Emerging Innovations in the Study of Disease

It’s late in 2020, and the world needs good news regarding the study of disease. Fortunately, there’s plenty of it—in relation to COVID-19, cancer, Alzheimer’s Disease, and an array of other threats to health and longevity.

Read on for a sampling of emerging innovations in the study of disease.

Regenerative Medicine, Gene Editing and Beyond

In December 2019, the World Economic Forum listed several cutting-edge, emerging technologies it calls most likely to revolutionize healthcare over the next decade.

The list includes work in these areas:

Single-Cell Analysis

Expected to be one of the first breakthroughs to come to fruition, single-cell analysis will, for the first time, allow scientists to study individual cells in their normal environments. The ability to determine which genes are turned on or off in individual cells, and to decode how immune cells attack healthy tissue, can transform how we approach autoimmune diseases and combat the deadly process of cancer metastasis.

Alzheimer’s Disease

Aided by new imaging techniques developed and optimized by the National Institutes of Health’s Brain Research through Advancing Innovative Neurotechnologies (BRAIN) initiative, research indicates that, within a decade, we will be able to identify individuals at high risk of Alzheimer’s Disease before symptoms even appear. Early interventions will slow or change the course of the disease, providing profound human and economic benefits.¹

Regenerative Medicine

This field of research looks at ways of replacing or regenerating human tissues and organs when they are damaged. Methods range from stimulating the body’s own repair mechanisms to growing tissues and organs in the laboratory. In a decade, regenerative medicine could change the course of chronic diseases such as diabetes, and eliminate the problems associated with tissue and organ transplants—including sourcing, waiting lists, tissue rejection and the need for anti-rejection drugs.¹

Cancer Immunotherapy

This radical new approach enlists the cancer patient’s immune system, with one promising strategy involving the collection and engineering of immune cells to produce special cancer-fighters called chimeric antigen receptors. This work has already saved the lives of adults and children with untreatable blood cancers, and sights are set on tougher targets including breast, prostate, colon, ovarian and pancreatic cancer.¹

Gene Editing to Cure Disease

Scientists have identified the molecular causes of nearly 6,500 human diseases, yet treatments currently exist for only about 500. By 2030, science will have begun to realize the promise of genetic technologies to treat and cure diseases that once seemed out of reach. Gene editing tools such as Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR-Cas) allow the correction of gene mutations, with a cure for sickle cell disease being one of its first targets.¹

Polio

Once a disease feared worldwide, polio is nearing eradication, with only three countries remaining that have never interrupted the transmission of wild poliovirus (WPV).

According to Innovation for Eradication, the seventh report in a series on U.S. support for global polio eradication, the greatest challenge to the eradication effort is now vaccine-derived poliovirus. OPV, the primary vaccine in use, is a weakened live virus vaccine. In populations with low vaccine coverage, the vaccine-derived virus can be passed among unimmunized populations, mutating as it circulates until it can cause outbreaks with the same paralyzing effects as those of wild poliovirus.

Researchers with the Global Polio Eradication Initiative (GPEI) are now fast-tracking development of what is called novel OPV (nOPV), a more stable form of the oral vaccine that is less likely to mutate into a disease-causing state. Introduction of nOPV will be paired with expanded, simplified environmental surveillance techniques which can detect and categorize poliovirus in sewage samples. This will tell the GPEI where poliovirus is present and whether the sample is wild or vaccine-derived. It also will be able to show whether the correct vaccine is being used in a particular area and if programmatic changes are required to boost immunity.³

COVID-19

In the summer of 2020, the U.S. Food and Drug Administration (FDA) publicized several innovative pursuits in progress, aimed at ending the pandemic. They include:

The COVID-19 Diagnostics Evidence Accelerator

The FDA is participating in a multi-stakeholder collaborative project to advance the development of diagnostics. The Diagnostics Evidence Accelerator is organized by the Reagan-Udall Foundation for the FDA in collaboration with Friends of Cancer Research.

“While there are current studies of viral diagnostic and antibody tests using traditional assessment methods, the Diagnostics Evidence Accelerator will allow the community to analyze both diagnostic and clinical data in real time, which has the potential to contribute to the scientific evaluation of diagnostic tools and medical interventions for COVID-19,” said Amy Abernethy, M.D., PhD., FDA Principal Deputy Commissioner.

The Diagnostics Evidence Accelerator is a companion project to the Reagan-Udall/Friends of Cancer Research Therapeutic Evidence Accelerator, which brings together leading experts in health data aggregation and analytics in a unified, collaborative effort to share insights, compare results and answer key questions to inform the collective COVID-19 response.⁵

Study of Viral Infection in Cancer Patients

In partnership with experts in healthcare data and analytics, the FDA is investigating characteristics and clinical outcomes of patients with cancer who are infected with SARS-CoV-2, the virus that causes COVID-19, in order to improve understanding of the disease in this population.⁴

The Coronavirus Treatment Acceleration Program (CTAP)

A special emergency program for possible therapies, CTAP uses every available method to move new treatments to patients as quickly as possible, while simultaneously determining whether they are helpful or harmful. The FDA continues to support clinical trials testing new treatments for COVID-19, in the pursuit of knowledge about their safety and effectiveness.⁴

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