Scientific paper supports concept of CarThera's intracranial ultrasound implant to disrupt blood-brain barrier

CarThera, a French company based at the Brain and Spine Institute (ICM), that designs and develops innovative ultrasound-based medical devices to treat brain disorders, today announces the publication in Science Translational Medicine of a scientific paper on initial successes in disrupting the blood-brain barrier (BBB) with the use of ultrasound. This has been achieved in association with teams from the Assistance Publique-Hôpitaux de Paris (the Greater Paris University Hospital) and the Pierre and Marie Curie University.

Making the blood vessels in the brain temporarily permeable with the use of ultrasound allows for increased delivery of therapeutic molecules – including chemotherapy drugs, which generally have little brain penetration. A world first achieved at the Pitié-Salpêtrière Hospital in Paris, this paves the way for major therapeutic possibilities not only for brain cancers, but also for neuro-degenerative diseases such as Alzheimer’s, in which effective molecules have so far been unable to penetrate the brain.

The article, entitled ‘Clinical trial of blood-brain barrier disruption by pulsed ultrasound’, reports the preliminary results in the first 15 patients included since July 2014 in a phase 1/2a clinical trial in relapsing glioblastoma (aggressive malignant brain tumors). The aim of the trial was to increase the permeability of the blood vessels of the brain by transiently opening the blood-brain barrier. Increasing the permeability of the BBB with SonoCloud improves the delivery of intravenously infused chemotherapy drugs into the brain. The device’s tolerability and safety profile is good, since it uses low intensity ultrasound similar to a diagnostic or imaging level.

“The blood-brain barrier is one of the last major frontiers of neuroscience. The publication of the trial results in one of the most prestigious US scientific journals is a major acknowledgment of this medical first,” said Professor Alexandre Carpentier, a neurosurgeon at Pitié-Salpêtrière Hospital, the inventor of the SonoCloud device and founder of CarThera.

“Science Translational Medicine published our research in record time,” said Dr. Ahmed Id Baih, principal investigator for the clinical trial and a neuro-oncologist at Pitié-Salpêtrière Hospital. “We are pleased to see the international scientific community recognizing advances in French research.”

“The publication of this scientific paper supports the SonoCloud concept. It will allow us to start working on subsequent clinical developments with a view to marketing the device. We plan to raise money in 2017 to fund a large-scale phase 2b/3 clinical trial in 200 patients; with centers in Europe and the US,” said Frédéric Sottilini, CEO of CarThera. “SonoCloud could be commercially available in 2020 for use in recurrent glioblastoma; with CE marking and FDA approval obtained in the meantime. At the same time, the company will carry out exploratory studies in other indications, including Alzheimer's disease.”

According to the company’s estimates, 250,000 patients worldwide are diagnosed with a brain tumor each year. More than 160,000 of them could benefit from the SonoCloud breakthrough (mainly those with primary brain cancers and some brain metastases of other cancers). This represents a market worth €1.5 billion ($1.7 billion).