The need for a personalized approach to restorative therapy

Incurable disease, such as certain cancers and neurodegenerative disease, among others, affects millions of persons worldwide. To date, most research and therapeutic interventions for these diseases have focused on limiting or slowing disease progression. At Above and Beyond Concierge Therapeutics, we pursue the restoration of function and the hope of a cure – through rapid and personalized novel disease characterization and therapeutic intervention development and testing.

Advances in medical science and technology have illuminated three findings that largely determine the future likelihood of significant prognosis First, for treatments to be successful they must account for individual variability – in genes, their environment and their lifestyle. Neurodegenerative diseases manifest differently from person to person. A gene mutation in one affected individual may not be present in another person with the same diagnosis. Secondly, radical improvements in prognosis and outcomes seem possible through translational medicine – appropriating technology or interventions from one disease and, with modifications, applying it to the treatment of other seemingly unrelated diseases. Finally, a structure must be on place to vastly accelerate the translation of personalized bio-assessment into person-specific therapeutic interventions that can scale accordingly.

These new realities – tailoring treatments to an individual’s biology and repurposing diagnostic and therapeutic interventions – underpin the science behind Above and Beyond’s mission. We focus on patients with incurable conditions at stages that are above modern medicine’s capabilities, matching the research we facilitate to the exact condition, disease, genetic, and non-genetic factors of the individual. Our unique approach blends private health management with rapid-paced individuated therapeutic-driven research. Our multidisciplinary Scientific Advisory Board brings over 100 combined years of unparalleled clinical and research expertise, each member having established groundbreaking discoveries in diagnostics, pharmacology, technology, and clinical procedures. Our ultimate aim is to accelerate the pace and quality of medical interventions, moving patients beyond protective offerings – the preservation of function – to the point where function and health can actually be restored.

 

Patient-derived restorative and curative science

The realization of this goal requires novel research based on front line science of the kind being led by Above and Beyond Concierge Therapeutics. We approach this through work in two parallel tracks – advancing therapies that delay disease progression while pursuing novel restorative, and ultimately curative, techniques. Above and Beyond has advanced gene therapy through the use of client-specific induced pluripotent stem cells (iPSCs) – reprogramming them to form healthy motor neurons or other desired cell types. The products of these efforts are extremely useful for disease modeling where it is possible to recreate elements of a patient’s disease in a laboratory dish, thereby vastly facilitating diagnostics and therapeutic tests such as drug screening. Potential drug candidates are also identified through transcriptomics, the identification of the complete set of RNA transcripts that are produced by a genome. A second major area of protective-oriented research undertaken by Above and Beyond Concierge Therapeutics involves the administration of Riluzole, an FDA-approved oral treatment for ALS, directly into the spinal cord (intrathecal injection). This novel route of administration for a previously approved drug, requiring new techniques and equipment, was shown to produce a ten-fold increase in drug concentration compared to the traditional oral route. A significant potential advantage to this approach is reduced liver impact by direct administration to the Spinal Cord. Sustained drug levels in the spinal cord tissue were recorded over time, and there were no observed adverse reactions and no brain- or liver-toxic effects.

Beyond motor neuron protection, our science is also directed toward neuromuscular restoration, allowing individuals to recover previously lost motor function. We are prioritizing two efforts in this regard: diaphragmatic pacing and fingertip control. Restoring function to diaphragmatic muscles would reduce or eliminate respiratory inefficiency, common to ALS patients with advanced disease. Rejuvenating fingertip neuromuscular ability would greatly facilitate an individual’s access to touch-screen technology through which communications and environmental manipulations can be greatly enhanced.

Through our expert staff, our incomparable multidisciplinary Scientific Advisory Board, and our premier biotechnology partners, Above and Beyond Concierge Therapeutics leverages today’s science to create tomorrow’s possibilities.

Event: Shifting aims – The First Neuromuscular Restoration Summit