Onco Actu du 2 août 2017

	1. Biologie
Human Genetic Variation May Complicate CRISPR [The Scientist] Slight sequence differences confound target sites in precision genome-editing, a study shows.
	2. Etiologie
Periodontal Disease is Associated with Higher Risk of Several Cancer Types [AACR] The researchers conducted a prospective cohort study of 65,869 women aged 54 to 86 who were enrolled in the Women’s Health Initiative Observational Study. The women self-reported periodontal disease on questionnaires administered between 1999 and 2003. The researchers monitored cancer outcomes through September 2013.
	3.1 Tabac
Protecting American Families: Comprehensive Approach to Nicotine and Tobacco [FDA] Remarks by Scott Gottlieb, M.D., Commissioner of Food and Drug Administration.
	5. Traitements
Pancreatic cancer trial: Early surgery boosts success rates [BBC News] The trial involved cutting average time to surgery for 32 patients from two months to just over two weeks - 31 had their tumours removed successfully. But it will be two years before doctors know if operating earlier extends life.
	5.10 Traitements - Essais
Broadening Clinical Trial Participation [Cancer Research Catalyst] In 1990, fewer than 3 percent of patients were enrolled in clinical trials—which spurred then president-elect of the American Cancer Society, Walter Lawrence Jr., MD, to write an editorial calling for more concerted efforts. More than 25 years later, clinical trial participation hovers around 5 percent.
After French drug trial tragedy, European Union issues new rules to protect study volunteers [Science] The guideline, which was issued on 25 July, will take effect in February 2018. It comes in the wake of a tragedy in a French drug study last year that led to the death of one man and serious neurological damage in four others. But some say the revision isn't going for enough.
	5.12.1 Immunothérapies - partenariats
Looking for an oncology reboot, Sanofi, Regeneron shoot for a fast and flashy FDA OK of a promising new PD-1 [EndPoint] In an outline of Sanofi’s R&D work for the Q2 call this week, research chief Elias Zerhouni was clearly excited by the “fast-to-market strategy in the PD-1 space” that has been set up with its big development partner Regeneron.
	5.12.6 Immunothérapies - AMM
Bristol-Myers Squibb's Opdivo nabs MSI-H colorectal cancer nod, but it can't match Keytruda's umbrella approval [FiercePharma] The approval matches a piece of the go-ahead Opdivo’s archrival Keytruda picked up in May. But Keytruda’s green light was wider, covering solid tumors in any part of the body as long as they bear the MSI-H genetic features.
Bristol-Myers Squibb Receives FDA Approval for Opdivo (nivolumab) in MSI-H or dMMR Metastatic Colorectal Cancer That Has Progressed Following Treatment with a Fluoropyrimidine, Oxaliplatin, and Irinotecan [BMS] Approval based on CheckMate -142, in which Opdivo demonstrated an objective response rate of 28% (95% CI: 17-42; 15/53) among patients who received prior treatment with a fluoropyrimidine, oxaliplatin, and irinotecan.
FDA grants nivolumab accelerated approval for MSI-H or dMMR colorectal cancer [FDA] The approval was based on data from Study CA209142 (CHECKMATE 142; NCT 02060188), a multicenter, open-label, single arm study conducted in 53 patients with locally determined dMMR or MSI-H metastatic colorectal cancer (CRC) who had disease progression during, after, or were intolerant to prior treatment with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy.
	5.2 Pharma
Acalabrutinib granted Breakthrough Therapy Designation by US FDA for the treatment of patients with mantle cell lymphoma [AstraZeneca] The FDA granted Breakthrough Therapy Designation based on the totality of clinical data from the acalabrutinib development programme, including data from the Phase II ACE-LY-004 clinical trial in patients with relapsed or refractory MCL.
US FDA accepts regulatory submission for acalabrutinib and grants Priority Review [AstraZeneca] The NDA is based on results from the Phase II ACE-LY-004 clinical trial, which evaluated the safety and efficacy of acalabrutinib in patients with relapsed/refractory mantle cell lymphoma (MCL) who have received at least one prior therapy.
AstraZeneca gets breakthrough status for blood cancer drug [Reuters] AstraZeneca acquired acalabrutinib after buying Acerta Pharma in 2015. The drug is being developed for a variety of cancer types.
	5.2.2 Pharma - Fusions & Acquisitions
Expecting Big Pharma buyers to come running after weakened AstraZeneca? Not so fast [FiercePharma] And then there’s the political pressure AstraZeneca has faced at home since Pfizer came calling. In a 2016 speech, U.K. Prime Minister Theresa May name-dropped the company and highlighted the need to "defend a sector that is as important as pharmaceuticals is to Britain.”
Despite blockbuster boasts for R&D work, pressure increases on Pfizer to do a megadeal [EndPoints] “Pfizer has taken a break on M&A for now and no-one expects them to make another move for AstraZeneca. But M&A is like a game of chess and nobody will go after AstraZeneca until Pfizer picks its next target,” one banker told Reuters.
	5.3 Traitements - FDA, EMA, NICE...
EMA encourages tailored development of medicines for older people [EMA] EMA invites comments on reflection paper by 31 January 2018.
United Kingdom’s withdrawal from the European Union ('Brexit') [EMA] The Agency will maintain the delivery of its highest and medium priority activities but has decided to scale back or temporarily suspend some of its lowest priority activities. A significant loss of staff as a consequence of the Agency’s relocation may lead to more restrictions than currently foreseen in the plan.
EMA, Let's Move to Lille [EMA in Lille] We are waiting for you!
Offers to host the European Medicines Agency (EMA) [European Council] The agency will need to be relocated in the context of the UK's withdrawal from the EU. There have been 19 offers to host the European Medicines Agency (EMA).
EU drugs agency cuts workload as prepares for Brexit disruption [Reuters] The European Medicines Agency (EMA) said on Tuesday its "business continuity plan" meant it would suspend work on a new web portal and an electronic submission project for drugs, as well as the development of a transparency roadmap.
	5.3.4 Traitements - AMM (FDA, EMA)
Celgene and Agios win FDA approval for targeted AML drug Idhifa [FiercePharma] Celgene entered its cancer-metabolism partnership with Agios back in 2010, picking up global development and commercialization rights to the candidate. The partners will co-market the drug in the U.S.
FDA Grants Approval of IDHIFA®, the First Oral Targeted Therapy for Adult Patients with Relapsed/Refractory Acute Myeloid Leukemia and an IDH2 Mutation [Celgene] IDHIFA was approved concurrently with the Abbott RealTime™ IDH2 companion diagnostic test, which is FDA-approved as an aid in identifying AML patients for treatment with IDHIFA.
Celgene, Agios win a landmark FDA OK for new AML drug Idhifa [EndPoint] The drug enasidenib will now be sold as Idhifa, concentrating on about 9% to 13% of the AML market.
FDA approves leukemia treatment developed by Celgene, Agios [Reuters] The drug, Idhifa, will have a monthly list price of $24,872, Celgene said in an email, noting that the median time on therapy for patients was 4.3 months in the trial to secure the FDA's approval.
FDA approves new targeted treatment for relapsed or refractory acute myeloid leukemia [FDA] Idhifa (enasidenib) is an isocitrate dehydrogenase-2 inhibitor that works by blocking several enzymes that promote cell growth. If the IDH2 mutation is detected in blood or bone marrow samples using the RealTime IDH2 Assay, the patient may be eligible for treatment with Idhifa.
	5.6 ESMO
Exelixis-Discovered Compounds to Be Featured in 10 Presentations at ESMO 2017 Congress [Exelixis] “We look forward to this year’s ESMO Congress where new data from the CABOSUN trial of cabozantinib in patients with previously untreated advanced renal cell carcinoma will be presented, as well as the final analysis from the study exploring cabozantinib in combination with nivolumab and ipilimumab in genitourinary tumors, including metastatic urothelial carcinoma.”
	6.1 Observation
Patients with Advanced Cancer who Experience Sudden Cardiac Arrest Have Significantly Lower Survival Rates Compared to Patients without Cancer [ASCO] “This study reinforces the fact that doctors need to have early and frank discussions with their patients about end-of-life care. While in some cases it may be appropriate to attempt to resuscitate people with advanced cancer, patients should know all the facts and understand their options ahead of time."
Exercise linked to lower mortality with early prostate cancer [Reuters] For these men, regular moderate or vigorous physical activity was associated with 31 percent to 37 percent lower likelihood of death during the study, compared to more modest amounts of exercise.
	6.10 Politiques
Brexit Health Alliance warns that UK/EU negotiations put patients at risk [Pharmafile] Last month saw the formation of the Brexit Health Alliance, a partnering of key pharma and life sciences organisations including the ABPI, the BioIndustry Association and NHS Providers, all dedicated to vocalising and protect their collective interests throughout the negotiation process for the UK’s exit from the EU.
	6.11 Patients
Naughty Crabby [Of Tumors and M.E.N.] I am writing this blog partly as a legacy for my son, the youngest in my family to carry the burden of multiple endocrine neoplasia type 1. One day he too might need to undergo a Whipple, although I suspect by then that: a) this blog will seem as antiquated to him as, say, an 8-track does to me now, and b) the medical profession will have mastered less invasive ways to tame the pancreas.
	6.7.1 Bioinformatique
Better drugs, faster: The potential of AI-powered humans [BBC News] Scientists working in tandem with artificial intelligence (AI) could slash the time it takes to develop new drugs - and, crucially, the cost - say tech companies.
The Business of Artificial Intelligence [Harvard Business Review] If managers aren’t ramping up experiments in the area of machine learning, they aren’t doing their job. Over the next decade, AI won’t replace managers, but managers who use AI will replace those who don’t.