Last Week in Science (9/4-9/10)

Welcome, readers! Here is a recap of the biggest news in healthcare and medicine from last week.

First Alzheimer’s Disease Treatment on the Horizon?

The investigational Alzheimer’s disease treatment adcumabab, a human monoclonal antibody produced by BioGen, has been shown to decrease amyloid plaques and slow cognitive decline in patients. Initial results from the trial were first presented at last year’s AD/PD 2015: International Conference on Alzheimer’s and Parkinson’s Diseases. They showed promising results, which were validated by a recent publish of results.

BioGen Alzheimers
PET scans of patient using adcumabab. The image on the left is before treatment, while the image on the right is after treatment; amyloid plaques (red) was greatly reduced.

As mentioned in an earlier post on our blog, the cause of Alzheimer’s disease has been a point of contention among scientists. Some believe that clumps of a protein called tau are the cause of cognitive decline associated with AD. On the other hand, some Alzheimer’s researchers have focused on another kind of protein clump known as beta-amyloid. This recent study with aducumabab has reinforced the theory that cognitive decline may in fact be caused by amyloid plaques in the brain.

The FDA has granted Fast Track designation to the drug this past week. Recruitment has already begun for the multi-country phase 3 trials known as ENGAGE and EMERGE. Each study is expected to include 1350 patients and to be completed in February 2022. There are still no treatments for Alzheimer’s disease on the market yet, which makes this drug a very exciting one to follow.

Severity of Autism Linked with Genetics and Ultrasound?

In a study published in Autism Research, for children with autism and a class of genetic disorders, exposure to diagnostic ultrasound in the first trimester of pregnancy is linked to increased autism severity.

Researchers at University of Washington (UW) Medicine, UW Bothell and Seattle Children’s Research Institute studied the variability of symptoms among kids with autism, not what causes autism. What they found is that exposure to diagnostic ultrasound in the first trimester is linked to increased autism symptom severity. The greatest link is among kids with certain genetic variations associated with autism; 7 percent of the children in the study had those variations.

FDA guidelines say that ultrasounds should only be used in the first trimester if it is a medical necessity.

Currently, FDA guidelines say that ultrasounds during the first trimester should only be used for medical necessities. “Our results,” says author Pierre Mourad, a UW professor of neurological surgery in Seattle, “are to bolster the FDA guidelines.”

Autism raises many questions; What is the cause of it? How should we treat it? Why are there so many different variations of it? This research is meant to answer the last question–variability of autistic traits among children may be connected to ultrasounds during the first trimester, in conjunction with certain genetic traits as well.

Sickle Cell Gene Therapy Making Progress

Sickle cell disease is a disorder that affects the oxygen-carrying protein hemoglobin, which results in a sickle shaped red blood cell. As you can imagine, this ultimately results in a number of health problems. These sickled cells live less long than those with healthy hemoglobin, leading to anemia. They also bind together, resulting in a blockage of blood flow that can cause severe pain and organ damage throughout the body (sickle-cell crisis).

Image result for sickle cell
Sickled red blood cells can cause major problems such as anemia and can cause excruciating pain in those affected.

Researchers have known that fetal hemoglobin typically stop production in the body after birth, but in 2008 scientists found that suppressing a gene called BCL11A—which acts as an “off” switch—could restart fetal hemoglobin production. In 2011, using this approach, the same group corrected sickle cell disease in mice, replacing much of the defective beta (“adult”) hemoglobin that causes sickling with healthy fetal hemoglobin. Unfortunately, suppressing BCL111A also causes other unwanted side-effects; BCL11A has a major role in blood stem cells. When it is silenced in mice with the sickle cell mutation, blood stem cells cannot engraft long-term in the animals’ bone marrow and eventually become depleted. This will almost certainly cause negative long-term effects that render the therapy useless.

To solve this problem the Dana-Farber/Boston Children’s team performed shrewd genetic engineering; they created a gene therapy virus that silences BCL11A selectively—only in precursors of red blood cells. Currently, the scientists are taking the final steps toward FDA clearance for a clinical gene therapy trial in sickle cell disease that is expected to begin in early 2017.

Deaths from Ovarian Cancer Falls Worldwide Due to Oral Contraceptive Use

Deaths from ovarian cancer fell worldwide between 2002 and 2012 and are predicted to continue to decline in the USA, European Union (EU) and, though to a smaller degree, in Japan by 2020, according to new research published in the leading cancer journal Annals of Oncology last week.

Increased use of oral contraceptives associated with decreased mortality from ovarian cancer.

The researchers believe that this decline in deaths caused by ovarian cancer is because of increased use of oral contraceptives. Most of the risk for ovarian cancer is related to the amount of time spent in ovulation. Oral contraceptives reduces this. Using oral contraceptives (birth control pills) decreases the risk of developing ovarian cancer, especially among women who use them for several years. Women who used oral contraceptives for 5 or more years have about a 50% lower risk of developing ovarian cancer compared with women who never used oral contraceptives.

Using data on deaths from ovarian cancer from 1970 to the most recent available year from the World Health Organization, the researchers found that in the 28 countries of the EU death rates decreased by 10% between 2002 and 2012. In the USA the decline was even greater, with a 16% drop in death rates from 5.76 per 100,000 in 2002 to 4.85 in 2012.

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