← All medicines

Doxorubicin Hydrochloride

Anthracyclines and related substances · given by injection · on the market since 1987

5
Leads
18.0M
People
15
Makers
70
How abandoned
01

What it's made of

Each functional group on the molecule does a specific, predictable job.

SETS WHERE IT GOES

Sugar ring

What it does. Extremely water-loving. Adding one to a molecule usually destroys its ability to cross the gut wall, which is why many such drugs must be injected.

What it opens up. If given by mouth it stays in the gut and acts there only — a targeting advantage for intestinal disease.

SETS HOW LONG IT LASTS

Phenol

What it does. The liver's favourite attachment point. It tags this group and flushes the molecule out, often within an hour.

What it opens up. Usually shortens the drug's life. Blocking or masking this position is the standard way to make a compound last longer.

CARRIES THE MAIN JOB

Four-ring fused core

What it does. Beyond its known job, this frame binds metal ions inside human enzymes that break down tissue scaffolding — an activity completely separate from its original purpose and active at much lower doses.

What it opens up. Below its usual dose it is not an antibiotic at all but a tissue-protection agent — gum disease, skin inflammation, aneurysm progression.

02

Where it can get to

Predicted from molecular weight, lipophilicity, polar surface area and hydrogen-bond donor count. A drug can only act where it distributes, so this constrains which conditions are addressable before any biology is considered. derived

Partly
Systemic circulation
Absorbed from the gut and distributed in plasma.
No
Central nervous system
Crosses the blood–brain barrier.
Partly
Intestinal lumen only
Poorly absorbed. Acts locally in the gut with minimal systemic exposure.
No
Skin
Crosses the stratum corneum. Viable as a topical.
Partly
Urinary tract
Cleared unchanged in urine. Local concentration exceeds plasma.
03

What it binds — and whether we can reach it · 1 of 1 reachable

Binding affinity is only meaningful against the plasma concentration reached at the approved dose. A target requiring higher concentrations cannot be engaged in a patient, whatever the in vitro potency. Most repurposing hypotheses fail at this step, and it is rarely checked before a trial is proposed. measured

Values are pAct — the negative logarithm of the molar concentration at which the drug half-occupies the target. Each whole step is a tenfold difference in potency: 9.0 = 1 nanomolar, 6.0 = 1 micromolar, 5.0 = 10 micromolar. Oral drugs typically reach 0.1–10 micromolar in plasma, so roughly 6.0 is the threshold below which a target cannot be reached at a tolerated dose. Figures come from DrugCentral’s curated binding data.
TOP2A
Enzyme · inhibitor
6
Just reachable

This molecule concentrates in urine. Local concentration therefore exceeds plasma, so targets that look unreachable on a plasma reading may still be engaged. assumed

Cytotoxic. Targets core cell-division machinery. Use is limited to conditions severe enough to justify that toxicity; risk-benefit inverts in chronic benign disease.

04

New uses, and who is already trying them

One card per candidate indication: the reasoning link by link, the evidence tier that governs how strongly it can be stated, and any registered trials.

5 of the 60 conditions linked to this drug’s targets have been checked against trial records so far. The rest are not ruled out — they are unchecked.

Not yet used for these — 4

neoplasm

Tried in people

It showed an effect in a human trial that was not followed up.

The condition
A benign or malignant tissue growth resulting from uncontrolled cell proliferation. Benign neoplastic cells resemble normal cells without exhibiting significant cytologic atypia, while malignant cells exhibit overt signs such as dysplastic features, atypical mitotic figures, necrosis, nuclear pleomorphism, and anaplasia. Representative examples of benign neoplasms include papillomas, cystadenomas, and lipomas; malignant neoplasms include carcinomas, sarcomas, lymphomas, and leukemias.
TOP2A · DNA topoisomerase II alpha
Key decatenating enzyme that alters DNA topology by binding to two double-stranded DNA molecules, generating a double-stranded break in one of the strands, passing the intact strand through the broken strand, and religating the broken strand (PubMed:17567603, PubMed:18790802, PubMed:22013166, PubMed:22323612, PubMed:27754753). May play a role in regulating the period length of BMAL1 transcriptional oscillation (By similarity). {ECO:0000250|UniProtKB:Q01320, ECO:0000269|PubMed:17567603, ECO:0000269|PubMed:18790802,
Phase 3 has run
A Phase 3 trial exists. Read its result before anything else.
NCT01547741 Docetaxel and Cyclophosphamide Compared · Ph 3 · 4242 people
+14 more on ClinicalTrials.gov
What would kill it. A null result in the completed Phase 3.

breast cancer

Tried in people

It showed an effect in a human trial that was not followed up.

The condition
A primary or metastatic malignant neoplasm involving the breast. The vast majority of cases are carcinomas arising from the breast parenchyma or the nipple. Malignant breast neoplasms occur more frequently in females than in males.
TOP2A · DNA topoisomerase II alpha
Key decatenating enzyme that alters DNA topology by binding to two double-stranded DNA molecules, generating a double-stranded break in one of the strands, passing the intact strand through the broken strand, and religating the broken strand (PubMed:17567603, PubMed:18790802, PubMed:22013166, PubMed:22323612, PubMed:27754753). May play a role in regulating the period length of BMAL1 transcriptional oscillation (By similarity). {ECO:0000250|UniProtKB:Q01320, ECO:0000269|PubMed:17567603, ECO:0000269|PubMed:18790802,
Phase 3 has run
A Phase 3 trial exists. Read its result before anything else.
NCT01547741 Docetaxel and Cyclophosphamide Compared · Ph 3 · 4242 people
+14 more on ClinicalTrials.gov
What would kill it. A null result in the completed Phase 3.

acute myeloid leukemia

Tried in people

It showed an effect in a human trial that was not followed up.

The condition
Acute myeloid leukemia (AML) is a group of neoplasms arising from precursor cells committed to the myeloid cell-line differentiation. All of them are characterized by clonal expansion of myeloid blasts. AML manifests by fever, pallor, anemia, hemorrhages and recurrent infections.
TOP2A · DNA topoisomerase II alpha
Key decatenating enzyme that alters DNA topology by binding to two double-stranded DNA molecules, generating a double-stranded break in one of the strands, passing the intact strand through the broken strand, and religating the broken strand (PubMed:17567603, PubMed:18790802, PubMed:22013166, PubMed:22323612, PubMed:27754753). May play a role in regulating the period length of BMAL1 transcriptional oscillation (By similarity). {ECO:0000250|UniProtKB:Q01320, ECO:0000269|PubMed:17567603, ECO:0000269|PubMed:18790802,
Phase 3 has run
A Phase 3 trial exists. Read its result before anything else.
NCT00002766 Comparison of Two Combination Chemothera · Ph 3 · 170 people
+14 more on ClinicalTrials.gov
What would kill it. A null result in the completed Phase 3.

small cell lung carcinoma

Tried in people

It showed an effect in a human trial that was not followed up.

The condition
Small cell lung cancer (SCLC) is a highly aggressive malignant neoplasm, accounting for 10-15% of lung cancer cases, characterized byrapid growth, and early metastasis. SCLC usually manifests as a large hilar mass with bulky mediastinal lymphadenopathy presenting clinically with chest pain, persistent cough, dyspnea, wheezing, hoarseness, hemoptysis, loss of appetite, weight loss, and neurological and endocrine paraneoplastic syndromes. SCLC is primarily reported in elderly people with a history of long-term tobacc
TOP2A · DNA topoisomerase II alpha
Key decatenating enzyme that alters DNA topology by binding to two double-stranded DNA molecules, generating a double-stranded break in one of the strands, passing the intact strand through the broken strand, and religating the broken strand (PubMed:17567603, PubMed:18790802, PubMed:22013166, PubMed:22323612, PubMed:27754753). May play a role in regulating the period length of BMAL1 transcriptional oscillation (By similarity). {ECO:0000250|UniProtKB:Q01320, ECO:0000269|PubMed:17567603, ECO:0000269|PubMed:18790802,
Phase 3 has run
A Phase 3 trial exists. Read its result before anything else.
NCT00011921 Chemotherapy Followed by Peripheral Stem · Ph 3 · 430 people
+14 more on ClinicalTrials.gov
What would kill it. A null result in the completed Phase 3.
Already used for these — 1

Established practice, listed so the method can be checked against what is already known.

plasma cell myeloma

Already in use

It is already used for this.

The condition
A bone marrow-based plasma cell neoplasm characterized by a serum monoclonal protein and skeletal destruction with osteolytic lesions, pathological fractures, bone pain, hypercalcemia, and anemia. Clinical variants include non-secretory myeloma, smoldering myeloma, indolent myeloma, and plasma cell leukemia. (WHO, 2001)
TOP2A · DNA topoisomerase II alpha
Key decatenating enzyme that alters DNA topology by binding to two double-stranded DNA molecules, generating a double-stranded break in one of the strands, passing the intact strand through the broken strand, and religating the broken strand (PubMed:17567603, PubMed:18790802, PubMed:22013166, PubMed:22323612, PubMed:27754753). May play a role in regulating the period length of BMAL1 transcriptional oscillation (By similarity). {ECO:0000250|UniProtKB:Q01320, ECO:0000269|PubMed:17567603, ECO:0000269|PubMed:18790802,
In routine use
Established practice for this condition, not a new candidate.
NCT03908138 RDD Versus VDD in Newly Diagnosed Patien · Ph 4 · 120 people
+14 more on ClinicalTrials.gov
What would kill it. A null trial, or pharmacokinetic data showing the drug does not reach TOP2A in the relevant tissue at tolerated doses.
05

Why nobody will fund it

15 companies make this across 30 approved products. With that many manufacturers the price has fallen to production cost. A company funding a trial could not recover it — competitors could market the same molecule for the new indication immediately, without contributing.

How abandoned it is70 / 100
0 to 100, calculated from manufacturer count, remaining patent or exclusivity, and whether any reformulation route to exclusivity is still open. 100 means no route to commercial return exists. The weights behind it are unsourced.
THE ONE WAY A COMPANY COULD STILL OWN IT
inhaledmodified-release

A new route or dosage form qualifies for a 505(b)(2) application, which carries three years of exclusivity for that formulation. It is the only remaining route to exclusivity on a generic molecule.

06

What it would cost to find out

Derived, not quoted. Events needed is 4(zα+zβ)²/ln(HR)²; enrolment is that divided by the event rate and follow-up. The 1/ln(HR)² term dominates: halving the detectable effect size roughly quadruples enrolment. derived

0.75
Lower = bigger effect = smaller trial
8.0%
2 yr
$32K
Unsourced placeholder
379
Events needed
3K
Patients
$93M
Trial cost
assumed
$14.70
Per person reached
assumed

Cost per person reached is the relevant metric for a non-commercial funder, since there is no revenue to set against the outlay.

07

Ask for ideas nobody has listed

Reasons from the functional groups and distribution above, excluding conditions already listed. Output is hypotheses for falsification, not findings. Not medical advice.

08

Who might pay for the trial

Trials of off-patent drugs are rarely funded by venture capital or pharma. They are paid for by government grants and disease foundations. Here is who has funded work on this molecule before, what federal mechanisms are open now, and which foundations fund trials in the disease areas it touches. measured

Federal grants that name this medicine
3,562 grants on NIH RePORTER
1R01CA308007-01A1(NCI · National Cancer Institute)Active
$582K (2026)
Investigating age-dependent factors in the development and treatment of adrenal cancer
PI: Basham, Kaitlin · Utah State Higher Education System--University Of Utah, Salt Lake City, UT · through 2031-05
5R01HL166810-03(NHLBI · National Heart, Lung & Blood Institute)Active
$759K (2026)
Monitoring Autophagy in the Heart and in Tumors Treated with Potentially Cardiotoxic Chemotherapy
PI: Chen, Howard H. · Tufts Medical Center, Boston, MA · through 2027-12
5R01HL168045-03(NHLBI · National Heart, Lung & Blood Institute)Active
$694K (2026)
Therapeutic Strategies to Mitigate Toxicities of Anthracycline-Based Therapeutics
PI: Sparreboom, Alexander · Ohio State University, Columbus, OH · through 2027-12
Open federal programmes that would fund this work

Open notices with an explicit mandate to fund work on approved or off-patent molecules.

RFA-CA-25-033National Institutes of HealthForecast — not open yet
Glioblastoma Therapeutics Network (GTN; U19 Clinical Trial Required)
RFA-FD-25-020Food and Drug Administrationcloses 05/16/2028
Reissue of RFA-FD-23-001- Clinical Studies of Orphan Products Addressing Unmet
FOR-FD-25-020Food and Drug AdministrationForecast — not open yet
Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R
FOR-FD-25-001Food and Drug AdministrationForecast — not open yet
Clinical Trials Addressing Unmet Needs of Rare Neurodegenerative Diseases (R01
RFA-TR-25-002National Institutes of Healthcloses 07/02/2027
Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not
PAR-25-374National Institutes of Healthcloses 05/07/2028
Translational Bioinformatics and Experimental Approaches to Advance Drug Repos
HT942526PRCRPCTADefense Health Agency Contracting Activity -closes 10/05/2026
DoW Peer Reviewed Cancer, Clinical Trial Award
PAR-26-049National Institutes of HealthForecast — not open yet
Establishing Pediatric CNS Pharmacodynamic Measures as Tools to Enable Psychia
Showing 8 of 990 currently open under this category on Grants.gov, matched to the disease areas this drug touches. derived Browse all 990 on Grants.gov ↗
Funders beyond the federal system

Grants.gov lists US federal money. Many off-patent drug trials are supported by disease foundations and nonprofits. These are matched to the body systems this drug touches. assumed

Matched by disease area, not by molecule — a foundation appearing here funds work in a field this drug touches.
09

Who manufactures it

Companies holding an approved abbreviated application for this molecule in the United States, from the FDA Orange Book. Supply already exists at scale, so a trial would not need a manufacturing programme — but any of these could also be a partner, or an objector.

Actavis IncAlembic Pharmaceuticals LtdAmneal Eu Ltd
15
Manufacturers
companies with their own approval
30
Approved products
distinct strengths and forms
1
Routes
injection
1987
Oldest product still sold
approval year of the earliest product still on the market
ALSO SOLD AS
DOXIL (LIPOSOMAL)DOXORUBICIN HYDROCHLORIDE (LIPOSOMAL)

Nothing on this page is medical advice. Candidate uses are unproven hypotheses derived from public data; established uses are listed for verification only. Figures carry their provenance inline — anything marked assumed or derived is not a measurement.

← Back to the full ledger