4 September 2015. A new challenge on InnoCentive is asking the public for a design of an efficient yet simple incinerator of medical waste generated during humanitarian emergencies. The competition has a total potential payout of $30,000 and a deadline for submissions of 4 November 2015.
InnoCentive in Waltham, Massachusetts conducts open-innovation, crowdsourcing competitions for corporate and organization sponsors, in this case Humanitarian Innovation Fund. Free registration is required to see details of the competition.
Humanitarian Innovation Fund sponsoring the challenge supports organizations seeking to make the delivery of humanitarian aid more effective and cost-efficient. For this competition, the fund is tackling the problem of medical waste that piles up in humanitarian emergencies, such as floods and armed conflicts, particularly in low-resource countries.
Without a means of effectively destroying medical waste, affected populations face more exposure to infectious diseases and fouling of air, ground, and drinking water. In many cases, medical workers in the field during emergencies burn the waste in empty fuel drums, but they rarely work well and can cause problems with pollution. More durable medical waste incinerators can be brought in, but they are often expensive and need skilled staff to set-up and operate.
The sponsors are calling for a medical waste incinerator design that is simple, efficient, and durable. The device should be economical to operate in low-resource regions, easy to transport, and be assembled with local skills and materials. In addition, the incinerator must meet specific fuel and temperature requirements.
InnoCentive calls this type of competition a theoretical challenge requiring a written proposal that fleshes out an idea before it becomes a proven concept. Proposals submitted in a theoretical challenge usually offer detailed descriptions and specifications showing how the ideas would work. Humanitarian Innovation Fund indicates it expects written proposals to run about 10 pages.
Proposals will be reviewed by a panel of experts from humanitarian aid organizations such as Oxfam and Médecins Sans Frontières (Doctors Without Borders). The sponsor says it plans to award three prizes of $5,000 each to the top ideas generated in the competition. A separate bonus prize of $15,000 will also be awarded to a winning proposal that can be immediately turned into a working prototype device.
Humanitarian Innovation Fund asks competitors receiving awards to grant a non-exclusive license to their intellectual property. Sub-licensing of intellectual property is possible for further development or manufacturing of the solution.
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(Public Domain Pictures/Pixabay)
4 September 2015. A synthetic antibody designed to prevent bleeding episodes among people with the most common type of hemophilia received a breakthrough therapy designation from the U.S. Food and Drug Administration. The pharmaceutical company Roche says FDA assigned the designation its biologic therapy code-named ACE910, now being tested in clinical trials.
Hemophilia is an inherited disorder, where the proteins needed to mix with platelets to allow blood to coagulate are missing. About 8 in 10 people with the condition have hemophilia type A, where the protein clotting factor 8 is missing. In type B, clotting factor 9 is missing.
ACE910 is an engineered, humanized antibody that simulates some of the functions of clotting factor 8, given once a week by injections under the skin. While ACE910 acts like factor 8, Roche says its structure does not generate reactions leading to formation of factor 8 inhibitors, a problem with traditional factor 8 replacement therapies. Formation of factor 8 inhibitors is a serious complication for some people with hemophilia, which makes control of their bleeding more difficult.
FDA assigns a breakthrough designation to therapies that address a serious condition and demonstrate through clinical evidence that the treatment is a substantial improvement over current methods. Once designated as a breakthrough therapy, a drug or biologic can receive early and frequent communications with FDA staff, quick resolution of questions, intensive guidance on drug development, and an organizational commitment from top managers.
The breakthrough designation was given to ACE910 as a preventive drug for people with hemophilia A and factor 8 inhibitors, based on an early-stage clinical trial with a small sample of individuals in Japan having severe cases of hemophilia A. The most recent report from the trial, given at a meeting of the International Society of Thrombosis and Hemostasis in June, shows ACE910 completely controlled bleeding in half of the participants (9 of 18) while receiving the therapy. Adverse effects among participants were considered mild or moderate, largely due to injection site reactions.
ACE910 was originally developed by Chugai Pharmaceutical Co. in Tokyo, and later licensed to Roche, which is now co-developing the therapy with Chugai. A late-stage clinical trial is planned to begin later this year among individuals with hemophilia A and factor 8 inhibitors. A separate trial with people having hemophilia A without factor 8 inhibitors is planned for 2016.
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Acute myeloid leukemia cells (Cancer Genome Atlas, NIH)
3 September 2015. Ontario Institute for Cancer Research and Structural Genomics Consortium are making an early-stage drug prototype freely available to the biomedical research community to further define its therapeutic capabilities. Financial and intellectual property aspects of the offer were not immediately disclosed.
The two research labs — both in Toronto, Ontario, Canada — jointly developed a small molecule compound that in lab tests already shows promise in blocking growth of breast cancer cells and some types of acute myeloid leukemia. The molecule, code-named OICR-9429, targets and stops activity of WD repeat-containing protein 5 or WDR5 protein associated with a number of regulatory processes in the body affecting cell growth.
Developers of OICR-9429 already collaborated with labs in the U.S. and Austria to find targets for the therapeutic molecule. In a study published yesterday in the journal Nature (paid subscription required), a lab at University of Pennsylvania in Philadelphia discovered OICR-9429 can counteract mutations of the TP53 gene associated with a wide range of cancer types, and in the study sharply reducing proliferation of breast cancer cells.
In a study published on 20 August in Nature Chemical Biology (paid subscription required), a team led by researchers at CeMM Research Center for Molecular Medicine in Vienna, Austria applied OICR-9429 to mutations of the CEBPA gene associated with about 9 percent of cases of acute myeloid leukemia that expresses a protein that interacts with WDR5. In lab tests, the researchers found OICR-9429 inhibited growth of acute myeloid leukemia cells expressing the mutation-associated protein.
Developers of OICR-9429 want to see more of these studies and quickly, which is the reason for making OICR-9429 available to fellow researchers. “In the time that it would normally take to negotiate a legal agreement to provide OICR-9429 to other research teams,” notes Cheryl Arrowsmith, chief scientist at Structural Genomics Consortium in a joint statement, “we have received results back from our collaborators showing that it can kill two different types of cancer cells.”
By making OICR-9429 readily available and insisting on researchers sharing the results, Ontario Institute for Cancer Research and Structural Genomics Consortium say they hope to speed the identification of targets for the compound and build on the common experiences of teams testing the compound. The two organizations are collaborating on other therapeutic molecules with which they expect to apply these same development methods.
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Gordana Vunjak-Novakovic (Columbia University)
3 September 2015. Engineers and medical researchers at Columbia University developed a technique that delivers small amounts of medications in liquid form to specific areas of lungs. The team led by biomedical engineering professor Gordana Vunjak-Novakovic published its proof-of-concept findings earlier this week in Proceedings of the National Academy of Sciences (paid subscription required).
The Columbia team is seeking a method to more carefully target a small volume of drugs to treat lung diseases such as cystic fibrosis, pneumonia, chronic obstructive pulmonary disease (COPD), and lung cancer. Current techniques deliver drugs for the lungs systemically with pills or inhaled aerosols that require a large dosages of medications. The high volume is needed for the drugs to reach their targets, but the quantities can also cause adverse side effects.
Vunjak-Novakovic’s lab in New York studies lung regeneration with stem cells and bioengineering methods, which led to its work on drug delivery to this highly complex organ. Postdoctoral researcher Jinho Kim, the paper’s first author, tracked the flow of liquid in a lab model of lungs with glass tubing representing the channels and stages the compound passes from the airway down to the individual air sacs.
Kim and colleagues then developed a mathematical model of drug delivery with liquids designed to have lower surface tension for easier absorption by the air sacs, a technique used sometimes with premature infants having weak weak lungs. The team used the math model to simulate delivery of minute volumes of drugs, less than 1 milliliter, to various parts of the lung and under varying conditions of ventilation.
The researchers tested the process in lungs of lab rats, delivering less than 1 milliliter of drug and enzyme solutions, with fluorescence imaging to record the results. The findings show the technique can deliver these small quantities to specific areas of lungs, depositing a thin film of liquids on the targeted locations.
“We envision that our micro-volume liquid instillation approach will enable predictable drug concentrations at the target site,” says Vunjak-Novakovic in a university statement, “reducing the amount of drug required for effective disease treatment with significantly reduced side effects.”
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Scanning electron micrograph of HIV particles infecting a human T cell (NIH.gov)
2 September 2015. A large-scale study of preexposure prophylaxis or PrEP, the use of antiviral drugs to prevent HIV infection, found the practice prevented new HIV infections among Kaiser Permanente clients in San Francisco over a 32-month period. Results of the study led by Kaiser Permanente epidemiologist Jonathan Volk, appear in yesterday’s issue of the journal Clinical Infectious Diseases (paid subscription required).
Kaiser Permanente is a California-based health care plan and provider that includes a research division conducting epidemiological and health services studies. In 2012, the U.S. Food and Drug Administration approved the combination of anti-viral drugs emtricitabine and tenofovir to prevent HIV among people at high risk of infections. Volk and colleagues looked into the efficacy of PrEP in a long-term, real world setting, which they say is so far evaluated only in clinical trials and demonstration projects.
The drugs emtricitabine and tenofovir are administered as a single pill taken daily, marketed as Truvada by Gilead Sciences. Truvada is a type of drug known as nucleoside reverse transcriptase inhibitors that works by blocking an reverse transcriptase enzyme.
Blocking the enzyme prevents the HIV virus from multiplying, and causes the amount of HIV in the body to decline. The drug is given along with advice to perform safe sex to reduce HIV infections in adults, even after infections are diagnosed, and is also approved by FDA to treat HIV infections.
The Kaiser Permanente team tracked referrals to its San Francisco medical center over a 32-month period. During that time, the center received 1,045 referrals for PrEP, of which 657 individuals began the regimen. Virtually all (99%) of the individuals were men having sex with other men, and averaged 37 years of age. People receiving PrEP medications were more likely to have multiple sex partners than individuals not taking PrEP drugs, but less likely to have a partner with HIV.
During the observation period, no new HIV infections were reported among PrEP participants, but sizable percentages of other sexually transmitted infections were reported. After 6 months, some 30 percent of PrEP participants reported at least one sexually transmitted infection, with that percentage rising to half (50%) after 12 months. Chlamydia, rectal infections, and gonorrhea were those reported most often.
Among people receiving PrEP, the length of time taking the drugs was about 7 months, but they report a mixed record of sexual behavioral change. After 6 months of use, about three-quarters (74%) of people receiving PrEP said their number of sexual partners did not change, while 15 percent said the number of partners decreased and 11 percent said the number of partners increased. Condom use, however, decreased among 41 percent of PrEP participants, while increasing among only 3 percent, and 56 percent going unchanged.
The authors recommend ongoing screening for sexually-transmitted infections, such as hepatitis C, as well as outreach to PrEP candidates other than gay men, including injected drug users, transgender women, and heterosexual men and women.
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(Vanessa Isabel, Wikimedia Commons)
2 September 2015. A study by medical researchers at University of Michigan shows sequencing of genomes and tumors of children with cancer provides their physicians with new information leading to changes in care given a large percentage of patients. The team led by Michigan pathology professor Arul Chinnaiyan published its findings yesterday in the Journal of the American Medical Association (paid subscription required).
Chinnaiyan and colleagues investigated a program at C.S. Mott Children’s Hospital in Ann Arbor, part of the University of Michigan health system, known as Peds-MiOncoSeq, a children’s version of an initiative to sequence the tumors and genomes of cancer patients to gain greater insights into mutations and other genetic causes of the patients’ cancer. Analyzing the sequence of nucleic acids in the DNA can reveal genetic variations among those sequences associated with disease conditions. This information can offer physicians more details about the cause of an individual’s condition, and lead to treatments needed to address it.
“This individualized genetic information helps us better predict what genetic change is driving a particular child’s tumor, what’s causing the resistance to the treatment, and how to predict response to certain treatments,” says pediatric oncologist Rajen Mody, the paper’s first author, in a university statement. “This knowledge can help us match each patient with the specific therapy most likely to benefit him or her.”
The study reported on results from 102 children and young adults, averaging 11 years old, enrolled in Peds-MiOncoSeq, who were among the program’s first participants. All of the participants had cancer of some form considered rare, relapsed, or unresponsive to treatment. Of the 102 participants, 89 percent had sufficient tumor tissue for analysis, with 7 in 10 participants (69%) having a solid tumor cancer, while the remaining 31 percent having a blood-related cancer.
Each of these individuals had their whole genome analyzed, as well as their tumors’ DNA and RNA — genetic instructions to cells transcribed from DNA. Families of the patients received counseling on the results the analysis, and the findings were reviewed by precision medicine panel, which made recommendations to the families and physicians.
Of the participants receiving genomic analysis, nearly half (46%) were offered findings of genetic anomalies that their physicians considered actionable, resulting in changes in diagnosis, identification of new drug targets, or genetic counseling for family members who may also be susceptible to inherited conditions. The results led to changes in treatments for 15 percent of patients, with 10 percent experiencing at least partial remission of their cancers for as long as 21 months. The authors note there was no control group to compare these result with standard cancer care.
The university spun-off a company, Paradigm Diagnostics, that performs genomic sequencing for cancer patients and clinical trials. Study leader Arul Chinnaiyan is a scientific advisor to the company.
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Artist depiction of Cas9 protein editing a gene (Jennifer Doudna, University of California – Berkeley)
1 September 2015. Intellia Therapeutics, a developer of therapies that edit human genomes to fix inherited disorders, raised $70 million in its second round of venture financing. OrbiMed HealthCare Fund Management led the funding round for the Cambridge, Massachusetts enterprise, with new and current investors taking part.
Intellia Therapeutics, founded in November 2014, is a spin-off from Caribou Biosciences, an early-stage life sciences company, that develops and commercializes CRISPR-Cas9 therapies. CRISPR, short for clustered, regularly interspaced short palindromic repeats, is adapted from a natural process used by bacteria to protect against attack by viruses, where a protein that deactivates or replaces genes binds to targeted RNA molecules generated by the genome. The RNA molecules then guide the editing protein, known as CRISPR-associated protein 9 or Cas9, to specific genes needing changes.
The company is harnessing CRISPR to engineer chimeric antigen receptor T-cells, immune-system cells that attract antigens producing antibodies, cultured and grown in the body, then infused back into patients to attack disease-causing cells. In addition, Intellia is working with hematopoietic or blood-forming stem cells, which they plan as well to culture and grow in the lab for transplant in patients.
OrbiMed HealthCare Fund Management, a life sciences and health care investment company in New York, led the new financing round. Participants in the funding include Intellia’s founding investor Atlas Venture and the pharmaceutical company Novartis. In January 2015, a reported in Science & Enterprise, Novartis licensed CRISPR-Cas9 technologies from Intellia and Caribou Biosciences, which included Novartis taking an equity stake in Intellia.
Also taking part in the financing were new investors Fidelity Management and Research Company, Janus Capital Management, Foresite Capital, Sectoral Asset Management, and EcoR1 Capital as well as other unnamed mutual fund and health care investors. Intellia says it plans to use the proceeds of the round to advance its pipeline, expand its gene-editing platforms, engage in new alliances, and expand its staff.
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Spherical nucleic acid (Mirkin Research Group, Northwestern University)
1 September 2015. National Cancer Institute is renewing its support for a Northwestern University research center advancing nanotechnology to design new cancer treatments. The Northwestern University Center for Cancer Nanotechnology Excellence is the recipient of a new five-year, $11.7 million grant from National Cancer Institute, part of National Institutes of Health.
The Center for Cancer Nanotechnology Excellence aims to develop cancer therapies based on research with nanoscale materials — 1 nanometer equals 1 billionth of a meter — from the lab of Chad Mirkin, director of the International Institute of Nanotechnology at Northwestern’s main campus in Evanston, Illinois. Mirkin and colleagues design nanoscale particles with nucleic acids, components of genetic substances such as DNA, called spherical nucleic acids. These nanoparticles are engineered to express chemical and physical characteristics that interact with cells in the body for diagnostics and treatments, or they can act as a scaffold to build therapeutics from more complex genetic molecules.
Northwestern’s Lurie Cancer Center in the university’s medical school in Chicago is a partner in the Center for Cancer Nanotechnology Excellence. Leonidas Platanias, director of Lurie Cancer Center, shares the project’s leadership with Mirkin. National Cancer Institute began supporting the Center for Cancer Nanotechnology Excellence in 2005.
The center brings together researchers from the fields of medicine, biology, chemistry, materials science, physics, and engineering to develop new types of nanoparticle-based therapies and diagnostics that can be tested in lab cultures and animals. The project also expects to engage partners in the business community to advance their discoveries into the clinic.
One of the spherical nucleic acids developed in Mirkin’s lab that reached commercialization stage is Nanoflares, a gold nanoparticle with single-stranded DNA flaring from out from the core. Nanoflares can detect live cancer cells in the blood stream before they form tumors. EMD Millipore, a division of the pharmaceutical company Merck, licensed Nanoflare technology from Mirkin’s lab and is taking it to market as an early cancer diagnostic tool.
The National Cancer Institute grant also covers educational programs in nanotechnology and cancer for undergraduates and medical students, as well as fellowships in nanotechnology and continuing education for clinicians.
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Prototype contact lens with sensors to detect and measure glucose levels in tears (Google Inc.)
31 August 2015. The pharmaceutical company Sanofi is collaborating with Google’s life sciences teams to develop technologies for improving the care of people with diabetes. Financial and intellectual property details of the partnership were not disclosed.
Diabetes is a chronic condition where the pancreas does not create enough insulin to process the sugar glucose to flow into the blood stream and cells for energy in the body. Type 2 diabetes is a disorder where the pancreas produces some, but not enough insulin, or the body cannot process insulin, and accounts for some 90 percent of all diabetes cases. Type 1 diabetes is an autoimmune condition where the body is tricked into producing little or no insulin. According to the International Diabetes Federation, diabetes affects 387 million people worldwide, of which 39 million are in North America.
The two companies plan to bring together their respective experiences to help people with diabetes better manage their conditions, reduce risks of complications, and improve outcomes. Sanofi, based in Paris, offers its expertise in developing diabetes treatments. The company has a number of diabetes management products on the market, including Afreeza, a form of insulin administered as an inhalation powder.
Google is contributing its experience with analytics and miniaturized electronics. One of Google’s current R&D projects is a smart contact lens with sensors built-in for analyzing the composition of tears to gauge blood glucose levels of people with diabetes. In July 2014, as reported in Science & Enterprise, Google licensed intellectual property rights to the smart contact lens to the pharmaceutical company Novartis for medical applications involving the eyes.
The Sanofi-Google collaboration is expected to result in better integration of health and wellness indicators related to diabetes including concentrations of glucose in hemoglobin, a protein in red blood cells, abbreviated HbA1c or A1c. Sanofi and Google are joining a field of new devices to improve care and management of diabetes, with implanted systems and mobile apps from other companies already being tested in clinical trials.
Innovations in life sciences is one of the target areas of Alphabet, Google’s parent company. In the corporate announcement of Alphabet, Google founder Larry Page specifically mentioned the smart contact lens that senses glucose as an example of the direction Alphabet wants to go.
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Nanostim device (St. Jude Medical)
31 August 2015. First reports from a clinical trial of a heart pacemaker placed inside the heart without wires shows after six months the device maintained effective pacing of patients’ hearts and worked without serious adverse effects in a vast majority of cases. Results of the trial testing the Nanostim device by St. Jude Medical were published yesterday in New England Journal of Medicine and presented at a meeting of the European Society of Cardiology in London.
Heart pacemakers are implanted in patients with an irregular or slower than normal heart rate, and generate normal-paced electrical impulses through thin wires into the heart called leads, which also sense the patient’s heart rhythm to send a compensating electrical signal. These conventional pacemakers are surgically implanted under the patient’s collarbone, with the leads running to the heart. The leads, however, need time to set requiring the patient to keep the area around the pulse generator inactive for a few weeks, to prevent disconnecting. Surgical implants also run a risk of infection around the implant site.
The Nanostim device is about one-tenth the size of a conventional pacemaker and self-contained with a battery, circuitry, and sensors sitting inside the heart, removing the need for wire leads. It is designed for patients needing to stimulate one chamber of the heart, a condition affecting 20 to 30 percent of patients requiring pacemakers.
The device is inserted with a minimally-invasive procedure using a catheter that sends the device through the femoral vein in the thigh into the heart. It can also be repositioned or retrieved after initial implant, such as for battery replacement. Science & Enterprise reported in March 2014 of an early-stage trial testing implantation of the Nanostim device.
The clinical trial, led by cardiology professor Vivek Reddy at Mount Sinai medical center in New York, tested the Nanostim among 526 individuals averaging 76 years of age enrolled at 56 sites in 3 countries, all of whom were diagnosed with heart rhythm problems in one chamber. The findings published and presented yesterday reported on experiences of the first 300 people in the group, looking primarily at Nanostim’s functioning over 6 months as well as any safety issues, including the ability to retrieve the device if necessary. The trial is still underway and aims to enroll 667 participants.
The findings show the device met both its functional and safety objectives among the large preponderance of cases. After 6 months, devices implanted in 90 percent of the 300 participants — 93 percent of the 289 patients with successful implantation — continued to meet sensing and pacing targets. Of participants tracked over 12 months, sensing and pacing threshold values improved from the time the devices were first implanted.
The authors say 504 or 96 percent of the total 526 individuals had successful Nanostim implantations, with 30 percent requiring repositioning of the device. Of the 300 participants tracked over the 6 months, 22 adverse effects were reported in 20 individuals, due to cardiac perforation, device dislodgement, vascular complications, and elevated pacing thresholds in 1 to 2 percent of cases for each type of problem. Similar rates of adverse effects were reported for all 526 participants.
Where devices needed to be retrieved, in 7 cases, the retrievals occurred without complications. Due to individual requirements of participants, 3 received new Nanostim devices, while 2 were fitted with conventional pacemakers, and 2 people with heart failure received cardiac-resynchronization therapy.
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