NorthWest Biotherapeutics Inc (NWBO)

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ILT,

Your post reminds me one of questions that Dr. Monica Bertagnolli answered during her nomination as NIH director.

Question 37. What opportunities do you see for more targeted research on rare and orphan diseases?

Answer 37. While individual rare diseases are uncommon, they collectively affect 25-30 million Americans. NIH has several programs that support rare disease research. The National Center for Advancing Translational Sciences (NCATS) established the Division of Rare Disease Research Innovation, which facilitates and coordinates NIH-wide activities involving research for a broad array of rare diseases. This division develops and maintains a centralized data base on rare diseases, coordinates and liaises with organizations worldwide concerned with rare diseases research and orphan products development, and advises the Office of the Director on matters related to NIH-
sponsored research involving rare diseases. One example of this research is Rare Disease Clinical Research Network, which is funded by NCATS and 9 other Institutes and Centers (ICs). If confirmed as NIH Director, I intend to continue the important work the agency is doing to address rare diseases.

Here is the part from the Nature article.

This study was funded in part by the National Institutes of Health NIH/NCI grant (R01CA123396), the UCLA SPORE in Brain Cancer (P50CA211015), NIH National Center for Advancing Translational Science - UCLA CTSI (UL1TR001881), the Parker Institute for Cancer Immunotherapy, and the Brain Tumor Funder’s Collaborative.

Here is something I want to repeat again.

Massive clinical trial on rare caners was initiated by NCI in January 2017 two yeas after the poly-iclc trial enrollment was finished with 23 patients enrolled (the original objective was to enroll 60 patients). GBM was not listed in the trial.

https://clinicaltrials.gov/study/NCT02834013

The trial was run through NCI's National Clinical Trials Network (NCTN). Dr. Monica Bertagnolli was active in NCTN. Seems to me that she introduced biomarker analysis into clinical trial. Do we have biomarker analysis in the current combo trial?

Now can we guess the reason that NCI had genetic profiling analysis on tissue samples from the p3 trial without Dr. Bosch involved? Can we also assume the reason that the Moonshot program was reinvigorated after the data lock and the enrollment of the last patient in the combo trial?

https://www.ncbi.nlm.nih.gov/geo/query/acc.cgi?acc=GSE249282

https://reporter.nih.gov/search/giVvKRz1ikqztvDXWEtuUw/projects?sort_field=fiscal_year&sort_order=desc

Bertagnolli also led initiatives within NCI’s National Clinical Trials Network. One of the first studies she ever conducted involved a molecular marker for colon cancer and its effects on tumor behavior. That marker was later found to predict better responses to immunotherapy.

https://nihrecord.nih.gov/2023/05/12/enthused-lead-nci-bertagnolli-has-plan

Her leadership in the NCI-funded Cooperative Groups Program (now NCI’s National Clinical Trials Network) has led to the integration of tumor-specific biomarkers in clinical trial protocols. More recently, her research on the APC gene and the role of inflammation in influencing its activity has transformed our understanding of how colorectal cancer develops.

https://www.cancer.gov/news-events/press-releases/2022/bertagnolli-nci-director

As a physician–scientist, she led translational science initiatives from 1994 to 2011 within the NCI-funded Cooperative Groups Program (now known as NCI’s National Clinical Trials Network), and from 2011–2022 served as group chair of the Alliance for Clinical Trials in Oncology, a National Clinical Trials Network member organization.

https://www.nih.gov/about-nih/who-we-are/nih-director/nih-director-monica-m-bertagnolli-md