Investing in Precision Medicine to Yield New Treatments for Neurodegenerative Diseases

Braeden William Lichti
5 min readNov 17, 2021


Advances in the collective genetic understanding of diseases, and the ability to identify disease biomarkers, is ushering in a new era of personalized medicine. Technologies such as CRISPR/Cas9 are also paving the way for improved, more tailored treatments targeted to a specific genetic marker of a disease. As our understanding of the molecular underpinnings of disease continue to improve, so, too, will the technologies at our disposal to treat them.

We’ve already seen the benefits of this type of personalized medicine in the cancer realm. Using a person’s (or disease’s) genes to drive cancer therapy is known as precision medicine. Precision medicine can help doctors identify high-risk cancer patients, choose treatment options, and evaluate treatment effectiveness. Precision medicine can also be used to prevent certain types of cancer, diagnose certain types of cancers early (leading to earlier treatment and better outcomes), and diagnose specific types of cancer more correctly.

As targeted therapies continue to advance, we will continue to see their impacts flow beyond that of the cancer realm. One area in which interest is ramping up is neurodegenerative diseases, which are chronic, progressive diseases affecting the brain and its constituent cells. Neurologic disease can be genetic, or caused by a stroke or brain tumor. Examples of neurodegenerative disease include Alzheimer’s Disease, Parkinson’s Disease, and Huntington’s Disease. These diseases have a genetic component, with specific genes playing a role in the development and progression of disease, especially in rare forms. Neurodegenerative conditions, like cancer, are devastating and costly. Collectively, neurodegenerative conditions cost people in the United States $655 billion in 2020.

Can we apply concepts from targeted therapies developed for cancer to create better outcomes for patients suffering from neurodegenerative diseases? What’s more, can precision medicine be used to treat other large unmet needs in the field of neurology, such as neuropsychiatry, pain, epilepsy, sleep disorders, and stroke?

Precision medicine in neuroscience and neurology is where many companies have dedicated their time and efforts. Three companies trading on the NASDAQ in this space that investors should research are Alnylam (NASDAQ: ALNY), Ionis Pharmaceuticals (NASDAQ: IONS), and Regeneron (NASDAQ: REGN).

Neuroscience research companies are clamoring to make use of the plethora of cellular and molecular biology data that is emerging about drugs and the patients who use them. There is much more information to be gleaned from diseases and patients than the genetics, which may not reveal information about the ways that genes are formally transcribed and expressed. Emerging technologies, therefore, also look at the RNA profiles of a drug response, patient, or disease state, called transcriptomics; and the set of proteins expressed by a cell, tissue, or organism, called proteomics. While a challenge with gene therapy is reimbursement by insurance providers, research is underway that can make gene therapies more common, and pave the way for more established insurance structures.

RNA targeting is an active area of research for neurodegenerative disease, with companies such as Skyhawk Therapeutics, Regeneron Pharmaceuticals, Alnylam Pharmaceuticals, and Takeda involved. By modifying genetic transcription via RNA technologies, these companies hope to develop novel treatments for disorders of the central nervous system. The study of RNA profiles in a given cell, tissue, or organism is known as transcriptomics, and this area will likely heat up as these researchers work to develop pioneering RNA technologies to target neurodegenerative disease.

Proteomics, or the study of the proteins expressed by a cell, tissue, or organism, will also play a role in precision medicine for neurological disorders. In June 2021, the United States Food and Drug Adminstration approved the first therapy addressing the underlying biology of Alzheimer’s disease. The drug, Biogen’s Aducanumab, is a monoclonal antibody therapy that works by clearing a substance known as beta-amyloid, a protein that scientists believe causes Alzheimer’s, from the brain. The drug, which was found to exhibit a unique proteomic profile upon treatment in mice, was the first approved for Alzheimer’s in 20 years, and while it is thought to be effective in a limited number of Alzheimer’s disease cases (namely, people in the early stages of Alzheimer’s), it represents a step forward in neurodegenerative disease research.

The FDA’s approval of Aduhelm, which was under an accelerated timeframe, has created more interest in the area of Alzheimer’s and Parkinson’s disease treatments. Scientists believe that a protein called tau is more closely associated with dementia than beta-amyloid, so they are also seeking to develop drugs targeting tau protein. In the realm of Parkinson’s disease, research is underway to target a compound called alpha-synuclein, which, like amyloid beta and tau protein in Alzheimer’s, is associated with cognitive decline in Parkinson’s disease. There are a number of approaches in development to target tau. Investors can expect many more biotech companies and venture firms moving into this space to develop innovative and alternative treatments.

This work is not without significant challenges. One obstacle in neurodegenerative research is creating drugs that can bypass the brain’s blood-brain-barrier, which keeps the brain safe from toxic substances or pathogens that would otherwise make their way into the brain. Another challenge is the fact that neurodegeneration affects a subset of neurons, which may have different levels of vulnerability to such disease. It is not yet fully clear which factors predispose certain neurons to develop pathology over others.

Yet as drug discovery continues to leverage the latest techniques in genomics, transcriptomics, and proteomics, and combinations of these technologies, this will unlock new potential for companies to create novel, increasingly personalized, therapies. For example, advances in genomics may provide insight into how neurodegeneration occurs in the brain.

Drug discovery in neurodegeneration also overlaps with that of other diseases, due to common disease pathways. For example, phosphatidylinositol 3-Kinase (PI3K) inhibitors are implicated not only in COVID-19 and breast cancer, but also Parkinson’s Disease. Stem cell therapies, which could benefit patients suffering from many conditions, can also have significant applications in the neurodegenerative realm. Stem cells could potentially be used to restore lost brain tissue, or to release compounds such as anti-inflammatory factors and growth factors supporting repair of the nervous system. Stem cell therapies, which are already in use for conditions such as cancer, could thereby restore function to neurodegenerative patients. Therefore, advances made in the treatment of other disease states could potentially innovate the field of neurodegeneration as well.