Top 5 Life Science News Stories of April 2017

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Apr 28, 2017, 07:03 ET

ROCKVILLE, Md., April 28, 2017 /PRNewswire/ -- This month a prize was awarded for a device that allows consumers to diagnose their own diseases, Roche got approval for a new first-in-class multiple sclerosis drug, and a task force softened its stance against prostate cancer screening. A study explored umbilical cord blood as a treatment for symptoms of autism and Illumina shared tumor variant data with a public database. These are some of the top life sciences news stories in April 2017. Kalorama's latest report in life sciences is PCR Markets for Life Sciences and Diagnostics.

Roche wins approval for multiple sclerosis drug
Roche received FDA approval late last month for a first-in-class drug to treat an aggressive form of multiple sclerosis (MS).
Ocrevus (ocrelizumab) is a monoclonal antibody that targets a type of B cell that attacks myelin and causes nerve cell damage in the brain. It has been approved to treat both the relapsing form of MS, which is the most common form, and also primary progressive MS, which affects only about 15 percent of MS patients. While more than a dozen treatments are now available for relapsing MS, this is the first drug approved for primary progressive MS. The drug's creator Genentech, part of the Roche Group, plans to charge $65,000 a year for the treatment, according to the Wall Street Journal. The drug is administered by intravenous infusion every six months.
Cord blood explored as autism treatment
PerkinElmer announced this month that a Phase I clinical trial, funded in part by its cord blood and cord tissue preservation business ViaCord, showed it is safe to use a child's own umbilical cord blood to treat symptoms of autism. The study results, published April 5 in the journal Stem Cell Translational Research, hinted at some improvement in social communication skills and other autism symptoms among the 25 children enrolled, as judged by both parents and clinicians, though the study had no control group for comparison.
ViaCord provided some financial support for the study and nearly half of the cord blood units, according to a PerkinElmer press release.
A randomized, double-blind Phase II trial is now enrolling patients to determine if a cord blood infusion can actually improve social communication skills in children ages 2 to 7 who have autism spectrum disorder. Umbilical cord blood is thought to contain cells that can alter brain connectivity and suppress inflammation. Researchers are also exploring umbilical cord blood infusions as therapy for cerebral palsy and brain injuries.
The U.S. Centers for Disease Control and Prevention estimates 1 in 68 school-age children have autism spectrum disorder. Medications are sometimes used to minimize symptoms, but there is no cure.
The Kalorama Information report Novel Autism Diagnostics examines the market for diagnosing autism spectrum disorders, and outlines many of the current approaches to autism testing, including molecular testing, pre-disposition screening, targeted sequencing-based testing, next-generation sequencing with modeling, genetic risk assessment / testing, cloud platform approaches, microarrays, end-to-end sequencing, gene variants, and blood-based genetic testing.
Illumina donates tumor variant data
Illumina announced this month it will donate a large bundle of information to the Clinical Interpretation of Variants in Cancer (CIViC) resource, an open access database of genetic variants linked to cancer.
Illumina will contribute more than 8,000 associations of somatic genetic alterations, which will triple the size of the current database.
"This genetic information represents a very valuable data set that is now available to researchers worldwide," said Obi L. Griffith, Assistant Professor of Medicine at Washington University School of Medicine, St. Louis in a statement. "CIViC's strategy is to break down information silos between academic and industry researchers. This donation shows that such a strategy can work."
Illumina made a similar contribution in October when it submitted more than 95,000 human genetic variants to the National Center for Biotechnology Information's open-access ClinVar database.
"The donation to CIViC and ClinVar represents our commitment to help researchers access and share the secure health data information they need to transform healthcare," said Sanjay Chikarmane, Senior Vice President and General Manager at Illumina in a statement.
Prize awarded for self-diagnostic devices
A team led by an emergency room physician from Pennsylvania won the $2.6 million Qualcomm Tricorder XPRIZE for developing a device that allows consumers to diagnose health conditions by themselves, without help from medical professionals.
The team, called Final Frontier Medical Devices, created a prototype called DxtER, which connects to a tablet or smartphone and uses artificial intelligence to mimic a doctor-patient conversation about symptoms. Once the device arrives at a tentative diagnosis, the consumer performs his own diagnostic tests, which may involve a urine or blood sample or sensors that monitor breathing or check the eardrum. It also has sensors that gather continuous vital signs data to aid in diagnosis.
The competition was inspired by a fictional device in the original Star Trek series called the Medical Tricorder, a computer with a deployable hand-held scanner that Dr. McCoy used to measure crew members' vital signs and symptoms.
About 300 teams entered the Tricorder XPRIZE competition, which began in 2013.
The second-place winner, an international team called Dynamical Biomarkers Group, received $1 million. The winners were announced at a ceremony April 12.
Both teams said they plan to continue develop their prototypes into devices that can be released commercially.
Task force looks more favorably on prostate cancer screening
The U.S. Preventive Services Task Force released a draft guideline April 11 on prostate cancer screening that upgrades its recommendation from D to C for prostate specific antigen (PSA) tests for men age 55 to 69.
The task force had previously recommended against PSA-based screening for all men because of the lack of proven benefit and the risk of harm from unnecessary treatment.
That recommendation changed to a C in the draft released this month because of new evidence of benefit, the task force said, including some evidence that PSA screening reduces the chance of death and of metastatic cancer. However, many more men will experience the harms from treatment than will experience benefits, the task force noted.
The C rating means the benefits and harms are closely balanced, so men should make their own decisions based on their values and preferences. The task force still gives a D recommendation to PSA testing for men older than 70.

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