"Right to Try" Signed into US Law

Many interesting medical news items have been released last week, but it seems worth underlining a regulatory development: A bill giving terminally ill patients the right to try experimental treatments not approved by the government has been signed into law in the US, BBC News reports.

The new "Right to Try" act allows patients with life-threatening diseases, who have exhausted approved treatments and clinical trial options, to bypass the FDA's application process for "compassionate use" of experimental drugs. Only the approval of the patient’s own physician and the drug manufacturer is required, and the new bill protects physicians and companies from legal risks.

In my own opinion, this development is unambiguously good. Therefore, I am surprised and saddened by seeing that many reactions are negative. I can only attribute this to partisan political reasons, which (again, in my own opinion) is unambiguously bad.

Single CAR T cell triggers remission in leukemia patient. Researchers at University of Pennsylvania School of Medicine have reported that a patient treated for chronic lymphocytic leukemia (CLL) in 2013 went into remission because of a single CAR T cell and the cells it produced as it multiplied. The patient has stayed cancer free in the five years since, with CAR T cells still present in his immune system. A study published in Nature indicates that the response is tied to where the CAR gene inserted itself into the patient’s T cell DNA, a key factor that may help improve response rates to the therapy.

Flexible tactile sensory system for prosthetics and robotics. Scientists at Stanford and Seoul National University have developed an artificial nervous system that could give prosthetic limbs or robots reflexes and the ability to sense touch. The research results, published in Science, are considered to be a step toward creating artificial skin for prosthetic limbs, restoring sensation to amputees and, perhaps one day, giving robots some type of reflex capability. A review, also published in Science, describes the development as a “neuromorphic” tactile sensory system based on organic, flexible, electronic circuits that can measure the force applied on the sensing regions, exploiting organic electronics that allow for three-dimensional printing of flexible structures that conform to large curved surfaces, as required for placing sensors on robots and prostheses.

Circuit repair mechanism in the retina restores neural connections after injury. Studying neural circuit repair in the retina of laboratory squirrels, scientists at UC Santa Cruz and Stanford University have found that neurons can make new connections to the right types of photoreceptors to restore selective connectivity after an injury. A research paper published in Current Biology indicates that neural cells left without a connection to a photoreceptor start to grow new dendrites and form new connections.

First 3D printed human corneas. Scientists at Newcastle University have 3D printed the first human corneas, prototyping a technique that could be used in the future to ensure an unlimited supply of corneas.. A study published in Experimental Eye Research describes how stem cells (human corneal stromal cells) from a healthy donor cornea were mixed together with alginate and collagen to create a “bio-ink” that could be printed. Using a simple low-cost 3D bio-printer, the bio-ink was successfully extruded in concentric circles to form the shape of a human cornea. It took less than 10 minutes to print.

Toward therapeutic options for age-related memory decline. Scientists at Translational Genomics Research Institute and Northwestern University have found indications that having resilient memory performance during aging could be inherited. A research paper published in Frontiers in Aging Neuroscience shows that “Super Agers” - people in their 80s and beyond who still have the memory capacity of those 30 or more years younger - have genetic changes in the MAP2K3 gene, and suggests that therapies targeting this gene could reduce age-related memory decline, and perhaps the threat of memory loss posed by Alzheimer’s disease.

New non-invasive laser treatment for myopia and astigmatism. Researchers at Columbia Engineering have developed a new promising non-invasive approach to permanently correct vision problems. A study published in Nature Photonics describes a femtosecond oscillator, an ultrafast laser that delivers pulses of very low energy at high repetition rate, for selective and localized alteration of the biochemical and biomechanical properties of corneal tissue. The technique, which changes the tissue’s macroscopic geometry, is non-surgical and has fewer side effects and limitations than those seen in refractive surgeries. The scientists are persuaded that the new approach could lead to treatment for myopia, hyperopia, astigmatism, and irregular astigmatism.