Fiscal Year 2022
Released June, 2021
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, HHS conducts research is conducted ethically and responsibly.
In the previous administration, the Office of the Secretary led this objective. The following divisions are responsible for implementing programs under this strategic objective: ACL, AHRQ, CDC, FDA, NIH, and OASH. HHS has determined that performance toward this objective is progressing. The narrative below provides a brief summary of progress made and achievements or challenges, as well as plans to improve or maintain performance.
Objective 4.3 Table of Related Performance Measures
By 2021, develop, optimize, and evaluate the effectiveness of nano-enabled immunotherapy (nanoimmunotherapy) for one cancer type (Lead Agency - NIH; Measure ID - SRO-2.1)
|FY 2018||Optimize properties of three nanoformulation for effective delivery and antigen-specific response in immune cells.||Developed, tested, and optimized, in animal models, three unique nanodelivery systems for effective anti-cancer immunotherapeutics||Target Met|
|FY 2019||Further optimize top two candidate nanoformulation for co-delivery of multiple antigens to enhance anti-tumor response in one animal model.||Further optimized two unique nanodelivery systems for effective anti-cancer immunotherapeutics in different animal models and showed promising results for consideration in clinical trials||Target Met|
|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.||Further optimized two nanodelivery systems that were identified as the top candidates. Researchers are testing both systems in cancer patients who have advanced stages of cancer.||Target Met|
|FY 2021||Further optimize the top candidate nanoformulation for co-delivery of antigens, adjuvants and immuno-modulators and evaluate its efficacy towards near and distance metastatic lesions in preclinical models with established tumors.||12/31/21||In Progress|
Nanoparticles are extremely tiny particles that can coat, attach to, or encapsulate drugs. Scientists use nanoparticles in drug delivery systems to enhance the effectiveness of cancer drugs, which include immunotherapies. NIH supports research to enhance existing immunotherapies with nanotechnologies and facilitate the development of new, more efficacious nano-based immunotherapies.
Results from recent studies have shown that optimizing nanoparticle drug delivery systems improves the effectiveness of cancer immunotherapy. The optimization process of both drugs and delivery systems involves many different steps, which include testing the drug systems in different animal models and in different stages of disease (e.g., localized tumors and tumors that have spread to other parts of the body). In FY 2019, two NIH-funded research teams further optimized two unique nanoparticle drug delivery systems. Their work provides additional evidence that these systems are effective in delivering drugs to different tumor types and at different stages of disease, which include stages when the tumors have spread to other parts of the body. Building on these results, in FY 2020, the two research teams evaluated the delivery systems in cancer patients to identify the most tolerated dose with minimal toxicity. In FY 2021, the research teams are testing the effectiveness of these delivery systems in treating patients with advanced stages of cancer.
By delivering drugs directly to the tumor, nano-enabled immunotherapy will help overcome current limitations with immunotherapy, such as development of treatment resistance, relapse, low response rates, and systemic side effects. This measure highlights foundational proof of principle studies resulting in clinical trials that could lead the way for viable targeted drug delivery systems for cancer. Nano-enabled immunotherapies could provide safer, more efficacious, and less expensive alternatives to traditional immunotherapies and bring exciting new treatment options to groups of cancer patients.
This measure is scheduled to discontinue beginning in FY 2022, but improving the effectiveness of cancer immunotherapy delivery will remain a priority for NIH.
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: NIH-funded investigators 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||NIH-funded investigators completed final analysis of an open-label extension study that built on the findings of an earlier trial and aimed to assess the continued safety of the dapivirine vaginal ring and study participants' adherence to its use.||Target Met|
|FY 2020||Strategy 1: Complete follow-up of participants in studies testing the safety, tolerability, and effectiveness of VRC01.||NIH-funded investigators completed follow-up of participants in two studies testing the safety, tolerability, and effectiveness of VRC01.||Target Met|
|FY 2021||Strategy 1: Analyze data of two studies testing the safety, tolerability, and effectiveness of VRC01 broadly neutralizing antibody (bnAb).||12/31/21||In Progress|
|FY 2022||Initiate an open label extension of two studies, HPTN 083 and HPTN 084, investigating the safety and efficacy of the long-acting injectable antiretroviral drug cabotegravir (CAB).||12/31/22||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.
Since FY 2016, NIH has funded two proof-of-concept studies to assess whether giving people without HIV an infusion of VRC01, a "broadly neutralizing antibody" or bnAb (capable of stopping a wide range of HIV strains from infecting human cells), every eight weeks was an effective way to protect against HIV. In FY 2020, the research teams completed the studies in Sub Saharan Africa, the United States, and South America and demonstrated that VRC01 prevented the acquisition of HIV strains that were sensitive to the bnAb. However, VRCO1 did not prevent the acquisition of HIV strains that were resistant to the bNAb (nearly 70% of the circulating strains in these regions), and there was no overall prevention efficacy when the VRC01 subgroups were compared with the placebo subgroup.
In FY 2021, NIH will initiate an open-label extension of two studies to investigate the long-term safety and efficacy of cabotegravir, a long-acting injectable drug recently approved by the FDA for treating HIV infection in adults. (An open-label extension study enrolls participants of a previous clinical trial and is designed to gather information about the long-term safety and tolerability of a potential new drug beyond the time period of the previous clinical trial.) In FY 2022, NIH aims to complete enrollment of both of these studies. Having a long-acting method for prevention, whether a broadly neutralizing antibody or injectable drug, would help reduce adherence issues and give people a safe, more discreet, convenient option for preventing HIV.
By 2023, 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|
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 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.||The ADSP Discovery Follow-Up Phase has begun to analyze genomic regions of interest using whole genome sequence data from ethnically diverse cohorts. The ADSP has continued its confirmation of genomic regions identified in the Discovery Phase of the project. Genetic data for all phases of the ADSP have been harmonized.||Target Met|
|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.||Data analysis for the ADSP Discovery follow-up Phase continued. Ongoing data analysis includes analysis from genomic regions of interest in ethnically diverse cohorts with increased sample size and data comparison on genomic regions of interest by ethnicity.||Target Met|
|FY 2021||Continue analysis of ADSP Discovery Follow-Up Study in ethnically diverse cohorts. Continue confirmation of genomic regions of interest from Discovery Phase and Discovery Follow-Up Phase in ethnically diverse datasets. Begin harmonization of phenotypic data with ADSP genetic data across multiple types of study approaches from large epidemiology and clinical cohorts that are outside of the ADSP.||12/31/21||In Progress|
|FY 2022||Continue analysis of ADSP Discovery Follow-Up Study in ethnically diverse cohorts. Continue confirmation of genomic regions of interest from Discovery Phase and Discovery Follow-Up Phase in ethnically diverse datasets. Continue harmonization of phenotypic data with ADSP genetic data across multiple types of study approaches from large epidemiology and clinical cohorts that are outside of the ADSP. Begin analysis of ADSP genetic data using artificial intelligence approaches.||12/31/22||In Progress|
There is an urgent need for effective interventions to prevent, delay, and treat Alzheimer's disease (AD). As many as 5.5 million Americans age 65 and older are living with AD. Available treatments do not target the underlying molecular pathways believed to be involved in AD's development; thus, they neither halt nor reverse disease progression.
The overall goal of the NIH-supported Alzheimer's Disease Sequencing Project (ADSP) is to identify genetic variants associated with risk of and protection from AD. More than 150 investigators from institutions across the globe participate in the project. In FY 2020, the ADSP continued its efforts to identify and confirm genes associated with AD and examine them in ethnically diverse populations. Ongoing data analysis includes analysis from genomic regions of interest in ethnically diverse cohorts with increased sample size and data comparison on genomic regions of interest by ethnicity.
In FY 2021 and continuing into FY 2022, the ADSP is expanding on the results of its prior-year efforts. Upon completion, the project will provide critical information to enable NIH and AD researchers to explore new, promising pathways for preventing and treating AD.