FY 2020 Annual Performance Plan and Report - Goal 4 Objective 3
Fiscal Year 2020
Released March, 2019
Topics on this page: Goal 4. Objective 3 | Objective 4.3 Table of Related Performance Measures
Goal 4. Objective 3: Advance basic science knowledge and conduct applied prevention and treatment research to improve health and development
HHS conducts, funds, and supports a broad and diverse portfolio of biomedical research in a range of scientific disciplines, including basic and translational research, to augment scientific opportunities and innovation for public health needs. HHS works to strengthen basic and applied science and treatment pipelines to assess potential health threats and bolster the fundamental science knowledge in these risk areas to expedite the development of therapies. As described in Strategic Objective 4.2, Expand the capacity of the scientific workforce and infrastructure to support innovative research, research is conducted ethically and responsibly.
The Office of the Secretary leads this objective. The following divisions are responsible for implementing programs under this strategic objective: ACL, AHRQ, CDC, FDA, NIH, and OASH.
Objective 4.3 Table of Related Performance Measures
By 2023, develop, optimize, and evaluate the effectiveness of nano-enabled immunotherapy (nano-immunotherapy) for one cancer type (Lead Agency - NIH; Measure ID - SRO-2.1)
|FY 2018||Optimize properties of 3 nanoformulations for effective delivery and antigen-specific response in immune cells.||Researchers developed, tested, and optimized, in animal models, 3 unique nanodelivery systems for effective anti-cancer immunotherapeutics.||Target Met|
|FY 2019||Further optimize top 2 candidate nanoformulations for co-delivery of multiple antigens to enhance anti-tumor response in one animal model.||12/31/19||In Progress|
|FY 2020||Further optimize the top candidate nanoformulation for co-delivery of antigens, adjuvants and immuno-modulators and evaluate its efficacy and long-lasting immunity (over 3 months) in preclinical models with established tumors.||12/31/20||In Progress|
Nanoparticles are extremely tiny particles that can coat, attach to, or encapsulate drugs. Nanoparticle drug delivery methods have been shown to alleviate some of the current limitations of and enhance the effectiveness of cancer drugs, which include immunotherapies. Therefore, NIH has launched several lines of research aiming to enhance existing immunotherapies with nanotechnologies or to facilitate the development of new, more efficacious nano-based immunotherapies.
In FY 2018, NIH-funded researchers developed and/or optimized delivery of nanoparticle-based drug formulations to cancer cells, which resulted in enhanced immune responses. One research team developed and optimized two unique nanoparticle drug delivery platforms. Both drug delivery systems cause an immune response that boosts the effectiveness of the immunotherapy tested. Another research team used nanotechnology to improve the effectiveness of a treatment combination using immunotherapy and radiation to treat cancer. They engineered a number of antigen-capturing nanoparticles (AC-NPs) and found the AC-NPs significantly improved the efficacy of the immunotherapy tested in a model of melanoma. In FY 2019 and 2020, NIH will continue to support the optimization and evaluation of novel nanoparticle drug formulations to further the clinical translation of nano-based drugs into medicine for treating cancer.
By 2022, evaluate the safety and effectiveness of 1-3 long-acting strategies for the prevention of HIV (Lead Agency - NIH; Measure ID - SRO-2.9)
|FY 2017||Strategy 1: Continue enrolling participants into two studies to test the safety, tolerability, and effectiveness of VRC01 as an intravenous prevention strategy.||Enrollment of participants continued for both studies.||Target Met|
|FY 2018||Strategy 2: Analyze primary results of a Phase 2a study examining the long-acting injectable, cabotegravir, for the prevention of HIV||Analysis of primary results has been conducted and results are in press.||Target Met|
|FY 2019||Strategy 3: Complete final analysis of an open-label extension study that builds on the findings of an earlier trial and aims to assess the continued safety of the dapivirine vaginal ring in a more real-world context and study participants’ adherence||12/31/19||In Progress|
|FY 2020||Strategy 1: Complete follow-up of participants in studies testing the safety, tolerability, and effectiveness of VRC01.||12/31/20||In Progress|
NIH-funded research has led to the identification of highly effective, non-vaccine prevention strategies that have the potential to significantly reduce HIV infection rates around the world. However, adhering to daily or near-daily dosing has proved challenging for both HIV-infected and uninfected individuals. Cabotegravir, an investigational drug that is available in long-acting injectable form, has shown promise for HIV prevention. In FY 2018, an NIH-supported study investigating the safety and acceptability of cabotegravir completed enrollment of 200 HIV-uninfected men and women in 8 cities in the United States and abroad. The results of the primary analysis are in press (to be published in the open-access journal PLoS Medicine), and the results of the extended phase have been analyzed and were presented at the HIV Research for Prevention 2018 Conference. This study represents an important next step in determining whether additional research should be done to assess the effectiveness of cabotegravir in preventing HIV infection. In FY 2019 and 2020, NIH will continue support clinical studies that assess the effectiveness of long-acting prevention strategies.
By 2020, identify risk and protective alleles that lead to one novel therapeutic approach, drug target, or pathway to prevention for late-onset Alzheimer’s disease (Lead Agency - NIH; Measure ID - SRO-5.3)
|FY 2014||Complete Discovery Phase whole genome sequencing and analysis of 582 family members from 111 families with late onset AD to identify genomic regions associated with increased risk of AD; sequencing of the coding regions of the DNA (whole exome sequencing) of 5,000 cases / 5,000 controls for both risk raising and protective loci; and whole exome sequencing and analysis of one individual from ~1,000 additional AD families to identify regions associated with increased risk or protection from AD.||Sequencing and an initial level of analysis were completed.||Target Met|
|FY 2015||Initiate Replication Phase to validate genes / regions of interest identified from case-control and family sequencing in ~50,000 samples from well phenotyped individuals by targeted sequencing and/or genotyping.||Sample selection for whole genome sequencing on additional multiply affected families was initiated. Planning of the Replication Phase has begun.||Target Met|
|FY 2016||Begin confirmation of genomic regions of interest identified in the Discovery Phase using samples from the Replication phase.
|Sample selection/sequencing Discovery Extension phases completed (4,000 additional whole genomes). Data analysis for Extension Phase initiated. Genomic Center for Alzheimer’s Disease funded (all ADSP quality control and data harmonization).||Target Met|
|FY 2017||Continue confirmation of genomic regions of interest in the Discovery and Replication phase datasets.
Continue harmonization of Discovery Phase and Replication Phase datasets.
|NIH met its target of confirming genomic regions of interest in the Discovery and Replication phase data sets and continues to harmonize the Discovery Phase and Replication Phase data sets.||Target Met|
|FY 2018||Continue confirmation of genomic regions of interest in the Discovery phase using samples from the Replication phase.
Continue harmonization of Discovery Phase and Replication Phase datasets.
Begin analysis of genomic regions of interest in the genomes of minority cohorts.
|NIH continued confirmation of genomic regions of interest in the Discovery Phase using samples from the Replication Phase, continued harmonization of Discovery Phase and Replication Phase datasets, and began analysis of genomes of minority cohorts.||Target Met|
|FY 2019||Begin analysis of genomic regions of interest in the ADSP Discovery Follow-Up Phase using whole genome sequence data from ethnically diverse cohorts.
Continue confirmation of genomic regions of interest in the Discovery Phase using samples from the Follow-Up phase.
Continue harmonization of Discovery Phase and Follow-Up Phase datasets.
|FY 2020||Continue analysis of ADSP Discovery Follow-Up Phase in ethnically diverse cohorts. Continue confirmation of genomic regions of interest from Discovery Phase and Discovery Follow-Up Phase in ethnically diverse datasets. Compare data on genomic regions of interest by ethnicity.||12/31/20||In Progress|
Effective interventions to prevent, delay, and treat Alzheimer’s disease (AD) are urgently needed. It is estimated that as many as 5.5 million Americans age 65 and older are living with AD, and many more under age 65 are also affected. Available treatments provide symptomatic relief and may slow symptoms of cognitive decline in some people for a limited time. However, they do not target the underlying molecular pathways believed to be involved in AD’s development; thus, they neither halt nor reverse disease progression.
In FY 2018, the NIH-supported Alzheimer’s Disease Sequencing Project (ADSP), the overall goal of which is to identify genetic variants associated with risk of and protection from AD, continued to confirm the involvement of previously-implicated genomic regions of interest in development of the disease. In addition, ADSP initiated additional genomic analysis to include racial and ethnic minority participants in the hopes of identifying risk factors that are associated with those groups. Using a combination of technologies, ADSP has identified a number of genetic “hubs” that may explain some of the basis for the development of AD. In FY 2019 and 2020, NIH will continue its efforts to identify risk factor genes associated with the disease.