Norman Swan: Alzheimer's disease is the commonest form of dementia, and the results of billions of dollars of research investment have been bitterly disappointing in terms of finding an effective treatment. However, experiments on mice by a group from the Dementia Research Centre at Macquarie University have come up with some promising findings. Professor Lars Ittner is the director of the centre. Welcome to the Health Report Lars.
Lars Ittner: Hello, how are you?
Norman Swan: Now, essentially you are targeting an enzyme which protects nerve cells in the brain from the toxic effects of amyloid beta and that's one of the two main substances which accumulate in Alzheimer's disease. What did you actually do in this study?
Lars Ittner: So in this we found prior to this study that this enzyme activity that protects the brain from Alzheimer's disease is actually lost in Alzheimer's disease. And we devised a gene therapy to replace the enzyme activity and bring the enzyme back into the brain cells.
Norman Swan: And these are mice which replicate Alzheimer's disease in some shape or form.
Lars Ittner: Yes, they are, so we genetically engineered them to develop Alzheimer's disease.
Norman Swan: And they were showing signs of memory and thinking problems?
Lars Ittner: Yes, so their ability to form memory and then store the memory over longer terms is compromised.
Norman Swan: And this was the gene for this enzyme?
Lars Ittner: So we brought backit's called the P38 gamma gene, which we brought back into the brains of these mice and that restored their ability to form memory.
Norman Swan: So you actually got healing?
Lars Ittner: So we were quite surprised because when you set out with these type of studies you expect at most that you stop the progression. But yes, we got far more than we set out for.
Norman Swan: People have tried gene therapy before from Parkinson's disease and other things, and it's quite hard to get the gene therapy into the brain. And of course Alzheimer's disease is quite widespread as opposed to Parkinson's disease. How do you get the gene therapy in reliably?
Lars Ittner: So from the early days of gene therapy done in Parkinson's disease, the vehicles that are used to bring the genes into organisms or in the brain in particular have improved, so these days we use modified viruses that we take advantage of their ability to infect brain cells, and they then deliver the genes for us.
Norman Swan: In the right place. Were there any side effects?
Lars Ittner: So we did toxicity studies as part of our study, and then you use incredibly high amounts of the virus, and we did not see long-term side-effects.
Norman Swan: How do you getthere's something called the bloodbrain barrier, the brain is a protected organ and it's quite hard for things to get into the brain because of this barrier, how did you get beyond that with these gene therapies?
Lars Ittner: So with the mice we can take advantage of a modified virus which has been selected to actually passage this naturally, but in humans you would do a single injection, it's like a lumbar puncture, it's at the base of your neck, and it's directly into the liquid around the brain, so you basically mechanically bypass the bloodbrain barrier.
Norman Swan: There have been very disappointing results. I mean, what happens in mice particularly in Alzheimer's disease does not necessarily happen in humans, and there's not a single amyloid beta therapy that has had much effect on the brains of the people with Alzheimer's disease. Why do you think this one might work in humans when others haven't?
Lars Ittner: So the problem with the amyloid beta is that it is now understood that this is a disease inducing pathology but is not required for the progression of the disease, and we are targeting here actually the tau protein specifically which is
Norman Swan: It's the other thing that
Lars Ittner: Exactly, and that is responsible for the progression of the disease, so it's actually moving away from the amyloid beta as a drug target which has failed in the past.
Norman Swan: Now, with COVID-19 around we are getting used to the language of clinical trials and accelerating trials. When are you ready to go to phase 1 which would be a safety trial in humans?
Lars Ittner: So preclinical experiments have actually been completed for this particular study, and the next step are in fact phase 1 clinical trials, and we are currently working with Macquarie University and their commercialisation arm to find the right partner to move forward into clinical trials.
Norman Swan: Fascinating. Well, we'll follow that up when you do. Thanks for joining us.
Lars Ittner: It was my pleasure.
Norman Swan: Professor Lars Ittner is director of the Dementia Research Centre at Macquarie University.
You've been listening to the Health Report, I'm Norman Swan, and I'd really enjoy your company next week.
View original post here:
- New CSL gene therapy could trigger a stock rerating - The Australian Financial Review - October 19th, 2020
- Cancer Gene Therapy Market Size & Share Insights on Growing Applications by 2026 - re:Jerusalem - October 19th, 2020
- Gene Therapy: Healing remedy or harmful hoax? The Knight News - The Knight News - October 19th, 2020
- Polyplus-transfection presents latest solution portfolio for gene therapy market to Alliance for Regenerative Medicine's Meeting on the Mesa -... - October 19th, 2020
- Taysha Gene Therapies Receives Rare Pediatric Disease Designation and Orphan Drug Designation for TSHA-102 as a Treatment for Rett Syndrome - BioSpace - October 19th, 2020
- Merck's New VirusExpress Platform Speeds Development of Cell and Gene Therapies - PharmiWeb.com - October 19th, 2020
- Axovant Gene Therapies Receives Rare Pediatric Disease Designation for AXO-AAV-GM2 for Tay-Sachs and Sandhoff Disease - GlobeNewswire - October 19th, 2020
- Ori Biotech's new cell and gene therapy platform raises 23m - BusinessCloud - October 19th, 2020
- Foundation to Fight H-ABC, University of Massachusetts Medical School and Yale University Initiate Gene Therapy Study Targeting Cure for Rare Disease... - October 19th, 2020
- 5-Year-Old Girl, Being Treated In French Gene Therapy Trial, Dies In US - NDTV - October 19th, 2020
- Orchard Therapeutics Receives Positive CHMP Opinion for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) - GlobeNewswire - October 19th, 2020
- Castle Creek Biosciences Announces First Patient Dosed in Phase 1/2 Clinical Trial of FCX-013 Gene Therapy for Treatment of Moderate to Severe... - October 19th, 2020
- Longview family rallies around 3-year-old with rare, fatal genetic condition - Longview News-Journal - October 19th, 2020
- Child dies in US during gene therapy trial - The News International - October 19th, 2020
- Tag: Gene Therapy - The Think Curiouser - October 19th, 2020
- Gene Therapy for Inherited Genetic Disorders Market Robust pace of Industry during 2018-2028 - The Think Curiouser - October 19th, 2020
- Disruptive Technologies and Mature Regulatory Environment Vital for Cell Therapy Maturation - BioSpace - October 19th, 2020
- Ori looks to advance closed cell therapy tech - BioProcess Insider - October 19th, 2020
- Histogen Appoints Moya Daniels as Executive Vice President and Head of Regulatory, Quality and Clinical Operations - BioSpace - October 19th, 2020
- EMA starts rapid review of Bluebird's gene therapy for rare disease CALD - - pharmaphorum - October 10th, 2020
- American Gene Technologies to Present at 2020 Virtual Cell & Gene Meeting on the Mesa - GlobeNewswire - October 10th, 2020
- Unchained Labs launches the ultimate gene therapy tool; tackle AAV titer, empty/full ratio and more with Stunner! - PRNewswire - October 10th, 2020
- Smartphone-connected pacemaker devices, experimental gene therapy among top 10 innovations at annual event - BioWorld Online - October 10th, 2020
- CellGenix Completes Facility Expansion to Stay Ahead of the Increasing Demand for GMP Raw Materials - b3c newswire - October 10th, 2020
- Cleveland Clinic Names Top 10 Medical Innovations; Sickle Cell Therapy Tops the List - HealthLeaders Media - October 10th, 2020
- Investigational Gene Therapy for Phenylketonuria Gets Fast Tracked - Monthly Prescribing Reference - October 10th, 2020
- Flexion Therapeutics to Present at the 2020 Cell & Gene Virtual Meeting on the Mesa - GlobeNewswire - October 10th, 2020
- Taysha Gene Therapies Partners with Invitae to Enable Rapid Access to Genetic Testing and Earlier Diagnosis of Patients with CNS Disease for Rare and... - October 10th, 2020
- Cancer Gene Therapy Market 2020 by industry trends, statistics, key companies growth and regional forecast - News by Decresearch - October 10th, 2020
- Bone Therapeutics, Link Health and Pregene to develop and commercialize the ALLOB allogeneic bone cell therapy platform in China and Southeast Asia -... - October 10th, 2020
- Axovant to Participate in Upcoming October Conferences and Provide AXO-Lenti-PD Program Update the Morning of October 6th - GlobeNewswire - October 10th, 2020
- Investigational Gene Therapy Fast Tracked for Duchenne Muscular Dystrophy - Monthly Prescribing Reference - October 10th, 2020
- Genprex to Present at the Alliance for Regenerative Medicine's Virtual Cell and Gene Meeting on the Mesa - Business Wire - October 10th, 2020
- Generation Bio to Present at 2020 Virtual Cell & Gene Meeting on the Mesa - GlobeNewswire - October 10th, 2020
- Gyrolab AAVX Titer Kit Introduced to Support Cell and Gene Therapy Market - Technology Networks - September 2nd, 2020
- Taysha Gene Therapies Builds Experienced Executive Leadership Team to Advance Pipeline of Gene Therapies for Monogenic CNS Disease in Both Rare and... - September 2nd, 2020
- First Gene Therapy Products Approved in Brazil - Lexology - September 2nd, 2020
- Gene Therapy Market is projected to be worth USD 10 Billion by 2030, growing at an annualized rate of over 40% - The News Brok - September 2nd, 2020
- Prevail Therapeutics to Present at Upcoming Investor Conferences - Yahoo Finance - September 2nd, 2020
- Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for MPS-I - BioSpace - September 2nd, 2020
- The Safety and Clinical Activity of Gene-engineered T-cell Therapy Targeting HPV-16 E7 in Epithelial Cancers - Pharmacy Times - September 2nd, 2020
- AGTC to Host Management Update on September 9 at 8:00 am ET to Discuss Planned Phase 2/3 XLRP Trial, Share Additional XLRP Data and Report Fourth... - September 2nd, 2020
- Qualitative Analysis and Competitive Industry, CCOVID-19 Scenario of Personalized Gene Therapy Treatment Market - The News Brok - September 2nd, 2020
- Sorrento Announces the Closing of Its Acquisition of SmartPharm to Build Next Generation G-MAB-Encoded Plasmid DNA For Cost-Efficient and In Vivo... - September 2nd, 2020
- In the face of COVID-19, cell and gene therapy space shows 'remarkable resilience:' report - FierceBiotech - August 10th, 2020
- Gene Therapy Promising in BCG-Unresponsive Bladder Cancer - Medscape - August 10th, 2020
- Evolution and Expansion of Therapies in the Global Cell and Gene Therapy Tools and Reagents Market 2020-2024 - PRNewswire - August 10th, 2020
- How Gene Therapy Helped Conner Run : Short Wave - NPR - August 10th, 2020
- Preparing for an influx of cell and gene therapy approvals - - pharmaphorum - August 10th, 2020
- Catalent's Harmans Site Approved To Manufacture AveXis' Gene Therapy - Contract Pharma - August 10th, 2020
- Sangamo and Novartis partner on gene therapies for autism - BioPharma-Reporter.com - August 10th, 2020
- Global Gene Therapy in Oncology Market 2020 Growth Statistics, New Opportunities, Competitive Outloo - PharmiWeb.com - August 10th, 2020
- Research shows promising results for LHON gene therapy - Ophthalmology Times - August 10th, 2020
- The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.90% during the period 20192025 - Yahoo Finance - August 10th, 2020
- Detailed Information on Gene Therapy Market 2020 | Covid-19 Impact Analysis | Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio,... - August 10th, 2020
- Global Cell & Gene Therapy Market Outlook and Forecast 2020-2025 - GlobeNewswire - August 9th, 2020
- Coronavirus Impact Editon of Gene Therapy for Mucopolysaccharidosis Market Research Study 2020 Future Development, Top Manufacturers, Technological... - August 9th, 2020
- The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.90% during the period 20192025 - Press Release - Digital... - August 9th, 2020
- Orgenesis Second Quarter 2020 Revenue Increases 55% Reflecting Progress of POCare Platform - GlobeNewswire - August 9th, 2020
- Regulatory Focus, July issue: Cell and gene therapy - Regulatory Focus - August 8th, 2020
- G-CON PODs Selected for Expression Therapeutics' Gene Therapy Manufacturing Facility - PR Web - August 8th, 2020
- CRISPR co-discoverer on the gene editor's pandemic push - Axios - August 8th, 2020
- Voyager Therapeutics Provides Update on AbbVie Vectorized Antibody Collaborations - GlobeNewswire - August 8th, 2020
- Gene therapy - Wikipedia - August 8th, 2020
- Gene therapy - Mayo Clinic - August 8th, 2020
- gene therapy | Description, Uses, Examples, & Safety ... - August 8th, 2020
- What Is Gene Therapy? How Does It Work? | FDA - August 8th, 2020
- 112M Fund Launched to Commercialize UCLs Gene Therapy... - Labiotech.eu - August 8th, 2020
- UK gene therapy pioneer tilts at $125m NASDAQ IPO - Business Weekly - August 7th, 2020
- Tessa Therapeutics Announces Results from Two Independent Phase 1/2 Trials of Autologous CD30 CAR-T Cell Therapy in Patients with Relapsed or... - August 7th, 2020
- Cancer Gene Therapy Market Latest Treatment Methodology 2020 to 2025 - Owned - August 7th, 2020
- Kriya Therapeutics announces the establishment of its internal manufacturing facility for process development and scalable cGMP production of gene... - August 7th, 2020
- Regenxbio announces positive one-year data on wet AMD gene therapy - Seeking Alpha - August 7th, 2020
- Covid-19 Forces Gene Therapy Companies to Shift... - Labiotech.eu - August 5th, 2020
- Cell and gene therapies: For biotechs, collaboration is key to successful innovation - BioPharma Dive - August 5th, 2020
- The Time for DMD Gene Therapy is Now: A Chat with the MDA - BioSpace - August 5th, 2020
- Gene Therapy Beats the Blood-Brain Barrier To Cure Cat Disease - Technology Networks - August 5th, 2020
- Analysis of COVID-19 Gene Therapy Market - Market Research Posts - August 5th, 2020
- World-first gene therapy reverses Alzheimer's memory loss in mice - New Atlas - August 5th, 2020
- Gene Therapy Market is projected to grow at an annualized rate of 45%, till 2030 - Market Research Posts - August 5th, 2020