Sick Kids scientists wielding the “breakthrough” gene editing technology CRISPR have snipped out a genetic defect in mice that causes a severe form of muscular dystrophy, eliminating all signs of paralysis in the animals.
A Food and Drug Administration panel opened a new era in medicine, unanimously recommending that the agency approve the first-ever treatment that genetically alters a patient’s own cells to fight cancer.
Whole genome sequencing of healthy individuals as a way to predict and prevent disease will, however, inevitably reveal variants that put them at risk for rare genetic conditions – only some of which have health implications. The MedSeq Project examined responses to such results, in both patients and doctors, and revealed “reassuring evidence that primary care providers can be trained to manage their patients’ sequencing results appropriately, and that patients who receive their results are not likely to experience anxiety connected to those results,” said Jason Vassy, lead author.
Two Canadian companies have made a splash advancing personalized medicine in both the US and Canada. Zymeworks raised $85 million in its IPO to advance the development of disruptive therapeutic platforms and biotherapeutics – the first Canadian venture-backed IPO in the life sciences sector since 2014. Additionally, Repare Therapeutics Inc. announced a US$68 million Series A…
LifeLabs has partnered with GenXys Health Care Systems to offer a personalized medicine testing service. Using cheek swabs to collect patients’ DNA, samples are submitted to LifeLabs where they are analyzed for 60 genetic variations that affect how a patient metabolizes and responds to medications. This will ultimately help physicians prescribe medications based on each patient’s specific medical and genetic profile.
A new study has revealed the pricing of cancer drugs is unrelated to effectiveness. Using frameworks developed by the American Society of Clinical Oncology and the European Society of Medical Oncology, Dr. Christopher Booth at Queen’s University studied all randomized controlled trials of new cancer drugs in non-small cell lung, breast, colorectal and pancreatic cancer over a four-year period. The authors concluded that to deliver optimal cancer care in a sustainable health system, the disconnect between drug cost and clinical benefit must be reconciled.
Last week, Merck & Co’s Keytruda became the first cancer drug ever approved by the FDA based on patients’ specific genetic traits, regardless of where in the body the disease originated. The approval, though restricted to advanced patients who have failed other therapies, marks a major advancement for precision medicine, where genetic biomarkers may determine the course of therapy rather than the origin of the cancer in the body. The hope is that one day advanced genetic information will be able to identify and select the best treatments for cancer patients.
Alexion is a powerhouse in the market for orphan drugs, a fast-growing pocket of the pharmaceutical industry that focuses on rare diseases. Some orphan drugs, such as Alexion’s Soliris, are highly effective, but they have caused a seismic shift in both treatment costs and sales practices. Many orphan drugs cost more than most new homes and, in some parts of the world, must be delivered by armed guards. With global sales expected to almost double by 2022 to $209 billion, this article examines the ethical and medical boundaries of rare-disease drugs.
The Canadian government intends to challenge the constitutionality of Bill S-201, The Act to Prohibit and Prevent Genetic Discrimination. The law, which was recently passed by Parliament and took effect May 4, adds genetic information to the list of protected data under the Privacy Act and the Personal Information Protection and Electronic Documents Act. It also amends the Canadian Human Rights Act to add genetic characteristics as a prohibited basis for employment, housing and provision of goods or services discrimination.
Ontario is investing more than $12 million to support new and existing programs that will improve maternal and child health in Ontario. Announced May 12th, the investment includes enhanced screening of all newborns with the addition of a new screen for hearing loss, enabling families to receive treatment or language and early literacy support sooner. The province also recently added a new screen for critical congenital heart disease, to detect a range of heart defects in newborns that could cause life-threatening symptoms.