New report shows more than 260 medicines in development to fight neurodegenerative diseases

With neurodegenerative diseases affecting millions of individuals in the United States, now is an important time to spotlight the increasing impact these diseases have on our country’s aging population and their families.

Neurodegenerative diseases occur when nervous system cells (neurons) in the brain, spinal cord and/or peripheral nervous system begin to deteriorate or become functionally impaired. As neurons deteriorate, people may first experience relatively mild symptoms, such as coordination issues or problems remembering names, but as more neurons are affected, symptoms progress and eventually some patients lose the ability to walk or function independently. Alzheimer’s disease, multiple sclerosis and Parkinson’s disease are the most common neurodegenerative diseases.

Because neurodegenerative diseases strike primarily in mid- to late-life and the risk of being affected increases dramatically with age, cases are expected to soar as many Americans are living longer and the population ages. If new and effective medicines are not found, more than 12 million Americans are projected to suffer from a neurodegenerative disease over the next 30 years. Neurodegenerative diseases alone already cost the U.S. economy more than $655 billion a year in medical expenses and economic losses.

Today, PhRMA released a new report detailing the more than 260 medicines in development for 29 neurodegenerative diseases, all of which are in clinical trials or awaiting review by the U.S Food and Drug Administration (FDA). While research and development (R&D) for neurodegenerative diseases has been challenging with more setbacks than success, biopharmaceutical innovation has resulted in significant new medical milestones and scientific advancements. The biopharmaceutical industry continues to work to explore new targets for earlier detection, treatments for development and ultimately, cures for neurodegenerative diseases.

The 261 medicines in development include:

• 85 medicines in development for Alzheimer’s disease, which affects more than 6 million Americans and, along with other dementias, will cost the American economy an estimated $355 billion in 2021.
  
  
• 64 medicines for Parkinson’s disease, which affects nearly one million Americans. Incidence of the disease is expected to rise to 1.2 million by 2030.
  
  
• 38 medicines for amyotrophic lateral sclerosis (ALS). About 5,000 people are diagnosed with ALS each year in the United States.
  
  
• 33 medicines for multiple sclerosis (MS), a chronic neurological disease that affects nearly one million Americans, a number that is substantially higher than previously reported based on a new 2017 study.
  
  
• 25 medicines for neurodegenerative genetic diseases, including Rett syndrome and spinal muscular atrophy (SMA). Both disorders are caused by a gene defect and affect about one in 10,000 children.
  
  
• 14 medicines for Huntington’s disease. There are more than 40,000 symptomatic Americans and another 200,000 at-risk of inheriting the disease with currently no cure for the disease.

Other neurodegenerative medicines in development target Alexander disease, Batten disease, Friedrich’s ataxia, and progressive supranuclear palsy and multiple system atrophy, among others.

While the pipeline for new treatments is strong, the battle to find successful treatments and cures against neurodegenerative diseases is challenging. For instance, R&D for these types of diseases is incredibly difficult because little is known about the biology of neurodegenerative diseases, there is a high failure rate for treatments and many neurodegenerative diseases are heterogenous conditions, meaning there are multiple root causes that make it difficult to diagnose and treat patients.

For decades, biopharmaceutical companies have focused on finding new treatments and cures for these diseases and hope is on the horizon as scientists continue to uncover how the central nervous system works at the molecular and genetic levels. Additionally, advancements in science and technology now allow the biopharmaceutical industry to leverage digital technology tools in R&D to improve drug development and speed access to medicines. Digital R&D is rapidly evolving with the potential to reduce barriers to innovation for novel medical products and therapeutics. Additionally, artificial intelligence/machine learning (AI/ML) platforms have been used to research neurodegenerative diseases and shown promise in early diagnosis, predicting the likely course of disease and aiding in the development of new therapies.

There has been tremendous progress in scientific and medical innovations for neurodegenerative diseases over the past decade, but continued efforts are needed to ensure America’s aging population is protected against potential life-altering threats. PhRMA and our member companies are dedicated to finding new ways to help those affected by neurodegenerative diseases and enable innovation.

To read the full neurodegenerative disease medicines in development report, click here.