(R. Nial Bradshaw, Flickr)
17 November 2016. First results from a late-stage clinical trial shows a synthetic antibody reduces the number of days with migraines per month among people with episodic or infrequent migraines. The synthetic antibody, called erenumab, is jointly developed by the pharmaceutical companies Amgen and Novartis that reported the results today.
Migraine is a neurological syndrome causing severe headaches along with nausea, vomiting, and extreme sensitivity to light and sound. In some cases, migraines are preceded by warning episodes called aura including flashes of light, blind spots, or tingling in arms and legs. The web site Migraine.com estimates 37 million people in the U.S. suffer from migraines, and cites World Health Organization data indicating migraines affect 18 percent of American women and 7 percent of men.
Migraines experienced 14 days a month or fewer are called episodic migraines, while chronic migraines are experienced 15 days a month or more. A study published 2013, however, suggests that individuals with episodic migraines, about 90 percent of those with migraines, who do not get adequate treatment are 3 times more likely to progress to chronic migraines.
Erenumab is designed to prevent migraines by limiting receptors for calcitonin gene-related peptide. This peptide and its receptors are expressed in a number of different cells throughout the central and peripheral nervous systems, and regulate inflammation and pain arising from the stimulation of nerve cells. Erenumab acts by inhibiting the peptide’s receptors, not the peptide itself, and is administered monthly by injections under the skin.
The clinical trial enrolled 955 participants with a history of episodic migraine at 129 sites in North America and Europe. Participants were randomly assigned to receive injections of erenumab, at doses of 70 or 140 milligrams, or a placebo once a month for 6 months. Study teams looked primarily at the average number of days per month participants experienced migraines, compared to a baseline measure taken before the injections.
The results show individuals receiving erenumab experienced fewer days per month with migraines compared to those receiving the placebo. Before the trial, participants average 8.3 days per month with migraines. Individuals receiving erenumab reported 3.2 and 3.7 fewer days per month of migraines, for injections of 70 and 140 milligrams respectively. Placebo recipients, however, experienced 1.8 fewer migraine days per month, a large enough difference from erenumab recipients to be statistically reliable.
Occurrences of adverse effects, say the companies, were similar for participants receiving erenumab or the placebo: common cold symptoms, inflamed sinuses, and upper respiratory tract infections. The adverse effects in this trial were much like those reported in other intermediate and late-stage trials of erenumab.
“The results of this study are important,” says Amgen vice-president Sean Harper in a company statement, “because they confirm the results from our previous studies and add to our body of research in episodic migraine. We look forward to working with regulatory authorities to pursue approval of erenumab and making this novel migraine prevention treatment available to patients and physicians.”
Amgen originally developed erenumab, but in August 2015 joined forces with Novartis to co-develop treatments for neurological disorders, including erenumab, as well as therapies for Alzheimer’s disease. Under their agreement, Amgen retains commercialization rights in the U.S., Canada and Japan for migraine drugs, and Novartis has commercialization rights in Europe and rest of world.
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David Scadden (B.D. Colen, Harvard University)
17 November 2016. A new enterprise, spun-off from Harvard University stem cell labs, aims to make stem cell transplants from bone marrow safer and more reliable. The new company, Magenta Therapeutics in Cambridge, Massachusetts, is also raising $48.5 million in its first venture financing round.
Magenta Therapeutics is licensing discoveries from the lab of David Scadden, professor of medicine at Harvard University and Massachusetts General Hospital, affiliated with Harvard. Scadden’s research teams study stem cells in bone marrow that transform into red blood cells, known as hematopoietic stem cells, particularly processes that disrupt development of healthy blood cells leading to leukemia and other blood disorders.
Bone marrow transplants to treat blood-related cancers today are risky procedures, first requiring elimination of the patient’s own stem cells before transplanting stem cells from donors. To take out the patient’s own stem cells often means techniques such as chemotherapy and radiation, which have adverse side effects, including damage to the immune system raising the risk of infections.
In June 2016, Scadden and colleagues reported on an alternative transplant process with engineered antibodies, rather than chemotherapy or radiation, that specifically targets receptors found only on blood-forming stem cells in lab mice induced with sickle-cell anemia. The antibodies succeeded in removing nearly all of the blood-forming stem cells, making it possible to transplant more than 90 percent of a donor’s stem cells, and correcting the sickle-cell anemia. The process also spared bone marrow and subsequent white blood cells from damage that maintained immune systems in the test mice.
Magenta Therapeutics is developing new techniques based on Scadden’s research to make bone marrow transplants safer for patients. The company plans to adapt the antibody-based process, but also employ faster and more efficient stem cell harvesting techniques from donors with growth factor proteins, and more productive stem cell culturing methods in the lab, to produce the quantity of cells needed for transplant. Scadden chairs the company’s scientific advisory committee.
Magenta incubated for the past year with life science investment companies Third Rock Ventures and Atlas Venture, that led the company’s first funding round providing $48.5 million. Jason Gardner, co-founder and CEO of Magenta, says in a company statement the technology can be applied to a variety of blood and immune-system disorders, including early stage cancers and autoimmune diseases, such as multiple sclerosis and scleroderma.
“There has been terrific innovation in stem cell science recently, and it is time to bring this forward to patients,” Gardner adds. “Our ultimate goal is to reboot the blood and immune systems safely to make a significant impact on the overall quality of life for a much broader group of patients that can benefit from transplant.”
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16 November 2016. A company with a technology for converting drugs given by intravenous infusion into pills and capsules was acquired Grünenthal Group, a provider of pain medications. Financial terms were not disclosed of the acquisition of Thar Pharmaceuticals in Pittsburgh by Grünenthal Group in Aachen, Germany.
Thar Pharmaceuticals provides a process for converting approved drugs given by intravenous infusion into oral drugs like tablets and capsules, making the medication easier to take and more convenient for patients. Thar says its technology, called Enhance, changes a drug compound’s physical characteristics, such as solubility, permeability, and melting point, as well as bioavailability, onset, and absorption properties in the body. However, interactions between the drug and proteins, and effects of the drugs on molecular functions are not affected.
Grünenthal’s main corporate focus is pain medications, and Grünenthal says Thar’s lead product will fit into its overall portfolio. The product, code-named T121 by Thar Pharma, is a reformulation of zoledronic acid, marketed as Zometa by Novartis, given as an IV infusion to treat complications of multiple myeloma causing pain or fractures in bones and hypercalcemia, or high blood calcium levels. Novartis also offers Reclast, a version of zoledronic acid to prevent or treat osteoporosis, given as an IV infusion.
Thar Pharma is developing T121 as a treatment for complex regional pain syndrome, or CRPS, a chronic pain condition in the limbs often resulting from injuries to affected limbs. CRPS is believed to be caused by a malfunctioning or damaged peripheral nervous system, sending nerve signals to and from the brain and spinal cord. Pain from CRPS is prolonged or constant, and can become severe. Affected limbs can also become discolored or swollen.
According to Thar Pharma, T121 received orphan drug designation from Food and Drug Administration as a CRPS treatment. In addition to preclinical studies, the company carried out an early-stage clinical trial of T121 among healthy postmenopausal women, testing the drug for safety and actions in the body, at various dosage levels, Thar Pharma is planning late-stage clinical trials of the drug as well.
Another pain drug, code-named T109 by Thar Pharma, is designed as a fast-acting non-narcotic therapy for acute pain, such as back or post-operative pain, now in preclinical tests. The company filed for an initial public offering of common stock in August 2016, raising $50 million.
Thar Pharma’s products and technology are expected to fit in well at Grünenthal Group. “We are expanding our development portfolio for treating patients with orphan, disabling diseases, says Grünenthal’s chief scientist Klaus-Dieter Langner in a company statement. “For CRPS, an often debilitating, extremely painful syndrome, truly efficacious treatments are lacking today.”
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Kerstin Stenson with the laser optic leads used in photoimmunotherapy (Rush University Medical Center)
16 November 2016. A new type of cancer treatment that precisely attacks tumors with synthetic antibodies activated by laser beams is being tested in an early-stage clinical trial. The treatment known as photoimmunotherapy is developed by Aspyrian Therapeutics Inc. in San Diego, with Rush University Medical Center in Chicago as one of the 5 trial sites.
In photoimmunotherapy, a light-sensitive chemical known as a photosensitizer is combined with synthetic antibody that seeks out and binds only to specific biomarkers expressed by the tumor cells. The combination of photosensitizer and antibody is infused in the patient, where it’s taken up by the tumors. After 24 to 72 hours, surgeons attach thin optical fibers to the tumors and send near-infrared laser beams to the tumors marked by the photosensitizers. Energy from the lasers expands the targeted tumor cells while weakening the cell walls, causing the tumor cells to burst and destroy.
The clinical trial is an early-stage test of photoimmunotherapy’s safety, as well as to determine the maximum safe dose, among 24 individuals with recurrent and head and neck cancer considered unresponsive to other treatments. The study is evaluating RM-1929 code-named by Aspyrian Therapeutics that licensed the technology from National Cancer Institute, targeting epidermal growth factor receptors often expressed on the surface of tumors in head and neck cancers.
RM-1929 contains a combination of cetuximab, an engineered antibody approved by FDA to treat head and neck cancers, and a fluorescent dye designed to combine with antibodies for photoimmunotherapy. Cetuximab is marketed as Erbitux by Eli Lilly and Co. Aspyrian says preclinical studies show RM-1929 causes rapid destruction of human cancer cells grafted onto mice, which also have longer cancer-free survival than conventional treatments.
The clinical trial is looking primarily at the safety of photoimmunotherapy and the maximum dose of the drug and lasers in the treatments in the participants, who are tracked for a month. Of particular concern are effects of the treatments on the skin. Earlier forms of light-sensitive treatments known as photodynamic therapy reported cases of skin burns, including sunburn from exposure to direct sunlight. The study teams are also tracking chemical actions of the drugs in the body, responses or reductions of tumors, overall survival time, and progression-free survival time.
Kerstin Stenson, professor of otolaryngology and director of the study at at Rush University Medical Center, says in a university statement, “This treatment is so unique and promising because its cancer cell-killing power is so selective and immediate.” Stenson adds that “Almost immediately, you can see the tumor start dying. It turns white and melts away.” And because RM-1929 remains inert unless activated by the laser, she notes, it causes almost no damage to surrounding cells.
In addition to Rush University, the trial is recruiting participants at Centura Health Research Center in Denver, Virginia Piper Cancer Institute in Minneapolis, University of Oklahoma Stephenson Cancer Center in Oklahoma City, Thomas Jefferson University in Philadelphia, and MD Anderson Cancer Center in Houston.
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Ava bracelet and smartphone app (Ava Science Inc.)
15 November 2016. A two year-old medical device enterprise making a fertility tracker for women is raising $9.7 million in its first venture finance round. The new funds collected by Ava Science Inc., based in Zurich and San Francisco, supplement the company’s original $2.6 million in seed funding raised in November 2015.
Ava is developing a tracking band worn like a bracelet on the wrist that identifies the fertile days in a woman’s cycle. The device is worn while sleeping at night, when sensors collect data for 9 physiological factors associated with the onset of ovulation and that correlate with the rise in reproductive hormones estradiol and progesterone. Those variables include resting pulse rate, skin temperature, heat loss while sleeping, heart rate variability, sleep quality and quantity, amount of movement while sleeping, breathing rate, perfusion of blood to tissues, and bioimpedence for data on skin hydration and perspiration.
The data are collected and synced to a smartphone app, which calculates the fertile window in a woman’s cycle, averaging 5.3 days that include the days leading up to ovulation and the ovulation day itself. Using the bracelet, notes Ava Science, avoids guess work, urine samples, ovulation tests strips, and body temperature measurements. The company says the Ava system qualifies as an FDA class 1 or low-risk medical device.
Ava Science says data for the algorithms in the device were collected in a year-long clinical study led by Brigitte Leeners, a reproductive endocrinologist at University Hospital in Zurich and scientific adviser to Ava. The company says findings from the study, reported at a professional meeting in Switzerland in June, show results from the Ava system matched 89 percent of the time with independent urine samples.
The company reported in September the first pregnancy in the U.S. attributed to the Ava system, since it began shipping this summer. Ava Science says a second clinical trial of the device is underway.
Polytech Ecosystem Ventures, based in Lausanne, Switzerland and Saratoga in California’s Silicon Valley, led the financing round. Blue Ocean Ventures and Global Sources, with existing seed investors Swisscom and ZKB, and other unidentified investors, took part in the round. Ava Science president and co-founder Lea von Bidder says in a company statement that Ava will use the funds for further product development, scale-up production of Ava bracelets to meet consumer demand, and to advance the company’s research.
“With these funds,” adds co-founder and CEO Pascal Koenig, “we’ll be able to further accelerate our traction, and continue on our mission of developing technology that improves the reproductive health of women worldwide.”
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(Gerd Altmann, Pixabay)
15 November 2016. An experimental synthetic antibody was shown in a clinical trial to markedly reduce triglycerides and the types of cholesterol that contribute to heart disease. Findings from the trial testing evinacumab, developed by the biopharmaceutical company Regeneron Pharmaceuticals, were presented yesterday by University of Pennsylvania medical professor Richard Dunbar at a scientific meeting of American Heart Association in New Orleans.
Triglycerides are a type of lipid, or natural oil, usually stored in the body’s fat cells to help provide energy, but when the intake of calories exceeds the body’s ability to store fat in cells, triglycerides can be released into the blood stream. High levels of triglycerides in the blood contribute to atherosclerosis, the hardening of arteries or thickening of artery walls, that increases the risk of heart disease and stroke, as well as pancreatitis, or inflammation of the pancreas. While different from cholesterol, triglycerides circulate in the blood with the help of lipoproteins, proteins that transport lipids, much like cholesterol.
Regeneron is developing evinacumab as a treatment for homozygous familial hypercholesterolemia, a rare inherited condition where the liver cannot naturally process lipoproteins, leading to abnormally high levels of cholesterol and triglycerides, often resulting in early occurrences of heart disease and stroke. Evinacumab is an injected therapy designed as an antibody that blocks Angiopoietin-like 3, or Angptl3, a protein limiting the activity of enzymes that break down and metabolize fats in the liver, thus stimulating production of triglycerides and cholesterol.
The clinical trial was an early-stage test of evinacumab’s safety and chemical activity in the body. The study team looked primarily for signs of adverse effects among individuals, but also measured triglyceride and cholesterol levels in the blood. Participants in the trial were randomly assigned to receive 1 of 6 different dosage levels of evinacumab, or a placebo.
At the meeting, Dunbar and colleagues reported the first results of the trial from 41 participants, 32 receiving evinacumab and 9 getting the placebo. Results show sharp drops in triglyceride levels among evinacumab recipients, as high as 64 to 73 percent for those receiving the highest doses, well beyond the reductions seen in current treatments. “Current medications such as fibrates or prescription fish oils effectively lower triglycerides,” says Dunbar in a university statement, “but leave much to be desired, each only lowering levels by 20 to 50 percent.”
Results also show lowered cholesterol levels, with the higher the dose of evinacumab, the greater the reductions. But reductions were reported in both the high-density lipoprotein or HDL cholesterol — considered good cholesterol — as well as low-density lipoprotein, or LDL, cholesterol, the kind that contributes to atherosclerosis and heart disease. Reductions in both kinds of cholesterol are considered consistent with limits on Angptl3 proteins.
Participants receiving evinacumab tolerated the treatments, with only headaches reported as adverse effects among 7 participants. The results, say the researchers, provide enough evidence to move forward validating evinacumab as a treatment for people with high triglyceride levels. “In the short term,” notes Dunbar, “profoundly lowering triglycerides may render hospital admissions less frequent in patients prone to pancreatitis, while long term, lowering triglycerides and associated cholesterol could also help reduce the risk of certain heart disease.”
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Heroin powder (Drug Enforcement Administration)
14 November 2016. An experimental long-acting drug was shown at least as effective, and in some cases superior, to the current standard of care for opioid use disorder. The first results of a late-stage clinical trial testing the drug code-named CAM2038 were released today by its developers Braeburn Pharmaceuticals in Princeton, New Jersey, and Camurus AB in Lund, Sweden.
Opioids work by reducing the intensity of pain signals to the brain, particularly regions of the brain controlling emotion, which reduces effects of the pain stimulus. Examples of leading opioid prescription pain medications are hydrocodone, oxycodon, morphine, and codeine. Heroin is also considered an opioid.
The U.S. Department of Health and Human Services says the country is in the midst of a “prescription opioid overdose epidemic.” In 2014, according to Centers for Disease Control and Prevention, more than 28,000 people died from opioid overdose, and at least half of those deaths involved a prescription opioid, with even more becoming addicted to prescription and illegal opioids. Heroin-related deaths also increased sharply, more than tripling since 2010, leading to more than 10,500 deaths in 2014.
CAM2038 is a formulation of the drug buprenorphine that acts on the same receptors in the brain as heroin and morphine, providing enough satisfaction of those receptors without producing the intense “high” or serious adverse effects of abused drugs. Camurus adapted its injection technology that the company calls FluidCrystal for CAM2038 that combines buprenorphine with a liquid lipid solution to form a gel that that can be injected, yet breaks down slowly in the body to allow for release over extended periods of time.
CAM2038 is designed for delivery via injections under the skin by clinicians, as part of anti-addiction therapy. The formulation of CAM2038 can be adjusted for the length of time buprenorphine needs to be released, in this case either over the course of one week or one month. In 2014, Braeburn Pharmaceuticals licensed from Camurus the North American rights to CAM2038 and the companies took part in three early- and intermediate-stage trials of the drug on 176 healthy volunteers and patients.
The clinical trial seeks to test the effectiveness of CAM2038 injections given weekly or monthly, compared to buprenorphine and naloxone taken daily by absorbing tablets under the tongue, considered the current standard of care. Some 428 individuals age 18 and over, suffering from and in treatment for opioid use disorder, took part in the trial. Participants were randomly assigned to receive either:
(1) Injections of CAM2038 once a week for 12 weeks, then once a month, in a higher dose, for another 12 weeks, plus placebo daily tablets, or
(2) Daily buprenorphine/naloxone tablets for 24 weeks, plus placebo injections first at once week, then once a month.
The study aims primarily to find if CAM2038 injections are at least equivalent to the daily buprenorphine/naloxone tablets in reducing opioid use, as determined by urine samples. The trial also measured overall percentages of samples without signs of opioids, as well as monitored participants’ vital signs and other general health measures as indicators of adverse effects.
The results show equal rates of response for both weekly or monthly injections and daily tablets, as determined by at least one-third of urine samples without opioids in the first 12 weeks, and two-thirds of samples opioid-free in the second 12 weeks. And while not the main objective of the study, participants receiving CAM2038 injections reported lower rates of urine samples with opioids in weeks 5 to 24, compared to individuals receiving the tablets.
The companies say safety profiles were similar for both treatments, with 3 percent of CAM2038 injection recipients and 6 percent of tablet recipients reporting adverse effects. Injection-site reactions were reported in 19 and 22 percent of the injection and tablet recipients respectively, none severe. No overdoses were reported among injection recipients, while 4 of the participants receiving the tablets had overdoses, 3 from heroin, but none were fatal.
Michelle Lofwall, Professor of behavioral science and psychiatry at University of Kentucky and the study’s lead investigator, says in a joint company statement, “If approved, the CAM2038 weekly and monthly injectable buprenorphine medications can improve how we treat opioid addiction and decrease the stigma associated with the medication that is in large part due to concerns about non-adherence and diversion.”
In November 2015, CAM2038 received Fast Track designation from the U.S. Food and Drug Administration. In May 2016, FDA approved a six-month buprenorphine implant for maintenance treatment of opioid dependence, made by Braeburn.
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(Avtar Kamani, Pixabay)
11 November 2016. A medical device company presented results of preclinical studies showing its drug-free electronic pulse technology encourages the immune system to kill and clear cancer cells. Pulse Biosciences Inc. in Burlingame, California reported on three studies involving its technology at a meeting of the Society for Immunotherapy of Cancer now underway in Oxon Hill, Maryland.
Pulse Biosciences applies electronic impulses to penetrate cell walls to encourage tumor cell death and induce a response from the immune system to kill more tumor cells. The company’s technology, based on pioneering research at Old Dominion University and Eastern Virginia Medical School, uses very fast electronic impulses of about 100 nanoseconds, but also in high voltages to penetrate cell walls and disrupt the cell’s functions. Because of the impulses’ high speed, says the company, the thousands of volts applied in the charges affect only the targeted cells, not surrounding cells or tissue.
The technology — which the company calls nano-pulse stimulation, or NPS, and says is drug-free — stimulates cell signals in tumors that promote the natural programmed death of cells, a process known as apoptosis. NPS, according to Pulse Biosciences, also enlists cancer-killing cells in the immune system to recognize and attack the same type of tumor cells, and clears them away.
The studies presented at the conference report on tests of the NPS technology in lab mice and with tumor cell lines in lab cultures that indicate the technology’s ability to stimulate immune-system responses similar to vaccines. A report titled, “Nanosecond pulsed electric field treatment of murine melanomas initiates an immune response and inhibits metastasis,” shows the effects of the technology in mice implanted with melanoma or advanced skin cancer tumors. The tumors were then removed either by surgery or with NPS treatments, followed by researchers injecting more melanoma cells into the mice. Results show mice receiving NPS treatments exhibited fewer metastatic or spreading cancers than mice whose tumors were surgically removed, suggesting the NPS treatments have an immunizing effect against metastatic cancer.
Another report titled, “Adaptive immune response to nano-pulse stimulation,” shows mice implanted with fibrosarcoma or soft-tissue tumors and treated with NPS showed more immune system cells infiltrating the primary tumor. In addition, similar tumors given to the mice 3 weeks later were completely rejected by immune-system cells in the mice.
A third report, “Nanosecond pulsed electric field treatment of tumor cell lines triggers immunogenic cell death,” shows NPS treatments on 3 separate tumor cell lines resulted in indicators of apoptosis in the treated cells. The authors suggest these results could explain the vaccine-like effects preventing tumor growth or spreading reported in the tests with mice, later challenged with new tumors.
“We believe the immune response observed in these recent studies,” says Pulse Biosciences CEO Darrin Uecker in a company statement, “further indicates that NPS may have the ability to prime the immune system and may be effective, and potentially even synergistic, when used in combination with other immuno-therapeutics, with the distinct advantage of not adding any drug related toxicity.”
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Seedless watermelon (Steven Depolo, Flickr)
11 November 2016. A review of studies evaluating techniques for producing seedless fruit concludes these techniques can contribute to higher yields that help ensure the security of food supplies. The findings from researchers at University of Exeter in the U.K. appear in today’s issue of the Journal of Applied Ecology.
A team from the lab of applied ecology professor Juliet Osborne, on Exeter’s Cornwall campus in Penryn, U.K., assessed the potential usefulness of parthenocarpy, the growing of fruit plants with neither seeds nor the need for pollination. A key factor in this review was the role parthenocarpic crop plants can play in improving yields to meet food security challenges from climate change and other causes.
Parthenocarpic fruits are often sought out by farmers since they do not require pollination, making them easier to grow, and in some cases like grapes or watermelons, are favored more by consumers. These varieties also produce pollen and nectar, thus can still support pollinators, such as bees. Growers traditionally achieved parthenocarpy through selective breeding, but more recently added such techniques as growth hormones and genetic modification.
Osborne and colleagues searched public databases for published studies of parthenocarpy between 1945 and March 2016, but also included patents and requested unpublished studies to counter any publication bias. From an original return of 161 articles, the researchers gleaned a total of 69 outcomes showing parthenocarpic effects on 18 pollinator-dependent crop species. Of the 69 outcomes, 31 represented applications of hormones, 29 used genetic modification, and 9 applied selective breeding. Tomatoes, sweet peppers, and eggplant were the species most studied.
The team aggregated the results of the studies and calculated their combined effects. The results show all of the studies with outcomes reported larger quantities of fruit produced with parthenocarpy, but the differences in yields from specific techniques, e.g. genetic modification or hormones — were not large enough to be statistically reliable. In addition, the extent to which the species studied were experiencing limited availability of pollen could not be determined, thus the absolute benefits of parthenocarpy compared to optimum pollination conditions could not be calculated.
Enough of the results from the review, however, show parthenocarpic techniques can produce higher yields, without compromising quality or nutritional value. These findings enable the authors to conclude that these techniques can be applied to improve yields where normal pollination methods are interrupted, as in recent cases of bee colony collapse.
Parthenocarpy can also be considered where climate change affects pollination patterns, or to improve food security by extending the growing season. Osborne notes in a university statement that, “Food security is a pressing global challenge and environmental and technological solutions should be used in tandem to ensure the best possible crop yields where they are needed most.”
The authors caution, however, that parthenocarpy is not a panacea for all problems with fruit yields, and should be used with conventional pollination methods to preserve the ecosystem supporting the crops. “It is of course vital,” says Jessica Knapp, a doctoral candidate in Osborne’s group and the paper’s first author, “that we still encourage and increase our native pollinator populations to ensure crops and wild plants can thrive as much as possible.”
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10 November 2016. A biotechnology company spun-off last year in a pharmaceutical industry merger is receiving small business research grants to advance its technology treating neurological disorders. Aptinyx Inc. in Evanston, Illinois is receiving a total of $2.85 million in 3 awards from National Institutes of Health to evaluate experimental compounds to treat neuropathic pain and age-related cognitive decline.
Aptinyx develops treatments for neurological disorders that result from the lack of plasticity in synapses, the part of neurons, or nerve cells, that pass signals with neurotransmitter chemicals in the brain and nervous system. The company’s technology focuses on a particular set of pathways known as N-Methyl-D-aspartate, or NMDA, receptor molecules found in synapses.
NMDA receptors help keep synapses flexible, which affects memory, learning, and development of the central nervous system. Aptinyx applies its platform to discover small-molecule, or low molecular weight drugs to treat disorders such as depression, neuropathic pain, migraine, traumatic brain injury, and epilepsy.
The new Small Business Innovation Research, or SBIR, awards, come from different institutes in NIH:
– National Institute of Neurological Disorders and Stroke is supporting R&D of Aptinyx compounds to treat neuropathic pain, with initial funding of $1.25 million this year. An additional $1.09 million is recommended for the second year, depending on availability of funds and project progress.
– National Cancer Institute is funding a separate neuropathic pain project, a preclinical investigation of Aptinyx’s modulators of NMDA receptors on neuropathic pain associated with chemotherapy-induced peripheral neuropathy. This type of neuropathic pain is a common side effect of many chemotherapy drugs, causing severe pain and often causing patients to discontinue chemotherapy.
– National Institute on Aging is supporting preclinical research into Aptinyx’s NMDA receptor modulators on memory loss and cognitive function in the aging brain. Age-related memory loss and cognitive decline are public health issues becoming more pronounced with increasing life expectancy and an aging population.
Aptinyx is conducted an early-stage clinical trial of its lead product code-named NYX-2925, a small-molecule modulator of NMDA receptors designed to enhance synaptic plasticity, in this case to treat neuropathic pain associated with diabetic peripheral neuropathy, a complication of diabetes. In September 2016, NYX-2925 received Fast-Track designation from the Food and Drug Administration.
The company was formed in September 2015 when drug maker Allergan plc acquired biotechnology company Naurex Inc. As reported in Science & Enterprise, Aptinyx was spun-off from Naurex to continue its work with NMDA receptors. Naurex itself was a spin-off enterprise from Northwestern University, founded by Joseph Moskal, a biomedical engineering professor at Northwestern University and director of the school’s Falk Center for Molecular Therapeutics. Moskal continues as chief scientist as Aptinyx.
“Based on years of research into the role of NMDA modulation in enhancing synaptic plasticity,” says Moskal in a company statement, “we know that our approach can have broad and diverse applications. We are eager to expand research into these indications, for which there is a clear unmet need for more effective and innovative treatment options.”
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