Scientific paper published today in Science Translational Medicine supports concept of intracranial ultrasound implant developed by CarThera
Research teams from AP-HP hospitals, UPMC University, INSERM laboratories and the ICM incubator performed this breakthrough on brain tumors, paving the way for broader applications in neurological disorders
Paris, France, June 15, 2016 – 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).
About SonoCloud
SonoCloud is an innovative medical device developed by CarThera. It is capable of emitting ultrasound on demand to achieve temporary permeability of the blood vessels in the brain, with the aim of increasing the delivery of therapeutic molecules. Created by Professor Alexandre Carpentier and developed in collaboration with the Laboratory of Therapeutic Applications of Ultrasound (Laboratoire Thérapie et Applications Ultrasonores, LabTAU) at INSERM, SonoCloud is an ultrasound implant inserted into the skull after a regular surgical procedure and activated prior to each round of chemotherapy. Two minutes of low intensity ultrasound emission is enough to open the blood brain barrier for a duration of 6 hours and to increase by five to seven times the concentration of therapeutic molecules delivered into the brain, with good tolerability.
About the blood-brain barrier
The walls of the blood vessels in the brain are impossible to cross for certain molecules, limiting neuronal exposure to toxic agents. While this is a desirable property, it is also a major impediment to treating brain diseases and disorders. 99% of potential therapeutic drugs are blocked by this vascular barrier, known as the blood-brain barrier, and are unable to penetrate the brain. Scientists have been researching ways to bypass this barrier for over 50 years.
About CarThera
CarThera designs and develops innovative therapeutic ultrasound-based medical devices for treating brain disorders. The company is a spin-off from AP-HP, Greater Paris University Hospitals, the largest hospital group in Europe, and Pierre and Marie Curie University (UPMC). CarThera leverages the inventions of Professor Alexandre Carpentier, a neurosurgeon at AP-HP who has achieved worldwide recognition for his innovative developments for treating brain disorders.
CarThera developed the SonoCloud®, an intracranial ultrasound implant that temporarily opens the blood-brain barrier (BBB). By increasing the permeability of the cerebral blood vessels using low intensity pulsed ultrasound, the concentration of therapeutic molecules that reach the brain can be increased by five to seven times, with no increase in dose or toxicity. A Phase 1/2a clinical trial is underway in glioblastoma, for which treatment options are very limited. The initial results are supported by a paper published in Science Translational Medicine, which confirms the safety and therapeutic potential of the SonoCloud device. The ultrasound-induced disruption of the blood-brain barrier is a world first; it offers a new treatment option for a wide range of indications, including Alzheimer’s disease.
Founded in 2010 by Professor Alexandre Carpentier, CarThera is based at the Brain and Spine Institute (Institut du Cerveau et de la Moelle épinière, ICM) in Paris, France, and has laboratories at the Bioparc Laënnec business incubator in Lyon, France. The company, led by Frédéric Sottilini (CEO), works closely with the Laboratory of Therapeutic Applications of Ultrasound (Laboratoire Thérapie et Applications Ultrasonores, LabTAU, INSERM) in Lyon. Since its inception, the company has received support from France’s Ministry of Research, the Ile-de-France region, the Bpifrance public investment bank, Medicen Paris Region and Lyonbiopôle.
www.carthera.eu
Special notes to reporters
To receive a copy of the paper, please contact Meagan Phelan, Office of Public Programs (202-326-6436) or send an email to scipak@aaas.org.
More information, including a copy of the paper, can be found online at the Science Translational Medicine press package. You will need your user ID and password to access this information.
The article ‘Clinical trial of blood-brain barrier disruption by pulsed ultrasound’ will appear in the June 15, 2016, issue of the journal Science, published by AAAS, the science society, the world’s largest general scientific organization.
See www.sciencemag.org and www.aaas.org
