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.
T4 - Translation to Communities - Part II (10:09)
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来自 University of Rochester 的课程
Introduction to Translational Science
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从本节课中
T4 - Translation to Communities and Participating in Clinical and Translational Research
The fifth lesson of Introduction to Translational Science provides an introduction to T4 research, which is research that translates findings from practices (T3 research) to communities, both domestic and globally. T4 research is often conducted through prevention and outcome studies, mass screening studies, and health policy studies.The sixth lesson of Introduction to Translational Science provides an overview of how interested individuals can participate in clinical and translational research; as an independent research, as a clinical research professional, and as a study volunteer. The final segment of the module demonstrates what a typical study volunteer will encounter if they are selected to participate in a clinical trial.
与讲师见面
Martin S. Zand, MD, PhDProfessor of Medicine – Nephrology
School of Medicine
[MUSIC]
One way that the c-file will have a significant impact will be
simply recruiting subjects actually.
That turns out to be quite a challenge.
So in HIV research, in many of the clinical trials right now, the interest is
really on high-risk populations who are typically hard to reach.
So for example, the two groups with the highest incidence of new HIV
infection right now are young black men who have sex with men, or
MSM is the often known in the vernacular.
And the other group are transgender individuals.
Both of those groups are very hard to reach, and so one has to build trust.
One has to find out where do they receive care.
How do you approach them in a way that's respectful and
likely to engage effectively?
And how do you bring them in as participates, as partners, in research.
So that turns out to be sufficiently complicated.
That having the see far involved with that is a real plus.
And as an example, where currently working with the country health department,
because many people receive care in that context,
figuring out how to work with them in a partnership.
Where maybe we can provide some things that will be useful to the county and
in turn, have an ability to interface with some people
who would be potentially high risk individuals with respect to HIV.
So that's one example.
Another example, would be, maybe in some of the projects we have funded.
We have a couple of projects where we've make pilot awards that have helped
people develop data and get additional funding.
And so, a couple of examples of that, might be, Kroupa Shah, for example,
she's is in Geriatrics in the Department of Medicine.
She's very interested in its relatively simple concept,
but complicated to actually make it fly.
And that's an exercise intervention in people with HIV.
And the notion there is that, one often thinks of people, or
one thinks of HIV historically as a disease that's characterised by
people who have experienced weight loss.
And back in the day, more typically in Africa than perhaps in the U.S.
that was true in the latest stages of HIV.
But in fact most people with HIV in this country will have a relatively
higher body mass index.
So that's not in fact the real scenario but
as a group folks with HIV are experiencing the accelerated aging.
So they don't necessarily function at the same physical level as
an H-matched individual who might be HIV negative.
They have a relatively elevated body mass index.
Often they may have fat redistribution sometimes
as a consequence of the antiretrovirals they take.
And these sort of put together a picture where, as a group then,
they experience an elevated rate of frailty,
which is typically associated with aging, but it pops up at an earlier age.
And so how do you intervene with that?
And a classical way is that you intervene through some sort of exercise program.
But that's much easier said than done.
It's sort of like weight loss, right?
Many people would lie to lose weight but actually making that work not so simple.
And in the same way with exercise, how do you know that it's working?
How do you get people really engaged?
So in that case for example, we're using self-determination theory as set theory.
That was developed in large part here out at the U of Roch.
Not something, not exclusively here, but
this really was one of the places where that theory grew up.
And the notion is to give patients more autonomy.
And by having more autonomy and a greater sense of ownership, they're more likely to
buy into whatever it is that you believe would in fact be beneficial.
And so in this case that's exercise and the other place that that project would be
interesting to take that project is to use sensors.
So there's a lot of interest these days.
Using data science and taking devices that generate data from patients in
real time and tell you what's going on.
So in this case for example, if you had an exercise intervention it would be valuable
to know Are they actually exercising?
And you'd know that if there were wearable sensors involved.
So that's something we're sort of thinking about down the line for that project.
And then maybe a third project would be one that Handy Gelbod's very
involved with.
And so that one is developing a new drug to treat an aspect of HIV infection.
So we have a lot of antivirals for HIV right now, and for
most individuals, if they are in care, then one can control viral
load pretty effectively most of the time on antiretroviral meds.
And I should add that in fact,
most people are not [LAUGH] experiencing control of their HIV infection.
That's a big problem in the field.
So, our ability to reach people in the continuum of care is a real issue.
You have several hundred thousand people who don't even know their infected.
Then you have an enormous group may know their infected but
their not receiving any kind of care.
And so it's a small minority of about the 1.2,
1.3 million people in the US in who have HIV.
It's a small minority whose is really controlled, which is a separate problem.
But in any rate, in theory, we have the anti-retroviral drugs that,
if one could bring those individuals into care, one could treat them,
or one could prevent infection in individuals at high risk to
cut down the growth of the epidemic and really starve it, so to speak.
But, one of the other pieces then is, if one is infected with HIV,
there is a significant neurologic component to the disease.
So, for example, executive function is often impaired.
An executive function is kind of our internal to do list.
It's the thing that tells me to keep talking to you right now, instead of go
check my cell phone or whatever else I might feel like feel like doing.
And so if you have a relatively poor controlling your executive function,
your ability to sort of adequately manage the tasks of daily living,
becomes more difficult.
That form of the disease has not gone away,
even with highly active antiretroviral therapy.
We can control the virus, but
we can't roll the clock back in terms of the neurologic component and
that seems in fact to continue and maybe progress slowly.
Superimposed on that you have this sort of natural aging process as well.
We all get kind of forgetful as we get older.
So you sort of layer these things together and it becomes a concern.
And so what is interested in is an adjunctive therapeutic,
meaning something you would take in addition to anti-retroviral meds.
And this would really be geared towards the cognitive aspects of the disease.
So, how do you preserve cognitive function?
How do you make sure people are really going to be able to carry
out the activities of daily living and
have sort of that functional executive activity in their brain.
And so that's what he's been very interested in.
So we spent a long time figuring our molecular pathways that we thought
were relevant.
Pathways that are involved in both inflammation in the brain and
also in protecting neurons from inflammatory damage.
And so once we identified a pathway then we went down the road of identifying small
molecules that have the ability to perturb that pathway, and
sifting through those until you arrive at some that have the proper --pharmacologic
properties that you could probably use them in people, ultimately.
And so, right now, that went through some animal model testing to figure out,
does it work in an animal model?
And has now sort of moved forward into a company to spin that out.
because that's where you're going to be able to attract investment.
To really do these pre-clinical safety toxicity studies that are very costly.
They cost a couple of million dollars typically, sometimes more.
And you have to do all of that before you're able to get what's called an IND,
or Investigational New Drug.
One doesn't use the word approval, but nonetheless, authorization, perhaps,
from the FDA, the Food and Drug Administration.
And once you have that in place, then you could do your human trial,
assuming you had the money.
And this is a space, this space between basic science and a clinical trial,
there's a space that the NIH really doesn't invest in terribly much.
And the logic behind that is that this is a space that industry does invest in.
So the logic is that NIH gets out of the way and there's a hand off to industry.
But that hand off is very hit and miss.
Sometimes it works really well, other times it's difficult, and
typically you have to develop your intellectual property,
your lead compound a long way to where industry will actually want to pick it up.
Because what often happens is industry doesn't want to touch something that's
risky, they want to have it de-risked, ideally not on their dime.
And once it looks de-risked enough, then they might pick it up.
So typically you have what are called value inflection points meaning things
where something's been derisked and it's passed a hurdle.
So for example if you were rewarded an IND, that would've passed a hurdle.
You now know that the drug is safe in animals.
It hasn't got any genotoxicity or anything else.
There will be a flag, and so the value of the intellectual properties increased and
the likelihood industry might pick it up's increased, but
you had to spend a couple of million to get to that point.
So that's the reason that you often have to form a company and
get outside entrepreneurial investment, and that can take time.
You have to do a lot of dog and pony shows Kiss a lot of frogs and hopefully,
somewhere along the line, somebody will write you a big check.
Or not, as the case may be.
[MUSIC]
