Audacious advances to discover new treatments for psychiatric brain disorders

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In recent years, the pace of the development of novel new treatments for brain disorders in both psychiatry and neurology, including psychiatric disorders, has been the subject of much worry and hand-wringing.1

Some major pharmaceutical companies have stopped research programs in neuropsychiatry to focus on other, “easier” therapeutic areas where they think the biology is better understood and therefore drug development is more feasible.

However, I am now more optimistic than I have been in many years that we are on the verge of a promising era of pharmacotherapy that will usher in far better prevention, diagnosis, and management of neuropsychiatric disorders, and a better outcome for our patients. Why the optimism? There is a series of converging trends that justify it.

Funding for basic neuroscience research. Governments all over the world have woken up to the fact that brain disorders will account for the largest economic impact unless new treatments are developed. This has spurred multiple initiatives to better understand the underlying neurobiologic mechanisms of the brain in health and disease.2

Renewed enthusiasm for brain disorders from small pharmaceutical and mid-size biotechnology companies. While some of the larger pharmaceutical companies have withdrawn from pursuing new treatments for psychiatric disorders due to the need to satisfy “shareholders,” small and nimble biotechnology companies have stepped up, seeing an opportunity in a field that is not overcrowded and still has an extensive unmet need. These companies are developing truly novel treatments and approaches that can differentiate from current treatments. These include:

  • rapid-acting antidepressants
  • targeting specific symptom domains of psychiatric disorders, such as cognition, apathy, or anhedonia, that currently have no adequate or effective treatment
  • novel therapeutic targets in a range of indications
  • nonpharmacologic approaches.

Leading companies in this space include Allergan and Blackthorn Therapeutics. These companies and others have publicly discussed their commitment to developing new treatments for psychiatric disorders.

But large pharmaceutical companies should not be discounted. Examples of advances by larger companies include the recent FDA “breakthrough designation” for the development of balovaptan by Roche, a medication with the potential to improve “core social interaction and communication” in patients with autism, and the work Johnson & Johnson is conducting with S-ketamine for depression and acutely suicidal patients.


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