Clinical Trial of Promising Treatment for Cystic Fibrosis Begins

A joint public-private effort led by scientists from the University of Illinois Urbana-Champaign and the University of Iowa, in collaboration with biotechnology company cystetic Medicines, has initiated a clinical trial for a potential breakthrough treatment for cystic fibrosis (CF). The inhalable molecular prosthetic, named CM001, aims to improve lung function in CF patients who do not benefit from current therapies. This study represents a significant step towards developing a safe and effective treatment for the progressive genetic disorder.

Clinical testing has commenced for a groundbreaking treatment for cystic fibrosis, led by scientists from the Carle Illinois College of Medicine at the University of Illinois Urbana-Champaign and the Carver College of Medicine at the University of Iowa, in partnership with cystetic Medicines, a biotechnology spin-out company. The trial focuses on an inhalable molecular prosthetic, CM001, which aims to enhance lung function in individuals with cystic fibrosis who do not respond to existing treatments.

The launch of this clinical trial marks an important milestone in a collaborative effort between the public and private sectors to develop a safe and effective treatment for cystic fibrosis. The genetic disorder is characterized by persistent lung infections that can cause severe long-term damage.

Lead researcher Martin Burke, a professor at CI MED, expressed optimism about the potential of the new treatment: “We’re hopeful that for those who have held their breath for far too long, this could be a first opportunity to regain ion-channel-like function in the airway and thereby address CF at its roots.” The research team, working in conjunction with scientists from the University of Iowa, aims to provide a treatment option that addresses the fundamental defect in CF.

Recently, the first human volunteers in New Zealand began receiving the new inhaled drug CM001, which acts as a molecular prosthetic by replacing missing or dysfunctional protein channels. The drug employs a novel inhaled dry powder formulation to directly target the lungs, allowing for consistent dosing. Burke explained the potential benefits of this approach: “With this method, people with CF could directly deliver this molecular prosthetic to their lungs where they need it most, hopefully increasing its efficacy and safety.” The ongoing clinical trial will evaluate the drug’s safety, tolerability, and movement within the body.

Cystic fibrosis is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which affects the production of the protein responsible for maintaining the balance of anions and fluid in the lungs and other organs. Dysfunctional CFTR protein leads to the formation of thick, sticky mucus in the lungs, resulting in breathing difficulties and frequent infections. While a class of drugs known as CFTR modulators can restore protein-channel function in many CF patients, those who produce little or no CFTR protein have limited treatment options.

Burke and his team believe that CM001 can act as a molecular prosthetic, replacing the missing protein and restoring ion channel function. The initial focus is on providing benefits to the 10% of the CF population that does not respond to CFTR modulators. Additionally, the concept of molecular prosthetics holds potential for treating all individuals affected by cystic fibrosis, regardless of their specific CFTR gene mutation.

Jeffry Weers, Chief Technology Officer of cystetic Medicines, emphasized the potential of molecular prosthetics in transforming the treatment landscape for cystic fibrosis. He stated, “The concept of molecular prosthetics has the potential to alter the treatment landscape for cystic fibrosis in a profound way; in theory, it should work for all people dealing with the disease, regardless of the type of mutation they present.”

The collaborative efforts between the University of Illinois Urbana-Champaign and the University of Iowa have been instrumental in advancing the molecular prosthetic approach to treating cystic fibrosis. The translational, multi-disciplinary approach adopted by the research teams has allowed for significant progress. Financial support from both public and private sectors, including a $25 million investment from Deerfield Management, has contributed to the development of this potential treatment. Non-profit organization Emily’s Entourage has also provided grant funding for early-stage research in the labs of Dr. Burke and Dr. Welsh.

The clinical trial has received assistance from individuals within the CF community, who provided cells through the University of Iowa’s Cystic Fibrosis Research Center. Agnieszka Lewandowska, a senior research scientist at UIUC and a member of Dr. Burke’s lab, expressed gratitude for their contribution: “We are grateful for the willingness of the CF community that provided cells through the University of Iowa’s Cystic Fibrosis Research Center that ultimately brought these compounds to clinical trial.”

Dr. Burke envisions that the impact of molecular prosthetics extends beyond cystic fibrosis, offering potential treatment options for a wide range of diseases caused by loss of protein function. He believes that success in treating cystic fibrosis could pave the way for a general approach to address such diseases. The development of molecular prosthetics is part of the broader mission of the Molecule Maker Lab at the Beckman Institute in UIUC, in collaboration with the physician-innovators at the Carle Illinois College of Medicine.

Results from the clinical trial are expected to be available in 2024.

Takeaway

  1. Scientists from the University of Illinois Urbana-Champaign and the University of Iowa, in partnership with cystetic Medicines, have initiated a clinical trial for an inhalable molecular prosthetic, CM001, aimed at improving lung function in cystic fibrosis (CF) patients who do not respond to current therapies.
  2. This joint public-private effort represents a significant step forward in developing a safe and effective treatment for CF, a progressive genetic disorder characterized by persistent lung infections that can lead to severe damage over time.
  3. The clinical trial involves human volunteers in New Zealand who are receiving the inhaled drug CM001, which acts as a molecular prosthetic to replace missing or dysfunctional protein channels in CF patients’ lungs. The novel inhaled dry powder formulation allows for targeted delivery and consistent dosing, potentially increasing efficacy and safety.
  4. The study focuses on addressing the fundamental defect in CF caused by mutations in the CFTR gene. By serving as a molecular prosthetic, CM001 aims to restore ion channel function and benefit the 10% of CF patients who do not respond to current CFTR modulators. The concept of molecular prosthetics holds promise for treating all individuals with CF, regardless of their specific CFTR gene mutation.

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