Pfizer and the Alzheimer's Drug Discovery Foundation are providing financial and technical support to researchers at Johns Hopkins University School of Medicine who are developing therapies for Alzheimer's, Parkinson's and other neurodegenerative diseases. The collaboration arose at a FasterCures Partnering for Cures conference.
FasterCures'TRAIN (The Research Acceleration and Innovation Network) was established to create opportunities for medical research innovators to discuss and tackle the challenges that cut across diseases. TRAIN convenes dozens of forward-thinking foundations to share best practices, tools and other learnings to encourage collaboration within the biomedical research ecosystem. Our goal is to encourage more entrepreneurial philanthropy in medical research, build more and better networks between patient foundations and other R&D stakeholders, and enhance the influence of patient foundations on policy issues affecting the R&D system. Learn more about TRAIN today.
Patients organizations are at the forefront of integrating patient perspectives in the development and delivery of medical products and access to care. Now, a wider array of activities are informed by patient perspectives, including setting priorities for early-stage research, selecting outcome measures, making benefit-risk and value assessments, and developing care guidelines. In our most recent publication, FasterCures looks at the early benefits gained as patient-centric practices take hold. Read the full piece to learn more about the journey with case examples and stories of impact from patient organizations, like the Michael J. Fox Foundation for Parkinson's Research, the Leukemia & Lymphoma Society and more.
Advances in BRAF and immune checkpoint therapies significantly improved overall survival between 2000 and 2012 for patients with melanoma brain metastases who received first-line systemic therapy at Moffitt Cancer Center, according to a study published in Cancer.
Researchers who conducted a small case-control study in Puerto Rico found more compelling evidence linking the Zika virus infection to the onset of Guillain-Barre syndrome based on survey responses from 47 cases, according to a research letter in the Journal of the American Medical Association. The study analysis established three risk factors for GBS, consisting of severe illness two months prior to the onset of GBS symptoms, laboratory documentation of Zika infection and positive test results for Zika confirmed by a reverse-transcription polymerase chain reaction test.
Researchers at the Kaiser Permanente Research Bank are mapping genomic data from thousands of patients, which they combine with EHR data to discover new insights into complex genetic challenges. Nazneen Aziz, executive director of the biobank, says there is much work to be done, explaining that only about 5,000 of 20,000 protein coding genes are understood and that the previously overlooked intergenic region, or DNA sequences between genes, is important to understanding complex or multi-genetic diseases, such as diabetes and obesity.
San Francisco-based startup Vir Biotechnology, led by former Biogen chief George Scangos, raised more than $500 million from the Bill & Melinda Gates Foundation as well as venture capital funds, institutional investors, individuals and sovereign wealth funds. Vir is partnering with Stanford, Harvard and Oregon Health & Science universities, the Fred Hutchinson Cancer Research Center, Alnylam and Visterra on infectious disease drug development, and has acquired Switzerland-based Humabs BioMed.
A $7.5 million grant issued by Gilead Sciences will be shared by five institutions in a second round of funding for Gilead's HIV cure grants program. Grant recipients include the Dana-Farber Cancer Institute and University of Massachusetts Medical School.
The challenges of orphan drug development require different clinical trial approaches, such as cross-over studies and randomized withdrawal studies, said officials of the FDA's Center for Drug Evaluation and Research. They also stressed the importance of natural history studies and other preclinical preparation.
Clinical trials that incorporate patient and caregiver input into their design could spur higher participation rates and better returns on investment, but certain details must not be overlooked, Sean Collins and colleagues wrote in JAMA Cardiology. The paper provides a framework for patient-centric trial design based on recent experience and helps demystify the approach, FasterCures Senior Fellow Bray Patrick-Lake and colleagues wrote in an invited commentary.
Twenty Americans and UK citizens with a virulent form of herpes virus flew to the Caribbean vacation destination of St. Kitts and Nevis in 2016 to participate in an experimental herpes vaccine study that had not been sanctioned by the FDA or any institutional review board. The researcher who put together the study has since died, how and where the vaccine was made are unknown, and patients who experienced adverse effects have no recourse.
FasterCures is an action tank that works across sectors and diseases to improve the effectiveness and efficiency of the medical research enterprise. FasterCures, a center of the Milken Institute, is nonpartisan and independent of interest groups.
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