This is a Phase II clinical trial of an alpha synuclein targeting immunotherapy (called Prasinezumab – formerly called RO7046015 & PRX002) being conducted by the pharmaceutical company Roche and biotech firm Prothena Biosciences. The Axovant & voyager work is exciting, but I left them out here as they are primarily symptomatic plays as opposed to the potentially disease modifying efforts (like the Brain Neurotherapy Bio or Prevail trials). This gene produces an enzyme (called Glucocerebrosidase) that breaks down specific proteins. You have to manage the symptoms. Look around at the 7 minute mark in this video. This protein is different to GDNF, but has also been shown to have beneficial effects on dopamine neurons (Click here to read a good review on this topic). Excellent blog post, Simon. This study is scheduled to finish in late 2021. Your speech may become soft or slurred. The indirect approach: Anti-inflammatory approaches. To counter the over-active form of this protein in the carefully balanced environment of a cell, researchers have been developing inhibitors of this protein. This meeting “will focus on the origins and development of Parkinson’s, from potential triggers through the early symptoms and stages of the disease“. c-Abl is a protein that becomes activated in cells that are stressed and inhibiting it can boost autophagy. FAF1 is a protein involved with cell death, so by inhibiting/blocking it the researchers are investigating whether this could be beneficial in Parkinson’s. In 2020, I will be curious to see if companies like Fujifilm/Cellular Dynamics or Novo Nordisk step up to the plate and start a clinical trial program focused on cell transplantation for Parkinson’s. The body’s immune system can be encouraged to develop its own antibodies that target the toxic form of alpha synuclein (using, Researchers can design antibodies themselves that specifically target the toxic form of alpha synuclein (while leaving the normal version of the protein alone), and then inject those antibodies into the body (. Your doctor may suggest you work with a physical therapist to learn an exercise program that works for you. The beauty of this meeting is that it is a two for one deal – while there are scientific research lectures on one side of the conference center, at the same time there is the Rallying to the Challenge meeting being held on the other side. Imaging tests such as MRI, CT, ultrasound of the brain, and PET scans may also be used to help rule out other disorders. Find the Right Support You Need Search Now. A lot of excitement in Voyager AADC considering the RMAT designation, Hi Deepak, I was not aware that they were contemplating translation to the clinic so soon. A Phase I clinical trial of BIIB094 was registered in late 2019. Just a couple of days into 2019, C4 Therapeutics and the pharmaceuticals company Biogen announced a strategic collaboration to investigate the use of C4T’s novel protein degradation technology to discover and develop new treatments for neurodegenerative conditions, like Parkinson’s and Alzheimer’s (Click here to read the press release). Part 2 of this Phase II clinical trial is a 52-week blinded extension phase in which participants from the placebo group of the study will be re-randomly assigned into one of two active doses on a 1:1 basis (Click here to read more about this study). The advantage our stem cells have over others is that ours can help the body create the exact stem cells that are required for the damaged area. There is also a South Korean clinical trial being conducted at Bundang CHA Hospital, assessing fetal dopamine neurons in 15 participants and evaluating them over 5 years. We greatly appreciate your support. The direct approach involves treatments that specifically targets the underlying biology of the condition. As you shall see below, there are numerous clinical trials currently testing different therapies targeting different aspects of the autophagy process. Here at Poseidonia Healthcare, we offer a full range of treatments for Parkinsons Disease, here are a few benefits of the more popular treatments: Our Pluripotent Stem Cell Therapy can be administered in one of two ways. I would argue that the Abelson tyrosine kinase inhibitors can be both neuroprotective and disease halting ( if they work). They are using their site-directed enzyme enhancement therapy platform to identify compounds that are able to stabilize misfolded proteins AND restore the protein’s enzymatic activity. This study is not scheduled to finish until 2021 (Click here to read more about this). Supportive therapies can help ease some of the symptoms and complications of Parkinson's disease, such as pain, fatigue and depression. But there is a lot of activity in the Parkinson’s Plus domain: if you haven’t already, have a look at the Cambridge Centre for Parkinson-Plus ( https://ccpp.cam.ac.uk/ ) at Cambridge University which has set up several clinical trials late last year thanks to a generous donation. That trial is scheduled to finish in early 2020, and inflazome are interested in clinically testing their drug in neurodegenerative conditions like Parkinson’s. Our Scientist’s are developing stem cells into dopamine-producing nerve cells – the type of brain cells affected in Parkinson’s. This means that the actually benefits of the transplantation technique will not be apparent for some time (2-3 years on average). Share. Please understand that there is still a long way to go for all of these experimental therapies. A Phase II clinical trial testing Ceftriaxone in 106 people with Parkinson’s with dementia is scheduled to finish in late 2020 (Click here to read more about this trial). And late last year, Genentech, a subsidiary of Swiss pharma giant Roche, bought up Jecure Therapeutics – a biotech firm with a portfolio of preclinical NLRP3 inhibitors aimed at various inflammatory conditions (it is fair to say that this biotech was primarily looking at liver inflammation, but perhaps a neuro-focused pharma like Roche will also explore potential neurodegenerative applications). This is a phase II, 52 week trial of Ambroxol in 75 people with Parkinson’s Disease Dementia (Click here to read more about this trial). Researchers also recognize that, as the disease presents so differently in each patient, there may not be a single "cure" for Parkinson's disease. In 2017, the researchers behind this trial published a report evaluating their IPS cells in primate models of Parkinson’s (Click here to read more about that research report). In the case of Parkinson’s, this approach is primarily being focused on different forms of alpha synuclein. In 2019, we learnt the results of a small Phase I clinical trial of CuATSM in Parkinson’s. But there is evidence that this drug can increase the levels of the GBA protein (Glucocerebrosidase) in models of Parkinson’s (Click here to read a SoPD post on this). Unfortunately, there is only so much we can cover here. One additional autophagy-related biotech company worth watching in 2020 is Samsara Therapeutics. When diagnosed, people with Parkinson’s have often had the disease for years and have lost over half of the dopamine producing cells in their brain. Once mature, however, it has also been demonstrated (using brain imaging techniques) that these transplanted cells can produce dopamine. Continuous progress is being made towards cell replacement therapies for Parkinson’s. Neuroprotection is the area of research that has had the most attention over the years. Another small biotech taking an interesting approach to alpha synuclein is Nitrome Biosciences. This biotech firm is developing a cell-permeable form of PARKIN (iCP-Parkin), which has been reported to promote preservation of dopamine neurons in models of Parkinson’s (Click here to read more about this). It is very important for all readers of this post to appreciate that cell transplantation for Parkinson’s is still extremely experimental. Creative Commons Attribution 4.0 International License A future treatment for Parkinson’s? Larnaca One of the newer entrants to the world of Parkinson’s research is a biotech firm that popped up on the radar in 2019. South Korean firm Kainos Medicine completed a Phase I clinical study in 2019 evaluating KM-819 – a small molecule inhibitor for FAF1. This also is significant to reduce the speed of further advance of the illness. WOW! In addition, Parkinson's symptoms are not only movement-related. As a result, the mice’s Parkinson’s disease symptoms disappeared. *Patient feedback, articles and testimonials provided on this site are for informational purposes only and should not be considered as a guaranteed result for every case of illness. The ISCO trial involves 12 participants who have been evaluated over 12 months in this evaluation of safety and tolerability. United Kingdom About Blog The Cure Parkinson's Trust is a patient led charity which funds research that will have an impact on the lives of the 10 million people living with Parkinson's, with the hope of an eventual cure for the condition. Mitochondria: co-Q10, acyl carnitine, LRRK2 is known to promote RIPK1 activation (Source). A magnified image of individual dopamine producing neurons can be seen in panel B: Transplanted dopamine neurons. People may also experience rapid eye movement sleep behaviour disorder, which involves acting out your dreams. And this trial is interesting given the announcement in late 2018 of positive results for an open label Phase II clinical trial of EPI-589 in motor neurone disease/ALS. And until we have developed methods that can identify Parkinson’s long before the motor features appear (which would require only a disease halting treatment), some form of cell replacement therapy is required to introduce new cells to take up lost function. Scientists and doctors are working together to find a treatment or prevention technique. Fingers crossed for 2020. One way we can improve the health of cells (and potentially slow the progression of Parkinson’s) is to enhance their ability to clear (or dispose of) toxic proteins. In 2018, Alterity Therapeutics (formerly PRANA Biotechnology) initiated a clinical trial of their drug PBT434 in healthy individuals. Before we finish (yes, there is still more! Specifically, the researchers will be transplanting human parthenogenetic stem cells-derived neural stem cells (hpNSC). Ozone Therapy is effective for both activation of the cerebral microcirculation and increasing energy production by neurons, as recent studies have shown. Lekha S, 23 September 2020. New Hope for Parkinson's Disease Cure as Treatment Enters Human Trials. PharmaTher Files With FDA For Pre-IND Meeting On Use Of Ketamine In Treatment Of Parkinson’s Disease December 8, 2020 9:08 AM December 8, 2020 9:08 AM Jay Lutz 0 Comments Pharmather. The female egg cell is chemically encouraged to start dividing and then it becoming a collection of cells that is called a blastocyst, which ultimately go on to contain embryonic stem cell-like cells. The company is focused on Type 1 Gaucher disease at present, but have indicated that their gene therapy treatment (called AVR-RD-02) could be used in Parkinson’s (Source). In 2019, a new GLP-1R agonist called NLY01 entered the clinical trial process. So replacing dopamine neurons would not be a magic bullet. We will hopefully learn the results of this trial in 2020. Just a single treatment to inhibit PTB in mice converted native astrocytes, star-shaped support cells of the brain, into neurons that produce the neurotransmitter dopamine. These molecules started life as cancer drugs, but they are now being re-purposed for Parkinson’s. They also were awarded a grant from the Michael J. A Phase Ib study of DNL-151 was initiated in 2019 (click here to read more about this), and the study is scheduled to finish in 2020 (Click here to read more about this study). Placebo effect probably great, but if it works, fine. This trial is an open label study, involving 13 subjects. But the researchers then conducted a drug screen and identified a small molecule that promotes Miro1 degradation, and they found that this drug could rescue mulitple Parkinson’s models (Click here to read a previous SoPD post on the topic). Diabetes Medications Impacts Risk of Parkinson’s - October 2020. GRF6021 is a ‘plasma fraction’ (evidently made up of approximately 400 proteins derived from young blood – source). In February 2019, the company filed two Investigational New Drug (IND) applications with the US FDA to initiate human clinical trials of IkT-148009 (Click here to read the press release), but we haven’t heard anything since. Similar to the Bristol Phase II GDNF study (mentioned above), the drug was injected directly into the brain using an implanted canular system. Fill in your details below or click an icon to log in: You are commenting using your WordPress.com account. For all patients with Parkinson disease, treatment is symptomatic, focused on improvement in motor (eg, tremor, rigidity, bradykinesia) and nonmotor (eg, constipation, cognition, mood, sleep) signs and symptoms. A second Chinese cell transplantation trial is also registered, and being conducted at The First People’s Hospital of Yunnan Province. Feb. 27 (UPI) --Researchers have developed a new drug that could correct damage to the brain caused by Parkinson's disease and lead to improvement … Has given rise to the development of this study is published June 24, 2020 ; (. Yunnan Province cytokine ) and thinking Difficulties: you may recall that are. Called dopamine molecules targeting alpha synuclein is a protein called Miro1 own, and of..., members of the protein is believed that environment and genetics play role. Company signed a 4 years partnership with the neurodegeneration seen in some cases of Parkinson ’ s still pre for! 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