CPI-613 in Treating Patients With Myelodysplastic Syndromes Who Failed Previous Therapy

This pilot clinical trial studies 6, 8-bis (benzylthio) octanoic acid (CPI-613) in treating patients with myelodysplastic syndromes who failed previous therapy. Sometimes when chemotherapy or biological therapy is given, it does not stop the growth of tumor cells. The tumor is said to be resistant to treatment. 6, 8-bis (benzylthio) octanoic acid may interfere with the growth of tumor cells and may be an effective treatment for myelodysplastic syndromes that did not respond to previous therapy.

PRIMARY OBJECTIVES:

I. To evaluate the safety and anti-cancer activities of CPI-613 in myelodysplastic syndrome (MDS) patients who have failed previous agents (such as decitabine \[Dacogen\], azacitidine \[Vidaza\], growth factors or lenalidomide).

OUTLINE:

Patients receive 6, 8-bis (benzylthio) octanoic acid intravenously (IV) over 2 hours on days 1 and 4 of weeks 1-3. Courses repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up for 5 years.

Eligibility Criteria:
Inclusion Criteria:

* Histologically or cytologically documented MDS of any risk group that has failed previous therapy (therapy failure is defined as patients who have been sufficiently treated with previous agents without response in the opinion of the treating physician, or whose disease has progressed or relapsed while on a hypomethylating agent)
* Eastern Cooperative Oncology Group (ECOG) performance status of =\< 3
* Expected survival \> 2 months
* Women of child-bearing potential (i.e., women who are pre-menopausal or not surgically sterile) must use accepted contraceptive methods (abstinence, intrauterine device \[IUD\], oral contraceptive or double barrier device) during the study, and must have a negative serum or urine pregnancy test within 1 week prior to treatment initiation
* Fertile men must practice effective contraceptive methods during the study, unless documentation of infertility exists
* Patients must have fully recovered from the acute, non-hematological, non-infectious toxicities of any prior treatment with cytotoxic drugs, radiotherapy or other anti-cancer modalities; patients with persisting, non-hematologic, non-infectious toxicities from prior treatment =\< grade 2 are eligible, but must be documented as such
* Aspartate aminotransferase (AST/serum glutamic oxaloacetic transaminase \[SGOT\]) =\< 3 x upper normal limit (UNL)
* Alanine aminotransferase (ALT)/serum glutamate pyruvate transaminase (SGPT) =\< 3 x UNL (=\< 5x ULN if liver metastases present)
* Bilirubin =\< 1.5 x UNL
* Serum creatinine =\< 1.5 mg/dL or 133 umol/L
* International normalized ratio (or INR) must be \< 1.5
* Albumin \>= 2.0 g/dL or \>= 20 g/L
* Mentally competent, ability to understand and willingness to sign an Institutional Review Board (IRB)-approved written informed consent form
* Have access via central line (e.g., portacath)

Exclusion Criteria:

* Serious medical illness, such as significant cardiac disease (e.g. symptomatic congestive heart failure, unstable angina pectoris, coronary artery disease, myocardial infarction within the past 3 months, uncontrolled cardiac arrhythmia, pericardial disease or New York Heart Association class III or IV), or severe debilitating pulmonary disease, that would potentially increase patients' risk for toxicity
* Patients with active central nervous system (CNS) or epidural tumor
* Any active uncontrolled bleeding or bleeding diathesis (e.g., active peptic ulcer disease)
* Any condition or abnormality which may, in the opinion of the investigator, compromise his or her safety
* Pregnant women, or women of child-bearing potential not using reliable means of contraception
* Fertile men unwilling to practice contraceptive methods during the study period
* Lactating females
* Life expectancy less than 2 months
* Unwilling or unable to follow protocol requirements
* A history of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, family history of long QT syndrome, etc.)
* Evidence of active infection or serious infection within the past month
* Requirement for immediate palliative treatment of any kind including surgery
* Prior illicit drug addiction
* Patients with large and recurrent pleural or peritoneal effusions requiring frequent drainage (e.g. weekly)
* Patients with any amount of clinically significant pericardial effusion
* Patients with known human immunodeficiency virus (HIV) infection; (Note: patients with known HIV infection are excluded because patients with an immune deficiency are at increased risk of lethal infections when treated with marrow-suppressive therapy, and because there may be unknown or dangerous drug interactions between CPI-613 and anti-retroviral agents used to treat HIV infections)
* Patients who have received radiotherapy, surgery, treatment with cytotoxic agents (except CPI-613), treatment with biologic agents, immunotherapy, or any other anti-cancer therapy of any kind, or any other standard or investigational treatment for their cancer, or any other investigational agent for any indication, within the past 2 weeks prior to initiation of CPI-613 treatment
* Patients that have received a chemotherapy regimen with stem cell support in the previous 6 months