FDA Approves IDE Application for Alpha DaRT in Recurrent Glioblastoma

• The FDA has approved an investigational device exemption (IDE) application for the start of a pilot study evaluating the Alpha diffusing alpha-emitters radiation therapy (DaRT) technology in patients with recurrent glioblastoma (GBM).
• Alpha DaRT works to enable highly potent and conformal alpha-irradiation of solid tumors by delivering radium-224 impregnated sources intratumorally.
• Preclinical trials have displayed Alpha DaRT’s potential.

An IDE application to begin a pilot study of the Alpha DaRT technology in recurrent GBM has been approved by the FDA, according to a press release from Alpha Tau Medical Ltd.¹

Up to 10 patients with recurrent GBM not amenable for surgical resection are expected to be enrolled in the trial in the US. Patients must have undergone a prior course of central nervous system radiation.

The primary end point of the study will be to evaluate the safety and feasibility of the Alpha DaRT treatment.

“This is very exciting news for patients suffering from glioblastoma, a highly aggressive form of brain cancer with an extremely poor prognosis and survival rate. In this trial, we will introduce a completely novel approach, which is specifically designed to deliver Alpha DaRT sources into brain tumors that are not amenable to surgery,” said Robert B. Den, MD, Alpha Tau chief medical officer, in a press release. “According to the National Brain Tumor Society, glioblastoma is one of the most complex, deadly, and treatment-resistant cancers, with an estimated average survival rate of only 8 months.”

Alpha DaRT is a novel approach to localized cancer therapy and the first localized anticancer therapy to use the specific therapeutic properties of alpha particles for the treatment of solid tumors.¹ This technique involves the intratumoral implantation of radium-224 impregnated sources.

The subsequent radioactive decay of radium-224 releases short-lived alpha-emitting daughter nuclides, which diffuse within the tumor microenvironment and deliver potent, localized radiation.² With a limited diffusion range, these alpha emitters aim to maximize tumor cell kill while minimizing exposure to surrounding healthy tissues.

One of the clinical advantages seen in preliminary clinical trials assessing Alpha DaRT, specifically in cutaneous and head and neck cancers, are high response rates. A first-in-human trial showed a 100% overall response rate, with a complete response rate exceeding 78%. Alpha DaRT has also shown efficacy in tumors previously resistant to conventional radiation therapy.²

Alpha DaRT also has a favorable toxicity profile, leading to mild adverse events and patient tolerability.

For superficial tumors, Alpha DaRT can be administered in a single, short session under local anesthesia, simplifying the treatment process and improving patient convenience. Further, both preclinical and clinical observations suggest that Alpha DaRT may induce an antitumor immune response.

“This IDE approval follows the FDA’s continued recognition of the huge promise that this technology holds for patients with recurrent glioblastoma; after having already received the FDA’s Breakthrough Device Designation, followed by acceptance into the FDA’s prestigious Total Product Life Cycle Advisory Program to accelerate the Alpha DaRT treatment to market and to the GBM patients who may stand to benefit greatly,” added Uzi Sofer, Alpha Tau chief executive officer, in the press release.¹

References:

1. Alpha Tau receives FDA approval to initiate a trial for patients with recurrent glioblastoma. News release. Alpha Tau Medical Ltd. April 2, 2025. Accessed April 2, 2025. https://tinyurl.com/jzvw4n9w

2. Alpha DaRT treatment. Alpha Tau Medical Ltd. Accessed April 2, 2025. https://www.alphatau.com/alpha-dart-cancer-treatment