's big
A new study finds it may take decades or even a whole lifetime to appear.
- Young Parkinson's disease patients may have just provided a vital clue for prevention.
- Misbehaving neurons that eventually produce the disease may be present at birth.
- A drug already on the market looks to be able to arrest Parkinson's progress.
Looking at lab-made dopamine neurons<img type="lazy-image" data-runner-src="https://assets.rebelmouse.io/eyJhbGciOiJIUzI1NiIsInR5cCI6IkpXVCJ9.eyJpbWFnZSI6Imh0dHBzOi8vYXNzZXRzLnJibC5tcy8yMjY0MjY3My9vcmlnaW4uanBnIiwiZXhwaXJlc19hdCI6MTYwNzM1MzM4MH0.XiXy3h-mkmhsa9qRhBQjZI6vC4H4RIJNPvqhAJKThEw/img.jpg?width=980" id="da827" class="rm-shortcode" data-rm-shortcode-id="55fa1d29b4eac838c761e4d4d2749b36" data-rm-shortcode-name="rebelmouse-image" />
Image source: Kateryna Kon/Shutterstock<p>The authors' investigation began with an examination of neurons based on cells from young-onset Parkinson's (YOPD) patients who had no known mutations. From the cells, <a href="https://stemcells.nih.gov/info/basics/6.htm" target="_blank">induced pluripotent stem cells</a> (iPSCs) were generated and differentiated into dishes containing cultures of dopamine neurons. Senior study author <a href="https://bio.csmc.edu/view/15154/Clive-Svendsen.aspx?_ga=2.53353324.577457197.1580439866-1085877963.1580439866" target="_blank">Clive Svendsen</a> says, "Our technique gave us a window back in time to see how well the dopamine neurons might have functioned from the very start of a patient's life."</p><p>The scientists observed lysosomes within the YOPD neurons malfunctioning. Since lysosomes are counted on as "trash cans" for unnecessary or depleted proteins, the castoff chemicals began to pile up. In particular, substantial accumulations of soluble α-synuclein, a protein implicated in different types of Parkinson's, were seen. </p><p>Says Svendsen, "What we are seeing using this new model are the very first signs of young-onset Parkinson's,"revealing that, "It appears that dopamine neurons in these individuals may continue to mishandle α-synuclein over a period of 20 or 30 years, causing Parkinson's symptoms to emerge."</p><p>The researchers also saw unexpectedly high levels of the enzyme protein kinase C in its active form, though what that has to do with Parkinson's, if anything, is unknown.</p>
PEP005<img type="lazy-image" data-runner-src="https://assets.rebelmouse.io/eyJhbGciOiJIUzI1NiIsInR5cCI6IkpXVCJ9.eyJpbWFnZSI6Imh0dHBzOi8vYXNzZXRzLnJibC5tcy8yMjY0MjY3OC9vcmlnaW4uanBnIiwiZXhwaXJlc19hdCI6MTYyODE1NDY2Nn0.s_vugAK_mY2uTZyLHB2hGGkGFvR9ssundcffzQLlVOQ/img.jpg?width=980" id="aefe4" class="rm-shortcode" data-rm-shortcode-id="64f4405fe0b7060d51c5534209c20cc1" data-rm-shortcode-name="rebelmouse-image" />
Image source: sruilk/Shutterstock<p>The researchers tested a number of drugs on the cultures to see if any might address the observed accumulations of α-synuclein. (They performed parallel tests of laboratory mice.) One drug, PEP005, which is already approved by the FDA for treating skin pre-cancers, did effectively reduce the α-synuclein buildup, both in the iPSCs and the mice.</p><p>Since PEP005 is currently administered in gel form for treating skin, the researchers are now exploring how the drug might be modified so it can be delivered directly to the brain. The team also plans follow-on research to see if their findings apply equally to forms of Parkinson's beyond YOPD.</p>
Scientists identify key compounds that may help prevent brain diseases like Alzheimer’s, Parkinson’s, Huntington’s as well as Lou Gehrig’s disease.