OnCo
bottlenecksBottleneck

Cachexia, toxicity and the limits of the patient

Patients often die of wasting or cannot tolerate the doses that would work. Treating the patient, not just the tumour, lags far behind.

Cancer cachexia, a syndrome of muscle and fat loss driven by tumour-derived and host inflammatory signals (IL-6, GDF15, activin), affects most patients with advanced pancreatic, gastric and lung cancer and is implicated in a large share of cancer deaths, yet anamorelin in Japan is the only approved drug anywhere and there is none in the US or Europe. Beyond cachexia, treatment-limiting toxicities determine what dose a patient can receive: neuropathy, cardiotoxicity, cytopenias, interstitial lung disease from ADCs, cytokine release and neurotoxicity from cell therapies, and fatigue. Supportive-care research receives a small share of funding relative to its effect on survival and quality of life, and effective interventions such as structured exercise and geriatric assessment are rarely prescribed. Treating the host is a therapeutic target in its own right.

majorbiology18 ideas to fix it
How big the problem is
Up to 20%
Share of cancer deaths in which cachexia is the immediate cause (estimate)
+5.6% body weight difference
Weight gain with the GDF-15 antibody ponsegromab vs placebo at 12 weeks in cancer cachexia (highest dose)
Substantial under-reporting for all six symptoms studied
Physician under-reporting of symptomatic toxicities compared with patient self-report across three randomised trials
Root causes
  • Cachexia is driven by systemic inflammation and neuroendocrine signalling that tumour-directed therapy does not address.
  • Supportive-care trials have no patent-protected asset to fund them and attract little industry investment.
  • Toxicity is graded by clinicians on the CTCAE scale, which under-captures patient-experienced symptoms.
  • Dose-finding uses maximum tolerated dose, so many regimens start above what most patients can sustain.
  • Nutrition, exercise and psycho-oncology are delivered inconsistently and rarely reimbursed as oncology care.
What is already being tried
  • Pfizer's ponsegromab (anti-GDF-15) produced weight gain and improved activity in a phase 2 trial and is in phase 3.
  • Anamorelin (Ono) is approved in Japan for cachexia in lung, gastric, pancreatic and colorectal cancer.
  • The Cancer Grand Challenges CANCAN team is dissecting the neural and metabolic circuits of cachexia.
  • ASCO published its first cachexia management guideline in 2020, and PRO-CTCAE (NCI) provides a validated patient-reported toxicity instrument.
  • Cardio-oncology and exercise-oncology services (including the CHALLENGE trial of structured exercise in colon cancer) are formalising host-directed care.
  • Scalp cooling, biosimilar growth factors and ILD-monitoring algorithms for ADCs are reducing treatment-limiting toxicity.
What breaking it looks like
An anti-cachexia drug improves survival or function in a phase 3 trial and is approved in the US and EU; patient-reported toxicity is collected in every trial and used for dose decisions; supportive-care interventions with survival benefit are reimbursed and delivered to most eligible patients.

Ideas to fix it

18top
early clinicalclinicsmall cost
A cheap old tablet to restore appetite

A low dose of an old, inexpensive tablet improved appetite and weight in a randomised trial of people with advanced cancer. It could be used almost everywhere tomorrow.

speculativephilanthropymedium cost
A dedicated programme for cachexia and treatment toxicity research

Wasting and side-effects kill or stop treatment for a large share of patients but attract almost no dedicated funding. This would create a standing programme for them.

early clinicalclinicmedium cost
A dietitian in every gastrointestinal and head and neck tumour board

Malnutrition is the commonest untreated complication in cancers of the gut, throat and pancreas. Putting a dietitian in the meeting where treatment is decided means it is seen and treated before chemotherapy starts, not after weight has been lost.

speculativeresearchsmall cost
Aggregated single-patient crossover trials for symptom and supportive treatments

For treatments of symptoms like nausea, fatigue or neuropathy, each patient can try the drug and a placebo in alternating periods and see what works for them; pooling many such personal trials gives a population answer too.

speculativephilanthropylarge cost
An ARPA-style programme to develop supportive-care drugs nobody else will

A supportive-care ARPA would be a well-funded, milestone-driven agency that develops drugs for nausea, nerve damage, mouth sores, fatigue and brain fog from cancer treatment, which the market has largely ignored.

speculativepatientssmall cost
Catch wasting early with a smart scale and a step counter

Slow weight loss and falling daily activity are the first signs of cancer wasting, and both can be measured at home. An alert could bring help months earlier.

early clinicalindustrymedium cost
Combine the new anti-wasting antibody with exercise and protein

A new antibody blocks the hormone that makes people with cancer lose appetite and weight. Weight regained as muscle, not fat, needs exercise and protein alongside it.

early clinicalresearchmedium cost
Confirm low-dose olanzapine for appetite and weight in advanced cancer worldwide

An Indian trial found a cheap antipsychotic pill in very low dose improved appetite and weight gain in patients with advanced stomach, lung and pancreatic cancer. It needs confirming and adopting worldwide.

speculativeclinicmedium cost
Dose chemotherapy by muscle mass, not body surface area

Chemotherapy doses are calculated from height and weight, a formula from the 1950s. Doses based on actual muscle mass may cause fewer severe side-effects.

being tested at scaleclinicmedium cost
Four weeks of training and nutrition before major cancer surgery, as standard

Getting fitter and better nourished before an operation reduces complications and speeds recovery. It is cheap, but only a few hospitals do it.

early clinicalregulatorsmall cost
Get the one approved appetite drug licensed beyond a single country

A drug that improves appetite and lean weight in cancer wasting is approved in Japan but almost nowhere else. Reviewing the existing evidence could widen access quickly.

early clinicalpayermedium cost
Pay for supervised exercise the way we pay for drugs

A large trial showed a structured exercise programme improved survival after bowel cancer. Almost no health system pays for it, so almost no patient gets it.

speculativeregulatorsmall cost
Qualify a physical function endpoint so anti-wasting drugs can be approved

Regulators are unsure what to accept as proof that an anti-wasting drug helps. Agreeing on a simple measure such as stair climbing would unblock the whole field.

preclinical evidencedatasmall cost
Read muscle loss automatically from scans patients already have

Every staging scan contains a precise measure of muscle mass that nobody looks at. Software could report it automatically and flag patients heading for wasting.

speculativeindustrysmall cost
Select cachexia trial patients by the hormone driving their wasting

Wasting has several causes. Measuring the specific hormone in each patient's blood would put the right patients into the right trial instead of mixing everyone together.

speculativeresearchmedium cost
Test protein and resistance training during immunotherapy

Muscle is an immune organ as well as a movement organ. Building it during immunotherapy might improve how well the treatment works, not just how patients feel.

early clinicalindustrylarge cost
Treat cachexia as a disease: GDF-15 blockade plus anabolic and nutrition bundles

The wasting that kills many cancer patients has had no effective drug. New antibodies against GDF-15 restored weight in early trials. Combine them with exercise and nutrition and test properly.

speculativeclinicsmall cost
Treat wasting like sepsis: a trigger, a bundle, an audit

Hospitals have fast, standard responses to sepsis and heart attacks. Cancer wasting has no such pathway, so it is noticed late and treated inconsistently.

What relieves it today

7top

Connected

51top

Pages like this

not linked directly; found by shared links

cancers

5

fronts

1

technologies

8

companies

1

institutions

5

terms

9

trials

3

ideas

19