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Sangamo And Pfizer To Collaborate In Developing ALS Gene Therapy

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Pfizer and Sangamo Therapeutics announced on Wednesday that they would partner in the development of a gene therapy to be used in treating ALS. This is a disease which affects the nerve cells contained in the spinal cord and the brain. Additionally the gene therapy to be developed by the two pharmaceutical firms will be used in the treatment of patients suffering from a brain disorder resulting from a gene mutation. About a third of patients suffering from hereditary ALS have this mutation in their genes.

The partnership between Pfizer and Sangamo has coincided with a gain in momentum for gene therapy after a couple of high profile failures decades ago. Recently the Food and Drug Administration updated guidance with a view to assisting in speeding up the development of gene therapies.

Rare type of blindness

Last month a treatment for a rare blindness developed by Spark Therapeutics was approved by Food and Drug Administration. This was the time a gene therapy aimed at treating an inherited disease was getting approved. The deal between Pfizer and Sangamo will see the latter get upfront payments totaling $12 million from the former. There will also be milestone payments of about $150 million.

Pfizer and Sangamo’s partnership comes in the wake of the former being accused by United Kingdom’s Advertising Standards Authority of flouting advertising regulations in its marketing campaigns. On January 3, the Advertising Standards Authority issued a ruling to the effect that an ad developed for Imedeen, a collagen beauty shot developed by Pfizer exaggerated its claims.

“We considered that because the claim in the ad stated a particular site of action (the deep dermal layer of the skin), … it went beyond the authorized claim,” said the UK advertising watchdog in its ruling.

Leukemia treatment

Also earlier this week in the United Kingdom Pfizer welcomed a decision by the National Institute for Health and Care Excellence to reassess the benefits of its treatment Besponsa, as a therapy for leukemia. Last year in August Pfizer had lodged an appeal after the National Institute for Health and Care Excellence rejected the drug as a therapy for adult patients suffering from refractory or relapsed CD22-positive B-cell precursor acute lymphoblastic leukemia.

The reasons National Institute for Health and Care Excellence gave for the rejection included the cost-effectiveness of the treatment. During its INO-VATE clinical trial the Besponsa treatment was found to double the complete remission rates vis-à-vis the current standard treatment.

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Potentially Dangerous Asian Tick Infested 8 States

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Asian Tick Infests 8 States

Entomologists issued a new warning about the rapid spread of a particularly aggressive tick this season. Initially discovered in New Jersey, the Long-horned tick has now been found in eight states, including North Carolina and as far west as Arkansas.

Scientists report the tick can infest a host rapidly, with femalesproducing thousands of tick eggs at a time asexually. Each tick will gorge itself on the host’s blood until the tick can barely move. The pest has been known to draw enough blood from animals to induce anemia.

Also known as the East Asian Tick, or its biological name of Haemaphysalislongicornis, the newcomer to America is not easily identified without a microscope. Its distinctive “long horns” are not visible to the naked eye.

Initially, authorities thought the first of the ticks werediscovered last summeron a sheep in New Jersey. When the sheep’s owner brought a sample of what she pulled off her pet, Department of Health officials discovered the sheep and her entire paddock were infested with the creatures.

Since the sheep had not traveled anywhere in years, officials are at a loss to explain how the tick came to be in New Jersey. The Health Department later confirmed that a tick discovered on a dog in another New Jersey county in 2013 was also a long-horned tick.

Despite an aggressive attempt to kill the ticks, which included sheering the sheep and removing vegetation from the paddock, additional long-horn ticks were discovered this spring. That prompted a state alert that the ticks had “successfully wintered”, surviving freezes, snow, and rain.

During routine tick monitoring, which can as simple as dragging sheets through meadows, more of the East Asian menace were discovered in Virginia, West Virginia, and North Carolina by June. Over the summer, New York, Arkansas, Maryland, and Pennsylvania also reported cases.

Asian Tick Infests 8 States

The good news about East Asian Ticks

So far, none of the long-horned ticks found in the United States carried any diseases. That is not to say they are completely harmless to your animals, which can be overwhelmed by the sheer number of ticks if not recognized quickly.

The exotic pests are controlled by the most common repellents and behaviors we use against out domestic ticks. Deet is the most effective chemical repellent.

Officials also remind everyone to use long sleeves, pants, and socks while walking through high grass and be sure to check yourself and children for ticks when you get home.

The bad news about East Asian Ticks

Even though no disease-carrying ticks have been found in the United States yet, these ticks are known to cause serious health issues in their native country.

Severe Fever with Thrombocytopenic Anemia (SFTA) and Japanese Spotted Fever are among several diseases spread by the tick in other countries. Both can be fatal if not treated promptly. It is not yet known if the East Asian tick can spread Lyme Disease.

Scientists warn dairy farmers to check livestock. The ticks can spread an animal disease called theileriosis, which causes decreased milk production in cows and potentially fatal blood loss in calves.

What you need to do about long-horned ticks

Pet owners and farmers should regularly check their animals for ticks. Learn how to safely remove them from animals (or yourself).

Every local health department accepts ticks for identification and disease testing. Place the tick in a plastic bag with a small stick, leaf, or a piece of cotton (to keep it from getting crushed) and deliver it to the health department.

If you find a tick on yourself, remove it and clean the area thoroughly with soap and water. Be attentive to any signs or symptoms of illness and be sure to tell your doctor about the tick. In most cases, even the most serious ailments can be headed off by prompt treatment.

 

 

 

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Moderate Drinking Can Help You Live A Little Longer?

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And now some good news for casual drinkers:  a new study claims that moderate consumption of wine and beer (and coffee) can actually increase lifespan.  Working with a group of 1,700 subjects older than 80, since 2003, a research team out of University of California-Irvine says that people who drank moderate amounts of alcohol and/or coffee lived longer than those who abstained [from either or both].  They also say they learned that those who live longer tend to be on the heavier side in their 70s than those who or on the underweight side. 

Now, to be sure, the researchers are still trying to understand these results  Lead study researcher Dr. Claudia Kawas notes, “I have no explanation for it, but I do firmly believe that modest drinking improves longevity,” in a presentation at the American Association for the Advancement of Science conference.  

She explains that the “The 90+ Study” has been tracking the health of these study subjects for roughly the last 15 years, adding, “Those who consumed approximately two glasses of beer or wine a day were 18 percent less likely to experience premature death.”

On the other hand—or, concurrently, perhaps—the researchers also found that those who consumed about two cups of coffee per day also decreased their risk for early death by 10 percent.  However, it is also very important to note that daily activity was also a factor that reduced premature death. This assuaged the researchers to suggest that keeping a healthy weight (not underweight) and engaging in regular physical activity and eating healthy can not only stave off premature death but also chronic disease. 

At the end of the day, Dr. Kawas reminds that they still do not really know why modest drinking—and a few extra pounds—seems to help people live a little longer, but this study soundly speaks for such a case. 

Other interesting facts they found include:

  • Those who are slightly overweight—not obese—reduced their odds of premature death by 3 percent
  • As few as 15 minutes and as many as 45 minutes of daily physical activity could reduce premature death by up to 11 percent
  • Spending about 2 hours a day on a hobby could decrease risk for early death by as much as 21 percent

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New 5-Cluster Classification for Diabetes

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For a long time, the medical industry has associated diabetes into two categories.  Type 1 diabetes, of course, is genetic; you are born with it. Type 2 diabetes is also known as Adult-Onset diabetes and is a lifestyle disease that evolves over time, associated with the body’s eventual resistance to insulin. 

More recent studies, however, now suggest that there are actually 5 different types of diabetes. Or, rather, Scandinavian researchers argue that the two types could further divide into five total clusters with different physiological and genetic distinctions.

From data on roughly 15,000 patients, taken from five cohorts in Finland and Sweden, the researchers used six standard measurements to identify these five clusters.  Essentially there are two mild forms and three severe forms.

The first form is basically Type 1 diabetes, attributed to a genetic predisposition. The remaining four dissect Type 2 diabetes into different degrees of severity.

  • Cluster 1:  Severe Autoimmune Diabetes (SAID)
  • Cluster 2:  Severe Insulin-Deficient Diabetes (SIDD)
  • Cluster 3:  Severe Insulin-Resistant Diabetes (SIRD)
  • Cluster 4:  Mild Obesity-Related Diabetes (MOD)
  • Cluster 5:  Mild Age-Related Diabetes (MARD)

According to consultant and Imperial College London clinical scientist Dr. Victoria Salem, most specialists have long pursued that are 2-tier diabetes definitions is “not a terribly accurate classification system.” She goes on to say, “There is still a massively unknown quantity – it may well be that worldwide there are 500 subgroups depending on genetic and local environment effects,” adding that this is definitely going to improve the way we think about diabetes as a disease.

Lead study author Leif Groop, MD, PhD, of the Lund University Diabetes Center in Malmö, Sweden, and Folkhalsan Research Centre, Helsinki, Finland, comments, 

“Existing treatment guidelines are limited by the fact they respond to poor metabolic control when it has developed, but do not have the means to predict which patients will need intensified treatment.”

Finally, McGill University’s Rob Sladek, MD, explains in an accompanying editorial, “This study moves us towards a more clinically useful diagnosis, and represents an important step towards precision medicine in diabetes. Nevertheless, the finding that simple parameters assessed at the time of diagnosis could reliably stratify patients with diabetes according to prognosis is compelling and poses the challenge of development of methods to predict outcomes of patients with type 2 diabetes that are more generalizable and comprehensive.” 

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