Posts with «disease & medical conditions» label

England’s NHS will provide artificial pancreas to thousands of diabetes patients

England’s National Health Service (NHS) said on Tuesday that “tens of thousands of children and adults” with type 1 diabetes will receive an “artificial pancreas” to help manage their insulin levels. The hybrid closed loop system — a sensor under the skin that sends wireless readings to an externally worn pump, which delivers insulin as needed — can help patients avoid the risks of type 1 diabetes without worrying about finger sticks or injections.

This isn’t the first device of its kind. Tandem makes similar insulin pumps in the US after it received FDA authorization in 2019. Gizmodo notes that another company called iLet got FDA approval for a similar device last year. Although the NHS hasn’t said which specific device(s) its program will use, what’s different here is the nation’s publicly funded health care system providing them for free rather than as an exclusive privilege for the well-to-do. (Sigh.)

The hybrid closed loop system starts with a sensor implanted beneath the skin, which continually monitors glucose levels at regular intervals. The sensor sends that data wirelessly to a pump, worn externally, which delivers the proper insulin dosage. The “hybrid” part of its name comes from the fact that some user input, including entering carb intake, is still required in the otherwise self-regulating system.

The government agency gave an ultra-precise figure of 269,095 people in England living with type 1 diabetes, highlighting how many folks could potentially benefit from the rollout. The NHS says local branches will begin identifying patients for the program starting on Tuesday.

“Diabetes is a tough and relentless condition, but these systems make a significant, life-changing difference — improving both the overall health and quality of life for people with diabetes,” Colette Marshall, chief executive of Diabetes UK, wrote in the NHS’s press release announcing the rollout. “This really is a landmark moment and we’ll be working with the NHS and others to ensure a fair rollout that reaches people as quickly as possible.”

This article originally appeared on Engadget at https://www.engadget.com/englands-nhs-will-provide-artificial-pancreas-to-thousands-of-diabetes-patients-203236067.html?src=rss

Dr. Garmin will see you now

There’s a reason smartwatches haven’t replaced clinically-validated gear when you visit the hospital — accuracy and reliability are paramount when the data informs medical procedures. Even so, researchers are looking for ways in which these devices can be meaningfully used in a clinical setting. One project in the UK has explored if a Garmin Venu 2 and dedicated companion app could be used to free up doctors and nurses, six minutes at a time.

The Six Minute Walk Test (6MWT) is used to diagnose and monitor a number of cardiovascular maladies. This includes conditions like Pulmonary Hypertension that, if left untreated, are eventually fatal. “[The test has been] a cornerstone of hospital practice and clinical trials for decades all around the world as [...] a marker of how well the heart and lungs are working,” explained project leader Dr. Joseph Newman. While a change in a blood test marker might be clinically relevant, Newman said “it’s probably more important to someone that they can walk to the shop and back.” The test requires a patient walk on a flat, hard surface for six minutes straight, which stresses the heart enough to measure its capacity. A professional tests the patient’s heart rate and blood oxygen levels at the start and end, and while it’s simple and reliable, "it’s not perfect,” according to Newman. “This is why we’ve looked to change it in two important ways," he said, "can we make it shorter [...] and digitize it for remote use?"

After all, six minutes is a lifetime in a clinical setting, and patients dislike having to schlep all the way to their hospital just to walk up and down a corridor. It’s why Newman and Lucy Robertson — both researchers at the Royal Papworth Hospital in Cambridge — began looking for ways to revolutionize the test. They wanted to see if the test could be shortened to a single minute, and also if it could be carried out by a patient at home using a Venu 2. The watch was connected to a secure and dedicated clinical trial platform built by Aparito – a Wrexham-based developer – for testing. This was then sent out to patients who were instructed to wear the watch and walk outdoors to complete their own tests. “They’re asked to walk on flat, even, dry, relatively straight roads rather than in laps or circuits,” said Dr. Newman, with patients walking at their own natural pace.

“We carried out a product appraisal early on in the research process and were open-minded as to the brand or model,” said Newman. “Garmin came out on top for a few reasons; we can access raw data as well as Garmin’s algorithmically-derived variables,” he said. Because the research was being funded by a charity, the British Heart Foundation, the watch had to offer good value for money. It helped that Garmin, because of its existing health research division, gave the team “confidence in the accuracy of the sensors,” not to mention the fact that Aparito feels that “the Garmin SDK is relatively easy to work with,” he said. But while Garmin is in use right now, there’s no reason this setup couldn’t eventually work with a number of other brands. “As long as the technology works, it’s accurate, reliable and patients accept it, then we’re not tied to any brand.”

There are several benefits in giving patients the ability to run the tests at home: it’s more representative of the demands of their actual life, and patients can retake the test at regular intervals, making it easier to track that person’s health over time. “We can see real value in providing patients with pulmonary hypertension with an app and smartwatch to monitor their progress,”said Dr. Newman. “It’s unlikely to ever fully replace the need for in-person hospital reviews, but it will likely reduce their frequency.”

The results of the study right now suggest cutting the test to one minute has no detrimental effect on its outcome or accuracy,and that patients are far more likely to run the test regularly if they’re able to do so at home. “It’s likely that the upfront costs of wearables [to a hospital] may be offset by the longer term reduction in hospital visits,” said Newman. If that turns out to be right, then it means clinicians can better focus their time and efforts where their expertise is more valuable.

This article originally appeared on Engadget at https://www.engadget.com/dr-garmin-will-see-you-now-160013340.html?src=rss

Samsung gets FDA approval for a sleep apnea feature on Galaxy Watch

The Food and Drug Administration has given the green light to a sleep apnea detection feature on Galaxy Watch devices in the US, Samsung has revealed. The company notes this is the first approval of its kind in the US — South Korean officials previously rubberstamped the feature for use in that country.

Samsung plans to add the sleep apnea monitoring tool to compatible Galaxy Watch wearables in the third quarter of this year. It will be available via the Samsung Health Monitor app.

The feature allows those aged 22 and older who have not been diagnosed with the condition to check for signs of sleep apnea using their smartwatch and phone. It looks for signs of moderate to severe obstructive sleep apnea (OSA) over a two-night monitoring period. Users will need to track their sleep for more than four hours twice over a ten-day period to use the feature.

OSA is a common, chronic condition that affects around 25 percent of men and a tenth of women in the US, according to the National Sleep Foundation. Those with the condition tend to stop breathing while they sleep, which can reduce their sleep quality, disrupt oxygen supply and lead to more daytime tiredness. Left untreated, "sleep apnea can compound the risk of cardiovascular diseases such as hypertension, coronary artery disease, heart failure, cardiac arrhythmia and stroke," Samsung notes. The company added that the feature should help more people detect moderate and severe forms of the condition, and for them to seek medical advice when they do.

Other versions of the feature, which ties into Samsung's other efforts to help its customers have a good night's sleep, have popped up in devices elsewhere. In 2020, Withings added a sleep apnea detection feature to its sleep tracking mat.

This article originally appeared on Engadget at https://www.engadget.com/samsung-gets-fda-approval-for-a-sleep-apnea-feature-on-galaxy-watch-172856948.html?src=rss

Vibrating belt that treats low bone density gets FDA approval

The FDA has provided clearance for a medical device called Osteoboost, a vibrating belt that improves bone density in patients with osteopenia. The device, which was developed by California-based startup Bone Health Technologies and in part with NASA, is the first medical device of its kind to get regulatory approval as a treatment option for postmenopausal women.

One in two older women who have experienced menopause gets osteoporosis (the disease that comes after prolonged and untreated osteopenia), which is characterized by porous bones that can easily fracture. The Osteoboost belt is designed to prevent bone density from reaching that stage through early intervention. It works by mechanically stimulating the strength of the bones in the hips and spine of a wearer and prevents the further progression of bone density disintegration. The blueprint for the technology comes from NASA research that was investigating ways to prevent bone density from weakening in astronauts that work in mostly zero gravity environments where deterioration becomes a concern.

The belt should be worn for 30 minutes every day or at least five times a week for it to fully take effect. It delivers a gentle vibration that makes it easy to be worn pretty much anywhere or at any time, such as during dog walks or while washing dishes. During clinical trials, CT scans showed that following the integration of the belt into a patient’s care plan, bone density visually improved over time. In a study backed by the NIH, women aged 50 to 60 lost 3.4 percent of their bone density by the end of 12 months without any intervention, while patients who wore the belt lost only 0.5 percent of their bone strength.

Current standards of care for preventing osteoporosis during the osteopenia stage are mostly lifestyle suggestions that can be hard to adhere to, such as a well-balanced and calcium-rich diet, frequent weight-bearing exercises and reducing the risk of falls. “Although lifestyle interventions such as exercise and diet are beneficial to bone, the effect is small. The Osteoboost shows promise in slowing the loss of bone density and strength and may fill the treatment gap,” Laura Bilek, a researcher who has studied the belt’s effectiveness said.

Osteoboost is still not yet available for sale, but you can sign up to get notified when the device is released. A company representative said they will begin shipping later this year and will accept pre-orders in the next few months. While the price is also still not disclosed, the representative told Engadget that the belt will be “affordable and accessible to the millions of patients who need it.” To get the device, you will need a prescription from your doctor — so pricing may vary depending on insurers and co-pays. Bone Health Technology said it is currently in talks with insurers regarding coverage for the medical device. While the price projection could have drastically changed, three years ago the CEO Laura Yecies told NS Medical Devices she believed the device could debut for about $800.

This article originally appeared on Engadget at https://www.engadget.com/vibrating-belt-that-treats-low-bone-density-gets-fda-approval-181552362.html?src=rss

MIT experts develop AI models that can detect pancreatic cancer early

Researchers at MIT’s CSAIL division, which focuses on computer engineering and AI development, built two machine learning algorithms that can detect pancreatic cancer at a higher threshold than current diagnostic standards. The two models together formed to create the “PRISM” neural network. It is designed to specifically detect pancreatic ductal adenocarcinoma (PDAC), the most prevalent form of pancreatic cancer.

The current standard PDAC screening criteria catches about 10 percent of cases in patients examined by professionals. In comparison, MIT’s PRISM was able to identify PDAC cases 35 percent of the time.

While using AI in the field of diagnostics is not an entirely new feat, MIT’s PRISM stands out because of how it was developed. The neural network was programmed based on access to diverse sets of real electronic health records from health institutions across the US. It was fed the data of over 5 million patient’s electronic health records, which researchers from the team said “surpassed the scale” of information fed to an AI model in this particular area of research. “The model uses routine clinical and lab data to make its predictions, and the diversity of the US population is a significant advancement over other PDAC models, which are usually confined to specific geographic regions like a few healthcare centers in the US,” Kai Jia, MIT CSAIL PhD senior author of the paper said.

MIT’s PRISM project started over six years ago. The motivation behind developing an algorithm that can detect PDAC early has a lot to do with the fact that most patients get diagnosed in the later stages of the cancer’s development — specifically about eighty percent are diagnosed far too late.

The AI works by analyzing patient demographics, previous diagnoses, current and previous medications in care plans and lab results. Collectively, the model works to predict the probability of cancer by analyzing electronic health record data in tandem with things like a patient’s age and certain risk factors evident in their lifestyle. Still, PRISM is still only able to help diagnose as many patients at the rate the AI can reach the masses. At the moment, the technology is bound to MIT labs and select patients in the US. The logistical challenge of scaling the AI will involve feeding the algorithm more diverse data sets and perhaps even global health profiles to increase accessibility.

Nonetheless, this isn't MIT’s first stab at developing an AI model that can predict cancer risk. It notably developed a way to train models how to predict the risk of breast cancer among women using mammogram records. In that line of research, MIT experts confirmed, the more diverse the data sets, the better the AI gets at diagnosing cancers across diverse races and populations. The continued development of AI models that can predict cancer probability will not only improve outcomes for patients if malignancy is identified earlier, it will also lessen the workload of overworked medical professionals. The market for AI in diagnostics is so ripe for change that it is piquing the interest of big tech commercial companies like IBM, which attempted to create an AI program that can detect breast cancer a year in advance.

This article originally appeared on Engadget at https://www.engadget.com/mit-experts-develop-ai-models-that-can-detect-pancreatic-cancer-early-222505781.html?src=rss

2023 was a big year for CRISPR-based gene editing but challenges remain

2023 was an important year for patients with sickle cell disease. Prior to CRISPR, the only cure for the life-long ailment was a bone marrow transplant, which is notoriously dangerous and costly. This month, the FDA approved Vertex’s “Casgevy,” a CRISPR-based therapy for the treatment of sickle cell disease in patients 12 and older. The landmark approval made the therapeutic the first genetically edited therapy to reach the general market.

Casgevy, which also received the greenlight from regulators in the UK for another blood disorder called beta thalassemia, works by being administered in a single-infusion of genetically modified stem cells to a patient. Clinical study participants that took Casgevy were free from symptoms associated with sickle cell disease, like periodic episodes of extreme pain due to blocked blood flow through vessels, for up to a year.

CRISPR, which modifies precise regions of a human’s DNA strands, was once thought to be a far off scientific innovation. Human cells were first modified using CRISPR in clinical trials in China back in 2016. Less than a decade later, these landmark approvals have set the stage for future nods by regulators for other CRISPR-based therapies that can treat things like HIV, cancers and high blood pressure. “Gene therapy holds the promise of delivering more targeted and effective treatments,” Nicole Verdun, director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research said in a recent press release.

ASSOCIATED PRESS

CRISPR-based gene editing can be designed as a therapeutic for a number of diseases. A scientist can either delete, disrupt or insert segments of DNA to treat conditions by either targeting specific genes or engineering new cell therapies. The editing process can occur ex vivo (outside the body), in the same way Casgevy does, or in vivo (inside the body). Using CRISPR, sickle cell patients’ blood stem cells are modified in a lab before they are re-infused via a single-dose infusion as part of a hematopoietic transplant.

Neville Sanjana, a core faculty member at the New York Genome Center and associate professor in the Department of Biology at New York University, runs the Sanjana lab, which develops gene therapies for complex diseases like autism and cancer. “One of the really fundamental characteristics of CRISPR is its programmability,” Sanjana told Engadget. While working at the Zhang lab at the Broad Institute of MIT and Harvard, Sanjana says he helped design the “guide RNA” that became the blueprint for Vertex’s Casgevy. “CRISPR screens can be powerful tools for understanding any disease or genetic trait,” Sanjana said. Right now, he said biomedical folks are focused on applying CRISPR-based therapies for really serious inheritable diseases.

While it does “set a precedent” to have these first CRISPR-based gene therapies out there, it could also mean that regulators and the general public will regard future innovations in the space as “less novel,” Katie Hasson, a researcher with the Center for Genetics and Society (CGS) told Engadget. The CGS is a public interest and social justice organization that is focused on making sure gene editing is developed and distributed for good. Hasson explained, it doesn't mean that because one got approved that all other innovative therapies to come after it will not get as much scrutiny.

LIVE NOW: The media call on the approval of the first gene therapies to treat sickle cell disease is happening now!

Stream it here ➡️ https://t.co/oNR5uwEW44 pic.twitter.com/KlGNsNce5J

— U.S. FDA (@US_FDA) December 8, 2023

Beyond therapeutics, gene editing has very broad applications for the discovery and understanding of diseases. Scientists can use CRISPR to explore the origins of things like cancer and pave paths for therapeutics and incurable diagnoses, but that's not all there is to it. Scientists still need to conduct “considerable experimental research” when it comes to bringing an actual therapeutic to fruition, Sanjana said. “When we focus on therapeutic activity at a particular site in the genome, we need to make sure that there will not be any unintended consequences in other parts of the genome.”

Still, the spotlight will always shine a brighter light on the flashy developments of CRISPR from a therapeutic standpoint. Currently, a new gene editing method is being developed to target specific cells in a process called “cancer shredding“ for difficult-to-treat brain cancer. Scientists have even discovered a pathway to engineer bacteria to discover tumorous cells. However, there are barriers to using CRISPR in clinical practice due to the lack of “safe delivery systems to target the tissues and cells.”

“Maybe by curing one disease, you might give them a different disease — especially if you think of cancer. We call that a secondary malignancy,” Sanjana said. While there is strong reason for concern, one cure creating a pathway for other diseases or cancers is not unique to CRISPR. For example, CAR T cell therapy, which uses an entirely different approach to cell-based gene therapy and is not reflective of CRISPR, is a lifesaving cancer treatment that the FDA discovered can, in certain situations, cause cancer.

“We definitely don't want any unintended consequences. There are bits of the genome that if you edit them by mistake, it's probably no big deal but then there are other genes that are vitally important,” Sanjana said. Direct assessment of “off-target effects” or events in which a gene edit incorrectly edits another point on a DNA strand in vivo is challenging.

The FDA recommends that after a clinical trials’ period of investigatory study looking at the efficacy of a gene editing-based therapy, there needs to be a 15-year long term follow up after product administration. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said that the agency’s approval of Casgevy follows “rigorous evaluations of the scientific and clinical data.” Right now, researchers are focused on improving the precision and accuracy of gene editing and having the proper follow up is absolutely well merited, Sanjana explained. “The process right now is a careful one.”

Hasson believes that the 15-year recommendation is a good start. “I know that there is a big problem overall with pharmaceutical companies actually following through and doing those long term post-market studies.”

That’s where new approaches come into play. Base editing, a CRISPR-derived genome editing method that makes targeted changes to DNA sequences, has been around since 2016. Drugs that use base editing have already made headway in the scientific community. Verve Therapeutics developed a gene edited therapy that can lower cholesterol in patients with a single infusion. At higher doses, Verve said the treatment has the potential to reduce proteins associated with bad cholesterol for 2.5 years. Base editing, like CRISPR, has many potential applications for treatment and discovery. For example, base editing could repair a gene mutation that causes childhood blindness. Researchers at Weill Cornell Medicine also found base editing could help understand what genetic changes influence a patient’s response to cancer therapies.

Base editors use CRISPR to bring another functional element to a specific place in the genome. “But it doesn't matter whether it's CRISPR cutting or base editing… any time you're modifying DNA…you would want to know what the off target effects are and you can bet that the FDA wants to know that too. You're going to need to collect data using standard models like cell culture, or animal models to show there are zero or near zero off-target impacts,” Sanjana said.

CRISPR-based therapies already show high therapeutic potential for conditions beyond sickle cell disease. From blood based treatments, to edited allogeneic immune cells for cancers, there are a number of human clinical trials underway or expected to start next year. Trials for gene-edited therapies that target certain cells for cancer and autoimmune diseases are expected to begin in 2024.

Boston Globe via Getty Images

It won't be until 2025 before we get a better understanding of how Excision BioTherapeutics’ CRISPR-based therapy works to treat HIV. The application of gene editing as a therapeutic for Alzhiemer’s is still in the early stages, with mice at the forefront of research. Similarly, University College London researchers proved that CRISPR has promise as a potential therapeutic for treatment-resistant forms of childhood epilepsy. In a recent study, a gene edited therapy developed in the lab was shown to reduce seizures in mice.

But the clinical process of getting CRISPR to safely and effectively work as it's intended isn’t the only hurdle. The pricing of CRISPR and related therapies in general will be a huge barrier to access. The Innovative Genomics Institute (IGI), a research group that hopes to advance ethical use of these gene editing in medicine, estimates that the average CRISPR-based therapy can cost between $500,000 and $2 million per patient. The IGI has built out an “Affordability Task Force” to tackle the issue of expanding access to these novel therapies. Vertex’s sickle cell treatment costs a cool $2.2 million per treatment, before hospital costs. David Altshuler, the chief scientific officer at Vertex, told MIT Tech Review that wants to innovate the delivery of the therapeutic and make it more accessible to patients. “I think the goal will be achieved sooner by finding another modality, like a pill that can be distributed much more effectively,” Altshuler said.

“Access is a huge issue and it's a huge equity issue,” the CGS’ Hasson told Engadget. “I think we would also like to look at equity here even more broadly. It's not just about who gets access to the medication once it comes on the market but really how can we prioritize equity in the research that's leading to these treatments.” The US already does a poor job of providing equitable healthcare access as it is, Hasson explained, which is why it's important for organizations like CGS to pose roundtable discussions about implementing guardrails that value ethical considerations. “If you support people having access to healthcare, it should encompass these cutting edge treatments as well.”

This article originally appeared on Engadget at https://www.engadget.com/2023-was-a-big-year-for-crispr-based-gene-editing-but-challenges-remain-160009074.html?src=rss

CRISPR-based gene editing therapy approved by the FDA for the first time

In a landmark decision, the FDA greenlit two new drugs for the treatment of sickle cell disease in patients 12 and older, one of which —Vertex’s drug Casgevy — is the first approved use of genome editing technology CRISPR in the US. Bluebird Bio’s Lyfgenia also is a cell-based gene therapy, however, it uses a different gene modification technique to deliver tweaked stem cells to the patient.

Both approvals cultivate new pathways for the treatment of sickle cell disease, which is an inherited blood disorder that is characterized by red blood cells that can’t properly carry oxygen, which leads to painful vaso-occlusive crises (VOCs) and organ damage. The disease is particularly common among African Americans and, to a lesser extent, among Hispanic Americans. Bone marrow transplants are currently the only cure for sickle cell disease, but they require well-matched donors and often involve complications.

#Breaking: The U.S. FDA approves our treatment for #SickleCellDisease. We are excited to make this treatment available to patients. Learn more: https://t.co/9k3p4c7Kyv pic.twitter.com/c5yeqvvv6n

— Vertex Pharmaceuticals (@VertexPharma) December 8, 2023

While both drug approvals use gene editing techniques, Casgevy’s CRISPR/Cas9 genome editing works by cutting out or splicing in DNA in select areas. Patients first have blood drawn so that their own stem cells can be isolated and edited with CRISPR. They then undergo a form of chemotherapy to remove some bone marrow cells, so the edited stem cells can be transplanted back in a single infusion.

Both drug approvals are based on studies that evaluated the effectiveness and safety of the novel therapies in clinical patients. With Casgevy, study participants reported that they did not experience “severe VOCs” for at least 12 consecutive months during the 24-month follow-up. Similarly, patients on Lyfgenia did not experience a “pain crisis” for six to 18 months after the therapy.

The FDA's decision comes shortly after UK regulators, as well as the National Health Regulatory Authority in Bahrain both approved Vertex’s Casgevy. The approval for a CRISPR-based treatment creates opportunity for further innovation in the gene editing space — for treatments ranging from cancers to heart diseases to Alzheimer’s. “Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited,” Nicole Verdun, director of the Office of Therapeutic Products at the FDA’s Center for Biologics Evaluation and Research said. Casgevy is still currently under review by the European Medicines Agency.

This article originally appeared on Engadget at https://www.engadget.com/crispr-based-gene-editing-therapy-approved-by-the-fda-for-the-first-time-200726474.html?src=rss

UK authorizes first gene therapy for treating sickle cell disease

In a landmark decision, the UK’s Medicines and Healthcare products Agency (MHRA) approved the use of a gene-editing therapy called Casgevy for patients with sickle cell disease and beta thalassemia — both of which are hereditary disorders related to genetic mutations of the red blood cells. The treatment, manufactured by Vertex, is the first-ever approved therapy that utilizes CRISPR-based gene editing technology to treat eligible patients.

The UK approval of the novel therapy is informed by two previous global clinical trials that indicated the treatment's efficacy. 97 percent of patients using Casgevy were relieved of severe pain associated with the blood disorders for at least 12 months after treatment during the trials. The results suggest that the gene editing treatment could replace the current standard for care. Stem cell therapy and bone marrow transplants are currently the only pathways to cure sickle cell disease and beta thalassemia, however, they involve a lot of risks.

Both sickle cell disease and beta thalassemia are blood disorders characterized by defective red blood cells that can’t carry oxygen, and require patients to get monthly blood transfusions that can be costly and time-consuming. Casgevy works by specifically targeting the genes in the bone marrow stem cells that produce faulty blood cells. For the treatment to work, a patient’s stem cells need to be extracted from their bone marrow, edited in a lab and then re-infused into the patient.

#Breaking: The UK Medicines and Healthcare products Regulatory Agency (MHRA) approves our CRISPR/Cas9 gene-edited treatment for #SickleCellDisease and #BetaThalassemia in Great Britain—a historic scientific milestone. Learn more: https://t.co/hFk7wzThTQ pic.twitter.com/bMcVN5Ln5c

— Vertex Pharmaceuticals (@VertexPharma) November 16, 2023

Despite its promising outlook, CRISPR-based therapies may not be easily available to the general public. Gene editing is an expensive endeavor. The Innovative Genomics Institute (IGI) estimates that the average CRISPR-based therapy will cost between $500,000 and $2 million per patient. The IGI has built out an ‘Affordability Task Force’ to tackle the issue of expanding access to these novel therapies.

Aside from costliness, gene editing therapies offer huge promise to innovate treatment pathways for rare conditions including neurodegenerative diseases, cancer and muscular atrophy. More importantly, this landmark approval for Casgevy “opens the door for further applications of CRISPR therapies in the future,” Prof Dame Kay Davies, a scientist from the University of Oxford, said. And new iterations of gene editing technologies may even surpass CRISPR in the future.

Casgevy is still being reviewed by regulatory agencies for safety standards in other countries, including the United States and Saudi Arabia. A marketing application, the first step towards approval for the therapy, was recently validated by the European Medicines Agency.

This article originally appeared on Engadget at https://www.engadget.com/uk-authorizes-first-gene-therapy-for-treating-sickle-cell-disease-184130989.html?src=rss

Researchers developed a gene-editing technology that reduces 'bad' cholesterol

In a trial run by Verve Therapeutics, a Cambridge–based biotech company, researchers discovered that a single infusion of a gene-editing treatment called VERVE-101 was able to reduce cholesterol levels in patients. This treatment was tested in individuals with hereditary conditions that made them susceptible to developing clogged arteries and heart attacks. Scientists were able to use CRISPR editing techniques to tweak liver gene cells. The researchers “turned off” a cholesterol-raising gene called PCSK9, which is found in the liver, in order to lower LDL-C — sometimes called "bad" cholesterol — which causes plaque to build up in arteries in the first place.

PCSK9 was lowered by as much as 84 percent in the cohorts that received higher infusion rates of the treatment. At those higher treatment doses, Verve scientists said that the reduction of those LDL-C-related proteins lasted 2.5 years in previous studies on primates.

Verve Therapeutics

From a clinical standpoint, this gene editing therapy has the potential to disrupt the current standard treatment for high cholesterol. The current go-to's include prescription statins and PCSK9 inhibitors, but they require strict adherence and can have bad side effects like muscle pain and memory loss.

CRISPR, while seemingly miraculous, is a long way from replacing daily medications though. According to Nature, two of the 10 participants in the study suffered from a “cardiovascular event” that coincided with the infusion. Verve says one was not related to the treatment at all and the second was “potentially related to treatment due to proximity to dosing.” The use of a gene-editing technology will always carry some risk because the edits could occur elsewhere in the genome.

Before a single infusion therapy for high cholesterol can reach consumers, the FDA mandates that the treatment will need to be studied for up to 15 years. Verve recently received FDA clearance for an Investigational New Drug Application for VERVE-101, meaning that the company can begin to conduct trials in the US. The current trials in New Zealand and the United Kingdom will look for willing clinical trial participants to expand the study.

This article originally appeared on Engadget at https://www.engadget.com/researchers-developed-a-gene-editing-technology-that-reduces-bad-cholesterol-170040293.html?src=rss

Study: Wearable sensors more accurately track Parkinson’s disease progression than traditional observation

In a study from Oxford University, researchers found that by using a combination of wearable sensor data and machine learning algorithms the progression of Parkinson’s disease can be monitored more accurately than in traditional clinical observation. Monitoring movement data collected by sensor technology may not only improve predictions about disease progression but also allows for more precise diagnoses.

Parkinson’s disease is a neurological condition that affects motor control and movement. Although there is currently no cure, early intervention can help delay the progression of the disease in patients. Diagnosing and tracking the progression of Parkinson's disease currently involves a neurologist using the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) to assess the patient's motor symptoms by assigning scores to the performance of specific movements. However, because this is a subjective, human analysis, classification can be inaccurate.

In the Oxford study, 74 patients with Parkinson’s were monitored for disease progression over a period of 18 months. The participants wore wearables with sensors in different regions of the body: on the chest, at the base of the spine and on each wrist and foot. These sensors — which had gyroscopic and accelerometric capabilities — kept tabs on 122 different physiological measurements, and tracked the patients during walking and postural sway tests. Kinetic data was then analyzed by custom software programs using machine learning.

Oxford

The sensor data collected by the wearables were compared to standard MDS-UPDRS assessments, which are considered the gold standard in current practice. That traditional test, in this study's patients "did not capture any change" while the sensor-based analysis "detected a statistically significant progression of the motor symptoms" according to the researchers.

Having more precise data on the progression of Parkinson's isn't a cure, of course. But the incorporation of metrics from wearables could help researchers confirm the efficacy of novel treatment options.

This article originally appeared on Engadget at https://www.engadget.com/study-wearable-sensors-more-accurately-track-parkinsons-disease-progression-than-traditional-observation-171132495.html?src=rss