The toll that neurological disorders exact on patients and their families is truly tragic. Yet, looking forward there is cause for real optimism for some conditions. I see the promise of an ever-growing range of new technologies and approaches applied to neuroscience research, and looking back I see that step by step, research does lead to successful new treatments.

NINDS now proudly unveils a new segment of our website dedicated to highlighting some of these successes, both past and recent. The site, “NINDS Contributions to Approved Therapies,” provides timelines and narratives documenting the development of treatments for neurological disorders for which NINDS played a critical role. The first two posts focus on tissue plasminogen activator (tPA) for acute ischemic stroke (approved by the FDA in 1996), and nusinersen for spinal muscular atrophy (approved in 2016). These two examples illustrate the many ways NINDS and the scientists we support contribute along the spectrum of therapy development – from basic science to understand disease mechanisms, to translational research to turn knowledge into new medicines and devices, and clinical trials to assess these therapies in people.   

The development of tPA revolutionized acute stroke care and is estimated to save $4 million for every 1,000 patients treated, due to improved outcomes. NINDS funded early studies that bolstered the rationale behind using tPA for acute ischemic stroke, led pivotal clinical trials that treated stroke patients with unprecedented speed, and even promoted public awareness of stroke and the benefits of timely treatment. The development of tPA also set the stage for further advances. Earlier this month, results from an NINDS-funded clinical trial showed that the time window for removing clots in acute ischemic stroke can be extended dramatically through the use of brain imaging to identify patients with salvageable brain tissue. As a more recent example, nusinersen is the first treatment approved for SMA, a leading genetic cause of death in infants and toddlers. NINDS and other NIH institutes supported research to uncover SMA’s genetic mechanisms, identify a treatment strategy, and facilitate later stage translational and clinical research. The history of nusinersen also highlights the central role of patients and non-profit organizations as partners in our shared mission to reduce the burden of neurological disorders.

I invite you to learn more about the development of these two treatments and to check back throughout the year as we add new success stories to this site.

Related Link:

NINDS Contributions to Approved Therapies

Friday, February 9, 2018