Examining How Biotherapeutic Approaches Can Be Used to Treat Metabolic Disorders

Biologics Development

Metabolic diseases can have devastating effects on patients. Can a biotherapeutic approach bring them relief?
Image source: Wikimedia CC user Nephron

Metabolic disorders, like diabetes, typically arise when a gene responsible for encoding a necessary enzyme is defective. Since enzymes are crucial for the body to function properly, it should come as no surprise that problems occur when these pathways are disrupted. Since they’re due to faulty genes, metabolic disorders have no cure and patients with these conditions have to observe strict dietary regimens to keep their symptoms in check. Such regimens can be difficult for patients to maintain and can severely impact quality of life.

A Biotherapeutic Approach Might Offer the Solution

Given the disadvantages of current treatment plans, metabolic disorders have become a topic of interest for biotherapeutic companies. Since these conditions are caused by malfunctioning enzymes, therapeutic proteins offer a potential treatment. Instead of relying on restrictive diets, biotherapeutics could be used to deliver the missing enzymes into a patient’s body.

For example, people born with the metabolic disorder phenylketonuria (PKU) lack the necessary enzyme to break down the amino acid phenylalanine. When phenylalanine builds up in the body, it causes severe problems like intellectual disability, developmental delays, hyperactivity and neurological problems.1 Since phenylalanine is found in protein, people with PKU must completely avoid foods like meat, milk, nuts and beans. To further complicate matters, certain artificial sweeteners release phenylalanine when digested. Given how many foods are on the “avoid” list, PKU patients often struggle with following their restrictive diet.

To solve this problem, researchers are looking into biotherapeutic alternatives. This past August saw the announcement of a partnership between two companies seeking to develop a novel biotherapeutic approach to treating PKU. The treatment builds on ActoBiotics, a bacterium that’s been engineered to secrete biotherapeutic proteins and peptides.2 While most therapeutics are administered via injection and intravenous infusion over extended periods of time, the ActoBiotics treatment is delivered orally, making it efficient as well as painless.

Ideally, ActoBiotics would deliver the key enzyme needed to break down phenylalanine, thereby preventing toxic buildup in the bodies. Such a breakthrough would vastly improve the lives of people with PKU. While it wouldn’t necessarily mean doing away with the careful diet, it could potentially ease diet restrictions and make a lapse in the diet regimen less serious.

The Development Process: Biotherapeutics in Action

Biotherapeutic development that involves building upon existing platforms has a different set of challenges than discovery. In the case of this potential treatment for PKU, we already know the delivery vehicle. Now the difficulty is determining the next steps. Can the bacteria be engineered to synthesize and secrete the key enzyme? If research cannot proceed past this stage, everything else is a moot point. Further, which receptor cells in the gastrointestinal tract should the engineered bacteria target? It’s important to select sites that provide an environment ideal for enzyme synthesis and secretion. And finally, firms need to make sure that when the biotherapeutic is administered orally, it proves effective. Does it actually reach the target receptor cells? If so, do they successfully secrete a functional enzyme that repairs the broken metabolic process? All of these questions must be answered and evaluated through careful research and recording.

Throughout this process, flexibly managing research is important and it’s equally crucial to avoid getting overwhelmed by data. Researchers need tools that enable them to analyze complex results and glean key insights to aid in the decision-making process. As part of the BIOVIA Biologics Solution, researchers have access to tools that equally support research and development workflows. The solution allows users to easily manage the discovery, formulation development, manufacturing and related testing of biotherapeutics. Contact us today to learn more.

  1. “Phenylketonuria (PKU),” Last updated November 26, 2014, http://www.mayoclinic.org/diseases-conditions/phenylketonuria/basics/definition/con-20026275
  2. “ActoBiotics,” Last updated February 1, 2013, https://en.wikipedia.org/wiki/ActoBiotics

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