Wed, Feb 22, 2017 | updated 09:17 PM IST

Drug found to treat rare genetic disorder

Updated: Aug 31, 2016 17:44 IST

WashingtonD.C.[USA], Aug. 31 (ANI): In a recent study researchers have discovered a type of drug delivery system that may offer new hope for patients with a rare, ultimately fatal genetic disorder Niemann Pick Type C1 and make what might become a terrible choice a little easier.

No treatment currently exists for this disease that affects about one in every 120,000 children globally, and results in abnormal cholesterol accumulation, progressive neurodegeneration and eventual death.

However, a compound that shows promise is now undergoing clinical trials, but it has major drawbacks, the high doses necessary also cause significant hearing loss and lung damage, as well as requiring direct brain injection.

New findings outline the potential for a nanotech-based delivery system to carry the new drug into cells far more effectively, improve its efficacy by about five times, and allow use of much lower doses that may still help treat this condition without causing such severe hearing loss.

The same system may ultimately show similar benefits for 50 or more other genetic disorders, especially those that require "brain targeting" of treatments.

Professor Gaurav Sahay said, "Right now there's nothing that can be done for patients with this disease, and the median survival time is 20 years."

Adding, "The new cholesterol-scavenging drug proposed to treat this disorder, called cyclodextrin or HPßCD, may for the first time offer a real treatment. But it can cause significant hearing loss and requires multiple injections directly into the brain, which can be very traumatic. I'm very excited about the potential of our new drug delivery system to address these problems."

The HPßCD drug is attached to an extraordinarily small, nanotech-sized lipid particle that can carry it into cells, where it helps to flush out cholesterol.

However, researchers were surprised to discover that the carrier itself also helped address the problem, while working in synergy with the drug it carries to greatly increase its effectiveness.

This should allow use of much lower dosages and possibly an easier delivery through intravenous injection, instead of brain injection.

In the form currently used, only 0.2 percent of the drug is able to cross the blood brain barrier.

In previous research with the HPßCD drug in animal models, the treatment did slow the progression of this disease, but did not reverse it. The disease focuses its damage on liver and brain cells.

In their report, researchers noted that this type of drug delivery system has several advantages, including prolonged circulation times, the ability to incorporate multiple drugs with different mechanisms of action, and a variety of "targeting ligands" that can help cross the blood brain barrier.

The research was published in Scientific Reports. (ANI)

Beware! Zika can be a reason for miscarriage

Updated: Feb 22, 2017 14:18 IST

Washington D.C. (USA), Feb. 22 (ANI): Beware to-be mothers! According to a recent study, Zika virus could lead to a greater risk of miscarriage in the early stage of pregnancy.

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Washington D.C. (USA), Feb. 22 (ANI): Women with a rare type of epithelial ovarian or peritoneum cancer, can now take sigh of relief.

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Washington D.C. [USA], Feb. 22 (ANI): Now don't blame those chips and chocolates for you childs extra pounds, as a study says around 35-40 percent of a child's is inherited from their parents.

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Drinking too much may age arteries over time!

Updated: Feb 22, 2017 06:03 IST

Washington D.C. [USA], Feb. 22 (ANI): Excessive boozing over the years might age arteries prematurely, especially in men, putting them at an increased risk for heart disease.

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Chronic knee pain may be treated online

Updated: Feb 21, 2017 06:41 IST

Washington D.C. [USA], Feb. 21 (ANI): A study has found out that an online intervention, combining home exercise and pain-coping skills training, provided substantial clinical benefits for patients suffering from chronic knee pain.

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Washington D.C. [USA], Feb. 21 (ANI): Only exercise is not enough to maintain that figure, which is an after effect of much toil and sweat!

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New Delhi [India], Feb. 20 (ANI): With rising life expectancy, increasingly sedentary lifestyles and surge in incidence of obesity, India is also witnessing a resultant rise in orthopedic problems as a natural corollary.

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Children inherit obesity from parents: Study

Updated: Feb 20, 2017 14:54 IST

Washington D.C. (USA), Feb. 20 (ANI): A study reveals that 35-40 percent of a child's 'Body Mass Index' - how fat or thin they are - is inherited from their parents.

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New Delhi [India], Feb 20. (ANI): Good news for all those lazy-headed not-really-a-cleanliness-freak out there! Scientists have found out that the obsession with hygiene could even be turning some beneficial bacteria found in the human gut into "endangered species".

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New Delhi [India], Feb 20. (ANI): In order to persuade someone to quit smoking, it is the 'emotions' that need to be triggered rather than inciting fear in an individual.

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No difference between good or bad diet: Study

Updated: Feb 20, 2017 05:54 IST

New Delhi [India], Feb 20. (ANI): Ever faced a choice between Brown and white bread?

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Washington D.C. [USA], Feb. 19 (ANI): To boost sustainability of livestock production, a study finds that gene editing - one of the newest and most promising tools of biotechnology - enables animal breeders to make beneficial genetic changes, without bringing along unwanted genetic changes.

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People with ADHD may have smaller brain volume

Updated: Feb 19, 2017 07:09 IST

New Delhi [India], Feb 19. (ANI): In a latest study it has been found out that people diagnosed with attention deficit hyperactivity disorder (ADHD) have smaller brain volume than those without the disorder.

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New Delhi [India], Feb 19. (ANI): What if we tell you that scientists can actually slow down the process of ageing. Sounds too good to be true right?

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New Delhi [India], Feb 19. (ANI): Lefty or righty? Well it was decided when you were still in your mum's womb!

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Dads-to be face greater risk of depression

Updated: Feb 19, 2017 06:53 IST

New Delhi [India], Feb 19. (ANI): Expecting a baby is always a joyous experience for both the mother and the father, however a latest study has found out that fathers-to-be can be at risk of depression symptoms if they feel stressed or are in poor health.

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New Delhi [India], Feb 19. (ANI): Do you find it difficult hearing out people at a noisy bar or a restaurant even though you have passed the hearing test with flying colors? Well, you might be secretly deaf!

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New Delhi [India], Feb 19. (ANI): What if we told you that rice has the potential of carrying arsenic and is more than hazardous to feed it to infants!

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Washington D.C. [USA], Feb. 18 (ANI): Now you can save your kid from surgery, as a study shows that antibiotics may be an effective treatment for acute non-complicated appendicitis in children, instead of surgery.

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Washington D.C. [USA], Feb. 18 (ANI): Attention new mommies, sing lullabies to your new born to feel more connected to your babies, suggests a study.

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