FY 2019 Annual Performance Plan and Report - Goal 4 Objective 3
Fiscal Year 2019
Released April, 2018
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.||Dec 31, 2018||In Progress|
|FY 2019||Further optimize top 2 candidate nanoformulations for co-delivery of multiple antigens to enhance anti-tumor response in one animal model.||Dec 31, 2019||In Progress|
Immunotherapy approaches have shown potential to overcome some of the limitations associated with standard treatments for advanced cancers (e.g., toxicity in high-dose chemotherapy). However, treatment resistance, relapse, and low response rates limit the utility of current immunotherapies and/or hinder the development of new immunotherapies. Nanotechnology drug delivery methods have been shown to alleviate some of the current limitations of 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-immunotherapies. In recent years, NIH-funded investigators designed various nano-carriers capable of: more effective delivery of antigens and other biomolecules, controlling drug distribution within a patient’s body, and enabling co-delivery of immunotherapies with treatment modulators to improve efficacy. It is important to develop and test multiple nano-approaches to realize the effectiveness of nano-enabled immunotherapy.
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||Dec 31, 2018||In Progress|
|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||Dec 31, 2019||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, including: 1) treatment as prevention or TasP, in which antiretroviral drugs (ARVs) are used to treat HIV-infected individuals and reduce the possibility that they transmit HIV to an uninfected heterosexual partner; and 2) pre-exposure prophylaxis (PrEP), a strategy in which healthy people routinely take one or more antiretroviral drugs to reduce their risk of getting HIV.
Current oral ARV formulations require adherence to daily or near-daily dosing strategies for both HIV treatment and prevention, and this has proved challenging for both HIV-infected and uninfected individuals. Sustaining adherence over time also becomes increasingly challenging. Furthermore, research studies have found adherence to PrEP regimens particularly challenging for women, especially young women in sub-Saharan Africa, who in 2014, accounted for more than half of the 25.8 million people living with HIV in that region. PrEP may only reach its full potential with agents that do not depend on daily or near-daily pill taking.
Therefore, the development of alternative agents for PrEP, more adherence-friendly schedules for currently available agents, and long-acting injectable agents, as well as alternative interventions for women, such as an intravaginal ring, could go a long way in increasing the acceptability and adoption of prevention strategies and in reducing the number of new HIV infections.
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.
Begin harmonization of data from Discovery phase datasets with data from Replication Phase for confirmation of regions of interest.
|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 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.
|Dec 31, 2018||In Progress|
|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.
|Dec 31, 2019||In Progress|
Effective interventions to prevent, delay, and treat Alzheimer’s disease (AD) are urgently needed. Today, research reports estimate that as many as 5.1 million Americans may have the disorder, and the number is expected to rise as the population ages. Drugs currently in use for AD provide symptomatic relief and may slow symptoms of cognitive decline for some people for a limited time, but they neither halt nor reverse disease progression because they do not target the underlying molecular pathways believed to be involved in AD.
The advent of genome wide association studies (GWAS) and other high throughput technologies have facilitated the recent identification of risk factor genes for AD. This measure highlights the work of the NIH-funded Alzheimer’s Disease Sequencing Project (ADSP), the overall goal of which is to identify genetic variants associated with risk and protection for AD.