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Posts Tagged ‘immunotherapy

Science Policy Around the Web – July 21, 2017

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By: Rachel F Smallwood, PhD

Source: pixabay

Cancer

Engineered Cell Therapy for Cancer Gets Thumbs Up from FDA Advisers

A panel of advisers has recommended that the FDA approve chimeric antigen receptor T-cell (CAR-T) therapy for treatment of acute B-cell lymphoblastomic leukemia. The committee unanimously agreed that the risk to benefit ratio was favorable enough to proceed with approval of the drug (tisagenlecleucel), manufactured by Novartis. CAR-T therapy utilizes a patient’s own immune cells to find and attack cancer cells. In a recent trial in humans, 82.5% of patients went into remission following treatment with the drug; there have also been promising results from its use in glioblastoma treatment. The treatment would specifically be for pediatric and young adult patients who did not respond well to initial treatments or who relapsed from being in remission.

Despite have strong positive effects, there are potential risks posed by CAR-T therapy. In the study mentioned above, almost half of the patients experienced an inflammatory reaction called cytokine release syndrome. Although all of those cases were treatable, the condition can be life-threatening. Novartis also reported neurological problems. Other CAR-T trials have had several deaths due to brain swelling, but those were in adult populations and were some differences in the therapies.

The FDA often does take the recommendations of its advisers, but there is much to consider in this decision. It would essentially be approving a living drug that is individualized to each patient; the patients’ own blood cells are sent to a manufacturing center, where they are genetically engineered to target leukemia cells. The cell population is then allowed to proliferate, and the entire process takes around twenty-two days. This process presents a quality assurance and control problem to the FDA. However, the target population typically has a poor prognosis and very few options, so the panel considers the potential for increased survival and quality of life to be worth the risks. (Heidi Ledford, Nature News)

Stem-Cell Therapy

Unapproved Stem-Cell Treatments Touted on Federal Database Clinicaltrials.Gov

ClinicalTrials.gov is an online database, curated by the National Library of Medicine and the National Institutes of Health, that logs clinical studies occurring around the country and allows them to be searched by patients, family members, healthcare providers, and researchers. The information on the site is provided by the researchers or sponsors of the individual studies themselves. It allows patients and healthy people to become aware of opportunities to participate in medical research. These studies involve a wide range of treatments, including drugs, devices, behavioral therapies, and procedures.

A recent study found that the database is being abused by clinics advertising for stem cell trials. These trials target individuals looking for treatment for a variety of conditions, and all of them charge for participation. There are very few FDA-approved stem cell therapies, and most clinics that utilize stem cell therapies assert that they do not need FDA approval since they are practicing medicine and do not substantially alter the stem cells (although that is disputed).  Since the researchers themselves indicate in the database whether they need FDA approval, there is little oversight to ensure these studies are correctly representing the risks and benefits of their treatment.

Although a disclaimer was added this spring that informs visitors that the presence of a trial in the database does not indicate government endorsement of it, many people do not realize that they could potentially be participating in a for-profit procedure that does not have the proper oversight to ensure patient safety. In one such case, three women were blinded who paid to receive stem cell therapy for macular degeneration. Most legitimate research studies will not require payment for participation, although travel and lodging costs associated with participation may be incurred.

While many patients may receive treatment at one of these clinics without an adverse event or even with a positive result, critics of these types of clinics are calling for regulation of entries into the ClinicalTrials.gov system. They assert that a federal resource for medical research should not be used to advertise for for-profit clinics that are utilizing therapies that have not been studied or reviewed for safety and efficacy. (Laurie McGinley, Washington Post)

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Written by sciencepolicyforall

July 21, 2017 at 10:08 am

Science Policy Around the Web – June 16, 2016

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By: Ian McWilliams, Ph.D.

Kris Krüg via Photo Pin cc

Climate Change

Underground injections turn carbon dioxide to stone

In the race to stop or reverse global warming, scientists in Iceland have discovered a method that could trap the greenhouse gas carbon dioxide (CO2) underground by turning the gas into stone. Researchers found that injecting CO2 into volcanic rock leads to the formation of carbonate minerals. Though the project, termed CarbFix, is still in development it could have important implications for climate change and for incentivizing clean energy policies.

Injecting CO2 underground into sedimentary rock (i.e. sandstone) or depleted oil wells has long been an attractive method of carbon capture and storage  (CCS), but there are concerns that fissures in the rock layers could lead to leakage of CO2 back into the atmosphere. In a joint effort, Icelandic, American, and French scientists proposed that injecting CO2 into layers of basalt could improve this method. Basalt is rock formed by volcanic activity and contains metals such as calcium and magnesium that can react with CO2 to form carbonate minerals, such as calcite, in a process known as carbonation. In this injection process, CO2 is first separated from gaseous emission then dissolved in water to form carbonic acid that is injected into basalt formations 400 to 800 meters below the surface in Iceland. A heavy carbon tracer was included in this mix so that the scientists could track the sequestration of carbon. Over a year and a half after their initial injections, the researchers found that 95% of the injected carbon had been converted to calcite and other minerals, surprising the researchers that carbonation could happen so quickly.

Though these results are promising, many hurdles remain. The carbonation process is resource intensive and requires large amounts of water and electricity, which may limit its environmental impact and commercial scalability. Additionally, while basalt is found around the world and could allow for the indefinite storage of billions of tons of CO2 with minimal leakage risk, introducing large amounts of CO2 into the ground could lead to unforeseen consequences. Although the researchers admit they were surprised by how quickly the CO2 turned to stone in their initial trials, it remains unknown how long the process will take on a commercial scale.

Members of CarbFix admit that the largest obstacle is still a financial one. With no price on carbon emissions, power companies have little to no incentive to pursue this technology. Still, the project has great potential.  Since we depend on fossil fuels and likely will for many more years, “we have to decarbonize our energy infrastructure” according to study author Juerg Matter. Matter further stated that “if it’s mineralized within a human lifetime, then we know we are on a successful pathway”. With improvement, turning CO2 to stone could become a viable strategy for controlling greenhouse gas emissions and curb the deleterious effects of our energy dependence. (Eli Kintisch, Science Magazine)

Cancer Therapeutics

U.S regulator says too many drug makers chasing same cancer strategy

Immunotherapy has revolutionized the field of oncology, but some worry that too many companies are focused on developing therapeutics that target the same pathways. At the American Society of Clinical Oncology’s annual meeting last week, Dr. Richard Pazdur, head of the Food and Drug Administration’s (FDA) Office of Oncology Drug Products commented on the abundance of therapies that disable the protein PD-1, which unleashes the immune response against tumors. So far Merck, Bristol-Myers Squibb, and Roche have all received FDA approval for therapies that target PD-1, and many more drug makers are developing similar strategies. Though these drugs have had remarkable success in the treatment of skin, lung, and bladder cancers, Pazdur encouraged companies to pursue other strategies and to invest in more novel drugs.

Pharmaceutical companies have countered that the future of cancer therapy is combining therapies to target multiple mechanisms of disease simultaneously and that approval for their own PD-1 targeting drug will allow for greater flexibility. Approximately 20 percent of patients treated with PD-1 blockers respond to treatment, with some having long-lasting remissions. However, a recent study demonstrated that combining two different immunotherapy drugs increased survival compared to treatment with either drug alone.  While most of the studies have been done in patients with advanced disease, Pazdur and other experts agree that earlier use could be beneficial for many diseases. Indeed, these treatments are currently being tested in a wide variety of cancers at earlier stages. Furthermore, there are significant costs associated with these therapies with an average price of $150,000 per year. Competition could also help lower the costs, leading to greater benefits for patients. (Deena Beasley, Reuters)

STEM Education

Genuine research keeps students in science

A new study has found that actively involving students in learning rather than in traditional lectures and labs can help lower the attrition rate in STEM fields. In a program at the University of Texas, instructors in the College of Natural Sciences replaced standard introductory courses with a three-course sequence that focuses on problem-solving and hypothesis-driven research. The program, called the Freshman Research Initiative (FRI), was launched in 2005 and has since seen thousands of students complete the program. Participants choose a research stream in which they will experience hands-on learning alongside faculty and graduate students. With numerous research topics that include everything from autonomous robots to genomics to white dwarf stars, students have the opportunity to explore unanswered questions and contribute to larger research projects in faculty-led laboratories.

Researchers have analyzed the results from this initiative and found that, while there was no statistically significant different in grade point average, 94% of FRI students completed a STEM degree compared to 71% of non-FRI students. Students from underrepresented minority groups also achieved high retention and graduation rates through the program, further demonstrating that the initiative could be a successful alternative to traditional internships. These results may provide insight into how to incentivize students to complete their education and could point the way for a cost-effective approach to reach President Obama’s challenge to produce 1 million more STEM-trained workers by 2020. (Jeffrey Mervis, Science Magazine)

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Written by sciencepolicyforall

June 16, 2016 at 11:00 am

Science Policy Around the Web – October 21, 2014

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By: Jennifer E. Seedorff, Ph.D.

photo credit: NIAID via photopin cc

Ebola Outbreak – Public Health

In the US, fear spreads faster than Ebola

Ebola is a scary, lethal virus. Luckily, “There’s a reason it’s not everywhere. It’s just not as easy to transmit as people think.” said CDC epidemiologist, Michael Kinzer. So far, the only people to become infected while living in the US are two health-care works that treated the initial patient while he was having severe symptoms, including vomiting and diarrhea. In Ebola, the amount of virus in the body is not the same throughout the course of the infection. As the disease progresses, the amount of Ebola virus present in the body and bodily fluids increases dramatically. Since Ebola is transmitted through direct contact with bodily fluids, individuals without symptoms are not contagious despite being infected with Ebola. However, fears of Ebola have led to what some believe to be overreactions, including a cruise ship that was turned away from port or a school that temporarily closed because an employee had traveled on a different flight that used the same airplane as an Ebola infected health-care worker. As Kinzer told the Guinea media this summer, “Ebola’s not transmitted by the air. Fear and ignorance are transmitted by the air.” (Joel Achenbach and Brady Dennis, Washington Post)

 

Infectious Diseases

US pauses new funding for controversial virus research

The White House has announced that it is pausing any funding for new Gain-of-Function studies on viruses, like influenza, MERS, or SARS, and has called for a voluntary moratorium on existing research projects. Gain-of-Function studies have been controversial both inside and outside the scientific community. These types of studies seek to understand what kinds of mutations are necessary for a virus to evolve to become more pathogenic or to be more easily transmitted in humans or mammels. Proponents argue that these studies help in pandemic planning and strategies for vaccine development. Opponents argue that these studies are generating viruses that have the potential to cause a pandemic if accidentally or intentionally released from the labs. Concerns have been elevated due to recent concerns over safety at high-level containment research labs. US policy for determining the risk/benefits and approval process for these types of gain-of-function will be evaluated by both the National Science Advisory Board for Biosecurity and by the National Academy of Sciences over the next year. (Jocelyn Kaiser and David Malakoff, ScienceInsider)

 

Cancer Research – Precision Medicine

Cancer Immunotherapy successful in Phase I/IIA clinical trial

Cancer Immunotherapy is a promising precision medicine approach for treating cancer, and was named Science magazine’s breakthrough of the year in 2013.   In a recent study, cancer immunotherapy was shown to be an effective treatment for acute lymphoblastic leukemia, a cancer of B-cells. This treatment worked well in patients who had failed traditional therapies, including some whose cancer had previously returned even after stem cell transplants. This study reported impressive, durable results six months after therapy, with 23 of 30 patients alive, 19 of 30 in complete remission, and with 15 of 30 receiving no additional therapy. In this particular version of cancer immunotherapy, a patient’s own T-cells were harvested, then genetic bits of information are added to their T-cells to help them recognize B-cells (which are the source of the cancer), the modified T-cells are given back to the patient, and then these genetically modified T-cells hunt and kill the cancerous B-cells. As with any therapy, this treatment does have side effects most, including destruction of healthy B-cells and Cytokine release syndrome, a systematic inflammatory response that can cause a high fever, a drop in blood pressure, and difficulty breathing. This study was published in the New England Journal of Medicine and was partially sponsored by Novartis which holds the license to develop this therapy. In July, the FDA designated this engineered T-cell treatment as a “breakthrough therapy” which should help expedite the development and regulatory review of this therapy. (Denise Grady, New York Times)

 

Written by sciencepolicyforall

October 22, 2014 at 3:20 pm