Recently, I spent an hour on a zoom call hosted by Zurich Children’s Hospital. What they have accomplished is world class.  

The Zurich Children’s Hospital has great clarity and focus plus the committed and competent leadership to implement the mission which is to find a cure for pediatric brain tumours. 

They have created a global network of the top researchers in pediatric brain tumours.

The network is a working community of highly collaborative researchers. They share. They inform. They work separately and together. This community is working with the mission of finding a cure for pediatric brain tumours. They are not concerned about fame or fortune. 

The researchers were present on the zoom call lead by the key researchers at Zurich Children’s Hospital. Drs Javad Nazarian from the DIPG/MG Center Zurich and Sabine Mueller from UCSF/ the Pacific Pediatric Neuro-Oncology Consortium were the clear leaders of the entire community. PNOC brings 225 clinicians and researchers together to push for breakthrough.  Without effective leadership this could not be accomplished. 

The other key participants were the executive directors of family foundations with an interest in pediatric brain tumours. Most had had a personal experience in having lost a child to this lethal disease. 

Zurich Children’s Hospital had created a distributed centre of excellence of the top research centres in pediatric brain tumours and the funding sources able to fund the community and the research machine which has over 30 different approaches to curing pediatric brain tumours. 

Some of these approaches include using novel agents that disrupt mitochondria in the cancer cells and sono-sensitization which uses a drug to sensitize the cancer cell to high intensity focused ultrasound. 

The other dimension of Zurich Children’s Hospital is their clinical outreach. Pediatric brain tumours are rare. To gain the critical mass of patients, the ZCH has a global clinical outreach service which provides clinical assessments and virtual expert opinions for children with brain tumours irrespective of where they live.  This allow the ZCH to have the cumulative experience with patients necessary to deeply understand the disease and to have enough patients for the many clinical trials that are organized at and by ZCH. 

So the combination of the top researchers in pediatric brain tumours, the biggest database of patients with pediatric brain tumours and a vast inventory of clinical trials with novel agents and technologies plus the committed funding sources presents the highest probability of finding a cure for pediatric brain tumours. Well done Zurich Children’s Hospital.

The post Zurich Children’s Hospital: A World Class Wonder In Addressing Pediatric Brain Tumours: Raymond Rupert appeared first on Raymond Rupert.

I’m a clinical trials geek. I keep hearing people talk about the seven to 10 years it takes to make a vaccine and how dangerous speeding this up might be. The word that keeps popping up is “rushed”, and it is making the average person nervous about vaccine safety. So, as a clinical trials doctor, I am going to tell you what I do for most of those 10 years – and it is not very much.

I’m not lazy. I submit grants, have them rejected, resubmit them, wait for review, resubmit them somewhere else, sometimes in a loop of doom. When I am lucky enough to get trials funded, I then spend months on submitting to ethics boards. I wait for regulators, deal with personnel changes at the drugs company and a “change of focus” away from my trials, and eventually, if I am very lucky, I spend time setting up trials: finding sites, training sites, panicking because recruitment is poor, finding more sites. I then usually have more regulatory issues and, finally, if my big pot of luck is not used up, I might have a viable therapy – or not.

At this point, it might get delayed because of questions over profitability or any number of other obstacles. I’m not even going to go into the years it normally takes to get the “preclinical” studies, the ones before the human trials, done.

So next time somebody expresses concern at the astonishing speed the vaccine trials have happened at, point out to them that 10 years isn’t a good thing, it’s a bad thing. It’s not 10 years because that is safe, it’s 10 hard years of battling indifference, commercial imperatives, luck and red tape. It represents barriers in the process that we have now proved are “easy” to overcome. You just need unlimited cash, some clever and highly motivated people, all the world’s trial infrastructure, an almost unlimited pool of altruistic, wonderful trial volunteers and some sensible regulators.

With all of this and the clock ticking on a global pandemic killing people by the second, it turns out we can do amazing things. The vaccine trials have been nothing short of a miracle. A revolution in how we do trials that when you think about it is perhaps not that surprising given our ability to innovate when we really need to.

Three vaccines have already broken cover and demonstrated efficacy higher than we had hoped

And we really need to – necessity being the mother of invention. Safety has not been compromised. All trials have been through the correct “phases” or process of any normal drug or vaccine. Hundreds of thousands of the very best of us volunteered and had an experimental vaccine. The world watched so closely that when a single person fell ill, we were all debating it.

To date, there has not been a single associated death related to Covid vaccines and only a handful of potentially serious events. Just imagine watching everybody in a small city for six months and reporting every single heart attack, stroke, neurological condition or anything that might be judged serious. How astonishing is this? It has been a triumph of medical science.

I haven’t even touched on the lucky confluence of timing that meant this all happened at a time when sequencing all the genes in a person or virus is so routine nobody bats an eyelid. This turbocharged the early preclinical science needed as the foundation stone of several new technologies at the right point to be exploited.

At this time, three vaccines have already broken cover and demonstrated efficacy higher than we had ever hoped. The bar was set by regulators at around 50 per cent. Both Moderna and Pfizer reported 95 per cent efficacy, and Oxford University reported 90 per cent efficacy for a particular dosage regimen. Safety data is still to follow, but the track record of vaccines is excellent, and I am an optimist.

None of this is to downplay the challenges still ahead. It is also not to say vaccines are without safety questions still to be answered. It has been, however, a triumph of good process and great people. I am confident that when regulators pore over the safety and efficacy data, closely followed by every interested scientist in the world, that vaccines will only be used if their benefits clearly outweigh the risks – and you should be confident too.

Mark Toshner is a director of translational biomedical research at the University of Cambridge. This article first appeared on The Conversation

The post Need For Vaccines At Speed: Mark Toshner clinical trials doctor & researcher appeared first on Raymond Rupert.