Our second daily report from the Huntington’s disease World Congress brings together all the live updates from our twitter feed. Video of the day’s Oz Buzz session – with news, interviews and features – will be available to watch at HDBuzz.net later this week.
Tuesday, September 13, 2011
8:44 – G'day from Melbourne: day 2 of the Huntington’s Disease World Congress. Video of yesterday’s live Oz Buzz session is now on youtube.com/user/hdbuzzfeed
8:57 – Once we have drugs, how do we know how much to give people? Karl Kieburtz suggests some techniques.
9:05 – We’ll be interviewing Robert Pacifici, Chief Scientific Officer of CHDI live on stage later. Send your questions about drugs & trials!
9:12 – ‘There is nothing more precious to a drug hunter than an observation made in the population you want to treat’ Robert Pacifici, CHDI
9:15 – So sign up for observational trials! PREDICT-HD and TRACK-HD are two options
9:31 – Another observational trial to consider – ENROLL-HD
9:33 – ‘There’s no such thing as a good or bad animal model of HD’ – they all tell us useful things about different aspects – Pacifici
9:34 – We must understand how our experimental drugs work, and what problems they’re targeting, if we’re to test them successfully – Pacifici
9:46 – Joaquim Ferreira – It’s time to face challenge of how to do drug trials in ppl with the Huntington’s disease mutation but no symptoms
9:48 – Ferreira – designing trials carefully can help us tell the difference between effects on symptoms, and altering the progression of HD
9:52 – Studies like PREDICT, TRACK and ENROLL are crucial for getting to trials in pre-onset HD, & getting enough participants – Ferreira
9:59 – Drug regulatory agencies are willing to consider new rules for testing drugs in HD before symptom onset – if the community is on board
10:04 – “There are an impressive number of things moving towards trials that were specifically designed with Huntington’s in mind” – Pacifici
10:47 – Jeff: Steve Finkbeiner has built robot microscopes to understand how mutant huntingtin kills cells. Really cool.
10:51 – Jeff is now reporting from the session on ‘Basic science: protein homeostasis’. Ed reporting on ‘Biomarkers’
10:53 – Ed: Functional MRI scanning reveals important brain changes in pre-onset HD. Could be important for PreHD trials- Dr Nellie Georgiou
11:02 – Jeff: Question your assumptions. Steve Finkbeiner says that things we once thought bad for neurons might actually be protecting them
11:07 – Ed: Brain scans have helped develop drugs in diseases like Parkinson’s and Alzheimer’s. We can learn from that – Dr Rachael Scahill
11:21 – Ed: TRACK-HD results show that the earliest brain changes in HD mutation carriers may be in the ‘white matter’ connections – Scahill
11:23 – Jeff: Danny Hatter has built labels that let scientists follow the huntingtin protein around in live cells
11:25 – Ed: More TRACK-HD results: shrinkage in specific brain areas is linked to movement control. Again, may help us test drugs – Scahill
11:28 – Ed: “It takes a lot of sleuthing to get measurements precise enough to reveal the effects of drugs” – Prof Julie Stout
11:38 – Ed: We’re “not too far off” being able to detect drug benefits on thinking skills in HD mutation carriers – Stout
11:40 – Jeff: Bev Davidson is working to develop “RNAi” therapies, which turn off the mutant huntingtin protein
11:42 – Jeff: Bev: Even partial reduction of mutant huntingtin has beneficial effects in HD mice – we don’t need to completely ‘silence’ it.
11:50 – Ed: TRACK-HD and ‘CAB’ project are giving us a toolkit of reliable & meaningful tests for studying cognitive problems in HD – Stout
11:58 – Jeff: Bev has been testing ‘RNAi’ silencing in monkeys, a critical step to setting up human trials. Results show beneficial effects
12:04 – Ed: Huntington’s involves many cell types, not just neurons – including immune system. A whole-body disease – Prof Paul Muchowski #WHCD
12:07 – Jeff: Ralf Reilmann and TRACK-HD have developed machines to measure subtle motor problems in HD, like tongue strength
12:11 – Ed: KMO inhibitor drug, acting on blood immune cells, extends lifespan of HD mice – Muchowski
12:12 – Jeff: Reilmann – subtle changes in movements occur early in people carrying the HD mutation, before the onset of full-blown HD
12:14 – Ed: Muchowski also working on drugs to target ‘cannabinoid’ receptors (there’s no direct evidence for marijuana benefits in HD though)
12:18 – Ed: HD mice genetically engineered to lack ‘CB2’ cannabinoid receptors perform worse on tests of movement function – Muchowski
12:21 – Jeff: Reilmann – novel machines to measure HD movement symptoms are already being used in a human HD drug trial in Europe
12:22 – Ed: Breaking news. a drug that activates CB2 receptors improves motor function and prolongs the lifespan of HD mice – Muchowski
12:23 – Ed: CB2 activator drug even improves symptoms in ‘late stage’ mice – Muchowski
12:24 – Ed: Surprisingly the CB2 receptor isn’t found in the brain – meaning the CB2 drug may be acting in the blood, like the KMO inhibitor
12:28 – Ed: Targeting the immune system directly with an antibody to the immune signaling molecule IL6 produces similar benefits – Muchowski
12:47 – Ed: Working in HD fruit flies, Juan Botas found calcium changes. Now using data networks to work out what it means for patients
13:52 – Ed now reporting from the ‘clinical care research’ session. Jeff reporting from ‘Basic science: systems & peripheral pathology’
13:55 – Jeff: Maria Bjorkqvist – HD is a whole body disease, not just a brain disease. Patients have problems in bone, fat, muscle & others
13:56 – Ed: Regular patient/carer education sessions improve anxiety, mood, coping strategies and quality of life in HD – Prof Raymund Roos
14:00 – Ed: Roos – We must not hide from difficult issues like end-of-life care and suicide. Silence is the enemy. Keep talking.
14:03 – Jeff: Bjorkqvist – Heart attacks kill a large percentage of HD patients – this could this be part of the disease, not coincidence
14:14 – Jeff: Richard Faull – People with HD have diverse symptoms, which causes different patterns of brain cell loss
14:16 – Jeff: Faull – Donated human brains from HD patients are a precious gift to scientists studying the disease
14:27 – Ed: Dr David Craufurd: We have good treatments for psychiatric problems in HD e.g. depression, anxiety & aggression. Speak to your doc!
14:47 – Jeff: George Rebec records the firing of brain cells from awake mice, and can see clear changes in firing patterns in HD mice
14:53 – Ed: Problems recognizing other people’s emotions are more widespread than previously thought in HD patients – Izelle Labuschagne
15:17 – Ed: Nobody can handle HD alone – true of at-risk people and care professionals – it takes a great team from early on – Dr Martha Nance
15:20 – Jeff: William Yang-Building mice with mutant huntingtin in limited brain areas to understand what parts of brain are important in HD
