The Big Picture - Part II (9:22)

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En provenance du cours de University of Rochester

Introduction to Translational Science

12 notes

University of Rochester

Introduction to Translational Science

12 notes

Translational science seeks to speed up the process of moving research discoveries from the laboratory into healthcare practices. Numerous scientific and organizational roadblocks can act as obstacles along the path of translation and ultimately hinder the speed of progress in medical research. The National Center for Advancing Translational Sciences (NCATS) was established by the National Institutes of Health (NIH) to transform and accelerate the translational research process, with the intended result of getting treatments to more patients faster. The field of Translational Science aims to bridge these gaps by: Developing new approaches, technologies, resources and models Demonstrating the usefulness of new approaches, technologies, resources and models Disseminating the resulting data, analyses, and methodologies to the broad scientific community Introduction to Translational Science is an introductory course that provides students with a broad understanding of translational science, the types of research that are conducted under the translational science umbrella, and how this research impacts the public at large.The course will compare and contrast current impediments to clinical research with the potential of translational science and will include selected case studies from the University of Rochester.

À partir de la leçon

Translational Science - The Big Picture and T1 - Translation to Humans

The first lesson in Introduction to Translational Science provides an overview of the basic definitions and key concepts related to clinical and translational science and the CTSA program. Students will learn about the different stages of translational research and the roadblocks that impede progress across the spectrum.The second lesson in Introduction to Translational Science provides an introduction to T1 research which is research that translates findings from basic (laboratory) research to humans through developing treatments and interventions. T1 research is often conducted through observational studies, case studies, proof-of-concept studies, phase I clinical trials, and phase II clinical trials

The Big Picture - Introduction (2:54)2:54

The Big Picture - Part I (7:41)7:40

The Big Picture - Part II (9:22)9:22

The Big Picture - Part III (5:20)5:20

The Big Picture - Part IV (7:00)6:59

Rencontrer les enseignants

Martin S. Zand, MD, PhD
Professor of Medicine – Nephrology
School of Medicine

All those therapies once approved,

are ready for translation into practice.

That's the T3 stage, Clinical Implementation Research.

The clinical implementation stage of translation

involves adoption of interventions that have been demonstrated

to be useful in a research environment into

routine clinical care for the general population.

These are the therapies that emerge into your doctor's office,

that diagnostic tests that emerge to define and diagnose disease at much earlier stages.

Everything from pregnancy testing to

genetic testing to looking at who responds to vaccinations,

to new drugs, new therapies for a whole spectrum of diseases, new diagnostic measures,

imaging, and medical devices like pacemakers and other diagnostic devices,

surgical devices for new operations.

All of those are included in Clinical Implementation Research.

Now, this stage involves several new types of investigations.

Those can include evaluating the results of the clinical trials.

Clinical trials are very focused and narrow

investigations under highly controlled situations.

You can think of T3 The Stage of Clinical Implementation Research into looking at

how those therapies perform when

applied to the general population who can benefit from them.

Also, new clinical questions and gaps in care can arise,

things that were not found in earlier T2 clinical trials can emerge.

Implementation research also might involve,

how best to deliver this care,

refining who those therapies apply to,

helping clinicians decide on standards for diagnosis and delivery of treatment.

All of these, sets the stage for thinking much bigger,

public health implementation, the T4 stage.

The T4 stage is about translating these therapies

into the health of the general public of communities,

of states, nations, and the world.

At this stage of translational research,

researchers study health outcomes at the population level.

We may have a great therapy for heart disease,

but does it help the whole population or just a segment of the population?

Does that therapy work in different areas of the world with different constraints?

How do we measure at a population level,

the effect of those therapies?

And how can we use those diagnostic methods to

prevent and diagnose illnesses so that we can treat them?

These findings also help guide scientists working to assess the effects

of current interventions and to loop back and develop new ones.

So while we think of

the T0 - T4 spectrum as a straight arrow progressing from one to the other,

each stage feeds back on the previous stages and it works to translate basic discoveries,

all the way up to population health implementation and improvement.

And at each stage,

we gain insights that can be applied forwards and backwards.

Organizes all of this.

In the United States,

that organizer is the National Institutes of Health.

The National Institutes of Health is the overriding Institute for Medical Research in

the United States and provides funding and guidance and

oversight in a whole spectrum of disease states.

There's the National Cancer Institute,

the National Eye Institute,

the National Institute for Aging,

the National Institute for dental and craniofacial research,

the National Institute for General Medical Science,

the National Institute for Medical Health and

the National Center for Advancing Translational Science.

The only Institute at

the National Institutes of Health with a verb in its name, advancing.

Let's talk a little bit about NCATS,

the National Center for Advancing Translational Sciences.

NCATS is an organization like any other organization.

There is a director who oversees a variety of

functions of the National Center for Advancing Translational Research.

There's an office of grants management and scientific review.

So, studies and centers that are funded by NCATS,

go through a review process where scientists and organizations submit

proposals for studies they would like to sponsor and research that they would like to do.

Now, many of these studies are organized by NCATS and

are used to direct and address national priorities for translational research.

For example, there's the Office of Rare Diseases Research.

The Office of Rare Diseases Research deals with

rare diseases and what are called orphan therapies.

Therapies that might not otherwise be developed without an intervention.

There's the Office of Policy Communications and Education.

And that office is responsible for developing educational material

to advance the capabilities of the National Translational Science Research workforce.

There's an Office of Strategic Alliances which

builds bridges between the foundation and industry.

There's the Division of Pre-clinical Innovation which looks at

innovations that could be moved from that the T1 - T2 stage.

There's the Division of Clinical Innovation that looks

at moving innovations from T2 - T3.

And of course, like any organization,

there's an Office of Administrative Management.

There are a variety of subprograms and initiatives that are sponsored by NCATS and

these are all organized around clinical and translational science.

Among the programs which I haven't mentioned,

there are some really cool stuff.

So for example, tissue chip testing for drug screening.

So, of course, we can't screen promising drugs for

rare diseases and any diseases that require treatment of cells in people right away.

We want to make sure that they're effective and safe and

we want to understand what they do to the cells.

So scientists have invented tissue on a chip.

These are tiny microchips that are

bioengineered to improve the process of

predicting whether drugs will be safe or toxic in humans.

They can be coated with cells,

they can be coated with cells from different tissues or proteins from different tissues,

and they can measure across a variety of tissues in this incredibly tiny device.

How those drugs affect processes that we're interested in treating?

There's also a Clinical and Translational Science Awards program.

This is a major program that funds scientific hubs, CTSA,

Clinical and Translational Science Awards at over 60 institutions across the country.

And this is the translational research highway that moves

discoveries from T0 all the way up to T4.

This structure was developed specifically for this purpose.

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