CNS disorders are a wide-ranging set of disorders in which the brain loses its normal functioning, limiting everyday ability to function. These may arise from various reasons-some are inherited, some result from damage, and some from infections. It contributes to the highest disability-adjusted life years. The current treatment options are minimal. Most of the disorders have no curative treatment available. For most CNS disorders, the development of effective drug candidates is challenging as clinical trials are often long-lasting and with considerable expense. The last few decades have been marked by increased research and development in neuroscience.

Positioning of Gene and Cell Therapies in Neurosciences

Decades of technological progress and careful research, Gene and cell therapy (GCT), which were previously viewed as the ‘future’ of medicine, are now all set to become a reality. Improving upon the currently available options. Gene and cell therapies hold the potential to provide a one-off cure. The major drivers that have enabled this vision are the advent of safe and effective vectors, newer gene-editing tools, and huge unmet needs for the treatment of devastating CNS disorders.

Race to be the first mover

The possibility of high end-rewards in form of high premium pricing and an untapped patient pool has ensured a busy pipeline activity in this space in hopes of a sharp increase in revenues. Currently, pharmaceutical companies such as Novartis, Hope Biosciences, Roche, Brainstorm Cell Therapeutics, Helixmith, Libella Gene Therapeutics, etc. are the key players that are expanding their pipelines to incorporate GCT to treat a range of CNS disorders. Besides, some pharmaceutical companies are also trying to be a part of this innovation by actively entering into collaborations with other players.

Early Victories

Zolgensma by Novartis became the first gene therapy to be approved by the FDA  for the treatment of spinal muscular atrophy. Its high price of USD 2.125 million per patient, adds it to the list of most expensive treatments. In the US, Zolgensma generated around USD 360.7 million in the first year of launch.

Gene & Cell Therapies and Key Indications

The key therapies currently in the development phase for different CNS indications are Amyotrophic lateral sclerosis: NurOwn (BrainStorm Cell Therapeutics), Engensis (Helixmith), Q cells (Q therapeutics), VM202 (Helixmith); Spinal cord injury: Neuro-Cells (Neuroplast), Umbilical Cord Blood Mononuclear Cell (StemCyte); Parkinson’s disease: Axo Lenti PD (Axovant); Alzheimer Disease: AAV-hTERT (Libella Gene Therapeutics), VY-AADC (Voyager Therapeutics). Analysts at DelveInsight estimate a combined market of USD 3,753.6 million in the United States by end of 2026, with a CAGR of 39.74% from 2018–2026.

Competition in the Market

The biggest competition for GCT is the development of antisense therapies. The classic example is Spinraza (Biogen), an antisense oligonucleotide that is the key contender to Zolgensma (Novartis) for the treatment of SMA. Both Spinraza and Zolgensma are expensive therapies However, Spinraza is more established being the first to enter the market in 2017. Zolgensma’s approval is in patients aged less than two, Biogen is also competing for those young patients. Nurture study results demonstrated that patients treated with Spinraza before symptoms matched development milestones typical for healthy babies.

Expected Roadblocks

GCT is usually priced at more than 30 times the median household income of an individual. One-time high payment may discourage payers who prefer significantly lower-cost treatment for a longer duration. Most gene therapies have been only developed recently and lack long-term efficacy data, making the long-term cost-effectiveness argument even more challenging. Besides, the therapy area also suffers from regulatory challenges. as the regulatory bodies currently lack historical process data; hence, making it hard for regulatory bodies to leverage regulatory experience. Also, the lack of guidelines around plasmid quality, animal-free material complicates the regulatory process. Lastly, there are no standardized guidelines that increase the risk of failure of novel gene therapies. Nonetheless, the promising benefits of GCT, have the potential to sideline current roadblocks and emerge as a breakthrough treatment for CNS disorders.