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Arimoclomol is an experimental drug compound developed by CytRx Corporation, a biopharmaceutical company based in Los Angeles, California. The orally administered drug is intended to treat amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, a neurodegenerative disease with no effective treatment.

Mechanism of Action

The drug is designed to stimulate a natural cellular repair pathway by activating compounds called “molecular chaperones.” Arimoclomol uses a unique 'molecular chaperone' co-induction mechanism. The small molecule drug candidate is believed to function by stimulating a normal cellular protein repair pathway through the activation of "molecular chaperones." Since damaged proteins called aggregates are thought to play a role in many diseases, CytRx believes that activation of molecular chaperones could have therapeutic efficacy for a broad range of diseases.


Arimolclomol has been shown to extend life in ALS-affected mice and was well tolerated in healthy human volunteers in a recently completed Phase I study. The drug was cited in the fifth annual report on "100 Great Investigational Drugs in Development," published in the March 2006 issue of the pharmaceutical industry magazine R&D Directions. CytRx is currently conducting a Phase II clinical trial at ten centers across the U.S.

Arimoclomol was discovered by Hungarian researchers, as a drug candidate to treat insulin resistance, and diabetic complications, such as retinopathy. neuropathy and nephropathy. Later the compound with other small molecules were synthetised and screened for further development, by Biorex (a firm seatled in Hungary) was sold to CytRx Corporation, who developed it toward a different, but very promising direction, from 2003.

Hungarian institutional code of Arimoclomol was: BRX-220.

Publication date of the patent: 2001-12-03 Publication number: SK11582001 Inventor: KURTHY MARIA (HU); BIRO KATALIN (HU); NAGY KAROLY (HU); UROGDI LASZLO (HU); CSAKAI ZITA (HU); SZILBEREKY JENO (HU); MOGYOROSI TAMAS (HU); TOROK MAGDOLNA (HU); KOMAROMI ANDRAS (HU); MARVANYOS EDE (HU); BARABAS MIHALY (HU); KARDOS MIHALYNE (HU); NAGY ZOLTAN (HU); KORANYI LASZLO (HU); NAGY MELINDA (HU) Applicant: BIOREX KUTATO FEJLESZTOE KFT (HU) Classification: - international: A61P3/10; C07D213/89; A61P3/00; C07D213/00; (IPC1-7): C07D213/89; A61K31/44; A61P3/10 - European: C07D213/89B Application number: SK20010001158 20000224 Priority number(s): HU19990000475 19990226; WO2000HU00015 20000224 Abstract of corresponding document: WO0050403 The invention relates to N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride, its stereoisomers and the acid addition salts thereof, pharmaceutical compositions containing the same, the use of these compounds in the treatment of pathological insulin resistance, and for the treatment of pathological conditions associated therewith, by simultaneous treatment and prevention of diabetes-induced chronic complications, especially retinopathy, neuropathy and nephropathy and/or with simultaneous increasing pathologically decreased peripheral neuroregeneration caused by diabetes and methods of treatment.

List of publications: Source: Key word: Arimoclomol in PubMed: 1: Traynor BJ, Bruijn L, Conwit R, Beal F, O'Neill G, Fagan SC, Cudkowicz ME. Neuroprotective agents for clinical trials in ALS: a systematic assessment.

       Neurology. 2006 Jul 11;67(1):20-7. Review.
       PMID: 16832072 [PubMed - indexed for MEDLINE]

2: Benn SC, Brown RH Jr. Putting the heat on ALS.

       Nat Med. 2004 Apr;10(4):345-7. No abstract available.
       MID: 15057226 [PubMed - indexed for MEDLINE]

3: Kieran D, Kalmar B, Dick JR, Riddoch-Contreras J, Burnstock G, Greensmith L.

       Treatment with arimoclomol, a coinducer of heat shock proteins, delays disease progression
       in ALS mice.
       Nat Med. 2004 Apr;10(4):402-5. Epub 2004 Mar 21.
       PMID: 15034571 [PubMed - indexed for MEDLINE]

Key word: BRX-220 in PubMed: 1: Kalmar B, Greensmith L, Malcangio M, McMahon SB, Csermely P, Burnstock G.

      The effect of treatment with BRX-220, a co-inducer of heat shock proteins, on sensory 
      fibers of the rat following peripheral nerve injury.
      Exp Neurol. 2003 Dec;184(2):636-47.
      PMID: 14769355 [PubMed - indexed for MEDLINE]

2: Kalmar B, Burnstock G, Vrbova G, Urbanics R, Csermely P, Greensmith L. R Upregulation of heat shock proteins rescues motoneurones from axotomy-induced cell

       death in neonatal rats.
       Exp Neurol. 2002 Jul;176(1):87-97.
       PMID: 12093085 [PubMed - indexed for MEDLINE]

3: Kurthy M, Mogyorosi T, Nagy K, Kukorelli T, Jednakovits A, Talosi L, Biro K.

       Effect of BRX-220 against peripheral neuropathy and insulin resistance in diabetic 
       rat models.
       Ann N Y Acad Sci. 2002 Jun;967:482-9.
      PMID: 12079878 [PubMed - indexed for MEDLINE]

4: Sebokova E, Kurthy M, Mogyorosi T, Nagy K, Demcakova E, Ukropec J, Koranyi L, Klimes I.

	Comparison of the extrapancreatic action of BRX-220 and pioglitazone in
       the high-fat diet-induced insulin resistance.
       Ann N Y Acad Sci. 2002 Jun;967:424-30.
       PMID: 12079870 [PubMed - indexed for MEDLINE]
5: 	Rakonczay Z Jr, Ivanyi B, Varga I, Boros I, Jednakovits A, Nemeth I, Lonovics J, Takacs T. 	

Nontoxic heat shock protein coinducer BRX-220 protects against acute pancreatitis in rats.

       Free Radic Biol Med. 2002 Jun 15;32(12):1283-92.
       PMID: 12057766 [PubMed - indexed for MEDLINE]

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