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Clinical Trial Begins for Liver Dialysis Device

Liver illustration

(Mikael Häggström, Wikimedia Commons)

24 July 2017. A clinical trial is underway in Europe testing a device that works like dialysis to clear toxins from the blood associated with liver failure, where a transplant is often the only option. The early-stage study, sponsored by Yaqrit Ltd., developer of the Dialive device, reported today enrollment of its first patient.

The Dialive device is designed for individuals with cirrhosis who encounter complications leading to a sequence of failures in the liver, a disorder known as acute-on-chronic liver failure. Yaqrit cites data from transplantation waiting lists showing between 200,000 and 300,000 people in Europe each year experience liver failure. About a quarter of those with liver failure are expected to die within 28 days without a transplant.

The condition results from bacteria in the gut that form toxins on their outer membranes called lipopolysaccharides. In healthy individuals, these toxins are cleared naturally, but in people with compromised livers the toxins build up, leading to inflammation, scarring, and loss of liver function. The Dialive filters a patient’s blood, removing lipopolysaccharides and exchanging damaged albumin, a prominent protein in blood serum, for healthy albumin.

The clinical trial is enrolling 24 individuals with acute-on-chronic liver failure at 7 locations in the U.K., France, Germany, and Spain. Participants are randomly assigned to receive either Dialive treatments for 10 days or the standard of care, which may vary depending on the severity of the patients’ condition. The study team is looking primarily at the safety of the Dialive device, in terms of serious adverse effects or the need to discontinue treatments because of the device.

However, the researchers are also evaluating performance of the Dialive, including removal of toxins and damaged albumin from the blood, and changes in liver functions compared to standard of care. In addition, the study team is assessing any changes in kidney, brain, and immune functions between the two groups of participants.

Yaqrit Ltd. is a spin-off enterprise from University College London, founded by Rajiv Jalan, professor of hepatology and part of the university’s health care engineering institute. The company, based in London, is commercializing research by Jalan and others, including the Dialive. Yaqrit is also creating a preventive and therapy for cirrhosis and nonalcoholic steatohepatitis, where fatty deposits build up in the liver. This treatment, called Carbalive, uses tiny packets of carbon beads delivered into the gut that bind to and remove lipopolysaccharides.

The clinical trial is part of a larger project funded by the European Commission called Aliver to develop the Dialive, and take the device through two clinical trials and regulatory approvals in Europe. If the first trial shows positive results, the second trial is expected to enroll 100 patients with liver failure at 18 sites in Europe.

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S&P 500 Tech Stocks Surpass Dot-Com Peak

22 July 2017. On Wednesday, Standard & Poor’s index of 500 stocks in the information technology industry closed at 992.29, its highest point since March 2000, at the height of the dot-com boom, when the index reached 988.49. Investment analysts attribute recent gains to better-than-expected profits for a number of companies in the technology sector.

Yesterday, the index closed down slightly at 991.97, which still represents an increase of more than 31 percent in the past year. The following chart is courtesy of Statista.

Infographic: S&P 500 Tech Stocks Surpass Dot-Com Peak | Statista You will find more statistics at Statista

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Calif. Agency Supporting Late Stem Cell Trial for ALS

Brain synapses illustration

(AllanAjifo, Wikimedia Commons)

21 July 2017. California’s agency supporting stem cell research for regenerative medicine is helping fund a clinical trial testing stem cell treatments for amyotrophic lateral sclerosis, or ALS. The trial is planned by Brainstorm Cell Therapeutics in Petach Tikva, Israel supported by a $15.9 million grant from California Institute for Regenerative Medicine.

ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder where neurons or nerve cells controlling muscles in the body begin to waste away, and can no longer send or receive signals from the brain or spinal cord. As the nerve cells stop functioning, the muscles in the limbs, and later speech and breathing muscles, begin weakening and eventually stop functioning. Most people with the disease die of respiratory failure.

Brainstorm’s NurOwn technology, licensed from Tel Aviv University, extracts stem cells from the patient’s bone marrow that are transformed into cells supporting development of nerve cells. These transformed stem cells, says the company, secrete proteins called neurotrophic factors that protect nerve cells, as well as encourage their growth and interactions with muscles. Because the original cells come from the patient, they have little risk of rejection by the immune system.

Brainstorm says the late-stage trial is in advanced planning stages, but will enroll some 200 individuals conducted a 6 sites in the U.S. The main efficacy measure is expected to be a standard ALS Functional Rating Scale that evaluates patients on their ability to perform various physiological functions, such as salivating and swallowing, and everyday activities like walking, handwriting, and cutting food.

Some of the trial’s participants are expected to be individuals with faster-progressing ALS who took part in an intermediate-stage study of NurOwn stem cell treatments, with results published in July 2016. As reported in Science & Enterprise, the results overall showed patients receiving NurOwn stem cells experienced smaller declines in function over 24 weeks from before the start of treatments, compared to other patients receiving a placebo. Participants in that trial with faster-progressing ALS reported higher functional rating scale percentages at each testing point, than patients receiving the placebo.

California Institute for Regenerative Medicine, or CIRM, funds research at institutions and companies in the state developing stem cell treatments for patients with unmet medical needs. The agency also supports studies by companies or institutions from outside California that conduct at least part of their research in the state. This suggests one or more of Brainstorm’s sites will be in California.

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Robot Designed to Grow into Shapes, Spaces

Robotic tube

Robotic tube extending through glued surfaces (Stanford University)

21 July 2017. Engineering researchers created a new type of robot that grows to extend its form into spaces like a vine, rather than move to different locations. A prototype growing robot, designed by a team from University of California in Santa Barbara and Stanford University, is described in the 19 July issue of the journal Science Robotics.

The system, known as a vinebot, is the product of the Collaborative Haptics and Robotics in Medicine lab led by mechanical engineering professor Allison Okamura. The researchers are seeking better ways for robotic devices to travel and navigate unfamiliar terrains and tight spaces while responding to both external conditions and operator controls. The team was particularly interested in alternatives to locomotion, which often requires moving parts and sometimes power supplies.

Okamura and colleagues used growing vines and nerve cells as models, which grow their length rather than move from one location to another. The team employs soft materials in a tube that extends its length through the process of eversion, or turning inside out like a pants pocket. The system is powered with air pressure, but water pressure could also be used. Onboard sensors and a camera help steer the tube, with a small bulb to light the way.

The vinebot tube, using thin inexpensive polyethylene materials, extends from a base equipped with an air compressor. A spool of material feeds the tube as it grows, controlled by a winching motor. The tube extends in segments of about 2 centimeters, with each segment controlled by an adhesive latch that remains closed. When the tube is pressurized, and the latch for the segment is at the leading tip of the tube, the latch opens allowing for the tube to extend.

The researchers used natural models such as fungi and pollen tubes for designing vinebot’s navigation. The tube has small control chambers along the sides that inflate to change the tube’s growing direction. To make a left turn, for example, the right-side control chamber inflates, lengthening that side of the tube, and moving the tip of the tube to the left. The team notes that no more air pressure or separate motors are needed to turn the tube, keeping the design simple. The tube also is able to to carry items for delivery. The researchers wrote software to control the vinebot, capturing images from the camera in the tip, with algorithms that process the images and alter the course of the tube almost instantaneously.

The team tested vinebot tubes over various challenging surfaces, including sticky surfaces like flypaper and glue, as well as nails. The nails could puncture the tube along its length, but the tube extends only from the tip, so as long as air pressure can reach the leading section, the tube continues to grow. Other tests squeezed the tube under a door about 10 percent of the tube’s diameter, and fit under and lifted a crate weighing 100 kilograms (220 lbs.).

Further tests sent the tube through the space above a dropped ceiling to demonstrate its ability to navigate around unfamiliar obstacles, while towing a cable. And in a separate demonstration, the team directed vinebot to extend up in the air to form a free-standing framework for sending out a radio signal.

This first prototype, says Okamura in a Stanford statement, is designed as much to understand the capabilities of the technology as much as build a working system. “Essentially, we’re trying to understand the fundamentals of this new approach to getting mobility or movement out of a mechanism. It’s very, very different from the way that animals or people get around the world.”

Okamura and first author Elliot Hawkes filed a provisional patent application for the technology. In addition to public safety applications, such as search and rescue, and inspection functions, the researchers believe the technology can also be applied to medical devices. One version of the device is as small as 1.8 millimeters, with potential applications in diagnostics and drug delivery.

In the following video, Okamura, Hawkes, and colleagues tell more about the vinebot.

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Start-Up Building Mobile Apps Combining DNA, Lifestyle

DNA in hand

(Gerd Altmann, Pixabay)

20 July 2017. A new enterprise in California is creating smartphone apps that provide lifestyle advice based on DNA analysis and activity tracking. The first app from Exploragen, in Rancho Santa Margarita, California offers customized guidance for improving one’s sleep that combines assessments of DNA and activity data related to sleep.

Exploragen says it plans to build mobile apps with algorithms derived from published scientific findings connecting genomics to physiological traits. The algorithms and apps will build in data on lifestyle factors provided by the users with features on the mobile device or entered on the screen.

The company’s first app, known as SlumberType, uses the DNA analysis to establish connections between one’s genetic code and basic sleep behavior, such as the amount of time it takes to fall asleep and length of time sleeping, as well as finding the most productive parts of the day. SlumberType also tracks activities that influence sleep such as caffeine and alcohol consumption, eating times, exercise, use of computers and phones in the evening, and yoga or meditation.

The analysis provided by SlumberType, says the company, will provide insights for the users into their sleep patterns based on relationships between DNA and these factors, such as metabolism of caffeine, as well as broader insights into the individual’s personality and how the person performs or manages tasks. The app, being written for the iPhone, also allows for personalized alarms and schedules, based on its analysis, to maximize an individual’s productivity reflecting insights derived from the app. No delivery date has yet been given for SlumberType.

The DNA analysis is provided by Helix, a company that performs personal genetic assessments from saliva samples sent in by customers. Helix stores the DNA analysis, then makes it available to partner enterprises, such as Exploragen, for further examination or incorporation with other data. In addition to Exploragen, Helix collaborates with personalized health units at medical centers including Duke University, Mayo Clinic, and Mount Sinai. Helix also partners with National Geographic for ancestry analysis, prenatal genetic testing, and even an app to help select the right wines.

Exploragen was founded last year by Ron Andrews, a veteran of life science technology companies Thermo Fisher Scientific, Life Technologies, Clarient, and Roche Molecular Diagnostics. Andrews is the company’s CEO, which in this case means chief exploration officer. Former board-certified genetic counselors Shannon Kieran and Sara Riordan are also company co-founders, now serving as heads of operations and science respectively.

“Consumers want to know how their body is functioning for optimal performance, and DNA is an important indicator of the body’s predispositions,” says Andrews in a company statement. “As people continue to gain access to their DNA, there will be more ways for them to make personal lifestyle choices based on what they discover.”

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Stem Cell Clinics Found Marketing in ClinicalTrials.gov

Stem cell clinic map

Map of continental U.S. with locations of stem cell clinics. Blue stars indicate hot-spot concentrations. Click on image to view full size. (Leigh Turner and Paul Knoepfler)

20 July 2017. A review of entries in  the U.S. government’s clinical trial registry shows commercial stem cell clinics are using the respected database to promote their services. The findings were revealed in a perspective article appearing in yesterday’s issue of the journal Regenerative Medicine (free registration required).

The article, authored by bioethics professor Leigh Turner at University of Minnesota in Minneapolis, tells how for-profit clinics marketing stem cell therapies for diseases such as diabetes and osteoarthritis register their services on ClinicalTrials.gov, a U.S. government listing of clinical studies. ClinicalTrials.gov is offered by National Institutes of Health as a service for patients, physicians, and researchers, and perhaps the leading authoritative database for current and past clinical studies.

As reported by Science & Enterprise in July 2016, Turner and stem cell researcher Paul Knoepfler at University of California in Davis found nearly 600 clinics in the U.S. claiming to offer stem cell treatments, many having dubious scientific or medical validity. Their analysis shows the clinics often provide treatments for orthopedic and pain disorders, but also for anti-aging and cosmetic applications, including face lifts and breast augmentation, as well as more serious conditions such as spinal cord injuries, and immunological, cardiac, pulmonary, vision, and urological diseases.

Turner says some of these clinics list their services on ClinicalTrials.gov under the guise of clinical studies, using terms such as “patient-sponsored” or “patient-funded” to indicate that people need to pay for participation. Legitimate clinical trials actively recruit participants, with taking part in the studies made free of charge. Individuals participating in clinical trials often need to pay for their own travel and accommodations to the trial sites.

His search through ClinicalTrials.gov uncovered 7 entries of patient-sponsored trials at 2 clinics in Florida and Texas using autologous, or a patient’s own, stem cells to treat type 2 diabetes, osteoarthritis, erectile dysfunction, critical limb ischemia — a serious form of peripheral artery disease — and chronic obstructive pulmonary disease, or COPD.

The author then matched the list of commercial stem cell clinics found in his article published in July 2016 to entries in ClinicalTrials.gov and found 5 of these companies with 11 entries in the registry. Because the trials are conducted by for-profit clinics, Turner suggests participation in the trials requires patients to pay. While the entries do not explicitly say individuals need to pay for participation, one company links its entries to an essay titled, “The Role of Patient Funded Clinical Research in Advancing Medical Care.”

Turner believes including pay-to-participate trials at clinics offering services with dubious medical or scientific value in ClinicalTrials.gov hurts the credibility of the service. Part of the responsibility lies with FDA that does not regulate autologous stem cell treatments, which largely shift cells and tissue from one part of the body to another, such as in cosmetic procedures. However, FDA issued draft guidelines for regulating biologic products such as those based on stem cells in October 2015, suggesting the agency will require its review of treatments going beyond cosmetic procedures.

The author also notes that FDA also does not permit charging patients to take part in clinical trials required for new drug applications, and charging fees to participants in trials can take place only under limited conditions. Turner suggests NIH that offers ClinicalTrials.gov coordinate its listings with FDA to make sure studies charging patients first have clearance from FDA.

Also, says Turner, NIH needs to better police the listings on ClinicalTrials.gov to find pay-to-participate entries. The site now includes a disclaimer, “Listing of a study on this site does not reflect endorsement by the National Institutes of Health.” NIH, adds Turner, needs to go well beyond that disclaimer to maintain the registry’s value.

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Patent Awarded for Exosome Capture Process

U.S. Patent and Trademark Office

U.S. Patent and Trademark Office in Alexandria, Virginia (A. Kotok)

19 July 2017. A company making blood filtering devices was awarded a patent for a process to capture tiny particles carrying proteins that suppress immune responses. The U.S. Patent and Trademark Office yesterday awarded patent number 9,707,333 to two inventors and assigned the patent to Aethlon Medical Inc. in San Diego.

Aethlon Medical develops blood filtering devices that remove viruses and toxins from the blood stream that interfere with immune system responses to diseases, including cancer. Among the targets for Aethlon Medical devices are exosomes, tiny — 40 to 150 nanometer — lipid-membrane containers in cells that gather up and secrete cytoplasm, the gel-like material outside the cell nucleus. While originally believed to carry out waste removal and other maintenance tasks, exosomes were shown in recent years to perform useful delivery functions carrying proteins and genetic material to other cells, and drawing increased attention from a range of biological disciplines.

The patent covers the company’s methods for capturing exosomes in whole blood and components, such as serum or plasma, in a iltering system outside the body. The exosomes in this case contain MHC-I or MHC-II antigens, proteins associated with suppressing the body’s immune responses to tumor cells, thus allowing the cancer to grow and spread. MHC-I and MHC-II antigens are also linked to autoimmune and neurodegenerative disorders.

The technology described in the patent includes a filtering device aided by antibodies with an affinity for MHC-I and MHC-II antigens. The process also uses absorbent materials to capture and remove exosomes, while allowing blood to flow through the device unimpeded. In addition, the system returns the filtered blood or components to the patient.

Exosome capture is a major focus for Aethlon Medical. A subsidiary of the company, Exosome Sciences, develops diagnostics for disease that process exosomes containing biomarkers, or molecular indicators of specific diseases. The lead product for Exosome Sciences is a test in development for chronic traumatic encephalopathy or CTE, a disease that develops in athletes playing collision sports, such as American football and rugby, as well as military veterans encountering repeated head impacts. The disease is associated with progressive degeneration of brain tissue, as well as a build up of abnormal protein deposits known as tau.

As reported in Science & Enterprise, Exosome Sciences is conducting a clinical trial of its blood test, recruiting former players from the National Football League as participants. James Joyce, the founder of Aethlon Medical and Exosome Sciences is a former NFL player, whose teammate in college died at age 45 from a drug overdose attributed to CTE.

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Start-Up Offering Precise Heart Rhythm Diagnostic

Heart in rib cage illustration

(CIRM.gov)

19 July 2017. A one year-old company in New York City is developing a device that assesses the severity of heart rhythm problems to prescribe a more precise course of treatment. Cardea Sciences, a spin-off enterprise from Mount Sinai medical school, received the a top prize in last month’s NYC Life Science Innovation Showcase.

Cardea Sciences was founded by Ya-El Mandel-Portnoy, a research fellow in emergency medicine at Mount Sinai, to commercialize a technology that more accurately evaluates atrial fibrillation, and identifies patients in the most danger of further heart damage. Atrial fibrillation is an irregular heartbeat or arrhythmia that can lead to stroke or heart failure, and affects some 2.7 million Americans, according to American Heart Association. With atrial fibrillation, heart muscle contractions in the upper chambers beat irregularly instead of in a regular rhythm, which can cause blood to pool and lead to blood clots, including those that move to the brain and cause a stroke.

The device being developed by Cardea Sciences is a bed-side heart monitor that measures differences in heart rate at various points in the body. These variations are called a pulse deficit, which occurs when the heart contracts inefficiently, such as in atrial fibrillation, with pulse waves not transmitted to peripheral sites in the body. The company says this more precise evaluation, if done early in diagnosis of atrial fibrillation or AFib, can highlight individuals at highest risk of of further heart damage, and spare people with a less dangerous outlook from unnecessary care.

“Cardea Sciences’ focus,” says Mandel-Portnoy in a Mount Sinai statement, “is on patient outcomes, empowering patients and clinicians to manage the disease by identifying those AFib patients who will not tolerate the arrhythmia well and will suffer from severe adverse events. This will help care providers optimize their course of treatment and reduce the number of readmissions, thus leading to a reduction in health care costs.”

At the NYC Life Science Innovation Showcase, Mandel-Portnoy received an award that provides lab and office space for Cardea Sciences at Alexandria LaunchLabs, a life sciences incubator in New York. Alexandria LaunchLabs provides health care and life science start-ups with mentoring, shared equipment, and seed-level financing, as well as office and lab facilities.

Cardea Sciences is currently part of E-Lab NYC, a program that provides business training and networking to scientists and engineers in the life sciences seeking to become entrepreneurs. Mandel-Portnoy became an E-Lab NYC fellow in December 2015.

In the following video, Mandel-Portnoy tells more about Cardea Sciences in a recent pitch competition.

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Biotech Licensing Viral T-Cell Immunotherapies

Human T-cell lymphocyte

Scanning electron micrograph of a human T-cell lymphocyte (National Institute of Allergy and Infectious Diseases, NIH)

18 July 2017. A company making treatments for viral infections that stimulate natural T-cells in the immune system is licensing a related technology from Baylor College of Medicine. While the agreement calls for initial and progress payments to Baylor from ViraCyte LLC in Houston over a number of years, further financial details including dollar amounts were not disclosed.

ViraCyte is a developer of synthetic virus-specific T-cells designed to stimulate immune responses fighting viral infections in people with compromised immune systems. Current anti-viral medications, says the company, have limited effectiveness in individuals without a robust immune system. ViraCyte notes that in early-stage clinical trials, more than 90 percent of participants in whom conventional treatments failed, respond to these anti-viral therapies, including elimination of the infections.

The company is currently testing two types of anti-viral treatments. Its Viralym line of therapies are derived from T-cell donors, from which are extracted immune stimulant proteins and benign virus fragments. ViraCyte says a small panel of donors can create Viralym anti-viral infection treatments for 95 percent of people with compromised immune systems from receiving bone marrow stem cell transplants. The company’s Prelym line is designed to prevent up to 5 viral infections, and derived from a patient’s specific T-cell donor.

The agreement with Baylor, also in Houston, gives ViraCyte an exclusive global license to a technology behind a collection of immunotherapies that target viruses by proteins on the cell surface called T-cell receptors. These proteins activate the immune response in T-cells to specific antigens. The licensed technology is expected to support ViraCyte’s infection therapies and preventive treatments.

Under the deal, ViraCyte is paying Baylor an initial licensing fee, with the university eligible for future maintenance fees, additional clinical and regulatory milestone payments, and royalties on net sales. Dollar amounts for these parts or the overall agreement were not disclosed.

The licensed technology is expected to help ViraCyte advance its lead product, Viralym-M, into later-stage clinical trials among children and adults with viral infections after receiving stem cell transplants.

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Plant-Based Diet Quality Key to Reducing Heart Disease

Basil, olive oil, tomatoes

(Conger Design, Pixabay)

18 July 2017. A review of health surveys going back to the 1980s reveals individuals consuming healthier plant-based diets are less likely to encounter heart disease than people eating less-healthy foods from plants or animals. The findings from the analysis appear online in yesterday’s issue of the Journal of the American College of Cardiology (paid subscription required).

A team led by postdoctoral researcher Ambika Satija at Harvard University’s school of public health sought to find evidence of beneficial effects from a diet of plant-based food with more whole grains and fresh produce, compared to diets with refined grains, potatoes, and sweetened drinks also derived from plants. In this paper, the researchers looked specifically for associations between quality of plant-based diets and heart disease.

The team created a numerical index where healthier foods derived from plants — whole grains, fresh fruits and vegetables, nuts and legumes, oils, tea and coffee — received positive scores, while less healthy foods derived from plants and animals received negative ratings. The researchers also devised an unhealthy plant-based food index, where refined grains, potatoes, sweetened desserts, and sugary drinks received positive scores and healthier foods derived from plants received negative scores.

Satija and colleagues applied their indexes to data from surveys of people working in health care. The surveys — Nurses’ Health Study, Nurses’ Health Study 2, and Health Professionals Follow-up Study — are administered to volunteers in the health professions going back to the 1970s. Participants in the surveys answer comprehensive health questions, then take follow-up inquiries every 2 years including items on lifestyle, health behaviors, and medical history. The team sampled between 43,000 to more than 92,000 records from each of the 3 surveys, beginning in the mid-1980s, who at the beginning were free of chronic diseases.

The samples represent more than 4.8 million person-years in the follow-up surveys. Those results uncovered 8,631 cases of coronary heart disease, a common heart disorder, where waxy plaques build up in blood vessels including the coronary arteries feeding heart muscles. The plaques narrow the arteries and can break away causing blood clots that slow or stop the flow of blood to the heart. The condition can lead to heart failure or heart rhythm problems.

The researchers found people in the surveys eating a healthier plant-based diet were less likely to suffer from coronary heart disease, while people consuming less-healthy food from plants were more likely to encounter heart disease. While the findings indicate an association and not necessarily a direct cause, the authors note that the correlation between a healthy plant-based diet and the absence of coronary heart disease is particularly strong.

An accompanying editorial in the same journal issue, published by American College of Cardiology, says that not all plant-based diets are equally healthy and but plant-based diets with whole grains, unsaturated fats and plenty of fruits and vegetables deserve more dietary recommendations.

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