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Novel Clinical Trial – First in United States – to Begin at UPMC and UPMC Hillman Cancer Center to Treat Glioblastoma with Innovative Photodynamic Therapy

February 6, 2024

A first of its kind clinical trial in the United States to treat glioblastoma will be launching in early 2024 at UPMC and the UPMC Hillman Cancer Center, which combines intraoperative photodynamic therapy (PDT) with the drug Pentalafen® and a new laser device Heliance®, both, developed by the French company, Hemerion Therapeutics®, sponsor of the clinical trial.

Leading the clinical trial at UPMC is principal investigator Jan Drappatz, MD, associate professor of Medicine in the Division of Hematology/Oncology. Dr. Drappatz also serves as director of the Adult Neuro-Oncology Program and holds a secondary appointment in the Department of Neurology. The neurosurgery co-investigator of the trial is Costas G. Hadjipanayis, MD, PhD, executive vice-chairman and L. Dade Lunsford Professor of Neurological Surgery at the University of Pittsburgh where he also is the director of the Center for Image-Guided Neurosurgery.

"UPMC is the first center in the U.S. to bring this promising treatment approach to clinical trial for GBM patients,” says Dr. Drappatz. “It combines a newly formulated compound that can be used to both fluorescently highlight the tumor and its boundaries for surgical removal, and then with the addition of the laser device and a specific wavelength of light, activate the same compound taken up by the tumor cells to kill them.”

A recent intraoperative PDT study was completed in France by the University of Lille showing safety, feasibility, and efficacy of the treatment in GBM patients.

"This study marks an acceleration in the clinical development of our therapeutic solution. We believe PDT can become part of the standard of care and help improve the quality of life and patient outcomes with this aggressive cancer," Maximilien Vermandel, CEO of Hemerion Therapeutics, the sponsor of the study, adds.

The Problem: Glioblastoma

Glioblastoma (GBM), a rare grade IV brain malignancy, is notorious for its aggressive nature and dismal prognosis. Most patients with GBM typically succumb to the disease within two to three years after diagnosis. GBM continues to be one of the most lethal forms of cancer, with limited treatment options that lack curative efficacy. The complex cellular composition of glioblastoma renders it resistant to conventional therapies. In order to make gains in length of survival after diagnosis, new therapeutic approaches are needed.

What is Intraoperative Photodynamic Therapy?

Intraoperative photodynamic therapy (PDT) has emerged as a promising technique in cancer treatment, especially in brain surgery. This approach involves the administration of a photosensitizing agent prior to surgical resection of the tumor, followed by the application of specific wavelengths of laser light after the resection is completed. The laser-generated light activates the photosensitizing agent and produces a form of oxygen that selectively targets any remaining cancer cells. This approach is able to target tumor cells while sparing healthy surrounding tissues.

“In our trial, we’re using for the first time the oral drug Pentalafen® for two purposes,” says Dr. Hadjipanayis. “First, we use it to identify the boundaries of the tumor for resection, and then when excited by a specific wavelength of laser light, we can use its properties to try and kill residual GBM cells not removed by surgery. Pentalafen® is unique in that it is designed specifically for GBM.”

New Clinical Trial: Design and Goals

The new clinical trial, titled "An Interventional, Non-Randomized, Phase 1/2, Light-Dose-Escalation Study," represents a significant step in developing and studying a new approach to glioblastoma treatment. This early phase study is designed to explore the safety and feasibility of intraoperative PDT using the newly created Pentalafen® and the Heliance® Solution device, following the maximal extent of resection (EOR) of the patient’s tumor.

It employs a "3+3" design to determine the maximum tolerated dose (MTD), which is essential for designing a future pivotal efficacy study. The trial will enroll a maximum of 12 patients across two light dose levels, with a close monitoring of dose-limiting toxicity (DLT) as defined by the NCI Common Terminology Criteria for Adverse Events.

Who is Eligible for Participation?

The study will be open to enrollment for male or female patients aged 18 to 69 years that have been newly diagnosed with grade IV glioblastoma. (

Study Procedure and Safety Measures

Participants enrolled in the study will be given oral Pentalafen® at a dose of 20 mg/kg 4 to 6 hours before their surgery for tumor removal.

During the resection phase of treatment, the neurosurgical team will identify the tumor cells using fluorescence of the Pentalafen taken up by the tumor’s cells.

After the resection of the patient’s tumor, the PDT procedure will be conducted using the Heliance laser device. First an intracavitary balloon is placed into the patient’s brain where the tumor was resected, and it is inflated to fill the cavity. Once placed and inflated, the laser is employed using a pulsed approach for 30 minutes at varying doses.

For the study, two patient cohorts will receive different laser light doses - 200 J/cm2 and 400 J/cm2, respectively. Patient enrollment in each cohort follows a stringent protocol to monitor adverse events and ensure safety, and this clinical trial involves an independent Data and Safety Monitoring Board (iDSMB) to review safety data and authorize the dose escalations.

After the patient’s surgery and PDT treatment, they will go on to receive standard of care therapies and follow-up as deemed appropriate by their treatment team.

“By leveraging the dual benefits of intraoperative visualization and photodynamic therapy, we are at the beginning of a new approach to GBM therapy,” says Dr. Drappatz. “The data from our study will be instrumental in shaping future therapeutic strategies for this challenging and devastating disease.”

Consultations and Patient Referrals

To consult about a possible patient referral for this study, please contact the UPMC Hillman Cancer Center Neuro-Oncology Center at 412-692-4724 or use our web contact form. You may also contact the UPMC Center for Image-Guided Neurosurgery by calling 412-647-0953.

Further Reading

Read the study protocol on (