We May Have Come So Far, But We Have Yet So Far To Go

The history of medicine, like so much of the history of everything human, is replete with examples of abuses of power (both physical and philosophical)—acts born of ignorance, stolen credit, and false credentials. One has only to recall the surgical methods developed by Dr. Marion Sims, an American physician, as he operated without anesthesia on enslaved Black women and the experiments conducted on the Tuskegee Airmen, Black military pilots who fought in World War II, from whom diagnoses were hidden and treatments were withheld all while considerable data were collected. So-called “studies” of many kinds conducted on specific ethnic, religious, or medically defined groups fill the annals of intra- and international affairs.

Since the Declaration of Helsinki, a statement of ethical principles for medical research involving human subjects adopted in 1964, we have truly come a long way. The ultimate aim and benchmarks of success were changes in international culture, the collective definition of common human decency, and proper conduct in regard to research on human subjects. You will note, however, that the practical road to that ideal began with regulations and policy; grassroots and committee-led enforcement of practice and reporting; and institutional and governmental oversight and enforcement of consequences for deficient practices.

But changing culture takes much longer. And rebuilding trust and establishing the kind of comfort level that allows people to speak out about important issues and to participate in a clinical trial or a research study takes even longer and requires human interaction on a personal level.

So, what has NINDS done to try to move the needle? How is NINDS making clear to people of color, and specifically Black people, that not only do their lives matter to us, their health, well-being, and voices as part of our NINDS family matter as well? Dr. Koroshetz, the NINDS director, has also published his thoughts in a new Director’s Message.

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May is Stroke Awareness Month

COVID-19 is on everyone’s mind these days and rightfully so. Everyone’s life has been turned upside down, with frequent handwashing, masks, working from home, and the closing of non-essential businesses. Doctors and scientists have learned so many new things from studying the novel coronavirus and the patients infected with it. You may be puzzled by how many of the body’s functions and organs appear to be affected by COVID-19, and, at first, doctors and scientists were too. But now they know that the normal body protein that allows the novel coronavirus to enter cells, ACE-2, is present on cells that are…well, just about everywhere in our bodies.

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What Is the Extramural Component of NINDS and How Does it Work?

In a previous column, I described NINDS as including the Office of the Director, and two components that serve, respectively, extramural and intramural research. In the next two columns, I will explain, in turn, how each of these components of NINDS work.

“Extramural research” occurs at institutions other than the NIH. NINDS and the other Institutes at NIH fund extramural research through grants, cooperative agreements, and contracts. In fact, NIH is the largest funder of “outside” (i.e., extramural) research in the world!

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COVID-19 and Those Served By the Mission of NINDS

While NINDS does our best to ensure that third party resources are vetted, the links below do not imply endorsement for any organization or product. We suggest that you research outside resources thoroughly before sharing.

The past two weeks have been very challenging for the whole world. A virus, a variation so new that none of us has immunity to it, has been infecting substantial fractions of the population and leading to the deaths of many people around the globe. We have learned new words, phrases, and abbreviations such as “COVID-19”, “social distancing”, and “PPE.” And we have put healthcare, facilities, and information technology professionals to the toughest test there is—one that puts their lives and lifestyles at risk to potentially save those of others.

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What exactly is “gene therapy”?

In recognition of Rare Disease Day, I thought I would spend some time discussing one of the promising avenues of treating rare diseases: gene therapy. You can learn more about NINDS efforts to address rare diseases from today’s post from NINDS Director Walter Koroshetz on his blog.

These days, you can hardly pick up a newspaper or listen to a radio without hearing something about gene therapy. Once the realm of science fiction, treatments that modify genetic material or its regulation are becoming a reality even as they are largely still experimental. But what is gene therapy; how do we separate the science from the myth; and what’s the big deal that prevents its being available to everyone who might benefit from it?

The term “gene therapy” refers primarily to three different kinds of treatments: treatment with sequences of genetic material called “antisense oligonucleotides”; insertion of a new gene into cells; and the editing of the genes that are already there. Each of these has their own potential applications and diseases and conditions for which they have been proposed as treatment. Each has different risks and side effects, some of which are yet unknown because they are largely experimental therapies. I will discuss each in turn.

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