Biotech Strategy Blog

Commentary on Science, Innovation & New Products with a focus on Oncology, Hematology & Cancer Immunotherapy

Posts tagged ‘Parkinson’s Disease New Products’

Yumanity logoWe recently wrote about Syros Pharmaceuticals, one of whose founders, Dr Rick Young is based at the Whitehead Institute of MIT in Cambridge MA.

Another biopharma start-up company being spun out from research done at the Whitehead Institute for Biomedical Research is Yumanity Therapeutics.

The company recently launched with Tony Coles as CEO and Ken Rhodes as Chief Scientific Officer. Their focus is on transforming drug discovery for neurodegenerative diseases caused by protein misfolding.

The scientific founder is Dr Susan Lindquist, who spoke with Biotech Strategy Blog about her research and the Yumanity approach to drug development.

The company is committed to “improving human conditions. That’s why we call it Yumanity. The Y is for yeast, but it really is focused on humanity,” said Lindquist.

Dr Linquist started her interview by noting that as we live longer, we are more likely to get neurodegenerative diseases, starkly noting the reality of the lack of progress in drug development in this area:

“There is really, right now, nothing that we can do about them. We just do not understand how to move the needle on these and it’s really becoming an absolute crisis and it is taking a very substantial section of our healthcare budget as it is. As we continue to make better inroads against cancer and HIV and all of the other ills of mankind, it’s just going to get worse, I think. Everybody is beginning to appreciate that there is going to be an economic disaster and that we are going to ruining the next generation in a way that, at this point, is going to be tragic.”

So what is the approach Yumanity is taking, in the hope of succeeding where others have failed?

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This month is Parkinson’s awareness month.  Following on from my recent interview (that you can read here & here) with Dr Todd Sherer of The Michael J. Fox Foundation for Parkinson’s Research, I was interested to read about progress being made on the road to towards targeted therapies.

The April 2011 issue of Nature Chemical Biology reports the development of a selective inhibitor of leucine-rich repeat kinase 2 (LRRK2), a gene that is mutated in some patients with Parkinson’s disease.

The team of researchers from Dana-Farber Cancer Institute, Harvard Medical School, University of Dundee, Scripps Research Institute and ActivX Biosciences applied a novel, screening strategy focused on selectively inhibiting LRRK2.

The result was the identification of LRRK2-IN-1, a novel analog that inhibits both wild-type and mutant LRRK2 kinase activity. The team confirmed the activity of LRRK2-IN-1 using human lymphoblastoid cells from a Parkinson’s disease patient with the LRRK2 mutation.

Unfortunately, LRRK2-IN-1 was unable to cross the blood-brain barrier, which means that it is not suitable for Parkinson’s disease.  However, this research is progress on the road to LRRK2 inhibition and the development of a targeted therapy in the future.

Moving forwards Parkinsons’ researchers may wish to consider combining new small molecules with nanoparticles that are able to cross the blood-brain barrier; this may be the way to deliver targeted therapies to the brain.


ResearchBlogging.orgDeng, X., Dzamko, N., Prescott, A., Davies, P., Liu, Q., Yang, Q., Lee, J., Patricelli, M., Nomanbhoy, T., Alessi, D., & Gray, N. (2011). Characterization of a selective inhibitor of the Parkinson’s disease kinase LRRK2 Nature Chemical Biology, 7 (4), 203-205 DOI: 10.1038/nCHeMBIO.538

Yesterday, I posted the first part of my interview with Dr Todd Sherer, Chief Program Officer at the Michael J Fox Foundation.

Next week, I will be posting the second part of the interview that discusses the significant research the foundation is funding on biomarkers that can help the diagnosis of the disease and monitor its progression.

If you are interested in learning more about the latest developments around Parkinson’s disease biomarkers, then you may wish to consider the April 27, 2011 webinar from the American Association for the Advancement of Science (AAAS) on the “Early Detection of Parkinson’s Disease: The Challenges and Potential of New Biomarkers.”

Moderated by Dr Todd Sherer, the webinar will discuss the only FDA approved biomarker, DaTscan that provides for imaging of dopamine transporters at dopaminergic nerve terminals in the nigrostriatal pathway.  It will also discuss the Parkinson’s Progression Markers Initiative (PPMI) that the foundation is funding.

Today is the deadline to take advantage of the early bird discounts on offer for this webinar.

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