How to get more patients involved in research | Startup seeks partnerships to identify new targets for old drugs | Off-the-shelf blood cancer gene therapies may be the next big thing
December 3, 2019
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New UNAIDS leader says governments must work to end HIV/AIDS epidemic
Ugandan diplomat and Oxfam director Winnie Byanyima has joined the Joint United Nations Program on HIV/AIDS as executive director. Byanyima says she believes the UNAIDS goal to end the HIV/AIDS epidemic by 2030 is achievable, but governments must remove barriers to HIV services among marginalized populations, improve women's rights and gender equality, resume funding and reduce threats to civil society.
Science (tiered subscription model) (11/27) 
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Patient Engagement
How to get more patients involved in research
Enrollment in clinical trials remains low despite high willingness among patients and significant investment in recruitment and retention, and sponsors might have greater success if they use patient engagement practices used in clinical care, write Marla Clayman of the American Institutes for Research, and colleagues. Patients need ample information, effective communication methods and a framework for judging the benefits, risks and personal value of clinical trial participation, they write.
Clinical Leader (12/3) 
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Science and Technology
Startup seeks partnerships to identify new targets for old drugs
Healx recently raised $56 million in a Series B financing round and wants to collaborate with rare disease patient groups to advance 100 treatments by 2025. The company's drug discovery platform uses natural language processing and other means to analyze publicly available and proprietary biomedical data to identify new uses for existing drugs.
Xconomy (11/26) 
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Off-the-shelf blood cancer gene therapies may be the next big thing
Off-the-shelf gene therapies are now considered the next big thing in treatments for leukemias and lymphomas due to the speed with which they can be produced compared with chimeric antigen receptor T-cell treatments, which often take two weeks to produce, a period of time that could see the patient it is intended for deteriorate or even die. One example of such a treatment discussed at the annual meeting of the American Society of Hematology was Fate Therapeutics' off-the-shelf CAR NK cell therapy, the first-of-its-kind treatment that targets multiple antigens.
Medscape (free registration) (11/28) 
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Protein-targeting antibodies may make stem cell transplants safer
Antibodies that target c-Kit molecules in blood stem cells could make transplants of blood stem cells safer and more widely available for treating immune-system and blood disorders, researchers say.
Nature (free content) (11/29) 
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Study: Blood test assesses multiple health conditions
Researchers have developed a blood test they call a "liquid health check" that can potentially assess multiple health conditions and evaluate a patient's risk for cardiovascular disease or diabetes using a single blood sample. The test, described in the journal Nature Medicine, searches plasma for DNA fragments called aptamers to determine the presence and concentration of certain proteins, then feeds the findings into a machine learning algorithm.
Engadget (12/2) 
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Finance and Economics
UCLA research teams awarded $8.7M for prostate cancer studies
Researchers at the University of California, Los Angeles, will use a five-year, $8.7 million grant from the National Cancer Institute to fund three translational projects for treating advanced prostate cancer. Research teams are working on a drug inhibitor to minimize tumor resistance, a chimeric antigen receptor T-cell therapy and ways to target the ONECUT2 gene.
Healio (free registration)/HemOnc today (12/1) 
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Survey shows need to educate payers on RWE for rare disease treatments
Health insurers and other payers in the US and Europe are willing to use real-world evidence in coverage decisions for rare-disease drugs, but a lack of both knowledge and data standardization are barriers, according to the results of a survey by Syneos Health. "Multistakeholder feedback points to the need for collaborative, well-structured exercises and standard-setting for payers to advance at the same velocity as other industry stakeholders," Syneos CEO Alistair Macdonald said in a statement.
The American Journal of Managed Care online (11/26) 
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Policy and Regulation
FDA issues final guide on adaptive trial designs for drugs, biologics
The FDA has finalized its guidance on adaptive clinical trial designs for drugs and biologics and the information the agency will review from the submission as part of new drug applications, investigational new drug applications, biologics license applications and supplemental submissions. The guidance cleared the recommendations for Bayesian adaptive designs and the expectations for the extent of prespecification necessary for adaptations to studies.
Regulatory Focus (12/2) 
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2019 marked by new therapeutic modalities, FDA leadership churn
This year was marked by mergers and acquisitions in the biopharmaceutical industry, the approval of more drugs for neglected diseases, and investment in next-generation cell therapies, gene editors and protein degraders. Though there has been some churn in FDA leadership, FasterCures Senior Fellow Bernard Munos says the agency's day-to-day work related to drug approvals continues apace, and the FDA is increasingly seen as an innovation catalyst.
Chemical & Engineering News (tiered subscription model) (12/2) 
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FasterCures, a center of the Milken Institute, is driven by a singular goal: to save lives by speeding up and improving the medical research system. The Milken Institute is a nonprofit, non-partisan think tank determined to increase global prosperity by advancing collaborative solutions that widen access to capital, create jobs, and improve health.
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