A study that explores whether vitamin D prevents asthma attacks in children is being criticized as unethical. All of the study participants have serious asthma and low levels of vitamin D, but only half of the participants in the trial are getting high doses of vitamin D, while the other half will receive a placebo, leaving their clinically low level of vitamin D untreated. (Boston Globe)
How to improve adjuvant clinical trials? Include only patients for whom current therapies do not work well
A limitation of modern breast cancer adjuvant trials is that there are often not enough events in the experimental arm to demonstrate an effect, so we don’t know if the new drug actually works. The authors propose a method to make adjuvant clinical trials more effective by including in the trial only patients for whom current therapies do not work well — only if their risk for cancer recurrence with current best therapies is higher than a prespecified risk. (MedicalResearch.com)
A “gatekeeping” statistical approach can avoid false-positive results in clinical trials
Clinical trials characterizing the effects of an experimental therapy rarely have only a single outcome of interest, but comparing a large number of outcomes using the significance threshold of 0.05 runs a high risk of falsely concluding that a significant effect is present when none exists. In a study with 16 secondary endpoints, the investigators used a serial gatekeeping approach (an alternative to the Bonferroni approach) for statistical testing to minimize the chance of false positive results. (The JAMA Network)
Why so little quality research on the risks and benefits of medical marijuana?
A growing number of Canadians are turning to medical cannabis, despite questions about its risks and effectiveness as a health product, as there has been little quality research into medicinal marijuana. Pharmaceutical companies fund clinical studies of their products, but marijuana producers have no incentive to conduct research and it’s difficult to create a plausible placebo for marijuana studies. (CTV NEWS Health)
Time to abandon our model for Phase 1 dose escalation studies?
The primary aim of dose-escalation trials (Phase 1 trials) is often to find the largest dose of a treatment that can be administered safely, often called the maximum tolerated dose (MTD). Rule-based designs such as the 3+3 design continue to be popular even though they can perform extremely poorly. The alternative to these rule-based designs are (Bayesian) model based approaches which use a statistical model to describe the dose-response relationship. The author lists several arguments why rule-based designs should not be used for Phase 1 dose escalation studies. (openaccessgovernment.org)
Cannabis therapy trials may overestimate beneficial effects
The benefits associated with medical cannabis therapy may be overestimated because of inadequate blinding in clinical trials. Many trials are flawed by inadequate blinding because subjects can distinguish between active cannabis and placebo. It is difficult to design a well-blinded study for cannabis therapy; in the meantime we should use caution when interpreting the findings of cannabis trials. (Psychiatry Advisor)
Guest blog. Using Exercise as Medicine: An Interview with Dr. Webb Smith, Clinical Exercise Physiologist at Le Bonheur Children’s Hospital in Memphis, TN
On June 27th, 2017, Tanya Verstak interviewed Dr. Webb Smith about how his clinic uses exercise as medicine to treat pediatric obesity at Le Bonheur Children’s Hospital in Memphis, TN. He speaks about his extensive background and training in exercise physiology has led him to develop a clinical program that Read more
Report by Ahmed Enany from SoCal Bio outlines impact of NIH cuts on CA
According to a new report by Ahmed Enany, CEO of SoCalBio, the 20% cuts in NIH budget proposed by the Trump administration may put health research and jobs at risk. In 2016, NIH allocated most of its funds to extramural research grants — $24.83 billion out of a $32 billion total budget. Of all states, California got the most, with $3.7 billion or 15% of NIH extramural grants. Most of this goes towards basic and applied research at non-profit educational institutions and research hospitals, with the top-receiving CA organizations being UCSF, Stanford University, UCSD, UCLA, and Scripps. San Diego organizations winning the largest NIH grants were UCSD, Scripps, and Sanford Burnham Institute. The top winning investigators were Eric Topol, Dennis Burton, and Howard Feldman, and the top funding foci were digital health and translational science, Aids, and Alzheimers. (slideshare.net, Ahmed Enany)
Microbiome therapy pioneer Seres restarts failed microbiome study
Seres Therapeutics has redesigned their SER-109 clinical study of a fecal transplant aiming to treat Clostridium difficile infection, which failed last July, and will re-initiate it, with FDA’s guidance. Seres is the first publicly held microbiome-focused company, and, if approved, SER-109 would be the first approved microbiome therapeutic in this new field of medicine. (Industry Dive)