DDW readers’ top drug discovery developments of 2023

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DDW’s Diana Spencer takes a look at the breaking news stories that were most popular with DDW readers over the last year.

Representing a dominant field of drug discovery, it isn’t surprising that breakthroughs in cancer research were popular with DDW readers in 2023, particularly strategies to overcome treatment resistance and prevent the spread of disease. Neuroscience was also a key area for the year for the audience, with a number of ground-breaking developments, including a new potential treatment for schizophrenia and positive results for psychedelics as an antidepressant.

  1. Compound that can block cancer metastasis

In DDW’s most-read news stoy of 2023, researchers from the University of Liverpool, UK, created a biomedical compound that has the potential to stop the spread of breast cancer.

Scientists from the Chemistry and Biochemistry Departments at the University of Liverpool and Nanjing Medical School in China discovered a possible way to block proteins that cause metastasis.

Read the story: Scientists create compound that can block cancer metastasis

  1. CAR-T therapy for solid tumours

In September, Leucid Bio was granted clinical trial authorisation by the UK Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase I/II clinical trial evaluating autologous NKG2D-targeted CAR-T cell therapy, LEU011, in patients with relapsed or refractory solid tumours.

The open-label, single patient cohort ascending dose trial followed promising preclinical data for LEU011 in a spectrum of solid tumour models.

Read the story: CAR-T therapy targets over 80% of human tumours

  1. Stanford false data accusations

In the biggest research ethics scandal of the year, the president of Stanford University in the US announced in July that he would resign after the university found that scientific papers he had published contained manipulated research data.

The scientific misconduct inquiry did not find neuroscientist Marc Tessier-Lavigne guilty of falsifying research personally, but said that papers he co-authored had “serious flaws”.

Read the story: Stanford president quits following false data accusations

  1. LDL cholesterol vaccine

In 2023, the first subjects were dosed in a Phase I clinical trial of VXX-401, an investigational vaccine designed to lower low-density lipoprotein (LDL) cholesterol, a known factor in heart disease.

The drug works by targeting proprotein convertase subtilisin/kexin type 9 serine protease (PCSK9).

Read the story: LDL cholesterol vaccine ‘could be a game-changer’

  1. A game-changing schizophrenia drug

A potential schizophrenia treatment, discovered at Vanderbilt University in Tennessee in the US, was cleared by the US Food and Drug Administration for use in Phase I clinical trials in December 2023.

NMRA-266, an allosteric modulator that works through a mechanism that has been clinically validated in the treatment of disorders like schizophrenia, will be developed by Neumora Therapeutics.

Read the story: Potentially game-changing schizophrenia drug enters trials

  1. Psychedelic antidepressant trial

Psychedelics were big news in 2023. In this story, Phase II interim data for psilocybin analog CYB003 in major depressive disorder (MDD) showed rapid, robust, and clinically significant reduction of depression symptoms.

Of the trial participants, 53.3% responded and 20% are in remission (i.e. no longer meeting the clinical definition of depression) after a single 12mg dose.

Read the story: Psychedelic antidepressant trial shows rapid and robust response

  1. New Covid-19 vaccines

With Covid-19 still never far from the headlines, in August Pfizer/BioNTech, Moderna and Novavax developed new Covid-19 vaccines believed to be effective against the fast-spreading EG.5 strain.

Variant of the month, EG.5 or ‘Eris’ was found in more than 50 countries. At the time it was the most common and fastest growing Covid-19 subvariant in the US, estimated to be responsible for around 17% of Covid-19 cases.

Read the story: New Covid-19 vaccines ready to slow spread of EG.5

  1. Analysis of 2023 clinical trials

Analysis of 13,490 clinical trials found that almost a third have been cancelled during Phase II amidst an overall decline in clinical development productivity.

Data analytics company Phesi released the results of its mid-year global analysis of all clinical trials conducted in 2023 to date. The analysis revealed that Covid-19 is no longer one of the top five most studied disease areas, and trial activity for depression had increased – becoming the fourth most studied disease globally.

Read the story: A third of clinical trials cancelled during Phase II

  1. Overcoming tumour resistance to treatment

New study results demonstrated the potential of a new drug to reprogramme the immune profile in the tumour microenvironment (TME) and convert resistant tumours to responders to Immune Checkpoint Blockage (ICB) therapies.

Purple Biotech shared the new results at the American Association for Cancer Research (AACR) Annual Meeting 2023 in Florida, US.

Read the story: New drug could help overcome tumour resistance to treatment   

  1. RNA therapy eradicates melanoma tumours

Investigators at the Icahn School of Medicine at Mount Sinai designed an RNA-based strategy to activate dendritic cells that eradicated tumours and prevented their recurrence in mouse models of melanoma.

The findings, which suggest that the approach has the potential to be effective against tumours that have already spread to other parts of the body and against different cancer types, were reported in Nature Nanotechnology.

Read the story: RNA strategy activates dendritic cells and eradicates melanoma tumours

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