Mirvakili and Hunter study characteristics of synthetic fibers to devise solutions for medical devices and other applications using fibers that respond to their environment. Many current approaches for these solutions, however, involve exotic or expensive materials, like yarns made from carbon nanotubes. These nanotech yarns have a long lifetime, but today are still too expensive. Other metal alloys can bend and return to their original shape, say the authors, but are not durable enough for everyday use.
In earlier work, Mirvakili and Hunter found ordinary nylon fiber, twisted into coils, could simulate some properties of human muscle. Nylon is an inexpensive polymer, easy to produce, and with a long life cycle. They found nylon could be configured to extend and retract in linear actions like human muscles, storing energy to a greater extent than natural muscle fibers.
The ability to direct nylon fibers to bend like human muscles, however, remained a challenge. Mirvakili and Hunter began with applying heat, which would then contract the nylon when it cooled. They discovered just heat alone was not enough, but directing the heat to one side of the fiber would bend the fiber in a desired direction. Taking the process further, the researchers found they could bend nylon fibers into complex shapes, including figure-eights, and movements.
While Mirvakili and Hunter started with ordinary nylon fishing line, they had to change the shape of the fiber and other properties to get the desired responses. They compressed the fiber’s shape to change its cross section from round to rectangular. They also experimented with different heat sources, including heat converted from electricity or chemical reactions, as well as laser beams.
In other tests, the researchers used a conductive paint that when exposed to an electric current heated a section of the fiber, causing the non-painted portion of the fiber to bend in the desired direction. In addition, their fibers lasted through more than 100,000 bending cycles, at speeds of more than 17 bending cycles per second.
The authors foresee the bendable nylon fibers used in medical devices, such as prosthetic devices or self-adjusting catheters to better fit the patient’s blood vessels or organs. Other potential applications are clothes or shoes that adjust to fit the wearers, or solar panels that respond to heat to keep directly facing the sun’s rays.
The following video tells more about this research.
23 November 2016. A clinical trial testing treatments that modify immune system cells to fight leukemia in adults was stopped after one of the patients died from inflammation in the brain. Juno Therapeutics Inc. in Seattle, the company developing the treatments and conducting the trial, says a second participant also developed cerebral edema, a life-threatening condition, and is not expected to recover.
The intermediate-stage clinical trial is testing Juno’s therapy code-named JCAR015 as a treatment for B cell acute lymphoblastic leukemia is a cancer of blood and bone marrow that progresses quickly, making an overabundance of immature lymphocytes, a type of white blood cell. It is the most common type of cancer among children, although it can also affect adults.
JCAR015 harnesses the cancer-fighting ability of a patient’s own T-cells, white blood cells in the immune system, engineered with a gene adding molecules known as chimeric antigen receptors to their DNA. Without these added weapons, cancerous cells evade the immune system, allowing tumors to grow unchecked. The chimeric antigen receptors added to T-cells, called CAR-Ts, are designed in this case to attack cancer cells identified with specific CD19 proteins. These CD19 molecules act as indicators of weakened B-cells in the immune system found with blood-related cancers. The modified T-cells are then infused back into the patient.
As reported in Science & Enterprise, Food and Drug Administration halted this same trial in July 2016 when two participants died after receiving the chemotherapy drug fludarabine as a pre-treatment to remove their original T-cells. Those patients, and a third individual, also developed cerebral edema and died. Juno later replaced fludarabine with the drug cyclophosphamide, and was allowed to continue the trial.
The company in this case stopped the trial itself and notified FDA. Juno says it is working with FDA and its own monitors to consider its next steps. The company adds that its other clinical trials and CAR-T products are continuing.
Until this study, Juno reported a string of favorable results in its early-stage trials, including those testing JCAR015. One of those trials reported in June 2016 with B-cell acute lymphoblastic leukemia patients showed high rates of response and remission, where all signs of leukemia disappear. However, the trial also showed high rates of adverse effects, including severe cytokine release syndrome that occurs when enzymes are emitted from cells targeted by treatments, which can cause flu-like symptoms such as fevers, nausea, and muscle pain, as well as neurological symptoms including hallucinations and delirium.
22 November 2016. The Feinstein Institute for Medical Research and Global Good Fund are evaluating an electronic device that stimulates nerves to stop bleeding, as a way to control postpartum hemorrhage. The device is known as a neural tourniquet, developed by Sanguistat Inc., a spin-off company from the Feinstein Institute in Morris, Connecticut, also taking part in the project.
Postpartum hemorrhage is a particular maternal health problem in developing regions, where maternal mortality rates exceed 1,000 women per 100,000 live births, and the disorder is responsible for an estimated quarter of those deaths. Postpartum hemorrhage is defined arbitrarily as blood loss of more than 500 milliliters following vaginal delivery or more than 1,000 milliliters following cesarean delivery. Diagnosis of postpartum hemorrhage is usually reserved for pregnancies that progress beyond 20 weeks.
Feinstein Institute is the research division of Northwell Health, a health care provider in Manhasset, New York, which conducts research on electronic nerve stimulation as a therapeutic technique. The institute says this therapy strategy is already being applied to neurological disorders including epilepsy, depression, and cluster and migraine headache. The neural tourniquet is a device that applies the same principle to dangerous bleeding episodes.
Sanguistat, founded in October 2015, is commercializing Feinstein Institute’s research neural stimulation and excess bleeding. The company says the neural tourniquet sends electronic signals through the vagus nerve, part of the involuntary nervous system controlling various bodily functions. The vagus nerve extends from the brain to the abdomen, and through multiple organs, including the spleen.
In the spleen, the signals help prime blood platelets, which when in contact with damaged tissue, begin forming clots to stop the bleeding faster than normal platelets. Sanguistat says its preclinical studies show a single treatment with the neural tourniquet can reduce blood loss by up to 70 percent in cases of trauma or hemophilia.
The alliance of Global Good Fund, Feinstein Institute, and Sanguistat will assess the utility of the neural tourniquet in clinical trials to treat postpartum hemorrhage. Global Good brings together government, business, and not-for-profit organizations to develop new technologies to improve the life of residents in the poorest regions in the world.
The organization is a partnership between Microsoft founder Bill Gates and new technology developer Intellectual Ventures in Bellevue, Washington. Intellectual Ventures acquires patent rights to new inventions, then arranges their licensing, financing, and development with business and not-for-profit organizations as sponsors.
“All postpartum hemorrhage deaths are tragic, but they are all too common in low-income countries, where many women suffer from anemia and lack access to the necessary obstetric care,” says David Bell, Global Good’s director of Global Health Technologies in a joint statement. “If this technology proves successful, the partnership with the Feinstein Institute and Sanguistat could mean the difference between life and death for mothers around the world who suffer from postpartum hemorrhage.”
22 November 2016. Drugs directly attacking cancer cell growth show high remission rates with a common form of leukemia, but a new study suggests the high costs of these drugs could impose difficult economic burdens on patients and the health care system. The analysis led by Jagpreet Chhatwal, a radiologist at Massachusetts General Hospital and Harvard Medical School in Boston, appears in yesterday’s (21 November) issue of the Journal of Clinical Oncology.
Chhatwal, a decision scientist in Mass. General’s Institute for Technology Assessment, applies tools of operations research, such as computer modeling and statistical analysis, to clinical decision-making and health care policies. In this study Chhatwal and colleagues analyze the costs of new small-molecule drugs designed to treat chronic lymphocytic leukemia that block protein signals supporting cancer cell growth.
Chronic lymphocytic leukemia, or CLL, is a disorder of the blood and bone marrow, where the bone marrow makes too many blood stem cells that fail to mature into healthy white blood cells. The overabundance of these leukemia cells crowd out the healthy blood cells, including other white blood cells that support the immune system as well as platelets and red blood cells. CLL is the most common type of leukemia in adults.
Earlier in 2016, Food and Drug Administration approved two small-molecule drugs — the kind that can be formulated into tablets or capsules — as treatments for CLL: ibrutinib as a first-line therapy, marketed as Imbruvica by Pharmacyclics and Janssen Biotech, and venetoclax, marketed as Venclexta by Genentech for relapsed or unmanageable CLL. Ibrutinib was originally approved in 2014 as a treatment for a specific type of CLL.
Also in 2014, FDA approved a combination of idelalisib, marketed as Zydelig by Gilead Sciences and rituximab, marketed as Rituxan by Genentech and Biogen, for relapsed and unmanageable CLL. (In March 2016, Gilead Sciences stopped 6 clinical trials testing idelalisib in combination with other drugs due to high rates of serious adverse effects.)
While these newer targeted drugs achieve higher remission rates and extended survival times, they come at a high financial price tag. The cost for one year of ibrutinib and idelalisib treatments, for example, is $130,000 and can continue for many years. In contrast, chemo-immunotherapies, the previous standard of care for CLL, range in cost from $60,000 to $100,000 per year, and the course of treatments last about 6 months at a time.
The researchers sought to better understand the economic implications of these new drugs for individuals and the U.S. health care system that pays for a large part of the drugs’ costs. To make these projections, the team developed a computer model that covered factors affecting use of the new drugs: patient characteristics and clinical course of treating patients, including different stages of therapy for CLL, based on outcomes of clinical trials. The researchers validated their model against published tables with survival and mortality data.
The model shows the number of people living with CLL in 2025 to increase, but the costs of the new drugs over this period to rise even faster. The authors project the number of people in the U.S. living with CLL to increase by 55 percent from 2011 to 199,000 by 2025. In that time, however the total annual cost of drugs to treat CLL is expected to rise nearly 7 times, from $740 million in 2011 to $5.13 billion in 2025.
As the new targeted therapies become the first-line standard of care, the lifetime cost per patient for drugs is projected to more than quadruple from $147,000 to $604,000. Out-of-pocket costs of these drugs for Medicare patients is expected to jump more than 6 times from $9,200 to $57,000.
The researchers applied a standard measure of economic valuation in health care known as quality-adjusted life year, an index that runs from 0.0 (death) to 1.0 (excellent health). The index considers number of years of life, as well as factors such as mobility, pain, and ability to carry on usual activities. The authors then calculated incremental cost-effectiveness ratios per quality-adjusted life year, for the newer targeted drugs and previous chemo-immunotherapies, converted to standard 2015 dollars.
The team calculated an incremental cost-effectiveness ratio for the newer targeted rugs of of $189,000 per quality-adjusted life-year, compared to chemo-immunotherapies, which far exceeds the benchmark willingness to pay threshold of $100,000 per quality-adjusted life-year. The authors urge more efforts to secure discounts of 50 to 70 percent for the new drugs rather than abandoning these promising treatments. Chhatwal notes in a Mass. General statement that, “high out-of-pocket costs for cancer care not only cause financial hardships for patients but also reduce their quality of life through anxiety, stress and worry about paying those large bills. Lower-income patients may be unable to afford these therapies at all.”
21 November 2016. A developer of feeds for farmed salmon says its algae-based feed supplements could arrest the sharp decline in omega-3 fatty acids discovered in a recent study. The company TerraVia Holdings in San Francisco makes an algae supplement for farmed fish that it says can substitute for oil from small fish normally eaten by salmon, but are disappearing.
TerraVia is responding to a study reported by the BBC and others in October 2016 that concentrations of omega-3 fatty acids in farmed salmon today are about half the level of farmed salmon caught 5 years ago. The study was conducted by the Institute of Aquaculture at University of Stirling in the U.K., led by Douglas Tocher, a biochemist at the institute. Tocher studies molecular biology and the role of genetics in regulating lipid and fatty acid metabolism and nutrition in fish, particularly those with omega-3 fatty acids.
Omega-3s are considered essential polyunsaturated fatty acids with benefits affecting the heart and brain. These substances are believed to reduce inflammation, and help improve heart health and cognitive functions. University of Maryland Medical Center cites data showing infants who do not get enough omega-3 fatty acids from their mothers during pregnancy are at risk for developing vision and nerve problems. And American Heart Association recommends eating foods high in omega-3s, such as salmon, twice a week.
Tocher and colleagues, however, found that people would need to eat twice as much salmon as before to get the same omega-3 benefits. “About five years ago,” Tocher told the BBC, “a portion of Atlantic salmon of 130 grams was able to deliver three-and-a-half grams of beneficial omega-3. This is actually our weekly recommended intake. Now, the level of omega-3 has halved.” Much lower levels of oil in fish feed is believed to be responsible for lower levels of omega-3 fatty acids in the farm-raised fish.
TerraVia is a provider of food, nutrition, and specialty ingredients based on algae. The company partners with agribusiness enterprise Bunge Ltd. to make a fish feed ingredient from algae known as AlgaePrime DHA designed for farmed raised fish. DHA is short for docosahexaenoic acid, a specific omega-3. AlgaePrime DHA, say the companies, is rich in long-chain or complex DHA similar to those found naturally in fish and olive oils, but in higher concentrations.
“AlgaPrime DHA contains approximately 3 times the level of DHA compared to fish oil,” says TerraVia vice-president Walter Rakitsky in a company statement. “One ton of AlgaPrime DHA is the equivalent of saving up to 40 tons of wild caught fish from our oceans on a DHA basis.”
AlgaPrime DHA is made in a joint venture with Bunge’s subsidiary in Brazil producing renewable edible oils. The companies say AlgaPrime DHA is a microalgae product from sugar cane waste fermented into omega-3 algae. The process, the companies add, uses low carbon and water inputs.
Red blood cells with sickle cell disease (NCATS.NIH.gov)
21 November 2016. Drug maker Novartis is acquiring Selexys Pharmaceuticals Corp., a developer of treatments for pain episodes associated with sickle cell disease. The agreement to acquire Selexys is expected to bring the Oklahoma City company’s shareholders as much as $665 million.
Selexys Pharmaceuticals’ lead product, code-named SelG1, is designed as an antibody to address a key protein believed responsible for damage to blood vessels from sickle cell disease and the pain that results. Sickle cell disease is a genetic blood disorder affecting hemoglobin that delivers oxygen to cells in the body. People with sickle cell disease have hemoglobin molecules that cause blood cells to form into an atypical crescent or sickle shape.
That abnormal shape causes the blood cells to break down, lose flexibility, and accumulate in tiny capillaries, leading to anemia and periodic painful episodes. The disease is prevalent worldwide, and affects 70,000 to 80,000 people in the U.S., including about 1 in 500 people of African descent.
Selexys Pharma developed SelG1 as a synthetic antibody that seeks out and blocks the actions of P-selectins, proteins that accumulate on the surface of endothelial cells lining the inside of blood vessels and in blood platelets. P-selectins are also believed to promote accumulations of white blood cells called leukocytes on blood vessel walls, leading to inflammation and leakage causing the severe pain experienced by people with sickle cell disease.
Selexys tested SelG1 in an intermediate-stage clinical trial among 198 individuals with sickle cell disease, randomized to receive high or low doses of SelG1, or a placebo. The first 2 treatments were given by intravenous infusion 14 days apart, then every 4 weeks for 50 weeks. The study team looked primarily at the rate of pain crises related to sickle cell disease over a period of 1 year, but also related indicators, such as time to first and second pain crisis, and reports of adverse events.
Researchers from the study team conducting the trial will report their findings on 4 December at the annual meeting of American Society of Hematology in San Diego. Results show trial participants receiving the higher dose of SelG1 had a 47 percent lower rate of pain crises from sickle cell disease than placebo recipients, a large enough difference to be statistically reliable. Dosage size appears to play a role, however, as individuals receiving the lower dose experienced a 33 percent lower rate of pain crisis, a difference not quite large enough for statistical reliability.
Some secondary indicators of effectiveness including median time to first or second pain crisis and annual rate of uncomplicated pain crisis were also reliably lower for higher dose participants. The most frequent adverse events reported by at least 5 percent of SelG1 recipients included joint pain, itching, vomiting, chest pain, diarrhea, fatigue, and a road traffic accident, among others. During the study, 5 deaths occurred, 3 people taking SelG1 and 2 receiving the placebo, but none of the deaths were deemed a result of the treatments.
The agreement is expected to return as much as $665 million to Selexys shareholders, from initial, acquisition, and milestone payments. In 2012, Novartis obtained an exclusive option to acquire Selexys, and the deal represents Novartis’s exercise of that option. Novartis says the clinical trial results were instrumental in the decision to acquire Selexys.
18 November 2016. A synthetic porous material developed to deliver drugs to the skin was shown in lab tests to prevent the growth of bacteria on its surfaces. The material known as Upsalite was developed in the nanotechnology lab of materials science and engineering professor Maria Strømme at Uppsala University in Sweden, which also conducted the study appearing this week in the journal ACS Omega.
A spin-off company from the university, Disruptive Materials AB, is commercializing Upsalite, where Strømme serves on the company’s board. Disruptive Materials markets Upsalite for drug delivery and dermatology applications, as well as for moisture absorption, filtration, and chemical processing.
Upsalite is a mesoporous material, where its pores range from 2 to 50 nanometers in diameter, from a synthesized magnesium carbonate compound. Because of its porosity, Upsalite has a large surface area giving it a high moisture absorption capacity. That property makes Upsalite useful with drug compounds having poor water solubility, thus promising for delivery of these drugs. Strømme’s lab first discovered Upsalite in 2013, and since showed the material to be well-tolerated by human skin.
In the new paper, Strømme and colleagues tested Upsalite’s effects on bacteria, in this case Staphylococcus epidermidis found on human skin. For most people, Staphylococcus epidermidis, or S. epidermidis, is a benign bacteria that causes no harm when in balance with other skin microbes. The most harmful condition most people will encounter from this bacteria is acne.
For individuals having weakened or compromised immune systems, however, S. epidermidis can cause serious complications, with infections sometimes arising in hospitals from infected devices such as catheters or artificial heart valves. Treatment options for these infections are limited, however, due to the appearance of resistant strains of the bacteria.
To test Upsalite’s effects of S. epidermidis, the researchers treated powder samples of Upsalite and three comparable materials — magnesium oxide, mesoporous silica, and basic magnesium carbonate — with resazurin, a fluorescent chemical that lights up when encountering metabolic activity of living organisms, such as bacteria. The team took measures of metabolic activity of these powders when exposed to S. epidermidis bacteria, as well as a saline solution that allowed bacteria to grow unchecked as a baseline.
The results show Upsalite stopped S. epidermidis bacteria from forming and growing almost immediately, which continued for 3 hours, comparable to magnesium oxide, but better than basic magnesium carbonate and mesoporous silica. The researchers attribute this bacteriostatic effect largely to Upsalite’s alkaline properties.
These and earlier results suggest Upsalite can be developed further into products worn or applied to the skin. “These newly found bacteriostatic properties,” says Strømme in a university statement, “combined with the ability to load and release molecules, for example fragrances from the pores in the material, are highly interesting for many applications.”
18 November 2016. Genomic analysis of tumors, a growing practice for diagnosing cancer in individuals, is shown to be feasible when conducted routinely throughout a cancer care center. A study by a team from Dana-Farber Cancer Institute in Boston that appears in yesterday’s (17 November) issue of the journal JCI Insight, also points out some gaps in applying genomic profile data, when put into practice with patients.
The study, led by Laura MacConaill, scientific director of Dana-Farber’s Center for Cancer Genome Discovery, evaluated the Profile program that offers genomic profiling of tumors to all Dana-Farber patients, regardless of age, cancer type, or stage of the cancer. The research team that included participants from a number of other universities and institutions reviewed the experiences of 3,727 Dana-Farber patients, and those at Brigham and Women’s Cancer Center and Boston Children’s Hospital. who agreed to take part in Profile during 2013, the program’s first year. The program to date analyzed about 15,000 tumors.
Data from the study show results of the institution’s OncoPanel platform that analyzes some 300 genes in parallel from each tumor sample. The analysis seeks to identify genetic alterations in tumors, as well as therapies targeted to address those alterations. Dana-Farber stores patient genomic profiles in a database linked to other clinical data that the institute says provides a rich resource for precision cancer medical research and practice.
Where OncoPanel could make a genomic profile of a tumor, nearly all (96%) returned results of some kind. Nearly three-quarters (73%) showed an alteration providing new insights or considered actionable. In 19 percent of the cases, the analysis pointed to currently approved cancer treatments. In the remaining cases, the analysis could point patients to clinical trials of experimental drugs or currently approved drugs being tested for other disorders. The paper also notes cases where Profile identified genomic alterations that responded to targeted therapies, and gave examples where the analysis resulted in a different diagnosis or treatment strategy.
MacConaill and colleagues, however, also identified a number of obstacles that need to be addressed by Dana-Farber and other institutions considering a program like Profile. Among all of the samples collected, only about half had sufficient tumor material available for analysis. In addition, only in about 10 percent of the cases did genomic profiles result in actual changes in patient care. The authors cite a number of factors in cancer care that must be coordinated with tumor profiling to make optimum use of the data, including the timing of the profile during the course of the patient’s disease progression, and access to targeted drugs and clinical trials.
Related to timing of the analysis is the amount of time needed to return results of tumor profiling. The authors report OncoPanel provided results in an average of 5.3 weeks, which Dana-Farber says is now down to 3 weeks. Genomic profiling of tumors is also expensive, “and this cost,” MacConaill notes in a Dana-Farber statement, “has been borne by our institutions.”
As reported on a number of occasions in Science & Enterprise, Foundation Medicine — a company spun off from Dana-Farber Cancer Institute, the Broad Institute, Harvard Medical School and MIT — commercializes a technology similar to Profile that examines and reveals genes altered in human cancers, and offers potential targets for therapies. Two of the paper’s authors, Matthew Meyerson and Levi Garraway, are co-founders of Foundation Medicine and serve as advisers to the company.
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.
Migraineis 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.
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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