- The ‘cruel joke’ of compassionate use and right to try: Pharma companies don’t have to comply (statnews.com)
...the FDA has a compassionate use program to allow people access to experimental drugs, it can’t compel a company to provide those drugs. The newly signed “right-to-try” law doesn’t either...the Food and Drug Administration calls its expanded access program, also known as compassionate use. It governs the use of an investigational medicine that has not been approved by the FDA outside of a clinical trial...Here’s how it is supposed to work. A physician caring for a patient with a terminal illness who has exhausted all other treatment options and isn’t eligible for a clinical trial appeals to the pharmaceutical company to provide an investigational drug that has undergone at least a Phase 1 trial, which studies the safety of a drug. If the pharmaceutical company agrees, the treating physician applies to the FDA for approval for expanded access to the investigational drug...Large pharmaceutical companies are notoriously risk averse when it comes to expanding access to medications that are still in the testing phase. Many refuse to grant access to investigational drugs outside of clinical trials...One fear they have is that an adverse event, like an injury or death — even if it is not directly due to the medication — will derail a company’s ability to push a drug forward for FDA approval...Pharmaceutical companies also worry that if an experimental medication is given to one patient through compassionate use, it must be given to all patients who request it. In the case of rare diseases...this could mean that a company would have trouble enrolling enough patients when it eventually opens a clinical trial...
- Getting Ahead with Modafinil: Is the Hottest New Smart Drug Safe? (mensjournal.com)
..latest wonder drug to make headlines sounds like a dream for ambitious workers: Modafinil, a narcolepsy pill, has been shown to make you a sharper thinker, a better decision maker, and generally more alert – whether or not you have a sleep disorder, researchers found few safety concerns or side effects from the drug…main problem…no long-term studies of safety and efficacy in healthy people,..Without safety studies,. .."we do not know that modafinil is safe in the long-term for people to use."
- Lab-Grown Mini Organs Could Speed Up Drug Discovery (forbes.com)
The thought of lab-grown organs conjures up Frankenstein-like imagery. The reality however, is somewhat less visually dramatic, with the term ‘organoids’ used to describe tiny 3D structures of human tissue, a millimeter or so in diameter...these tiny lumps of cells are creating a lot of excitement in the world of medical research...Cells in dishes and animal models have been used for preclinical testing of drugs for decades. Success in these experiments is a key hurdle for any new medicine to overcome before being given the green light for all-important human clinical trials...Organoids are most commonly made either from a small sample of tissue needled out of a person or from stem cells cultured in a cocktail of nutrients intent on pushing them towards becoming a particular tissue type. So far, organoids have been made resembling several tissues including lung, liver, brain, kidney and intestine...as a relatively new innovation they are being used to investigate dozens of conditions from infectious diseases to cancer.... A study published last year in Science Translational Medicine by scientists at the University Medical Centre, Utrecht generated organoids formed from the rectal tissue of 71 people with cystic fibrosis and exposed them to experimental drugs. By observing changes in the organoids, the scientists accurately predicted which patients would respond to the therapies in just one week at a cost of around $1200 per patient. The results were so convincing that a positive organoid test is now considered sufficient evidence for insurance companies to fund the new therapies in the Netherlands...
- Organ-on-a-chip startup Emulate raises $28.75M Series B (medcitynews.com)
Cambridge organ-on-a-chip startup Emulate has raised $28.75 million in a Series B round, with aims to commercialize its "Human Emulation System" on the R&D circuit. The idea, as with most lab-on-a-chip technologies, is to enhance in vitro research so as to make animal and in-human studies more efficient – using algorithms and microfluidics to accurately predict human response to medicines, chemicals and diseases...The financing will help expand Emulate’s organ-on-a-chip portfolio. Currently, it’s got organ emulations of the lung, liver, intestine and skin – but it plans to branch into organs like the kidney, heart and brain. Beyond that, however, is the concept of developing specific labs-on-chips to address important disease states, such as cancers, disorders of the intestine and microbiome, and infectious disease...Emulate wants its technology to operate as a plug-and-play system in the hands of product development teams at pharmaceutical, chemical, food and consumer products companies...