A Potential Remedy for COPD? Utilizing Patients’ Own Lung Cells Shows Promise
In a notable breakthrough, researchers have harnessed the therapeutic potential of patients’ own P63+ lung progenitor cells to mend damaged lung tissue in individuals grappling with Chronic Obstructive Pulmonary Disease (COPD). This groundbreaking development has resulted in tangible improvements in the quality of life for affected patients. Preliminary outcomes from the initial phase of clinical trials have yielded promising results, showcasing enhancements in lung function, diminished breathlessness, and improved exercise capacity. The research is now advancing into the second phase of clinical trials.
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Researchers have, for the first time, demonstrated the feasibility of rejuvenating injured lung tissue in individuals afflicted by Chronic Obstructive Pulmonary Disease (COPD) by employing their own lung cells.
A recent study, presented at the European Respiratory Society International Congress held in Milan, Italy, unveiled the transformative potential of an experimental treatment. The treatment, tested on 17 participants during a phase I clinical trial, resulted in enhanced respiratory capabilities, increased walking distances, and an overall elevation in the quality of life for these individuals.
COPD, a severe respiratory ailment responsible for claiming the lives of approximately three million people worldwide each year, entails the progressive deterioration of lung tissue. Current treatment methods merely alleviate symptoms by widening airways to facilitate improved airflow, utilizing medications known as bronchodilators.
To explore novel approaches to combating COPD, researchers have turned their attention to stem cells, renowned for their ability to differentiate into diverse cell types within the body. Additionally, progenitor cells, derived from stem cells, possess the unique capacity to differentiate exclusively into cells corresponding to their tissue or organ of origin. These cells are typically enlisted by the body to repair and replace damaged tissue. However, the outcomes of previous investigations into stem cell therapy, particularly in COPD cases, have yielded conflicting results.
Professor Wei Zuo, hailing from the School of Medicine at Tongji University in Shanghai, China, and serving as the chief scientist at Regend Therapeutics Ltd in China, has been at the forefront of research exploring the potential of P63+ lung progenitor cells to regenerate COPD-damaged lung tissue.
During an address at the congress, Professor Zuo remarked, “Stem cell and progenitor cell-based regenerative medicine may be the biggest, if not the only, hope to cure COPD. P63+ progenitor cells are known for their ability to regenerate the tissues of the airways, and previously we and other scientists have shown in animal experiments that they can repair the damaged epithelial tissue in the alveoli – the tiny air sacs in the lungs that play a crucial role in the exchange of gases between air breathed in and the blood supply to the lungs.”
In this inaugural phase I clinical trial, researchers embarked on a quest to assess the effectiveness and safety of extracting P63+ progenitor cells from the lungs of 20 COPD patients. These cells were subsequently cultivated in the laboratory to produce millions more, before being reintroduced into the patients’ lungs through transplantation.
Professor Zuo elucidated, “In our trial, 35% of the patients had severe COPD and 53% had extremely severe COPD. Usually, many patients with such severe COPD will die quite quickly if their disease progresses. We used a tiny catheter that contains a brush to collect the progenitor cells from the patients’ own airways. We cloned the cells to create up to a thousand million more, and then we transplanted them back into the patients’ lungs via bronchoscopy in order to repair the damaged lung tissue.”
Out of the cohort of 20 patients, 17 underwent this transformative treatment, while three served as the control group. Within 24 weeks of treatment initiation, the patients were rigorously evaluated to gauge their tolerance to the treatment and its overall efficacy.
Remarkably, all patients exhibited excellent tolerance to the cell treatment. After 12 weeks, the median diffusing capacity of the lungs (DLCO), a critical metric assessing gas exchange between the lungs and bloodstream, surged from 30% before treatment to 39.7%, further climbing to 40.3% at the 24-week mark among treated patients. Additionally, the median distance covered in a six-minute walk distance test (6MWD) expanded from 410 meters prior to treatment to an impressive 447 meters after 24 weeks. The median score in a quality-of-life evaluation (St George’s Respiratory Questionnaire or SGRQ) diminished by seven points, indicating a notable improvement. Notably, in two patients presenting with mild emphysema, a form of lung damage traditionally deemed permanent and progressive, the treatment facilitated the repair of the lung damage.
Professor Zuo affirmed, “We found that P63+ progenitor cell transplantation not only improved the lung function of patients with COPD but also relieved their symptoms, such as shortness of breath, loss of exercise ability, and persistent coughing. This means that the patients could live a better life, usually with a longer life expectancy. If emphysema progresses, it increases the risk of death. In this trial, we found that P63+ progenitor cell transplantation could repair mild emphysema, making the lung damage disappear. However, we cannot repair severe emphysema yet.”
With these compelling results in hand, researchers are preparing to embark on a phase II trial to further assess the treatment’s effectiveness within a larger patient cohort. Notably, this trial has received approval from China’s National Medical Products Administration (NMPA), the equivalent regulatory body to the US Food and Drug Administration (FDA). Consequently, the treatment remains unavailable to COPD patients and their physicians at large.
Professor Zuo conveyed his optimism, stating, “However, with more doctors and patients participating in our clinical trial, we may develop the treatment more quickly so that it can benefit patients sooner. A similar therapeutic strategy is also being tested in patients with lethal lung fibrotic diseases, including idiopathic pulmonary fibrosis. We are going to test the treatment’s efficacy in larger groups of people with more lung diseases. We hope to develop the treatment for clinical use within about two to three years.”
Professor Omar Usmani, heading the European Respiratory Society group focused on airways disease, asthma, COPD, and chronic cough at Imperial College London (UK), provided insight into the significance of these findings, stating, “The results from this phase I clinical trial are encouraging. COPD is in desperate need of new and more effective treatments, so if these results can be confirmed in subsequent clinical trials, it will be very exciting. It is also very encouraging that two patients with emphysema responded so well. A limitation of this study is that the uptake of the progenitor cells when they were transplanted back into the patients is uncontrolled. So we do not know whether the lungs of some patients responded better to the transplantation than others. We hope this information may become apparent in future studies.”
Reference: “Autologous transplantation of P63+ lung progenitor cells for chronic obstructive pulmonary disease therapy” by Wei Zuo et al, 12 September 2023, European Respiratory Society International Congress 2023.
This research was generously supported by Regend Therapeutics Ltd.
Table of Contents
Frequently Asked Questions (FAQs) about COPD Treatment
What is COPD, and why is it significant?
COPD, or Chronic Obstructive Pulmonary Disease, is a severe respiratory condition characterized by progressive lung tissue damage. It’s significant because it affects millions worldwide and currently has limited treatment options.
How does the experimental treatment using P63+ lung progenitor cells work?
In this treatment, P63+ progenitor cells are extracted from the patient’s own lungs, multiplied in the lab, and then reintroduced into the patient’s lungs. These cells have the potential to repair damaged lung tissue.
What were the key findings of the phase I clinical trial?
The trial demonstrated that the treatment was well-tolerated by patients. Lung function (DLCO) improved from 30% to 40.3%, and walking distance (6MWD) increased from 410 to 447 meters after 24 weeks. Quality of life, as measured by the SGRQ, also improved.
Are there any limitations to this treatment?
The study noted that it couldn’t repair severe emphysema. Additionally, the uptake of progenitor cells during transplantation varied among patients, which may require further investigation in future studies.
What’s the next step in advancing this treatment?
Researchers plan to conduct a phase II trial with a larger patient group to further evaluate its effectiveness. Approval from regulatory authorities is also needed to make the treatment available to a wider COPD patient population.
