Wednesday, 22 April 2015

This Is What Sitting for Too Long Can Do to Your Body

With the continuous progress of technology, we need to put less and less effort to get the things done. And it’s not only about jobs which are getting more and more computerized every day; it’s about our lifestyle in general.

If in the recent past we had to wash the dishes and clean the floor ourselves, now there are people who have a dishwasher and a robotic vacuum cleaner which can do that for us. I’m not saying it’s bad to take advantage of the privileges of technology (I’m a fan of technology myself), but the problem is that, as a result, there are those of us who sometimes tend to avoid physical activity and end up sitting for more hours a day than our parents and grandparents did.

Let’s take a look at some statistics. Approximately 80% of the Americans use the Internet on a daily basis, and there is at least one computer in 90 % of the homes. Of course, it’s not only about the Internet usage or home computers. Imagine how many Americans do office jobs that require sitting in a chair for at least eight hours a day.

The real problem is that even after a long working day, many people continue spending their free time in front of a screen, watching TV, playing video games, browsing the Internet or chatting with friends online.

So when it comes to health problems, the statistics show that 40% of people who suffer from back issues tend to spend long hours sitting at the computer. You may think that back problems are the only major consequence of a sedentary lifestyle. Unfortunately, it’s not that simple, and recent research reveals that too much sitting is linked to a number of health problems, including obesity, heart disease and diabetes.

Here is an infographic that explains the health problems associated with spending too much time in a chair. There are some recommendations on the proper sitting position and exercises too.

I hope this information will help those who sit for many hours a day improve their lifestyle. 

Sunday, 19 April 2015

Are Health Apps Helpful? Steven Novella


There seems to be an app for everything, although we are just at the beginning of this new technology. It is a very recent phenomenon that many people in industrialized nations are walking around with a hand-held networked computer. This creates a new opportunity – to have constant access to applications that can help us run our lives. Even though there are already millions of apps, we really are just beginning to explore this opportunity.

One category of apps that seem to have a great deal of potential are health-related apps. There are apps to help people count calories, track their migraines, track their exercise, or even look up medical information or take a crack at diagnosing their own symptoms. There are apps to help you quit smoking or using alcohol, manage your medications, track your diabetes, or to provide some automatic therapy for mental illness.

The UK’s NHS maintains a list of approved health apps with hundreds of vetted apps you can browse.

In there are some useful ideas, and not-so-useful ideas. Trial and error will sort that out over time, and there are probably some killer health apps waiting to be developed.

How evidence-based are typical health apps, however? How would we even study that question?

A recent survey asked UK physicians, mostly primary care doctors, their personal experience with patients using health apps. The result are in line with my own personal experience. Basically, apps that help patients track or monitor their own health or habits are helpful. This is the low-hanging fruit of health apps.

However, apps that provide information or advice to patients are problematic. The same is true of any health information targeted to the general audience, whether in books, magazines, the internet, or now apps. Less than 5% of those surveyed found such apps helpful.

The problem is, such sources of information are rarely able to put the information into an individual medical context. What ends up happening is that patients match their own symptoms to possible diagnoses, and will often match the symptoms to some horrible fatal disease. This makes patients understandably anxious, and often may motivate them to request extra testing just for reassurance.

Not all symptoms are created equal, however. Some are non-specific and may match hundreds of possible conditions. Others require individual assessment – not every twitch is a Parkinsonian tremor. It takes years of training and experience to know how significant and predictive specific symptoms are, and how likely specific diagnoses are in turn.

I am not arguing that information is a bad thing. Rather, it is critical how information is presented. Presenting information in such a way that encourages or enables naive attempts at self-diagnosis is counterproductive. Further, useful resources will anticipate common misconceptions or misinterpretations, strongly counsel for caution in interpreting symptoms, and stress the need for expert opinion.

The possible benefits of general access to health information is that people will be better informed about their own health and theoretically will be able to make better decisions about how to manage conditions that can be managed at home, and when to consult a physician.

The possible harms are that such access to information will drive anxiety, false diagnoses, and unnecessarily increase use of medical resources.

There is another aspect to this, of which I have personally seen the positive and negative. Patients who come into an office visit armed with information they have read on the internet sometimes have a leg up, already have some useful background information, and ask well-informed questions. More often, however, they are filled with misinformation, or have questions about conditions completely unrelated to what they have.

It is difficult to estimate the net effect this has on the efficiency of office visits (something very critical to the overall efficiency of the health care system). I spend a lot of time undoing damage done by misleading websites.

The bottom line is that apps and websites that provide information are a double-edged sword. We are far from an optimal use of this technology, and there doesn’t even seem to be a way to control the information that is out there. Vetting and seals of approval are helpful, but these are difficult to execute and maintain, and often are infiltrated by special interests.

A thorough review of health apps is not possible here. A 2012 estimate put the number of health apps at 40,000.  There are already hundreds of papers assessing the utility of the apps. The ones that seem to be the most useful are those that enhance patient self-monitoring and behavior. Even there, it is not clear if they improve health outcomes.

Conclusion

Health apps are exploding in number and diversity, in a largely haphazard free-market way. There are some benefits to this model, and some useful apps are likely to emerge. I routinely recommend apps for tracking migraines, for example.

It will take time, however, to sort out the real effects of having a marketplace with tens of thousands of health apps, and which approaches are helpful and which are not. I do think health apps deserve more attention from researchers and healthcare experts. There is likely vast untapped potential there, but also pitfalls (I guess I can’t avoid the pun of the “killer app”).

Overall I’m excited by the possibilities, especially as the technology advances with the possibility of smart phone accessories that can actually monitor health parameters such as blood pressure, oxygen levels, blood sugar, and more. Monitoring and tracking are likely to be the most useful applications. Information will always be tricky – there is no app that can make a non-expert into an expert.

سائبر کرائم بل آزادی پر خطرناک حملہ کیوں ہے؟


گذشتہ چند ماہ پاکستان میں ڈیجیٹل حقوق کے لیے کام کرنے والوں کے لیے جدوجہد سے بھرپور رہے۔ یہ لوگ بھلے ہی ملک میں موجود طرح طرح کے سماجی کارکنوں کے سامنے اقلیت میں ہیں، لیکن ان کے سامنے اب پاکستان الیکٹرونک کرائم بل (PECB 15) موجود ہے، جسے منظور کروانے کے لیے حکام پوری کوشش کر رہے ہیں۔

فی الوقت یہ بل قومی اسمبلی کی قائمہ کمیٹی برائے انفارمیشن ٹیکنولاجی نے منظور کرلیا ہے، اور اب یہ آسانی سے آگے بڑھتا دکھائی دے رہا ہے۔

اس کا پس منظر ہمیں یہ سمجھنے میں مدد دے گا کہ یہ بل اتنی تیزی سے منظوری کے مراحل کیوں طے کرتا جا رہا ہے، اور یہ مجوزہ بل کیوں بحیثیتِ شہری ہمارے لیے اہمیت کا حامل ہے۔

دسمبر 2014 میں ڈیجیٹل حقوق کے لیے کام کرنے والے ایک گروپ 'بولو بھی' نے اسلام آباد ہائی کورٹ میں ایک پٹیشن دائر کی، تاکہ ویب سائٹس پر پائے جانے والے مواد کی جانچ پڑتال کے لیے قائم کی گئی بین الوزارتی کمیٹی (IMCEW) کی آئینی و قانونی حیثیت کو چیلنج کیا جا سکے۔

بولو بھی نے رائٹ ٹو انفارمیشن، یعنی جاننے کے حق کے تحت دائر کی گئی درخواستوں کے تحت یہ پایا تھا کہ IMCEW کی کوئی قانونی حیثیت نہیں تھی، لیکن پھر بھی وہ انٹرنیٹ پر موجود مواد کو بلاک کرنے کے احکامات جاری کر رہی تھی، چاہے وہ توہینِ مذہب پر مبنی یا غیر اخلاقی ہوں یا نہ ہوں۔

جب عدالتوں کو اس بات پر یقین ہوگیا کہ یہ کمیٹی غیر آئینی ہے، تو اسے مارچ میں تحلیل کر دیا گیا۔ لیکن جیسا کہ توقع ظاہر کی جا رہی تھی، ہمارے وزیرِ اعظم نے بیان جاری کردیا کہ پاکستان ٹیلی کمیونیکیشن اتھارٹی (پی ٹی اے) کے پاس اب آن لائن مواد چیک اور بلاک کرنے کا اختیار ہوگا۔ وزیرِ اعظم چیک اور بلاک کرنے کے مرحلے میں شفافیت اور جوابدہی کی عدم موجودگی کو نظرانداز کر گئے۔

پی ٹی اے کا قیام ایک پارلیمانی ایکٹ کے ذریعے عمل میں لایا گیا تھا، جس کا مطلب ہے کہ پی ٹی اے کو حاصل اختیارات کا تعین قانون کرے گا، نہ کہ وزیرِ اعظم کا جاری کردہ بیان۔

اور یہی وجہ ہے کہ اس بل کے بارے میں جاننا ہمارے لیے ضروری ہے۔

مجوزہ سائبر کرائم بل کی شق 31 کے مطابق پی ٹی اے کو

"اختیار حاصل ہے کہ وہ معلومات کے کسی بھی نظام (انفارمیشن سسٹم) کے ذریعے کسی بھی مواد تک رسائی کو ناممکن بنائے، یا اس مواد کو ہٹا سکے، اگر وہ سمجھے کہ ایسا کرنا اسلام کی عظمت، یا پاکستان یا اس کے کسی حصہ کی سالمیت، سلامتی یا دفاع، غیر ممالک کے ساتھ دوستانہ تعلقات، امن عامہ، تہذیب یا اخلاق کے مفاد کے پیشِ نظر یا توہینِ عدالت، کسی جرم [کے ارتکاب] یا اس کی ترغیب کے حوالے سے ضروری ہے۔"

ان مبہم الفاظ کے ذریعے پی ٹی اے کو تقریباً ہر طرح کا مواد ہی بلاک کرنے کی آزادی مل جائے گی۔

مثال کے طور پر امریکا یا سعودی عرب پر تنقید 'دوسرے ممالک کے ساتھ دوستانہ تعلقات' کے مفاد میں روکی جا سکتی ہے۔ اس طرح کوئی بھی اخبار، آن لائن میڈیا، یا سوشل میڈیا پر موجود کوئی بھی مواد فوراً بلاک کیا جا سکے گا۔

اس حوالے سے سپریم کورٹ کے کسی بھی حکم، یا اس مواد کی جانچ پڑتال کی ضرورت نہیں ہوگی کہ آیا وہ غیرقانونی اور نقصاندہ ہے بھی یا نہیں۔ اسے بس انٹرنیٹ سے ہٹا دیا جائے گا۔

ہم نے فہد حسین کے اماراتی وزیر کے بیان پر لکھے گئے کالم کے ساتھ ایسا ہوتا ہوا دیکھا ہے۔ آج اگر کوئی مواد بلاک ہوتا ہے، تو ہم اسے چیلنج کر سکتے ہیں، لیکن ایک دفعہ یہ بل پاس ہوگیا، تو
ہم اس حوالے سے کچھ نہیں کر سکیں گے۔

میں اس حوالے سے کئی مثالیں پیش کر سکتی ہوں، کہ اوپر دی گئی وجوہات کو بنیاد بناتے ہوئے کیا کیا بلاک یا بین کیا جا سکتا ہے، لیکن ہم پہلے ہی جانتے ہیں کہ پاکستان میں قومی سلامتی کے لیے نقصاندہ، یا دہشتگردی قرار دے کر کیا کیا چیزیں بلاک کی جا چکی ہیں۔

اور آن لائن مواد کو بلاک یا اس پر پابندی کے خلاف اپیل کا آئینی حق ہم سے چھیننے کے لیے حکومت Prevention of Electronic Crimes Act 2015 پاس کرنا چاہتی ہے، جس کے ذریعے پی ٹی اے کسی بھی طرح کے مواد کو معلومات کے کسی بھی ذریعے (موبائل فون، ٹی وی، ایکس باکس، پلے اسٹیشن وغیرہ) پر بلاک کر سکتی ہے، اور آپ اس حوالے سے کچھ نہیں کر سکیں گے۔

لیکن چیلنج کرنے کے ہمارے حق کے علاوہ اس بل نے ہماری آزادیء رائے اور اظہار پر بھی پابندی لگانے کا طریقہ ڈھونڈ لیا ہے۔

آئینِ پاکستان کا آرٹیکل 19 کہتا ہے کہ

"اسلام کی عظمت، یا پاکستان یا اس کے کسی حصہ کی سالمیت، سلامتی یا دفاع، غیر ممالک کے ساتھ دوستانہ تعلقات، امن عامہ، تہذیب یا اخلاق کے مفاد کے پیشِ نظر یا توہینِ عدالت، کسی جرم [کے ارتکاب] یا اس کی ترغیب سے متعلق قانون کے ذریعے عائد کردہ مناسب پابندیوں کے تابع، ہر شہری کو تقریر اور اظہارِ خیال کی آزادی کا حق ہوگا، اور پریس کی آزادی ہوگی۔"

یہاں پر یہ بتانا ضروری ہے کہ انسانی حقوق کی ایک مشہور ویب سائٹ Article19.org پاکستان کے ایک مخصوص انٹرنیٹ سروس پرووائیڈر کی جانب سے بلاک کی جاچکی ہے، اور جب ان سے پوچھا گیا، تو ان کا کہنا تھا کہ ایسا پی ٹی اے کے احکامات پر کیا گیا۔ جبکہ یہ بل کی منظوری سے پہلے 15 اپریل 2015 کو تھا۔

آخر کیسے آزادی اظہار کے لیے کام کر رہی کوئی ویب سائٹ اسلام کی عظمت یا ملکی سلامتی، دفاع، اور خودمختاری کے لیے خطرہ بن سکتی ہے؟ جواب یہ ہے کہ ایسا کچھ بھی نہیں ہے، بلکہ اس سے صرف حکومت کو تکلیف ہوتی ہے، اور بس۔

یہ مجوزہ بل یہی کرے گا؛ یہ ہم سے اپنی رائے کے آزادانہ اظہار، لکھنے، کارٹون بنانے، خاص طور پر سیاسی کارٹون بنانے، یا کسی کی اجازت کے بغیر تصاویر اپ لوڈ کرنے کو ناممکن بنا دے گا۔

اور صرف یہی نہیں، آپ کسی شخص کی اجازت کے بغیر اسے ٹیکسٹ میسیج یا ای میل تک بھی نہیں بھیج سکیں گے۔ سائبرکرائم کے اس سیاہ قانون کا تازہ ترین ڈرافٹ پڑھیے، اور آپ خود جان جائیں گے۔ دنیا کے دیگر ممالک میں اسپیمنگ، یعنی بلا اجازت پیغامات بھیجنے کو مختلف طریقے سے ہینڈل کیا جاتا ہے، اور سزا صرف تب دی جاتی ہے جب ایسا تجارتی مقاصد کے لیے کیا جائے، لیکن یہاں پر معاملہ بالکل الٹ ہے۔

اور اگر آپ پر اس بل کے تحت کوئی الزام عائد کیا جاتا ہے، تو یا تو وارنٹ صرف رسمی ہوگا، یا پھر ہوگا ہی نہیں۔ اس بل میں وارنٹس کے متعلق الفاظ اتنے مبہم ہیں، کہ ایسا لگتا ہے کہ انہیں صرف رسماً شامل کیا گیا ہے تاکہ یہ تاثر دیا جا سکے کہ دفاع کرنے والوں کو شفاف ٹرائل کا حق حاصل ہوگا۔

قانون کے تحت انٹرنیٹ سروس پرووائیڈرز 90 دن کے لیے آپ کا ڈیٹا محفوظ رکھنے کے لیے پابند ہوں گی، اور اس لیے انہیں آپ کی براؤزنگ ہسٹری، یا دیگر ایسی معلومات جسے ہم کبھی ذاتی اور خفیہ سمجھتے تھے، ظاہر کرنے کے لیے مجبور کیا جا سکتا ہے۔ اور اگر آپ پر اس قانون کے تحت کیس ہوجائے، تو آپ کو تفتیشی حکام کو اپنے تمام ڈیٹا، چاہے آن لائن ہو، یا آپ کے کمپیوٹر یا دیگر ڈیوائسز میں موجود ہو، رسائی دینی پڑ سکتی ہے۔


ڈیٹا کے تحفظ کے کسی قانون کی عدم موجودگی، اور مبہم الفاظ پر مشتمل ایک سائبر کرائم قانون کو متعارف کروانا ملک میں شہری آزادیوں اور کاروباروں کے لیے نقصاندہ ثابت ہوگا۔

مجوزہ بل میں موجود یہ شقیں تقریر کی آزادی، اظہار کی آزادی، میڈیا کی آزادی، بلاوجہ تلاش اور گرفتاری سے تحفظ، اور کاروبار کی آزادی چھین لیتی ہیں۔ اس بل کی شکل و صورت سے میڈیا آؤٹ لیٹس بھی اس قانون کا نشانہ بن سکتے ہیں۔

اس بل کو ڈرافٹ کرنے والے شاید یہ بھول گئے ہیں کہ پاکستانی شہری کو کچھ حقوق حاصل ہیں، اور ان حقوق کی خلاف ورزی کرنے والا کوئی قانون پاس نہیں کیا جا سکتا۔ لیکن اس بل کا اسمبلی فلور تک پہنچنا تشویش کا باعث ہے۔

ایسا لگتا ہے کہ قانون سازوں کو اس بات کا اندازہ نہیں ہے کہ یہ قانون ہمارے آئینی حقوق کے خلاف ہوگا۔ یہ بہت اہم ہے کہ ہم بطورِ شہری اس بات کو سمجھیں کہ اس بل کے ساتھ مسئلہ کیا ہے، اور اس بل کی حمایت کسی صورت بھی نہ کریں۔

ہمیں بولنا ہوگا۔ آخر اب نہیں، تو کب؟

Wednesday, 15 April 2015

FDA and Homeopathy

FDA( Food and drug administration) and Homeopathy



The skeptical community is abuzz with the announcement by the FDA’s announcement that they are reviewing the “regulatory framework” of homeopathic products and are open to public input. We have written about this at Science-Based Medicine, and as you can imagine, this is a serious topic of discussion among the editors.

Background

The FDA regulates food, drugs, medical devices, supplements, and cosmetics for the purpose of protecting the public health and safety. Congress created the FDA and determines its powers. In the 1938 FDA act, one Senator, Royal Copeland, who was a physician and homeopath, included in the bill that the provision that the Homeopathic Pharmacopeia of the United States (HPUS) would be included in the list of official drugs.

What this means exactly is that homeopathic products are automatically considered drugs by the FDA. Further, any new homeopathic product added to the HPUS in a supplement also counts. All homeopaths have to do, therefore, to get a homeopathic product listed as a drug by the FDA is write it down in one of their supplements to the HPUS. That’s it. No research is necessary, no assurance of safety or efficacy.

However, that does not mean that the FDA has to approve the homeopathic remedy. They have the authority to require evidence for safety and efficacy, just like they do with real drugs. They simply chose not to enforce this authority. Apparently they thought it wasn’t worth the trouble 70 years ago so they simply outsourced the regulation of homeopathy to the homeopaths.

I can see the logic behind this. At the time homeopathy was such a small industry, and their products are literally just water or sugar pills, so why waste limited FDA resources?

However, homeopathy has grown into a multi-billion dollar industry. I would argue it is a multi-billion dollar scam. Pharmacies sell homeopathic potions on the shelf right next to real medicines, with claims that they treat specific diseases. Most people don’t even know what homeopathy means, and may not look for the small print indicating that the cold remedy they are buying is homeopathic.

Better Late than Never

Now, finally (I hope), the FDA has apparently realized that they are completely neglecting their mandate to protect the public from harmful or worthless medical products when it comes to homeopathy. If I would guess, I think the FDA understands that homeopathic products are utterly worthless pseudoscientific nonsense. They can’t possibly work, and when studied they don’t work.

If they couldn’t figure this out by themselves, the UK government recently did a massive review and concluded that homeopathy is nothing more than witchcraft. More recently the Australian government did a similar review and found that homeopathy does not work for anything.

There are indications that the FDA is serious about reform. They actually contacted the Committee for Scientific Investigation (CSI) and asked them to send an expert skeptic to their public hearing. There is also a period for written public commentary, and we at SBM are preparing a statement (which we will publish as an open letter also, so stay tuned).

Here’s the preview, however – it seems to me that the simplest thing for the FDA to do is to simply do their job. They have the authority to regulate homeopathic products like drugs, so do it. Don’t outsource to the homeopaths. Require evidence for safety and efficacy for all homeopathic products. It that’s simple. If they did that, homeopathic products would disappear overnight.

Well, I suspect they would give manufacturers a grace period of 1-2 years to provide such evidence, and if they didn’t provide adequate evidence by the deadline their products would be removed from the pharmacy shelves.

The homeopaths, of course, are going to fight back. What, however, are they going to say? Up to now they have been claiming that homeopathy works, and many even claim that the evidence shows that it works. Now they either have to admit that there isn’t evidence showing that it works, or that the FDA requiring evidence is OK.

Their only other option is to make some sort of conspiracy claim, that some nefarious force is using the FDA to shut down homeopathy. So, of course, I suspect that is what they are going to do. They will complain that homeopathy can’t be studied like regular drugs, and existing studies are not fair or adequate.

The thing is – they actually make the case for the FDA properly regulating homeopathic products. Homeopaths argue that you need a homeopath to individualize a remedy for each patient. While this is BS, if it were true that would be an argument against the over-the-counter homeopathic industry. In fact homeopaths routinely throw the homeopathic product industry under the bus to explain away the negative clinical trials of homeopathy.

Therefore, by their own logic, the OTC homeopathic industry is unscientific and is selling ineffective nostrums. So let the FDA regulate them away. The FDA does not have the power to regulate the practice of medicine, just the sale of medical products.
Conclusion

I hope that this move by the FDA is the historic opportunity it appears to be – meaning that the FDA is sincere and will listen to reason. Homeopathy is a scam that has been operating under the nose of the FDA for decades. They can end it with a stroke of the pen.

Now is the time to put the pressure on.

FDA – do the right thing.

Tuesday, 14 April 2015

Transatlantic lessons in regulation of mitochondrial replacement therapy

Transatlantic lessons in regulation of mitochondrial replacement therapy.

Mutant mitochondrial DNA (mtDNA) gives rise to a broad range of heritable clinical syndromes (1). A cure for those affected remains out of reach (1). However, recently developed mitochondrial replacement therapy (MRT) has raised the prospect of disease-free progeny for women carriers (2–4). Moreover, the feasibility of replacing mutant oocytic or zygotic mtDNA with a donated wild-type counterpart in humans has now been firmly established (2–4). In the United Kingdom, legislation regulating the clinical application of MRT, now 10 years in the making, has recently been approved by the House of Commons (5) and the House of Lords (6). The regulatory vetting of MRT in the United States, under way for a year, remains a work in progress (7). Here, we compare and contrast the regulatory history of MRT in the United Kingdom and the United States and examine potential lessons learned.
THE UK REGULATORY EXPERIENCE. In the United Kingdom, matters relating to reproductive technologies are wholly governed by the Human Fertilisation and Embryology Authority (HFEA), an independent regulatory agency established by Parliament through the Human Fertilisation and Embryology Act of 1990 (HFE Act). Among its responsibilities, the HFEA licenses and monitors human embryo research. In 2005, the HFEA issued a research license to the Newcastle Centre for Mitochondrial Research to “investigate the feasibility of using IVF-based techniques to prevent transmission of mitochondrial disease” (8, 9). At that time, the conduct of clinical trials of MRT was prohibited by the HFE Act. However, in 2008, Parliament amended the law to empower the HFEA to license and monitor clinical trials of MRT subject to subsequent parliamentary approval.

In 2011, the HFEA convened an expert panel to review the “effectiveness and safety of mitochondrial transfer” (10). The panel concluded that the “evidence currently available does not suggest that the techniques are unsafe” (10). Updated reports issued in 2013 and 2014 reached similar conclusions. In 2012, the HFEA launched a public consultation process on the social and ethical implications of MRT, the outcome of which revealed “general support for permitting mitochondria replacement in the UK, so long as it is safe…and is done so within a regulatory framework” (11). The Nuffield Council on Bioethics and the Ethics Committee of the British Medical Association reached similar conclusions. On 3 February 2015, the House of Commons voted to approve the conduct of MRT under the auspices of the HFEA (5). The House of Lords followed suit on 24 February. Clinical trials could commence as early as 29 October 2015.
Ethical questions discussed in the United Kingdom revolved around the safety of the procedure for newborns, the matter of parenthood, and the notion that MRT represents germline modification and thus crosses a “red line.” Although a full review of the ethical discussions in the United Kingdom is outside the purview of this policy piece, we would point out that several members of Parliament noted that MRT is more about changing a “battery pack” than it is about genetic modification (5). Although the term “three-parent” reproduction has been used in the public debate, as per the recent parliamentary vote, mitochondrial donors have not been accorded legal parental status. It follows that the identities of prospective donors are not to be disclosed to “mitochondrial donor-conceived persons” (5).
On the matter of germline modification, during the House of Commons and House of Lords debates, some MRT opponents suggested that the legislative action under consideration would violate the European Union Directive on clinical trials that states that “[n]o gene therapy trials may be carried out which result in modifications to the subject's germ line genetic identity.” Proponents who won the day argued that HFEA approval of MRT would not violate this directive and/or that the directive did not apply to MRT, but their view is thus far untested in court (5).
THE U.S. REGULATORY EXPERIENCE. The United States, unlike the United Kingdom, lacks a specific governmental entity whose sole charge it is to regulate reproductive technologies. In the case of MRT, jurisdiction has been asserted by the Food and Drug Administration (FDA) Office of Cellular, Tissue, and Gene Therapies of the Center for Biologics Evaluation and Research, whose task it is to oversee “human cells used in therapy involving the transfer of genetic material by means other than the union of gamete nuclei” (12). Under FDA's existing regulations, approval of the therapeutic use of MRT will require the conduct of phased clinical trials pursuant to an Investigational New Drug application (IND) (12).
The FDA has never officially considered or approved early-phase clinical trials of MRT. In fact, its 2 July 2013 draft guidance for industry for the “design of early-phase clinical trials of cellular and gene therapy products” (published for public comment) makes no mention of MRT (13). The first tremor in this status quo was felt early in 2014 when the FDA convened the Cellular, Tissue, and Gene Therapies Advisory Committee to discuss “oocyte modification in assisted reproduction for the prevention of transmission of mitochondrial disease” (7). Although there was no official conclusion, Reuters quoted the chairman of the committee as summarizing the session in the following way: “Several panelists felt ‘there was probably not enough data in animals … to move on to human trials without answering a few additional questions’” (14).
The FDA has since commissioned an ad hoc committee of the Institute of Medicine (IOM) to weigh in on the “Ethical and Social Policy Considerations of Novel Techniques for Prevention of Maternal Transmission of Mitochondrial DNA Diseases” (15). Two of the planned committee sessions will be open to the public. No further FDA action is expected until the IOM report has been released, which is estimated to be 19 months after the start date of September 2014. In the interim, any relevant INDs submitted would remain on hold.
The recent debate over MRT in the House of Commons and elsewhere went well beyond safety considerations to explore ethical arguments and matters of conscience. In the United States, some of these same issues have been discussed in the popular press, but a full consideration of the ethics and not just the science of MRT in the United States has yet to occur.

TRANSATLANTIC LESSONS. The regulatory paths of the United States and the United Kingdom toward MRT have diverged considerably, with the United Kingdom clearly further along in the decision-making process, having approved the clinical use of MRT. Why is there such a transatlantic difference? This is not an academic question, because MRT represents but one of a growing complement of novel reproductive technologies, many of which will require expert regulatory adjudication.
The first transatlantic distinction of note concerns the regulatory agency charged with overseeing extant and leading-edge reproductive technologies. In the United Kingdom, the regulatory adjudication of MRT has been relegated to the HFEA. Absent a comparable U.S. agency, the task of regulating MRT has fallen to the FDA, the mandate of which encompasses all therapeutics. Even the FDA Office of Cellular, Tissue, and Gene Therapies—the designated proximate overseer of MRT—is entrusted with a diverse portfolio of cellular, tissue, and gene therapeutics. It follows that UK regulators, unlike their U.S. counterparts, view MRT as a circumscribed outgrowth of related and highly familiar technologies (e.g., in vitro fertilization) rather than as a therapeutic (5). In contrast, U.S. regulators are proceeding on the premise that MRT constitutes a drug and/or a biological product. We posit that the narrow framing of MRT by the HFEA is partially responsible for the expert nature of the UK regulatory paradigm.
The second transatlantic dissimilarity harkens back to the prevailing national values and mores regarding reproduction and especially research involving the derivation and destruction of a human embryo. In the United States, scientific research with human embryos has long been controversial, and federal funding in this area has been constrained. This is in part due to the fact that, in the United States, such research has been tangled in electoral and religious contestation over abortion. No such impediments cloud the regulatory adjudication of MRT in the United Kingdom, where public funding for human embryo–derived stem cell generation and human embryo research through day 14 of development has been legalized.
The third material transatlantic difference concerns the relative weight assigned to public consultation on regulatory issues of substance. The UK public consultation process was an extensive outsourced multimethod (e.g., surveys and workshops) effort on a national scale lasting 6 months. The U.S. regulatory approach has thus far been largely limited to a conversation among experts, with relatively brief sessions open to the public (7).
A fourth transatlantic variance revolves around the framing of MRT as a beacon of national scientific prowess. For better or worse, the parliamentary debate has proceeded with an air of national pride. Even those opposed to MRT noted their admiration for the worldclass work of the Newcastle group (2, 5). We believe that this national sense of pride may have swayed some votes in support of MRT. No such sentiment has been sweeping the United States, even though U.S. scientists have made equally vital contributions to this field of inquiry (3, 4).
CONCLUSIONS. This examination of the different approaches taken to the regulation of MRT in the United Kingdom and the United States leads us to reexamine the wisdom of burdening the FDA with the regulatory adjudication of MRT, as opposed to adopting an HFEA-like paradigm (16). In the eyes of some, the regulatory oversight of reproductive technologies in the United States leaves much to be desired. Yet others are content with the status quo, in which reproductive technologies are not directly licensed (as in the United Kingdom) but instead are left to what can be characterized as self-regulation by the medical profession and its representative associations. However, with the MRT challenge looming and others not too far behind, it may be time to renew the national conversation as to the rules that should govern this terrain. Understandably, the outcome of such conversation is far from certain. Because some forms of MRT involve embryo destruction, approval in the United States will be embroiled in the prolife/prochoice divide (17–19). It remains an open question whether an initiative to reform the regulatory oversight of reproductive technologies in the United States can be realized without capsizing under its own weight and the force of the political winds.
References and Notes

↵ O. Russell, D. Turnbull , Exp. Cell Res. 325, 38 (2014). CrossRefMedlineWeb of ScienceGoogle Scholar
↵ L. Craven et al ., Nature 465, 82 (2010). CrossRefMedlineWeb of ScienceGoogle Scholar
↵ M. Tachibana et al ., Nature 493, 627 (2013). CrossRefMedlineWeb of ScienceGoogle Scholar
↵ D. Paull et al ., Nature 493, 632 (2013). CrossRefMedlineWeb of ScienceGoogle Scholar
↵ www.parliament.uk, Parliamentary business; Publications & records; Human Fertilisation and Embryology, 3 February 2015; http://www.publications.parliament.uk/pa/cm201415/cmhansrd/cm150203/debtext/150203-0002.htm#15020348000001
↵ G. Vogel , Mitochondrial gene therapy passes final U.K. vote, Science, ScienceInsider, 24 February 2015,; http://news.sciencemag.org/biology/2015/02/mitochondrial-gene-therapy-passes-final-u-k-vote
↵ U.S. Food and Drug Administration, Advisory Committees, 2014 Meeting Materials, Cellular, Tissue and Gene Therapies Advisory Committee, 25–26 February 2014; www.fda.gov/AdvisoryCommittees/CommitteesMeetingMaterials/BloodVaccinesandOtherBiologics/CellularTissueandGeneTherapiesAdvisoryCommittee/ucm380047.htm
↵ Human Fertilisation and Embryology Authority, HFEA grants licence to Newcastle Centre at LIFE for Mitochondrial Research, 8 September 2005; www.hfea.gov.uk/671.html
↵ Human Fertilisation and Embryology Authority, Newcastle Fertility Centre at Life, R0152: Pluripotency, reprogramming and mitochondrial biology during early human development, 24 September 2014; www.hfea.gov.uk/1564.html
↵ Human Fertilisation Embryology Authority, Scientific review of the safety and efficacy of methods to avoid mitochondrial disease through assisted conception (2011); www.hfea.gov.uk/docs/2011-04-18_Mitochondria_review_-_final_report.PDF
↵ Human Fertilisation and Embryology Authority, Mitochondria replacement consultation: Advice to Government, March 2013.
↵ U.S. Food and Drug Administration, Vaccines, Blood, and Biologics. Letter to Sponsors/Researchers, Human cells used in therapy involving the transfer of genetic material by means other than the union of gamete nuclei, 6 July 2001; www.fda.gov/BiologicsBloodVaccines/SafetyAvailability/ucm105852.htm
↵ Department of Health and Human Services, Food and Drug Administration, Fed. Regist. 78, 39736 (2013); www.gpo.gov/fdsys/pkg/FR-2013-07-02/pdf/2013-15797.pdf
↵ S. Begley , U.S. FDA weighs evidence on producing ‘three-parent’ embryos, Reuters, 25 February 2014; http://uk.reuters.com/article/2014/02/25/us-usa-health-ivf-idUKBREA1O1WL20140225
↵ The National Academies, Current Projects System, Ethical and Social Policy Considerations of Novel Techniques for Prevention of Maternal Transmission of Mitochondrial DNA Diseases (2014); www8.nationalacademies.org/cp/project.

Monday, 13 April 2015

Ultrasound for Alzheimer’s disease Steven Novella

Ultrasound for Alzheimer’s Disease
 Steven Novella.


A new study published in Science Translational Medicine concerning a possible new treatment for Alzheimer’s disease is getting quite a bit of play on social media. While it is an interesting study, and excitement over any scientific study is great to see, I also think it’s important to always put such studies into a reasonable context (which is rarely done well).

Alzheimer’s disease (AD) is a devastating chronic degenerative brain disease in which neurons slowly die over years, causing memory loss of eventually overall cognitive impairment. Nancy Reagan described her husband Ronald’s AD as a “very long goodbye.” My grandmother died of AD, and it’s likely many of her relatives did as well, but in her it was confirmed at autopsy. About 75% of all dementia cases are due to AD:

The pooled data of population-based studies in Europe suggests that the age-standardized prevalence in people 65+ years old is 6.4 % for dementia and 4.4 % for AD.3 In the US, the study of a national representative sample of people aged >70 years yielded a prevalence for AD of 9.7 %.

In short, this is a common and serious disease. Most people will have a family member or know someone with AD. Further, as our population ages the incidence of AD will increase as a matter of course.

At present there is no cure for AD or treatment that significantly alters the course of the disease. Current medications are symptomatic, moderately increasing function but not altering the slope of cognitive decline. Research is making steady progress, but the disease has proven to be very complex. Researchers now recognize that AD is a result of a complex combination of genetics, environmental, and lifestyle factors. It has also been well established that a prominent feature of AD is the collection of Amyloid Beta protein, which is normally soluble. The protein then undergoes misfolding, and the misfolded protein clumps together, resulting in toxicity that eventually kills the neuron. That is not the whole story, however, and researchers are still trying to tease out further details.

Because there is so much active research into AD I frequently see press releases promising some sort of AD breakthrough – a new piece to the puzzle falling into place, a new way to diagnose AD early, or a potential new treatment approach. These are often genuine scientific advances, but they are incremental, and rarely directly lead to a new therapeutic intervention, or still require years of research to see how they will pan out. It’s easy, however, to get caught up in the headlines promoting every new tiny advance.

The latest research from Science is a good example. The study is, Scanning ultrasound removes amyloid-β and restores memory in an Alzheimer’s disease mouse model, by Gerhard Leinenga and Jürgen Götz. They used ultrasound to reduce the blood brain barrier and stimulate microglia, cells which would normally clean up Amyloid Beta. They found that in 75% of the mice studied there was a significant decrease in Amyloid plaques, and some of those mice had improved function on memory testing in a maze.

This is a genuinely exciting result, as ultrasound is a novel and rather non-invasive treatment option. The results are also very promising. But here now are the caveats that any responsible science journalist should note.

The mouse models of AD are not perfect models of the human disease. Specifically, in some mouse models of AD it seems that the clumped Amyloid Beta causes cognitive impairment, but it is unclear if this is also the case in human AD. While clearing plaque may slow decline, and can conceivably improve function of still living neurons, it will not restore dead neurons, so any loss of memory or cognitive function due to dead neurons is permanently gone.

In fact some researchers believe that in humans the Amyloid plaques are more of a downstream effect of AD and not the underlying cause. It is not clear how important they are in causing AD itself.

The current study should be considered preliminary and exploratory. This is the kind of research that is very interesting and useful to other researchers, but should be of very limited interest to the general public. Most research at this stage does not translate to a specific treatment in humans.

I certainly hope that this research does bear fruit. I would love nothing more than for there to be an effective treatment for AD. This approach may also apply to other neurodegenerative diseases. It is just way too early to tell.

This is also a general pattern in the way scientific research progresses vs how it is reported. Battery and solar technology are very similar – you can read just about every week about some new breakthrough in battery or solar technology, but the reporting rarely puts the preliminary research into proper context, and then you never hear about these amazing breakthroughs again. Meanwhile, the science continues to progress incrementally. Incremental advances (the reality), however, do not make good headlines. Breakthroughs (rarely the reality) do.

Friday, 10 April 2015

To influence others is pivotal to nursing leadership

To influence others is pivotal to nursing leadership........
Eileen P. Williamson,
 MSN, RN

Leaders have many traits we admire, from competence and confidence to direction and determination. We describe them with words like strength, honesty, integrity, enthusiasm, commitment and engagement. We see them as thinkers and doers, organizers and risk takers. We admire their vision and the objectives they believe in and set for themselves and others; we look to them to move others ahead and help them succeed.

They are the ones who head projects and people and make themselves not just responsible but accountable for outcomes. They plan strategies and measure successes in their work with others and even in the work they do alone. They keep their eyes on the goals they have set and ensure sustained movement toward them. They move and motivate and above all, they influence.

Leaders sound a lot like nurses to me. In fact, the very essence of our work as nurses makes us leaders, regardless of what our ID badges say. We don’t have to be called directors, administrators or executives to lead. Staff or management, as nurses we all are called to leadership.

We look at nursing leadership where it begins: at the bedside. We highlight various education and mentorship programs around the country — some didactic, others one-on-one mentorship models — aimed at helping nurses become better leaders.

The call to leadership isn’t simply getting others to do things, it’s influencing them to want to do those things. Some say great leaders are born; others say they’re made. Either way, the best ones are those who have influence.

For leadership tips read “Rising Star gives critical tips on leadership” or “Program develops critical care, leadership skills.”