Media Coverage
- Drug discovery for Parkinson’s and related disorders (Chung*, Khurana* et al., Science 2013; Tardiff et al., Science 2013; Mittal et al., Science 2013; Vincent et al., Cell Reports 2018): Coverage of our back-to-back foundational Science papers in 2013 included this article in the Boston Globe and this very comprehensive overview in the Science of Parkinson’s blog. Ultimately, the drug discovery platform for Parkinson’s disease we described in these papers led to the development of a drug YTX-7739 for Parkinson’s disease and related disorders. The drug was developed by Yumanity Therapeutics, a company co-founded by Vik Khurana with Susan Lindquist and others in 2014. YTX-7739, profiled in Alzforum and here, is a steoaroyl-co-A desaturate inhibitor, and is the first drug to target lipids for Parkinson’s disease. After positive top-line phase1B results, the drug was acquired by Janssen (Johnson and Johnson) for further development. The Khurana lab has also contributed its stem cell platform to other efforts in drug discovery, including one that is considering a class of asthma drugs, beta-2 adrenergic agonists, for treatment of Parkinson’s disease. This collaboration with the Scherzer laboratory at BWH was published in Science in 2017 and profiled in numerous media outlets, including Nature. A drug clenbuterol arising from this work is currently in clinical trial.
- The first genome-scale combined genetic and physical interaction map of a proteinopathy (Khurana*, Peng*, Chung* et al., Cell Systems 2017; Chung*, Khurana* et al., Cell Systems 2017; Jarosz and Khurana Cell 2017). Our first systematic “mapping” of alpha synucleinopathy, described in detail in our Research synopsis, was covered by Whitehead Institute News, Alzforum and Science of Parkinson’s blog among other websites.
- A new function for the Parkinson’s protein alpha-synuclein in mRNA metabolism and gene regulation (Hallacli et al., Cell 2022): Dr Erinc Hallacli in our lab led a study that identified a surprising new function for alpha-synuclein. Traditionally thought of as a protein involved in synaptic vesicle trafficking, the membrane-interacting domain of alpha-synuclein has an altogether different function in the cytosol, namely to regulate the degradation of mRNAs and hence the expression of our genes. The new function also has disease-relevance. You can read more about this paper, published as a Featured Article in the June 2022 edition of Cell, in Alzforum, the Harvard University Gazette, the New York Stem Cell Foundation, the American Parkinson’s Disease Association, Parkinson’s News Today, the Science of Parkinson’s blog among other news sources. The paper was also profiled in Nature Reviews Neuroscience and Nature Chemical Biology.
- A new diagnostic test for synucleinopathies: Dr Anastasia Kuzkina in our lab routinely uses a method (invented by Dr Claudio Soto) called “seeded amplification” to amplify one toxic form of the protein alpha-synuclein from our patients with Parkinson’s disease, multiple system atrophy and dementia with Lewy bodies. Recently, the Michael J Fox Foundation published the most comprehensive study to date on its Parkinson’s Progression Markers Initiative (PPMI) cohort. Vik was interviewed for an article in STAT News that covered their paper.
- Environmental Toxicants in Parkinson’s disease: We have been awarded two grants from the Department of Defense to study how environmental exposures, in combination with genetic factors, can lead to Parkinson’s and related disorders. Vik was interviewed by the New York Times on the use of paraquat in late 2016, spurring continued research in the lab on this important question. A recent collaborative effort with Beate Ritz, led by Drs Kim Paul at UCLA and Rich Krolewski (in our and Lee Rubin’s groups) has identified 10 additional pesticides that are directly toxic to Parkinson patient dopaminergic neurons. Our Nature Communications paper has received coverage in the press, including by US News and World Report, Parkinson’s News Today and Medical News Today. Read the BWH press release.
Parkinson’s disease 101
This “Parkinson’s Disease 101” seminar for the Harvard BrainScience Initiative provides an introductory primer to Parkinson’s disease and touches on themes that relate to research in the lab.
A new function for the protein alpha-synuclein in mRNA regulation: Implications for Parkinson’s disease
In the following video for the American Parkinson’s Disease Association, and Harvard Gazette article, Vik and team member Erinc Hallacli discuss new findings from the lab that link the Parkinson’s protein alpha-synuclein to mRNA and gene expression regulation, and the wider implications of these findings for understanding and treating Parkinson’s disease.
Tailor-making a gene therapy for a young boy with a neurodegenerative movement disorder
A short video by team member Asia Korecka-Roet describes an ASO genetic therapy project for a young boy with DRPLA (dentatorubropallidoluysian atrophy), a polyglutamine expansion-driven neurodegenerative disorder. In this project we are testing the gene therapy in stem-cells generated from the patient prior to clinical trial. The aim is to optimize the genetic treatment.
Sculpting a research roadmap for MSA
Vik is proud to be a member of the Board of Directors of the MSA Coalition. As the Scientific Liaison of the Board, he has been charged to enact a research roadmap through a series of Collaborative Research Cores. View the press release for the genetics Core G. In this video he describes this collaborative plan for MSA research.
A new approach for clinical trials in synucleinopathies like PD and MSA
In this video, Vik describes a new approach he and others in the BWH Movement Disorders Division are taking for clinical trials. In a process known as “deep phenotyping” we will evaluate our patients with clinical scales, biometrics and PET/MRI imaging in conjunction with genome sequencing and stem-cell degeneration. This approach allows us to understand our patient’s clinical trajectory before they receive a drug in order to determine whether that trajectory is improved after the patient is given a drug. Some patients may respond, others may not. Heterogeneous responses may be missed in conventional randomized clinical trials. We hope that one day treatment response (or non-response) can be predicted by simply testing the drug in neurons and glia generated from our patients.
