NorthWest Biotherapeutics Inc (NWBO)

[jondoeuk]

But there are no other companies that have the treatments that can make stage IV cancer patients have pathologic complete response with no surgery, no chemo and no radiation.

I guess you need reminding that all the patients in the DCVax-L trial under surgery and chemoradiotherapy, before being randomised to either receive temozolomide (chemo) plus DCVax-L or temozolomide plus placebo https://translational-medicine.biomedcentral.com/articles/10.1186/s12967-018-1507-6

As for DCVax-Direct, not a single patient responded https://aacrjournals.org/clincancerres/article/24/16/3845/277824/Cytokines-Produced-by-Dendritic-Cells-Administered

Assume LP did file for CNPV pilot program, this could explain why MHRA hasn't granted the approval yet.

It doesn't.

Did someone mention before that MHRA had cleared backlog?

Not only is the backlog cleared, but the MHRA already laid out plans for (strict adherence to) target days. They are meeting every application within their stipulated timeframe now. They are 100% compliant, except for NWBO's DCVax-L.

Looking at July 2025 for example, 100% of licence applications are being assessed within the statutory 210-day timescale. The average time is around 167 days. Even for ''new active substance'' the application average is around 191 days, which is still within the 210-day target https://www.gov.uk/government/publications/mhra-performance-data/mhra-performance-data

A breakthrough treatment of this magnitude has to be approved by FDA first.

NWBO has not filed a BLA with the FDA.

Also, the trial of DCVax-L was negative. While the original primary endpoint of PFS was not statistically different between the groups (SOC plus placebo vs. SOC plus DCVax-L), it was numerically longer in the placebo group. The hazard ratio for DCVax-L was 1.1 as well. So the trend was towards harm.

The original secondary endpoint was OS, but it couldn't be analysed due to the crossover. However, a number of the placebo control group patients did not crossover. If DCVax-L works, there should have been a strong trend towards benefit. But based on the company hiding the data, I am almost certain there was no such trend. I think the opposite is true. There is no reason they could not report the data (like they did for PFS), other than they know people would not like it. Instead, survival was derived from an untrustworthy (cherry-picked), post-hoc analysis that was implemented and conducted years and years after the trial had already been completed!

On top of this, we need to remember the history of the DCVax-L trial (non-linear and quite complex over 17(+) years). The trial itself morphed into a phase three (it started as a phase two). Enrolment was halted due to lack of funds (around the 2007/2008 financial crisis). Enrolment was restarted before the number of patients was upsized at least once. There was also at least one modification to the original primary endpoint.

Not long after the first efficacy IA in 2015, there was an FDA halt that was not initially disclosed by NWBO. Because of the halt, enrolment could not be completed. The Chair of the DSMB resigned as well. The trial did not hit a 2/1 ratio of treatment/placebo either. The endpoints were changed. They discarded the placebo control group. The external controls were not specified in advance. There is no patient-level data either. Any one of those would be cause for major concern. But to find them all in one trial is unheard of!

Finally, dozens upon dozens of independent experts have raised serious concerns about the DCVax-L trial and data, including Prof. Roger Stupp, an internationally renowned medical oncologist and neuro-oncologist and a leading authority on primary and metastatic brain cancers.

Prof. Roger Stupp is one of the most influential figures in neuro-oncology. Prof. Stupp is widely recognised as the architect of modern glioblastoma treatment standards and has led multiple clinical trials, from first-in-human phase one to pivotal phase registration studies, helping redefine the standard of care on two different occasions, and was instrumental in identifying MGMT promoter methylation as a predictive biomarker for benefit from chemotherapy. His commentary therefore carries considerable weight, not only because of his scientific stature, but also because what he has done embodies the benchmark by which glioblastoma trial design and outcomes are measured. Against this backdrop, his critical evaluation represents more than an expert opinion - it is a signal to the wider oncology community about how the therapy stands relative to established standards of evidence and care.

New FDA approval pathway for n-of-1 therapies coming soon, Prasad says
https://endpoints.news/new-fda-approval-pathway-for-n-of-1-therapies-coming-soon-prasad-says/

For gene therapies.