Genetics / Genomics – SciPol Weekly, November 25 – December 1


BusinessKorea – S. Korea to Expand Stem Cell Research Range, Genetic Scissors Permission

At the second meeting to break down regulations chaired by Prime Minister Lee Nak-yon at the Advanced Institute of Convergence Technology in Suwon, on November 30, the Ministry of Health and Welfare said, “We will expand the allowable range of embryonic stem cell and genetic scissors research to the same level as advanced countries.” Under the current bioethics laws, various restrictions are imposed on research and development of embryonic stem cells, including human embryo, and somatic cell-based gene therapies. The research on embryonic stem cells is only allowed for 20 types of rare and incurable diseases, while that on gene therapies is allowed for severe diseases such as genetic disorder, cancer and AIDS. 

RAPS – Cures Act Implementation: Gottlieb Says Faster Cancer Drug Approvals Coming for Secondary Indications

In opening comments, Gottlieb told the committee that the agency is working on a proposal whereby cancer treatments already approved for one indication might be able to win approval for a second indication based on a supplemental application that relies on “a more targeted data set, like a single-arm study. This may be suitable, for example, when there’s a clear and outsized treatment effect, and the second indication concerns the same disease as the first one, but for a new setting. For example, a targeted drug approved for a third-line use that shows benefit in a second-line indication,” Gottlieb added.


Business Insider – A startup looking to reverse type 1 diabetes just raised $114 million

A startup that wants to change the way we treat type 1 diabetes just raised $114 million.  Semma Therapeutics, a company that got its start in 2014, is researching ways to use stem cells that act like key cells responsible for regulating blood sugar levels in the bodies of healthy people. The aim is to treat — and effectively cure — type 1 diabetes. Treating type 1 diabetes is —  in theory —   straightforward: If you could find a way to replace the beta cells in the body, the body would start producing its own insulin again. The plan is to use this funding to get Semma's treatment, which has been tested in animals, into human trials. 

Houston Chronicle – Critics wary of stem cell clinics that promise big results but have little regulation

Martha Barden hobbled around in so much pain - barely able to navigate stairs, always calculating distances she could walk - that her husband told her she needed knee replacement. The Friendswood woman didn't disagree, but she wasn't keen on surgery, which hadn't done wonders for some of her friends and made her nervous at 65 years of age. She was more interested in the promise of stem cell therapy, touted for an ability to rejuvenate aging or injured joints.

Medical Economics – Consumer genomics: Next direction or next distraction?

I’ve noticed in my own patient base a significant mood shift over the last several years. Patients are more interested in knowing their health risks than ever before. An example would be the recent FDA approval of  the new genetic risk panel from 23andMe, a direct-to-consumer genetic testing company. This is the first step in what will become patient access to ordering their own exome and full genome testing. It spotlights the murkiness of what happens once the results are in.

NBC – What you’re giving away with those home DNA tests

It sounds like such a fun holiday gift idea: a DNA test that can tell your sister-in-law whether she really has Native American ancestors, or one that promises to craft your friend a perfect diet based on his genes. Home DNA tests are likely a big seller for the next few weeks, but privacy experts say consumers should be cautious, and New York Sen. Chuck Schumer said this past weekend that he was asking the Federal Trade Commission to "take a serious look at this relatively new kind of service and ensure that these companies can have clear, fair privacy policies."

NPR – Gene Therapy Shows Promise For A Growing List Of Diseases

Eli Wheatley and Christian Guardino are among a growing number of patients whose lives are apparently being saved or radically improved by gene therapy. Wheatley, 3, of Lebanon, Ky., and Guardino, 17, of Patchogue, N.Y., were both diagnosed with what were long thought to be incurable genetic disorders. In the past, Wheatley's condition would have probably killed him before his first birthday. Guardino's would have blinded him early in life. But after receiving experimental gene therapies, both seem to be doing fine.

PR Newswire – PreventionGenetics announces integration with FDNA's deep learning platform, Face2Gene to increase the diagnostic power of genetic testing

"PreventionGenetics uses the most advanced technologies available to ensure patients of all kinds get the answers they need," said James Weber, Ph.D., founder and president of PreventionGenetics. "With the cost of sequencing continuing to decline, and technologies like FDNA's Face2Gene improving the insights available from testing, patients and providers are poised to benefit greatly." Since the product's inception, Face2Gene has analyzed facial images of tens-of-thousands of patients for more than 2,500 syndromes, and non-facial phenotypes for more than 10,000 syndromes, linking the analysis to the disease-causing genetic variations.


Big Think – This is Why Some People Naturally Have a Harder Time Losing Weight

Like many Americans, I struggle with my weight and have trouble losing it. I’ve always had something of a belly. Exercise and dieting have left me frustrated. I usually hit a wall after losing about 20 lbs. A new study finds that it might not be my lifestyle, but my genetic makeup. The scientists in this study do peg the growing worldwide obesity epidemic to over-nutrition and an increasingly sedentary lifestyle. Even so, “numerous familial studies also provide strong evidence for heritable contributions to obesity,” study authors write. Over 100 genes have somehow been implicated. But how much they impact weight and how they work exactly has been unclear, until now.

MedicalXpress – Researchers devise improved gene-editing process for Duchenne muscular dystrophy

Regenerative medicine researchers at UT Southwestern Medical Center developed an improved and simplified gene-editing technique using CRISPR/Cas9 tools to correct a common mutation that causes Duchenne muscular dystrophy. "We think these advancements will be valuable for the field and can help us move closer to tackling this disease in humans," said Dr. Eric Olson, Director of the Hamon Center for Regenerative Science and Medicine and Co-Director of the Wellstone Muscular Dystrophy Cooperative Research Center at UT Southwestern.

NPR – Scientists Train Bacteria To Build Unnatural Proteins

Scientists say they have created a partly man-made bacterium that can produce proteins not found in nature. This new life form, the latest development in a field called "synthetic biology," could eventually be used to produce novel drugs. The Scripps Research Institute's Floyd Romesberg and colleagues have been pushing toward this goal for well over a decade. Three years ago, they announced that they had added two more letters to the genetic alphabet of a bacterium: To DNA's familiar A, T, C, and G, they added X and Y. – Largest genetic study of mosquitoes reveals spread of insecticide resistance across Africa

Reported today (29 November) in Nature, this genetic resource will be used to develop new tools for monitoring resistance and managing insecticide use, and for designing novel control methods. The study by researchers from the Wellcome Trust Sanger Institute and their collaborators also discovered that wild mosquitoes collected in Africa were genetically far more diverse than had been thought. This helps to explain how mosquitoes evolve insecticide resistance so quickly.

The New York Times – Gene Therapy Hits a Peculiar Roadblock: A Virus Shortage

Biotech companies have exciting plans to introduce treatments that may be transformative, sometimes curing genetic diseases with a single treatment. And the firms are itching to test their products. But they are struggling to obtain a critical component of the therapy: the disabled viruses used to slip good genes into cells that lack them.


Science – So much for the abominable snowman. Study finds that ‘yeti’ DNA belongs to bears

In the folklore of Nepal, the yeti looms large. The creature is often depicted as an immense, shaggy ape-human that roams the Himalayan hinterlands. Purported sightings over the years, as well as scattered “remains” secreted away in monasteries or held by shamans, have hinted to some that the yeti is not merely a mythical boogeyman. But science has not borne this out so far. Previous genetic analyses of a couple of hair samples collected in India and Bhutan suggested that one small stretch of their mitochondrial DNA —the genetic material in a cell’s power-generating machinery that’s passed down only by females—resembled that of polar bears. 

Wired – The Most Promising New Cancer Treatments in a Century Have Arrived — But Not for Everyone

In 1891, a New York doctor named William B. Coley injected a mixture of beef broth and Streptococcus bacteria into the arm of a man with an inoperable neck tumor. The patient got terribly sick—developing a fever, chills, and vomiting. But a month later, his cancer had shrunk drastically. Coley would go on to repeat the procedure in more than a thousand patients, with wildly varying degrees of success, before the FDA shut him down. Though he didn’t have the molecular tools to understand why it worked, Coley’s forced infections put the body’s immune system into overdrive, allowing it to take out cancer cells along the way. While the FDA doesn’t have a formal definition for more modern immunotherapies, in the last few years it has approved at least eight drugs that fit the bill, unleashing a flood of money to finance new clinical trials.

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Philippe Semeria, CC BY 3.0