The risks of developing many neurodegenerative diseases, including Alzheimer’s disease and amyotrophic lateral sclerosis, better known as ALS or Lou Gehrig’s disease, increase significantly with age. However, there have been very few significant technological advances in treating these devastating diseases over the last few decades.
The Search For Effective Treatments For Neurodegenerative Diseases
Researchers continue to search for life-altering treatments for neurodegenerative diseases that will lead to significantly improved patient outcomes. In ALS, the ultimate goals of treatment include slowing the progression of the disease and thereby extending the patient’s life.
However, the treatments that are currently available either treat only the symptoms of ALS or only extend the patient’s life by a matter of months rather than years. In fact, the high unmet need for effective ALS treatments has led the Food and Drug Administration to approve a drug without clear evidence that it works and to reconsider another drug candidate that also lacks evidence of efficacy.
In the case of Alzheimer’s, most of the drugs approved by the FDA are for those with mild-to-moderate disease. As a result, those with the worst symptoms may be left with very few options that will have meaningful impacts on their symptoms.
New Ways To Treat Long-Running Diseases
Even the standard of care for many neurodegenerative diseases leaves much to be desired. Thus, researchers have been searching for new ways to treat these illnesses in hopes of uncovering treatments that are more effective.
One of the more recent developments in the area of drug research has been a focus on inflammation as the root of all evil in many diseases — rather than a side effect of them. However, there may be a myriad of ways to target inflammation in neurodegenerative diseases.
As a result, many biotech firms are developing drug candidates that take very different approaches to treating them. One of the most promising and newest approaches to targeting ALS, Alzheimer’s, and, potentially at some point, autoimmune diseases, involves tapping into the body’s regulatory T cells, also known as Tregs.
Howard Berman, Ph.D., CEO and Chairman of Coya Therapeutics (NASDAQ:COYA), shares more details on how Tregs can be used to treat ALS and Alzheimer’s, with the potential for additional indications like autoimmune diseases.
Coya touts itself as the “most clinically advanced company focusing on Treg-modulating therapies.” The company estimates its total addressable market at more than $7 billion, including patients with ALS, frontotemporal dementia (FTD), lupus and other indications.
What are regulatory T cells and what do they do?
Tregs are a type of lymphocyte, which is a type of white blood cell, that modulates the body’s immune response. They are essential anti-inflammatory immune cells that play a role in regulating the body’s functions and establishing equilibrium.
Healthy Tregs act on multiple immune cells to reduce the release of pro-inflammatory cytokines, which include a variety of substances secreted by immune-system cells that then affect other cells. In addition to inhibiting the release of pro-inflammatory cytokines, their other main functions include improving the body’s natural inflammatory mechanisms and reactions. Tregs also help maintain self-tolerance, which is the immune system’s ability to recognize its own antigens as non-threats while responding to foreign substances.
What happens when Tregs become dysfunctional?
Dysfunctional Tregs can cause an inflammatory state that leads to neurodegenerative, autoimmune and metabolic diseases. In fact, Treg dysfunction is a core driver of neurodegeneration and has been associated with progression of ALS and other neurodegenerative diseases, including Alzheimer’s and frontotemporal dementia (FTD).
Dysfunctional Tregs also increase the burden of disease, which takes into consideration the health, social, political, environmental and economic factors to determine the cost a disease exerts on the individual patient and the society as a whole.
What role does inflammation play in neurodegenerative diseases, and how do dysfunctional Tregs cause inflammation?
Inflammation plays a critical role in neurodegeneration by reducing certain populations of neurons. For example, in ALS, inflammation in the nervous system causes a loss of motor neurons, which connect the brain to the spinal cord and the spinal cord to the muscles throughout the body.
As the inflammation progresses, more motor neurons die, reducing the brain’s ability to initiate and control muscle movement. Eventually, patients lose the ability to move, speak, and even breathe and eat, and ALS is usually fatal within two to five years of symptoms beginning.
Given their regulatory functions, dysfunctional Tregs can cause inflammation by either hampering the body’s immunity to a disease, like in cancer and infections, or failing to control unwanted immune responses, like in autoimmune diseases or allergies. In neurodegenerative diseases like ALS, it’s the latter situation.
Dysfunctional Tregs fail to control the body’s unwanted inflammatory response, so by boosting their function, we aim to control the inflammation and, by association, the disease. In ALS, dysfunctional Tregs have been linked to reduced survival and the rate of decline
Tell us about your current pipeline
Our drug candidates fall into two series: COYA 200 and COYA 300. Our focus is currently on the COYA 300 series, particularly COYA 302 for ALS.
In the second half of this year, we plan to initiate a Phase 2 trial in ALS and file an investigational new drug (IND) application for COYA 302. We’ve found that COYA 302 significantly expands the suppressive function of Tregs as early as four weeks after treatment is initiated and maintains a significantly increased Treg function.
COYA 302 also boosted Treg numbers as early as four weeks after treatment was initiated and maintained a higher number over the course of treatment. It also enhanced the suppression of oxidative stress and pro-inflammatory biomarkers over 48 weeks.
We also have just released proof-of-concept data on COYA 301 to treat Alzheimer’s this month. Our COYA 200 platform series is for neurodegenerative, autoimmune and metabolic diseases. We’re still gathering pre-clinical in-vivo efficacy data and working on disease-specific validations in animal models.
What are the latest results from your clinical trials?
We conducted a proof-of-concept open-label study for our drug candidate COYA 302 in ALS patients, and it’s now in the IND-enabling stage, which involves the preparations for filing an investigational new drug application. We plan to initiate a larger clinical trial for COYA 302 later this year.
We presented the latest results from our proof-of-concept study in March at the Muscular Dystrophy Association’s MDA Clinical and Scientific Conference in Texas. COYA 302 is designed to increase Treg activity and decrease the activity of pro-inflammatory immune cells, thereby reducing neuroinflammation.
In the study, four ALS patients received the treatment for 48 weeks, and researchers followed them for an additional eight weeks following the end of the trial period. Before they started the trial, the patients’ scores on the Revised ALS Functional Rating Scale (ALSFRS-R) were declining by about one point monthly.
During the study, the patients recorded no average decline on the scale from their baseline to 24 weeks and “minimal” average decline from baseline to 48 weeks. We’re now planning a larger, well-controlled Phase 2 clinical trial for our Treg-based therapy later this year.
We also just presented proof-of-concept data for COYA 301 in eight patients with mild-to-moderate Alzheimer’s disease indicating that the therapy was well-tolerated, increased Treg function in the blood, and resulted in statistically significant improvements in cognitive function, as measured by the Mini Mental State Examination (MMSE).
The author of the introduction, Michelle Jones, also edited this interview for clarity on behalf of Quantum Media Group, LLC and their client, Coya Therapeutics Inc.
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