Study links Alzheimer’s disease with circular RNA – National Institute on Aging

Certain loops of ribonucleic acid circular RNAs in the brain are associated with the development of Alzheimers disease, according to a study reported recently in Nature Neuroscience. Because these circular RNAs can be detected not just in the brain but in cerebrospinal fluid and blood, they have the potential to be developed into lab tests to detect Alzheimers before symptoms appear.

RNAs are the molecules that carry instructions from genes to create proteins in the body. Most research has been done with the linear form, and scientists are just beginning to learn how the body uses circular RNA, especially in the brain. Using modern sequencing technologies, investigators at Washington University in St. Louis analyzed the complete RNA content in brain samples and compared circular RNAs in people with and without Alzheimers disease.

The investigators first compared the RNA sequences from brain tissue donated by 83 people who had Alzheimers with samples from 13 healthy people. Then they compared the RNA sequences from brain tissue samples from 89 people with Alzheimers, 66 probable or possible cases, and 40 controls obtained from the NIA-supported Accelerating Medicines PartnershipAlzheimers Disease (AMP-AD) Biomarkers Project.

In each of these two datasets, more than 3,500 circular RNAs were detected and analyzed. The researchers discovered that certain circular RNAs are strongly associated with the development of Alzheimers. In the first dataset, three circular RNAs, including one known as circHOMER1, were significantly associated with three Alzheimers traits: having a diagnosis of Alzheimers, a quantitative measure of dementia severity at the end of life, and the number and distribution of tau tangles throughout the brain.

The second dataset replicated those findings: 28 circular RNAs, including circHOMER1, were significantly associated with all three traits. Together, these findings suggest there is a significant association between the expression of certain circular RNAs like circHOMER1 in the brain and Alzheimers traits.

Next, the research team analyzed circular RNAs in samples from 21 brains donated by people with early-onset Alzheimers who were part of the NIA-supported Dominantly Inherited Alzheimer Network, which is a study to identify biomarkers to predict the development of Alzheimers. In these people with early-onset disease, the magnitude of changes in RNA expression was even greater than in the first two datasets. This finding suggests even more strongly that the correlation between certain circular RNAs and disease is meaningful and not merely a coincidence. However, it is not enough to suggest causation.

The investigators also analyzed circular RNAs in samples from people who had mild or no dementia but whose brains showed signs of Alzheimers. After comparing results to those from healthy controls and those with dementia from Alzheimers, the research team found evidence for early changes in circular RNA before the people had substantial symptoms of Alzheimers.

Taken together, these results suggest that specific circular RNAs might have the potential for use as biomarkers to detect Alzheimers disease before symptoms appear. Future studies are needed to better understand the functions of the circular RNAs identified in these analyses.

This research was supported in part by NIA grants R01AG044546, P01AG003991, RF1AG053303, R01AG058501, U01AG058922, RF1AG058501, R01AG057777, K01AG046374, K23AG049087, P50AG05681, P01AG03991, P01AG026276 and UF1AG032438.

These activities relate to NIAs AD+ADRD Research Implementation Milestones: Milestone 2.A Disease Mechanisms and Milestones 9.B and 9.F Biomarkers.

Reference:

Dube U, et al. An atlas of cortical circular RNA expression in Alzheimer disease brains demonstrates clinical and pathological associations. Nature Neuroscience. 2019; 22(11):1903-1912. doi: 10.1038/s41593-019-0501-5.

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Study links Alzheimer's disease with circular RNA - National Institute on Aging

Beebe Healthcare expands partnership with Jefferson Neuroscience Network – Milford Beacon

Beebe Healthcare announced it will expand its partnership with the Thomas Jefferson Neuroscience Network, to include a robotic teleconferencing unit in Beebes Emergency Department.

The Jefferson Expert Teleconsulting, or JET, unit is the regions first university-based, high-tech mobile robot system for acute stroke.

When a patient comes into Beebes Emergency Department with a suspected stroke, the emergency teams spring into action to run tests and get a medical history on the patient. As soon as a stroke is suspected, the JET protocol is started.

In addition to being used for patients who come in through the Emergency Department, the JET protocol will also be used for patients who have a stroke while already admitted to the hospital.

The stroke robot provides Jefferson Network hospitals with 24/7 access to vascular neurologists and neurosurgeons for emergency consultation services. The goal is to complement the care provided by community neurologists and/or emergency physicians to those patients presenting with symptoms of a stroke.

The process starts with a phone call from Beebes emergency team to the Jefferson Neuroscience Network to reach the on-call stroke neurologist.

Then the neurologist connects with the Beebe team via a mobile robotic system in the emergency room. The mobile robotic system allows the neurologist to speak directly to the team, the patient, and family members via secure videoconference technology. This allows the Jefferson neurologist to gather information, as well as to conduct a neurologic examination on the patient. The Jefferson neurologist can also review test results and medical history while on the teleconference.

This system allows us to be more efficient when treating stroke patients, said Nick Perchiniak, of Sussex Emergency Associates, the team providing care in Beebes Emergency Department. When it comes to stroke, time is brain, so it is very important to be able to diagnose and treat a stroke patient quickly. The Jefferson robot allows us to have quick access to their neurology experts within minutes of a patients arrival.

Once the immediate treatment plan is put into action, the Beebe and Jefferson medical teams are able to make decisions about the best next steps for the patient, including transport to Jefferson if necessary.

This is going to be especially helpful at our new South Coastal Health Campus Emergency Department, said Perchiniak. We will have the mobile robot there as well so that if a patient comes in with a possible stroke, we can activate a stroke consultation with Jefferson quickly and get the patient the help they need.

From South Coastal, patients could be taken by helicopter to Jefferson or to Beebes Margaret H. Rollins Lewes Campus. In Lewes, the patient could also be taken by helicopter to Jefferson.

In addition to the Jefferson stroke robot and videoconference system, Beebes Emergency Department also uses similar technology or telemedicine for psychiatric or behavioral health patients to connect with specialists in the region, and for children, the Emergency Department is able to teleconference with pediatric specialists at A.I. duPont/Nemours.

This takes our relationship with Jefferson to a new level, said Lynn Toth, cardiovascular medical specialist at Beebe. It will be a great resource for both our medical teams and for the community. The program gives us nearly immediate access to renowned stroke experts, which can only improve the way we care for our community.

For more, visit beebehealthcare.org.

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Beebe Healthcare expands partnership with Jefferson Neuroscience Network - Milford Beacon

Could we ever create an AI as smart as the human brain? – Telegraph.co.uk

For the last three decades our best models of reinforcement learning in AI and neuroscience have focused almost entirely on learning to predict the average future reward, he says.

But this doesnt reflect real life when playing the lottery, for example, people expect to either win big, or win nothing no one is thinking about getting the average outcome.

The work has significance in some key areas. For one, it could lift the lid on whats happening neurologically with conditions like addiction and depression.

If some neurons are reducing dopamine, or, as Dabney puts it, thinking in pessimistic terms, there may be a situation in which they take the reins, shifting the brains entire outlook to a pessimistic one. Seems like a pretty good characterisation of what depression involves, he says.

The finding is also a rare example of AI shedding light on the way the brain works, validating the work being done by researchers to get AI to work just as the brain does. And with researchers expecting this to be a more common occurrence over the next decade, there could be an acceleration in the understanding of the way the brain works, and the subsequent advancement of AI.

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Could we ever create an AI as smart as the human brain? - Telegraph.co.uk

Connecting Fear and Reward in the Mammalian Brain – Technology Networks

When you expect a really bad experience to happen and then it doesnt, its a distinctly positive feeling. A new study of fear extinction training in mice may suggest why: The findings not only identify the exact population of brain cells that are key for learning not to feel afraid anymore, but also show these neurons are the same ones that help encode feelings of reward.

The study specifically shows that fear extinction memories and feelings of reward alike are stored by neurons that express the gene Ppp1r1b in the posterior of the basolateral amygdala (pBLA), a region known to assign associations of aversive or rewarding feelings, or valence, with memories. The study was conducted by Xiangyu Zhang, a graduate student, Joshua Kim, a former graduate student, and Susumu Tonegawa, Professor of Biology and Neuroscience at RIKEN-MIT Laboratory of Neural Circuit Genetics at the Picower Institute for Learning and Memory at MIT and Howard Hughes Medical Institute.

We constantly live at the balance of positive and negative emotion, Tonegawa said. We need to have very strong memories of dangerous circumstances in order to avoid similar circumstances to recur. But if we are constantly feeling threatened, we can become depressed. You need a way to bring your emotional state back to something more positive.

Overriding fear with reward

In a prior study, Kim showed that Ppp1r1b-expressing neurons encode rewarding valence and compete with distinct Rspo2-expressing neurons in the BLA that encode negative valence. In the new study, Zhang, Kim and Tonegawa set out to determine whether this competitive balance also underlies fear and its extinction.

In fear extinction, an original fearful memory is thought to be essentially overwritten by a new memory that is not fearful. In the study, for instance, mice were exposed to little shocks in a chamber, making them freeze due to the formation of fearful memory. But the next day, when the mice were returned to the same chamber for a longer period of time without any further little shocks, freezing gradually dissipated and hence this treatment is called fear extinction training. The fundamental question then is whether the fearful memory is lost or just suppressed by the formation of a new memory during the fear extinction training.

While the mice underwent fear extinction training the scientists watched the activity of the different neural populations in the BLA. They saw that Ppp1r1b cells were more active and Rspo2 cells were less active in mice that experienced fear extinction. They also saw that while Rspo2 cells were mostly activated by the shocks and were inhibited during fear extinction, Ppp1r1b cells were mostly active during extinction memory training and retrieval but were inhibited during the shocks.

These and other experiments suggested to the authors that the hypothetical fear extinction memory may be formed in the Ppp1r1b neuronal population and the team went on to demonstrate this vigorously. For this, they employed the technique previously pioneered in their lab for the identification and manipulation of the neuronal population that holds specific memory information, memory engram cells. Zhang labeled Ppp1r1b neurons that were activated during retrieval of fear extinction memory with the light-sensitive protein channel rhodopsin. When these neurons were activated by blue laser light during a second round of fear extinction training it enhanced and accelerated the extinction. Moreover, when the engram cells were inhibited by another optogenetic technique, fear extinction was impaired because the Ppp1r1b engram neurons could no longer suppress the Rspo2 fear neurons. That allowed the fear memory to regain primacy.

These data met the fundamental criteria for the existence of engram cells for fear extinction memory within the pBLA Ppp1r1b cell population: activation and reactivation by recall and enduring and off-line maintenance of the acquired extinction memory.

Because Kim had previously shown Ppp1r1b neurons are activated by rewards and drive appetitive behavior and memory, the team sequentially tracked Ppp1r1b cell activity in mice that eagerly received water reward followed by food reward followed by fear extinction training and fear extinction memory retrieval. The overlap of Ppp1r1b neurons activated by fear extinction vs. water reward was as high as the overlap of neurons activated by water vs. food reward.

And finally, artificial optogenetic activation of Ppp1r1b extinction memory engram cells was as effective as optogenetic activation of Ppp1r1b water reward-activated neurons in driving appetitive behaviors. Reciprocally, artificial optogenetic activation of water-responding Ppp1r1b neurons enhanced fear extinction training as efficiently as optogenetic activation of fear extinction memory engram cells. These results demonstrate that fear extinction is equivalent to bona fide rewards and therefore provide the neuroscientific basis for the widely held experience in daily life: omission of expected punishment is a reward.

What next?

By establishing this intimate connection between fear extinction and reward and by identifying a genetically defined neuronal population (Ppp1r1b) that plays a crucial role in fear extinction this study provides potential therapeutic targets for treating fear disorders like PTSD and anxiety, Zhang said.

From the basic scientific point of view, Tonegawa said, how fear extinction training specifically activates Ppp1r1b neurons would be an important question to address. More imaginatively, results showing how Ppp1r1b neurons override Rspo2 neurons in fear extinction raises an intriguing question about whether a reciprocal dynamic might also occur in the brain and behavior. Investigating joy extinction via these mechanisms might be an interesting research topic.

Reference

Zhang et al. (2020) Amygdala Reward Neurons Form and Store Fear Extinction Memory. Neuron. DOI: https://doi.org/10.1016/j.neuron.2019.12.025

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Phoenix Childrens Is the First-Ever Health System in the U.S to Use Medtronic Stealth Autoguide Cranial Robotic Guidance Platform for Neurosurgery -…

Pediatric Health System First to Use Groundbreaking Cranial Robotic System in a Patient Surgery

DUBLIN and PHOENIX, Jan. 16, 2020 (GLOBE NEWSWIRE) -- Nationally ranked pediatric leader, Phoenix Childrens Hospital, is the first-ever health system in the U.S. to receive and deploy the newly FDA-cleared Medtronic Stealth Autoguide platform. Medtronic, a global leader in medical technology, chose Barrow Neurological Institute (BNI) at Phoenix Childrens as its first partner using this robotic technology. The highly advanced surgical tool is intended for use with the Medtronic StealthStation system, and Phoenix Childrens Hospital will use it in surgery for pediatric patients suffering from a range of neurological conditions.

Phoenix Childrens is proud to invest in the best possible technology for use while we provide outstanding care to children, said Daniel Ostlie, M.D., surgeon in chief and chair of Surgery at Phoenix Childrens. We are committed to being at the forefront of surgical innovation and having the most advanced solutions for pediatric patients.

BNI at Phoenix Childrens surgical staff have undergone comprehensive training with the Medtronic team as they prepared to use the Stealth Autoguide robotic guidance system in patient neurosurgery cases in early January.

The Stealth Autoguide is a tremendous addition to the neurosurgical teams tools at Phoenix Childrens, said P. David Adelson, division chief of Neurosurgery and director of BNI at Phoenix Childrens. Neurosurgery is such an intricate specialty, and having this technology at our fingertips perfectly aligns with our mission to provide state-of-the-art care to improve the health and quality of life for the children we see here.

Ranked a top pediatric neuroscience, neurosurgery and neurology program by U.S. News & World Reports Best Childrens Hospitals, BNI at Phoenix Childrens is eager to combine its deep bench of clinical talent with Medtronics cutting-edge innovation.

With our new technology deployed, we are thrilled to work with Phoenix Childrens and to support their mission of providing exceptional care for pediatric patients, said Dave Anderson, vice president and general manager, Enabling Technologies, which is part of the Restorative Therapies Group at Medtronic.

Phoenix Childrens Hospital Foundation received funding for the Stealth Autoguide from close community partners who support Phoenix Childrens in providing the best care by advancing pediatric medical solutions.

"We are extremely appreciative of the communitys support of the Stealth Autoguide, said Steve Schnall, senior vice president at Phoenix Children's Hospital Foundation. We are grateful to the Del E. Webb Foundation, Thunderbirds Charities, and WINGS, the womens auxiliary board of Phoenix Childrens, for investing in this state-of-the-art technology."

About Phoenix Childrens HospitalPhoenix Childrens Hospital is Arizonas only childrens hospital recognized by U.S. News & World Reports Best Childrens Hospitals with rankings in all ten specialties. Phoenix Children's provides world-class inpatient, outpatient, trauma, emergency and urgent care to children and families in Arizona and throughout the Southwest. As one of the largest childrens hospitals in the country, Phoenix Childrens provides care across more than 75 pediatric specialties. The Hospital is poised for continued growth in quality patient care, research and medical education. For more information about the hospital, visithttp://www.phoenixchildrens.org.

About Barrow Neurological Institute at Phoenix Children's HospitalBarrow Neurological Institute at Phoenix Children's Hospital heals children with neurological and mental health diseases and disorders so that they can have a happy and healthy quality of life by offering the most comprehensive inpatient and outpatient neurological care and services to infants, children and teens. Recognized by U.S. News & World Report as a leading Neuroscience Center for our collaborative and comprehensive approach to clinical medicine, Barrow at Phoenix Children's is largest pediatric neuroscience center in the Southwest. This is due in large part to BNIs commitment to education and research, along with the integration of pediatric neurosurgery, neurology, psychology, psychiatry, neurodevelopmental pediatrics and rehabilitation in the global care of children. Specialized medical equipment, pediatric patient rooms and pediatric specialists, in addition to a family-centered focus, make the institute and hospital uniquely qualified to treat complex neurological disorders in pediatric patients. For more information, visit:http://barrow.phoenixchildrens.org.

Story continues

About Stealth AutoguideTheMedtronic Stealth Autoguideis designed for accurate positioning of instruments to support a variety of neurological procedures. The technology can generally be used as a tool during stereoelectroencephalographies (sEEG), biopsies, and Visualase procedures. The Stealth Autoguide combines advanced software, navigation and instrumentation to enable accuracy during surgical procedures.

About MedtronicMedtronic plc (www.medtronic.com), headquartered in Dublin, Ireland, is among the worlds largest medical technology, services and solutions companies alleviating pain, restoring health and extending life for millions of people around the world. Medtronic employs more than 90,000 people worldwide, serving physicians, hospitals and patients in more than 150 countries. The company is focused on collaborating with stakeholders around the world to take healthcare Further, Together.

Any forward-looking statements are subject to risks and uncertainties such as those described in Medtronic's periodic reports on file with the Securities and Exchange Commission. Actual results may differ materially from anticipated results.

-end-

David T. YoungMedtronic plcPublic Relations+1-774-248-2746

Ryan WeispfenningMedtronic plcInvestor Relations+1-763-505-4626

Erica SturwoldPhoenix ChildrensMedia Relations+1-602-933-5871

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Phoenix Childrens Is the First-Ever Health System in the U.S to Use Medtronic Stealth Autoguide Cranial Robotic Guidance Platform for Neurosurgery -...

Nerve Stimulation Therapy Could Cut Fibromyalgia Pain – HealthDay

THURSDAY, Jan. 16, 2020 (HealthDay News) -- For people with the mysterious chronic pain condition fibromyalgia, researchers say nerve stimulation may offer some relief.

In a recent study, use of TENS -- transcutaneous electrical nerve stimulation -- during movement or activity was shown to significantly reduce pain associated with fibromyalgia after just four weeks.

Dr. Lesley Arnold, who was not involved with the new study, lauded its outcomes. "The improvements in pain and fatigue were remarkable," she said. Arnold, a professor of psychiatry and behavioral neuroscience at the University of Cincinnati College of Medicine, suggested that the study may help guide physicians in use of TENS for symptom management.

TENS uses a battery-operated machine to deliver electrical currents via electrodes attached to the skin. This is believed to activate nerve pathways that inhibit pain.

People with fibromyalgia are encouraged to engage in physical activity as a way to manage their symptoms. But, paradoxically, movement can be painful for them.

In the study, researchers randomly divided more than 300 women with fibromyalgia into three groups: active TENS; placebo (sham) TENS; or no TENS. Those in the TENS groups were instructed to use the device over four weeks, at home, for two hours daily during activity. The patients were told to apply the device's electrodes to two specific areas along the back -- one upper and one lower -- and to activate the machine at a modulated, or varying, frequency at the highest intensity bearable.

After four weeks, participants in the active-TENS group reported significant reductions in movement-and-resting pain and fatigue, especially compared to the no-TENS group.

Lead study author Kathleen Sluka, a University of Iowa research professor, said this study represents the culmination of years of animal research in which she and co-investigators experimented with variations in the use of TENS. Over time, the investigators learned that alternating between low and high frequency worked best to provide pain relief. So too did cranking up the machine to its highest intensity possible. "Strong, but not painful," she said.

While Sluka sees promise in the study's results, she was careful to note that TENS is not a cure for fibromyalgia.

"This is another tool that's not a drug that patients can use to manage their pain," one that may allow someone to reach for something other than ibuprofen or opioids as a first line of defense, Sluka said.

That TENS is readily available and comes with minimal risk also make it appealing. TENS devices can be bought over-the-counter at pharmacies for less than $50. As far as adverse effects, slight irritation at the site where the electrodes were applied was the primary adverse effect reported. And that is minor and easily adjustable, Sluka noted.

Superstar Lady Gaga shined a light on fibromyalgia when she canceled several concert tour dates in 2017 and 2018 due to reported flare-ups.

The condition is believed to affect about 10 million Americans, according to the National Fibromyalgia Association.

As researchers continue to look for safe and effective ways to alleviate the pain of fibromyalgia, much about it remains a mystery.

Diagnosis is usually based on a patient's medical history and exam; there's no blood test or other biomarker to identify it. And while its biological origins are unclear, most experts believe it is associated with alterations in the central nervous system that affect how the body responds to pain.

Management of the condition, which affects women disproportionately, has improved in recent years, said Arnold. It's being identified earlier; general awareness of the condition has improved; and, recently, a few medications have been approved by the U.S. Food and Drug Administration to manage it.

"But there's still a great deal of unmet need," Arnold said.

The report was recently published online in the journal Arthritis & Rheumatology.

More information

There's more on fibromyalgia at the National Fibromyalgia Association.

SOURCES: Kathleen Sluka, P.T., Ph.D., research professor, University of Iowa, Iowa City; Lesley Arnold, M.D., professor, psychiatry and behavioral neuroscience, University of Cincinnati College of Medicine; Nov. 18, 2019, Arthritis & Rheumatology, online

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Nerve Stimulation Therapy Could Cut Fibromyalgia Pain - HealthDay

Functional Ultrasound Advance Could Improve Brain Tumor Removal – Technology Networks

Researchers of CUBE (Center for Ultrasound Brain imaging @ Erasmus Medical Center) have managed to image live cerebral blood flow during awake brain surgery using functional Ultrasound (fUS). This cutting-edge technique could aid neurosurgeons in their effort to remove brain tumors without damaging surrounding functional brain tissue. Additionally, the researchers demonstrate that the technique can also pinpoint the healthy, functional areas in the brain during surgery. Their work was recently published in Frontiers in Neuroscience.

During tumor removal, it is essential to prevent damage to the surrounding healthy tissue. In brain tumors this might even be more critical, as damage to brain tissue may cause speech deficits, motor problems or worse. Using fUS, as implemented by CUBE, it is now possible to image both the vascular structure of a tumor as well as specific functional brain areas during awake brain surgery. Pieter Kruizinga and Sadaf Soloukey from CUBE: For the first time, we now have access to a technique with which we can image the living brain directly and with an unprecedented level of precision.

Functional Ultrasound (fUS) displays very small changes in blood flow. For tumor tissue, this means that we can see feeding vessels of the tumor in extreme detail, allowing for tumor delineation. For healthy tissue, this technique gives us immediate access to brain activity. During awake brain surgeries we could also ask our patients to perform tasks such as speaking and moving. By identifying exactly those areas where the blood flow follows the pattern of the functional task, we can determine whether or not that brain area is involved. As such, we can image the eloquent areas of the brain and inform the surgeon which brain areas to avoid.

Kruizinga and Soloukey were especially impressed by their language-related measurements: We asked our patients to think about words, which we could then display live in our ultrasound images. This means we can now actually image the thought of language using ultrasound. This observation has an impact that reaches even further than just the neurosurgical domain.

For the future, the CUBE-team has high hopes. Kruizinga and Soloukey: We are now in the middle of finalizing our last technical developments: we aim to make live 3D images in the OR, and compare them to conventional pre-operative MRIs. We are also working towards live and continuous imaging of the brain during the full length of the surgery, to give the neurosurgeon direct feedback on the procedure. The true breakthrough will be when we manage to image the brain without the need for skull removal, as is now still necessary. These types of challenges are especially ones we are good at solving at the Erasmus MC in Rotterdam.

Reference: Soloukey, S., Vincent, A. J. P. E., Satoer, D. D., Mastik, F., Smits, M., Dirven, C. M. F., Strydis, C., Bosch, J. G., van der Steen, A. F. W., De Zeeuw, C. I., Koekkoek, S. K. E., & Kruizinga, P. (2020). Functional Ultrasound (fUS) During Awake Brain Surgery: The Clinical Potential of Intra-Operative Functional and Vascular Brain Mapping. Frontiers in Neuroscience, 13. https://doi.org/10.3389/fnins.2019.01384

This article has been republished from materials provided by Erasmus Medical Center. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Functional Ultrasound Advance Could Improve Brain Tumor Removal - Technology Networks

Cerevel Therapeutics Initiates Phase 3 Program of Tavapadon for the Treatment of Parkinson’s Disease | Small Molecules | News Channels -…

DetailsCategory: Small MoleculesPublished on Wednesday, 15 January 2020 09:35Hits: 556

Studies to Enroll Approximately 1,200 Patients to Determine Effectiveness of Tavapadon Across the Full Spectrum of Early- and Late-Stage Parkinsons

BOSTON, MA, USA I January 14, 2020 ICerevel Therapeutics, a company dedicated to unraveling the mysteries of the brain to treat neuroscience diseases, today announced the initiation of its registration-directed Phase 3 program evaluating tavapadon in patients with Parkinsons disease. The company plans to conduct three 27-week trials designed to evaluate the efficacy, safety and tolerability of fixed doses (TEMPO-1) and flexible doses (TEMPO-2, TEMPO-3) of tavapadon as either monotherapy in patients with early-stage Parkinsons disease or as adjunctive therapy to levodopa in patients with late-stage Parkinsons disease who are experiencing motor fluctuations. A fourth 58-week, open-label, safety extension trial will also be conducted as part of the program.

Parkinsons disease affects approximately 10 million people worldwide, and there remains an important need for better and more effective therapies across the spectrum of this debilitating disease, said Raymond Sanchez, M.D., chief medical officer of Cerevel Therapeutics. We believe tavapadon has the potential to improve outcomes for patients with both early-stage and late-stage Parkinsons. It is our expectation that the innovative design of each of these Phase 3 trials will allow us to demonstrate tavapadons ability to improve patients motor symptoms and functioning. We anticipate data from these trials to be available beginning in the second half of 2022.

The three double-blind, randomized, placebo-controlled, parallel-group Phase 3 clinical trials will enroll patients ages 40 to 80 years with either early-stage Parkinsons disease (TEMPO-1, TEMPO-2) or patients with late-stage Parkinsons disease who are experiencing motor fluctuations on levodopa treatment (TEMPO-3). Approximately 1,200 patients will be enrolled across all three trials. The primary endpoint of the TEMPO-1 and TEMPO-2 trials is the change from baseline in the Movement Disorder Society-Unified Parkinsons Disease Rating Scale (MDS-UPDRS) Part II and Part III combined score. The primary endpoint of the TEMPO-3 trial is the change from baseline in total daily on time without troublesome dyskinesias.

In each of the three 27-week trials, participants will be randomized to tavapadon or placebo groups. In the TEMPO-1 trial, study participants will be titrated up to a fixed dose of either 5 mg once daily (QD) or 15 mg QD of tavapadon. In the TEMPO-2 and TEMPO-3 trials, participants will be titrated upward to a dose of between 5 mg and 15 mg QD in a flexible dosing paradigm.

The TEMPO-1 and TEMPO-2 trials have already initiated screening of patients, and the TEMPO-3 trial will begin screening later this year.

About Tavapadon Tavapadon is a potent, orally-bioavailable, selective partial agonist of the dopamine D1 and D5 receptors. This investigational therapeutic is being evaluated for the once-daily symptomatic treatment of Parkinsons disease.

About Parkinsons Disease Approximately 10 million people worldwide are living with Parkinsons disease, according to the Parkinsons Foundation. The disease is characterized by a progressive degeneration of dopaminergic neurons (the main source of dopamine) leading to a loss of critical motor and non-motor functions. Symptom severity and disease progression differ between individuals but typically include slowness of movement (bradykinesia), trembling in the extremities (tremors), stiffness (rigidity), cognitive or behavioral abnormalities, sleep disturbances and sensory dysfunction.1 There is no laboratory or blood test for Parkinsons disease, so a diagnosis is made based on clinical observation,2 which may contribute to an underestimation of the incidence of the disease.

About Cerevel Therapeutics Cerevel Therapeutics is dedicated to unraveling the mysteries of the brain to treat neuroscience diseases. The company seeks to unlock the science surrounding new treatment opportunities through understanding the neurocircuitry of neuroscience diseases and associated symptoms. Cerevel Therapeutics has a diversified pipeline comprising five clinical-stage investigational therapies and several preclinical compounds with the potential to treat a range of neuroscience diseases, including Parkinsons, epilepsy, schizophrenia and substance use disorder. Headquartered in Boston, Cerevel Therapeutics is advancing its current research and development programs while exploring new modalities through internal research efforts, external collaborations or potential acquisitions. For more information, visit http://www.cerevel.com.

1 J Neurol Neurosurg Psychiatry. 2008;79:368-376. doi:10.1136/jnnp.2007.131045. 2 Cold Spring Harb Perspect Med. 2012;2:a008870.

SOURCE: Cerevel Therapeutics

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Cerevel Therapeutics Initiates Phase 3 Program of Tavapadon for the Treatment of Parkinson's Disease | Small Molecules | News Channels -...

Surveying all the Proteins on a Neuron’s Surface – Howard Hughes Medical Institute

Scientists have found a new way to home in on the proteins covering a particular cells surface. The feat offers insight into how brain cells form intricate networks during development.

As if casting a tiny net, a new technique has rounded up all the proteins on the surface of neurons in the brains of fruit flies. The roundup uncovered 20 new molecules involved in wiring the developing brain.

The find furthers scientists understanding of how neurons in the brain form complex networks, researchers report January 16, 2020, in the journal Cell. And it demonstrates for the first time that this protein-finding method actually works in intact brain tissues not just cells grown in the lab, says study coauthor and Howard Hughes Medical Institute Investigator, Liqun Luo.

Thats important because the tissue environment is crucial for cells development, and lab cell cultures cant replicate it. Until now, scientists had no way to monitor all the proteins on cell surfaces in complex tissues like the brain. The new approach provides a way to survey this previously mysterious landscape.

What really blew me away was the biological follow-up, says biochemist Matthias Mann of the Max Planck Institute of Biochemistry. Luos team was able to find a trove of proteins whose biological role was previously unknown, says Mann, who was not involved with the work.

Cell surfaces are incredibly dynamic places, especially for cellular communication, says Luo, a neurobiologist at Stanford University. In the nervous system, proteins on the surfaces of nerve cells help the cells find each other and link up. Luos team wanted a complete view of the proteins that direct connections in the developing fly brain. The researchers focused on proteins involved in forming olfactory networks, which control a flys sense of smell.

Luo, along with his doctoral student Jiefu Li and collaborators at Stanford and the Broad Institute of MIT and Harvard, modified a method pioneered by study coauthor Alice Ting. In this method, called proximity labeling, researchers use an enzyme to add a molecular tag to a particular protein of interest, plus all the neighboring proteins. Researchers can then identify the tagged proteins using a chemical analysis called mass spectrometry.

Luos team added a new twist to the technique. They made the enzyme target proteins on fruit fly olfactory neurons at a particular point in brain development: when neurons are making decisions about which connections to form. The team compared the proteins present in adult cells with those present in the developing brain. The difference is actually very striking, Luo says.

The team identified 20 proteins that were more abundant on the surfaces of developing neurons and knocked them down one by one to see if their absence had an effect on brain wiring. Surprising even to the researchers, all 20 were involved in wiring the fly olfactory network. Whats more, many of the proteins they found hadnt even been known to play a role in neural development.

Luo and Li hope their approach will be useful for researchers in other fields as well. Li says it could be applied to immunology as well as to understanding how organs develop or modeling disease. For example, he adds, cell surface proteins are altered in cancer cells, so profiling those proteins could help scientists understand how cancer cells behave within tissues.

Id love to use [this technique], says Joshua Sanes, a neuroscientist at Harvard University who was not involved in the research. Like Luo, Sanes (who is a member of HHMIs Scientific Review Board) is interested in how neurons form the precise patterns of connections that lead to all the complex neural circuitry underlying behavior. But he studies the brains of mammals, not flies. So, first, Sanes says, the method will have to be optimized for mammalian cells a goal that has so far been elusive.

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Citation

Jiefu Li et al. Cell-Surface Proteomic Profiling in the Fly Brain Uncovers New Wiring Regulators. Cell. Published online January 16, 2020. doi: 10.1016/j.cell.2019.12.029

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Surveying all the Proteins on a Neuron's Surface - Howard Hughes Medical Institute

The CEO of Impossible Foods, the startup behind the wildly popular veggie burger backed by Serena Williams and Katy Perry, shares the biggest piece of…

captionImpossible Foods CEO Pat Brown holds up an Impossible Burger 2.0, the new and improved version of the companys plant-based vegan burger that tastes like real beef.sourceRobyn Beck/Getty Images

Impossible Foods CEO Pat Brown is on a mission to make meat obsolete, and it looks like hes off to a pretty promising start.

Impossible Foods wildly popular plant-based burger can now be found at thousands of restaurants across the United States, and the company is bringing a new faux-sausage breakfast sandwich to Burger King by the end of the month.

Plus, last spring, the company raised $300 million in a Series E round led by Temasek and Horizon Ventures, who were joined by more than a dozen superstar investors ranging from Serena Williams to pop icon Katy Perry and rapper Jay-Z, bringing its valuation to $2 billion.

Suffice it to say that Browns bleeding meatless burger has caught on. But Brown didnt necessarily have any of these milestones in mind before starting Impossible Foods. Rather, the key to starting a successful company has less to do with business-oriented goals like fundraising and retail partnerships and more to do with the problem youre aiming to solve, says Brown.

The main thing I would say to people who are entrepreneurial is, pick a problem that matters to the world, Brown, who is in his 60s, said to Business Insider when asked what advice he would give to his 20-year-old self. Really, that solves a big problem in the world, and dont talk yourself out of it.

Brown started Impossible Foods in 2011 when he was on sabbatical from his roles as a biochemistry professor at Stanford Universitys medical school and an HHMI investigator. But he says he wishes he had a better understanding of the meat industry earlier on in his career so that he could have started Impossible Foods sooner.

If I would have realized how catastrophic the use of animals in the food system was when I was in my 20s, instead of going into biomedical research, I would have gone right to working on this problem, he says.

Impossible Foods recently unveiled its first new foods since debuting the original Impossible Burger in 2016: Impossible Pork and Impossible Sausage. The company is testing a new Burger King breakfast sandwich that includes the meatless sausage at 139 locations in the US, but it has not said when Impossible Pork will be launching.

Impossible Foods decided to go with pork for its next major product expansion for two reasons: its the most widely eaten meat in the world, according to the Food and Agriculture Organization of the United Nations, and Impossible Foods hopes to cut back on the detrimental effects that pig farming can have on the environment.

Were not going to solve the problem by declaring war on the incumbent industry or telling people to change their diets, Brown said in a previous interview with Business Insider. The only way to do it is by making products that do a better job of delivering what consumers value from meat and these other foods.

All told, even if your company fails, youll at least know your efforts have gone toward a worthy cause if you choose to address a meaningful problem, Brown says.

If you think you have the capability of coming up with a useful solution to the problem, thats the big opportunity, he said. I feel like the world does not need more gadgets to collect data on everyone, Alexa-enabled toothbrushes or whatever. Do something actually useful.

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The CEO of Impossible Foods, the startup behind the wildly popular veggie burger backed by Serena Williams and Katy Perry, shares the biggest piece of...