Most academic discoveries die before anyone tests them in people because nobody funds the middle step.
Between a validated target or lead compound in an academic laboratory and a first-in-human trial lie medicinal chemistry optimisation, GMP manufacturing, toxicology, formulation and an IND or CTA dossier, work that costs several million dollars, is not publishable, and is funded by neither research grants nor, until the asset is de-risked, by companies or venture capital. Of highly promising basic science claims published in leading journals, a small minority reach licensed use decades later. The consequence is that promising ideas stall for years or vanish, and that the ideas that do cross are selected for commercial attractiveness rather than for medical need. Public translational programmes (NCI Experimental Therapeutics, NCATS), charity drug-development units, academic-industry alliances and non-dilutive philanthropic capital exist but are small relative to the flow of discoveries.
Companies shelve drugs that were safe but failed in the disease they tried. An oncology commons cataloguing discontinued assets with their mechanism, human pharmacokinetics, safety data and reasons for discontinuation, under template access terms, would let others test them where they might work, as NCATS and AstraZeneca schemes have shown.
Investors will not back a single university drug because most fail. A fund that finances fifty of them at once in exchange for a small slice of each one's future royalties spreads the risk enough to attract capital.
The studies needed before a first human trial cost a few million and no grant pays for them. A dedicated fund would decide in weeks and take a small share of any future revenue.
Thousands of tests that could predict who benefits from a treatment are published and never validated. A fund would pay for the boring but essential confirmation studies in independent patient groups.
Before any academic compound gets money for pre-trial studies, it would have to show activity in a standard panel of patient-derived tumour models run by an independent centre, so weak candidates are stopped early.
Academic first-in-human cancer trials recruit slowly because each hospital repeats ethics and regulatory review. Twenty to thirty academic phase 1 units across Europe, North America and Asia would share protocol templates, one mutually recognised review, a joint safety committee and harmonised contracts, so a trial opens at every site within weeks and rare molecular subtypes can be pooled.
Drugs fail for distinct reasons: wrong target, drug never reached it, unacceptable toxicity, unselected population or poor trial design. A shared machine-readable taxonomy applied to every discontinued oncology programme in public pipeline databases would show where the system breaks, as AstraZeneca and Pfizer's own attrition analyses did.
Running an early trial properly requires monitors, data managers, safety reporting and regulatory filings that universities cannot afford from commercial providers. A public not-for-profit would do this work at cost.
Build a drug company that does not need profits, modelled on the ones that developed new tuberculosis and sleeping-sickness drugs, to take on rare, paediatric and undruggable cancers.
Build a shared, not-for-profit clinical unit that runs early combination trials to a standard recipe, so that small companies and academics can test pairs without building their own trial machinery.
Before investing in a full trial, give a few patients a tiny dose of a new compound and use scans and blood tests to see whether it reaches the tumour and hits its target. Fund these small studies as a matter of routine.
Tools that show surgeons where the tumour ends during the operation could cut the number of patients who need a second operation, but none has been properly tested at scale. A network would run those trials and pay on results.
Companies and public funders would jointly pay for standardised experiments that confirm or refute new cancer targets, sharing all results openly, so nobody wastes years on a target that does not hold up.
Universities often refuse to be the legal sponsor of a first-in-human trial because they cannot afford the insurance and liability. A shared public insurance pool would remove that block.
Thousands of cancer compounds that stopped development for portfolio rather than scientific reasons sit unused in university freezers and company archives. A public catalogue listing each asset's mechanism, stage, data, reason for stopping and licensing contact, with a standard research licence and a brokerage function, would let academic and non-profit developers adopt them.
Promising academic cancer discoveries stall because nobody funds the expensive step from lab to first human trial. Build a shared public facility that does exactly that step, repeatedly.
Companies hold thousands of well-characterised drugs that could help rare cancers, but each request takes a year of legal negotiation. One standing agreement would unblock it.
Between diagnosis and surgery there are usually a few weeks. Giving a new drug in that window and comparing the tumour before and after surgery shows whether it hits its target in real people, quickly and cheaply.
New surgical tools, imaging probes and radiotherapy hardware invented in universities rarely attract investors. A dedicated fund would pay for prototyping, safety testing and first-in-human studies.
Most new cancer imaging agents and radioactive drugs start in university hospitals. A network sharing production, quality files and regulatory paperwork would get them into multi-centre trials years faster.
Many cancer lab results cannot be reproduced, and trials built on them fail. Fund an independent institute that re-runs important experiments before anyone spends millions on humans.
Academic labs find new tumour targets but cannot turn an antibody into an antibody-drug conjugate or a bispecific without licensed linker and payload technology. A shared platform would provide that at no cost for first trials.
You cannot study a cancer without a laboratory model of it, and most rare cancers have none. A funded bank that makes and shares models would unlock research.
Three genetic classifications of the commonest aggressive lymphoma exist and none of them yet decides anyone's treatment. The trial that would change that has not been run.
When a company stops developing a cancer drug for business rather than safety reasons and leaves it idle for two years, it would be obliged to offer the rights, data package and remaining drug supply to qualified non-profit or academic developers on pre-set terms, keeping the right to resume. This turns the stalled-asset registry's listing into a duty.
Bowel cancer in people under 50 is rising by 2 to 8 percent a year on both sides of the Atlantic and nobody knows why. The strongest lead is a toxin made by some gut bacteria whose damage signature is three times more common in young patients and is stamped on the colon early in life. If that is the cause, the fix is in childhood, not in a screening programme.
Fast grants for oncology would be a fund that decides within two days on small grants for quick, decisive experiments in cancers or questions that mainstream funders neglect, modelled on the pandemic-era Fast Grants.
Investors avoid genuinely new cancer drugs because most fail in mid-stage trials. A public insurance scheme would repay part of the loss when a first-in-class drug fails honestly, making the bet worth taking.
European law already lets hospitals make advanced therapies for their own patients. Pair that with a shared outcomes registry so academic CAR-Ts and similar treatments can prove themselves without a commercial licence.
Doctors who could turn discoveries into trials are buried in clinical work. Hospitals would guarantee them research time and recover the cost from the trials and grants they bring in.
Before a new cancer drug from a university is given to people, a separate laboratory should have repeated the main experiment showing it works.
Cancer charities would fund companies to hit specific development milestones, as the cystic fibrosis charity did to create Kalydeco, and take a royalty they reinvest in the next drug.
A drug that works in one laboratory's mice often fails elsewhere. Running the confirmatory animal study as a randomised, blinded, multi-laboratory trial, as stroke and amyotrophic lateral sclerosis research has done, would catch this before a human trial; oncology has no such standing infrastructure.
Clinical trials get expert statistical review; the laboratory studies that justify them usually do not. Paying statisticians to review these papers before they influence a trial would catch errors early.
Dogs get cancers that closely resemble human ones, with real immune systems and years of natural history. Treating them, with owner consent, can test drugs in a way mice cannot.
Building a cell-therapy factory costs tens of millions, so most good academic ideas never reach patients. Shared public facilities would give them a route to the clinic.
A public investment fund would match private money in the riskiest early trials of truly new cancer drugs, taking a small share of future royalties so that taxpayers gain when the bets pay off.
Build a handful of publicly-funded centres where academic discoveries can be manufactured to clinical grade and written up for regulators, so good ideas do not die for lack of a factory and a filing.
The US government already runs a small programme that turns academic cancer discoveries into drugs ready for human trials. Scale it up tenfold and copy it in other countries.
Robotic labs guided by AI that design experiments on tumour models, run them, read the results and design the next ones, around the clock, with every result published openly.
Before spending millions to turn a lab finding into a drug, spend a little to have an independent lab check it is real. Funders would reserve a small slice of money for exactly this.
Personalised cancer vaccines and cell therapies need a manufacturing run for each patient, and universities cannot afford their own plants. Regional closed, automated, modular GMP facilities offering slots to academic trials at cost, with common release testing and a shared quality system, modelled on the UK Cell and Gene Therapy Catapult centre, would let academic groups run these trials.
Postdocs who make a translatable discovery usually leave it behind when their contract ends. A two-year fellowship with salary, a project budget of $250,000 to $500,000 and mentors from drug development, regulation and the clinic would pay them to turn it into a candidate drug, diagnostic or device, with the option to license it or found a company.
The shape of nearly every protein is now available to any researcher in seconds instead of years, which shortens the path from a cancer target to a designed molecule. It does not by itself produce drugs: binding pockets, dynamics and cellular context still need experiment.
Many exciting laboratory findings that motivate drug programmes are weaker or less reliable than published, which helps explain the high failure rate of drugs entering clinical trials. It argues for pre-registration, detailed methods, data sharing and independent replication before major translational investment.
One of the two foundational genetic classifications of diffuse large B-cell lymphoma. Neither has yet changed what a patient receives outside a trial, but together they are the reason precision-medicine trials in this disease now select by genetics rather than by cell of origin.
DepMap is the lookup table drug hunters use to ask: which cancers would die if we blocked this gene, and how would we recognise them? It generated targets such as WRN and PRMT5-MTAP now in clinical trials, and it is public.
This paper is the proof-of-concept for a living drug: a single infusion of a patient's own engineered T cells could eradicate leukaemia that had survived chemotherapy, transplant and antibody therapy. It defined cytokine release syndrome and its antidote, tocilizumab, and revealed antigen-loss relapse. It led directly to the first approved gene-modified cell therapy three years later.
The proof, in people, that castration-resistant prostate cancer is usually still androgen-driven. It renamed the disease (hormone-refractory became castration-resistant) and it opened the line of drugs that now dominate treatment at every stage from first diagnosis of metastatic disease onwards.
One bottleneck page and twelve idea pages on OnCo cite this paper by its DOI; this record gives the citation a page of its own so a reader can follow it without leaving OnCo. Read the abstract above alongside the citing pages listed under Related; the record was created automatically from the Europe PMC entry and its figures have not been checked by hand.
The second driver in lung cancer, and the one that proved the first was not a special case. It also established mutual exclusivity as a working assumption: a tumour usually has one driver, so finding it tells you what to give.
Shares Forty-eight-hour small grants for bold experiments in neglected cancers, Ellison Institute of Technology, Worldwide Cancer Research, Larry Ellison.
Shares Pet dogs with spontaneous cancer as a bridge before human trials, Multi-centre randomised animal trials before committing to a human trial, Cancer Center at Illinois, A funded organoid and PDX panel as the go/no-go gate before IND-enabling money.
Shares An independent replication institute that re-tests key preclinical cancer findings before trials, Paid independent statistical review for preclinical papers that inform trials, Independent replication of the key experiment before first-in-human academic trials, Multi-centre randomised animal trials before committing to a human trial.
Shares An independent replication institute that re-tests key preclinical cancer findings before trials, Multi-centre randomised animal trials before committing to a human trial, A funded organoid and PDX panel as the go/no-go gate before IND-enabling money, A pre-competitive consortium to validate or kill academic targets before licensing.
Shares A public registry of stalled academic assets and shelved company compounds, An open engineering platform for academic ADCs and bispecifics, A pre-competitive consortium to validate or kill academic targets before licensing, A shared compound library that rare cancer researchers can actually use.
Shares Scale up public drug development that takes academic assets to phase 1, AlphaFold 2: predicting protein structures to near-experimental accuracy, First imatinib trial: a pill that switched off the enzyme driving chronic myeloid leukaemia, Defining a Cancer Dependency Map: which genes each cancer cell line cannot live without.
Shares AlphaFold 2: predicting protein structures to near-experimental accuracy, Isomorphic Labs, Generate:Biomedicines, Insilico Medicine.