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GB2189391A - Use of nucleoside analogue and uptake inhibitor therefor in anti-cancer compositions - Google Patents

Use of nucleoside analogue and uptake inhibitor therefor in anti-cancer compositions Download PDF

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Publication number
GB2189391A
GB2189391A GB08630714A GB8630714A GB2189391A GB 2189391 A GB2189391 A GB 2189391A GB 08630714 A GB08630714 A GB 08630714A GB 8630714 A GB8630714 A GB 8630714A GB 2189391 A GB2189391 A GB 2189391A
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Prior art keywords
nucleoside analogue
inhibitor
administration
nucleoside
analogue
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GB08630714A
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GB8630714D0 (en
GB2189391B (en
Inventor
Oswald Morton
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Boehringer Ingelheim Pharma GmbH and Co KG
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Dr Karl Thomae GmbH
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Publication of GB8630714D0 publication Critical patent/GB8630714D0/en
Publication of GB2189391A publication Critical patent/GB2189391A/en
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/06Pyrimidine radicals

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

There is disclosed a new use of a nucleoside analogue which is transferable across the blood brain barrier and/or of a nucleoside analogue cell uptake inhibitor which is substantially non-transferable across the blood brain barrier for the manufacture of a therapeutic agent for use in a method of treatment of the human or animal body to combat cancer cells within the central nervous system by the administration to said body of a said nucleoside analogue and a said inhibitor. Especially useful are cytosine arabinoside as nucleoside analogue, and dipyridamole as the uptake inhibitor.

Description

SPECIFICATION Use of Chemical Compounds The present invention relates to the treatment of cancerous cells in the central nervous system (CNS) and the use of nucleoside analogues and nucleoside analogue cell uptake inhibitors for the manufacture of therapeutic agents for use in such a treatment.
In cancer therapy, and in particular in the treatment of leukaemia, there is a severe danger of relapse following the initial treatment, e.g. by chemotherapy or surgery, of the affected tissues.
One of the factors likely to cause relapse is the migration of cancerous cells to apparently unaffected areas that may occur before or during the treatment of the tissue diagnosed as affected.
Migration of cancerous cells into the CNS is considered to be one particularly important cause of relapses occurring especially with acute leukaemia.
Therefore following the initial treatment of tissue diagnosed as cancerous, efforts may be made to sterilize the CNS, i.e. to destroy or inactivate cancerous cells therein.
This has been done particularly with acute leukaemia following initial chemotherapeutic treatment and to this end chemotherapeutic and/or radiotherapeutic methods have been used.
In the chemotherapeutic sterilization of the CNS, nucleoside analogues, such as cytosine arabinoside (Ara-C) have often been used. Such compounds are active against cancer tissue, especially leukaemia cells, lymphomas and brain tumours. In the case of Ara-C, while its mode of action is not yet fully understood, it is effective against cells in the Sphase and thoughtto betaken up bythe cells and incorporated into nucleic acid metabolism thereby preventing cell replication. However, while the nucleoside analogues are particularly effective against certain cancer cells, bone marrow cells may also be inhibited and destroyed and it is bone marrow suppression that is the main dose-limiting toxicity of intravenously or subcutaneously administered Ara-C.
Ara-C is not taken up effectively on oral administration and so in CNS sterilization the administration route will be either systemic (i.v. or s.c.) or intrathecal (i.t.).
Intrathecal administration can lead to relatively poor distribution of the nucleoside analogue in the CNS and can be painful and distressing, especially to young patients. However, since Ara-C is relatively poorly transported into the CNS, high systemic doses have been required to ensure that cytotoxic concentrations are achieved in the CNS and such doses are limited of course by the bone marrow toxicity of systemic Ara-C.
Thus it is an object of the present invention to provide a form of therapy by which effective CNS sterilization can be achieved using systemic doses of a nucleoside analogue without causing undue damage to bone marrow tissue.
The present invention is based on the concept of a combined therapy wherein as well as the cytotoxic nucleoside analogue there is administered to the patient an agent which inhibits cell uptake of nucleoside analogues.
According to one aspect of the present invention we therefore provide the use of a nucleoside analogue which is transferable across the blood brain barrier and/or of a nucleoside analogue cell uptake inhibitor which is substantially nontransferable across the blood brain barrier for the manufacture of a therapeutic agent for use in a method of treatment of the human or animal body to combat cancer cells within the central nervous system by the administration to said body of a said nucleoside analogue and a said inhibitor.
According to a further aspect of the present invention we provide a method of treatment of the human or animal body to combat cancer cells within the central nervous system by the administration to said body of a cytotoxic or cytostatic amount of a nucleoside analogue which is transferable across the blood brain barrier and of a cell uptake inhibiting amount of a nucleoside analogue cell uptake inhibitor which is substantially non-transferable across the blood brain barrier, said inhibitor, and preferably also said nuceloside analogue, not being administered directly into the central nervous system of said body.
In accordance with the invention, the nucleoside analogue is preferably administered intravenously or subcutaneously although the invention should be understood to encompass administration by oral and rectal or by other parenteral administration modes whereby an effective concentration of the nucleoside analogue in the CNS, and in the cerebrospinal fluid (CSF) in particular, may be achieved. Where the nucleoside analogue is Ara-C, however, administration will generally be subcutaneous or, preferably, intravenous.
For the inhibitor, the administration mode should be such as to provide a concentration in the blood or at the bone marrow which is effective to prevent undue damage of bone marrow cells by the nucleoside analogue. Depending on the nature of the inhibitor, oral, rectal, intravenous or subcutaneous administration may be contemplated, however oral and/or intravenous administration will generally be preferred.
The nucleoside analogue used according to the present invention is preferably a pyrimidine or purine analogue, such as those listed in Goodman and Gilman "The Pharmacological Basis of Therapeutics", 6th Edition, Chapter 55, pages 127S1287, however pyrimidine analogues and particularly Ara-C, a pyrimidine analogue, are especially preferred.
The inhibitor of cell uptake of the nucleoside analogue may be any physiologically tolerable substance having this effect. A particularly preferred compound is dipyridamole (which is available from Boehringer Ingelheim Ltd. of Bracknell under the registered Trade Mark Persantin).
Dipyridamole acts to inhibit cell uptake of nucleosides and nucleoside analogues, and of Ara-C in particular, apparently by hindering the transfer of the nucleoside or nucleoside analogue through the cell walls, the hindering effect being greater for ingress than egress.
In various in vitro studies (see for example King et a/. "Modulation of cytarabine uptake and toxicity by dipyridamole" Cancer Treatment Reports, Vol. 68 No. 2, February 1984, pages 361-366) it has been shown that co-administration of dipyridamole and Ara-C serves to protect bone marrow cells from the cytotoxic effects of Ara-C. In such studies however it has also been shown that dipyridamole also protects cancerous, e.g. leukaemia, cells from Ara-C.
However dipyridamole, unlike Ara-C and the other suitable nucleoside analogues, does not cross the blood-brain barrier and thus co-administration of dipyridamole and Ara-C on one side of the bloodbrain barrier will suppress the cytotoxic effects of Ara-C on that side of the barrier while allowing Ara Cto inhibit replication of cancerous cells on the other side of the barrier.
A further beneficial result flows from this coadministration ofthe nucleoside analogue and the nucleoside analogue cell uptake inhibitor in that, with its uptake on the blood side of the barrier suppressed, the rate of decrease of the nucleoside analogue's concentration in the blood is lowered and thus to transfer the same quantity through the blood-brain barrier into the CNS may require a smaller initial dose of the nucleoside analogue when administered with the inhibitor than when no inhibitor is administered.
In the combined CNS sterilization therapy of the present invention, the precise dosage of nucleoside analogue will of course depend i.a. upon the precise nucleoside analogue used, the administration route, the patient's size and the severity of the condition undertreatment. Nevertheless, where Ara-C is used the dosages will generally be about 4.3--12.9 mg/ m2/day (eg. as 10--30 mg/m2 (approximately 0.3--0.9 mg/kg bodyweight) given three times weekly) if administered intrathecally and 1-6 g/m2/ day (eg. as 0.5 to 3 g/m2 each 12 hours) if administered intravenously.The nucleoside analogue administration will generally be for period of up to 7, preferably 3 to 5 days; however, in many cases treatment may conveniently be terminated when sampling of the cerebro-spinal fluid (CSF) shows cancerous cells no longer two be present.
The CNS sterilization may form only one part of a cancer treatment, e.g. to prevent relapse in the treatment of acute leukaemia, and in that case the CNS sterilization will generally be effected following the chemotherapeutic or radiotherapeutic treatment of the primary cancer site(s). In the case of the chemotherapy of acute leukaemia, CNS sterilization according to the invention will generally be effected about 12 weeks after chemotherapy of the primary site has started.
The dosage of the inhibitor of nucleoside analogue cell uptake will again be dependent on various factors such as the precise nature of the inhibitor and the nucleoside analogue, the administration route, the patient's weight, and the condition and sensitivity of the patient's bone marrow. Generally, the half life of the inhihitor in the blood and the uptake from oral administration of the inhibitor and of the nucleoside analogue will differ, often extremely as is the case with dipyridamole and Ara-C, and thus it may be convenient to select a different administration mode for the two active agents in the combined therapy of the invention.
Where the inhibitor is dipyridamole, the initial administration may conveniently be by oral administration followed if desired by an occasional oral or occasional or continuous intravenous "topping up" during the period during which the nucleoside analogue is continuously administered.
In general, for dipyridamole, the dosage should be chosen to maintain a blood level of about 2 llg/ml, preferably 3--4 IlM/i, especially about 3.5 pM/I during the period during which the nucleoside analogue is present in the blood at active or high levels. For this purpose it may be sufficient to administer 100 to 200 mg/kg dipyridamole p.o.
initially, followed by iv administrations of 10 mg/2 ml dipyridamole bolus each 6 hours for 6 doses.
Alternatively, dipyridamole may for example be administered intravenously with an initial bolus e.g.
of 20--100 mg followed by an infusion e.g. of 30 to 150 mg, the nucleoside analogue being administered during the infusion stage.
The nucleoside analogue and the inhibitor may each be formulated for administration according ho the present invention with a pharmaceutical carrier or excipient. In this respect substances and additives conventionally used in galenic formulations may be used. Injection solutions containing one or both of the active ingredient(s), conveniently dissolved in water for injections, are particularly preferred.
The following Examples are provided to illustrate the invention in a non-limiting fashion: Example I Patient Details: Bodyweight: 70 kg Condition: Acute lymphoblastic leukaemia.
Treatment Details: (A) Primary Treatment 300 mg/day of Ara-C administered intravenously, as 300 mg over 30 minutes, for 5 days.
(B) CNS Sterilization Commence 12 weeks after start of the primary treatment.
Nucleoside analogue: Ara-C-administer intravenously 3--8 g over 24 hours each days for 5 days.
Inhibitor: dipyridamole-administer orally 100--200 mg 1-2 hours before initial nucleoside analogue dose. Thereafter administer 100 mg orally each 8 hours starting immediately after the initial nucleoside analogue dose and terminating after the final administration of the nucleoside analogue, or 10 mg/2 ml dipyridamole as an i.v. bolus each 6 hours for 6 days.
Formulation Details: Arn-C0 mg in 2 ml or 100 mg inS ml of water for injections (optionally containing 0.9% w/v benzyl alcohol).
Dipyridamole-tablets containing 25 and 100 mg dipyridamole for oral administration, and solutions containing 10 mg in 2 ml water for injections for intravenous administration.
Example II Patient Details: Female suffering relapse from acute lymphocytic leukaemia.
Treatment Details: CNS Sterilization (i) Nucleoside analogue: Ara-C-administer intravenously 0.5g/m2 over 1 hour.
(ii) Inhibitor: dipyridamol-initial i.v. bolus of 20 mg with a subsequent i.v. infusion of 30 mg over one hour.
Intravenous administration of nucleoside analogue and inhibitor are effected over the same one hour period.
Example Ill Patient Details: Male suffering from leukaemia.
Treatment Details (in Addition to Routine Leukaemia Therapy): CNS Sterilization (i) Inhibitor: dipyridamoleinitial i.v. bolus of 40 mg with a subsequent i.v. infusion of 30 mg over 30 minutes.
(ii) Nucleoside analogue: Ara-C-initial i.v. bolus of 330 mg/m2 with subsequent i.v. infusion of 666 mg/m2 over 30 minutes. The nucleoside analogue is administered during the i.v.
infusion of inhibitor.

Claims (7)

1. Use of a nucleoside analogue which is transferable across the blood brain barrier and/or of a nucleoside analogue cell uptake inhibitor which is substantially non-transferable across the blood brain barrier for the manufacture of a therapeutic agent for use in a method of treatment of the human or animal body to combat cancer cells within the central nervous system by the administration to said body of a said nucleoside analogue and a said inhibitor.
2. Use as claimed in claim 1 for the manufacture of a therapeutic agent comprising said nucleoside analogue in an intrathecally, intravenously or subcutaneously administrable form.
3. Use as claimed in either one of claims 1 and 2 wherein said nucleoside analogue is a pyrimidine or purine analogue.
4. Use as claimed in any one of claims 1 to 4 wherein said nucleoside analogue is cytosine arabinoside.
5. Use as claimed in claim 1 for the manufacture of a therapeutic agent comprising said inhibitor in an orally, intravenously, rectally or subcutaneously administrable form.
6. Use as claimed in any one of claims 1 to 5 wherein said inhibitor is dipyridamole.
7. Use as claimed in claim 1 substantially as herein defined with reference to the Examples.
GB8630714A 1986-04-16 1986-12-23 Use of chemical compounds Expired GB2189391B (en)

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GB868609290A GB8609290D0 (en) 1986-04-16 1986-04-16 Chemical compounds

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GB2189391A true GB2189391A (en) 1987-10-28
GB2189391B GB2189391B (en) 1989-11-29

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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0457671A3 (en) * 1990-05-14 1992-04-08 Fidia S.P.A. Dipyridamide for the treatment of diseases of the central or peripheral nervous system
WO2011085473A1 (en) * 2010-01-13 2011-07-21 Linda Penn Treating cancer with statins and compounds having dipyridamole activity

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0457671A3 (en) * 1990-05-14 1992-04-08 Fidia S.P.A. Dipyridamide for the treatment of diseases of the central or peripheral nervous system
WO2011085473A1 (en) * 2010-01-13 2011-07-21 Linda Penn Treating cancer with statins and compounds having dipyridamole activity

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Publication number Publication date
GB8630714D0 (en) 1987-02-04
GB8609290D0 (en) 1986-05-21
GB2189391B (en) 1989-11-29

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PCNP Patent ceased through non-payment of renewal fee

Effective date: 19991223