Drug Development

Although the established therapies have considerably improved the lives of patients with this disease, their application is limited by the number of undesired side-effects. In particular, the main side-effect is usually the suppression of bone marrow stem cells. Many cytostatic agents therefore continue to damage the bodily organs which have already been badly damaged by the disease itself. In addition to this, the suppression of the stem cells resulting from the therapy negatively affects the ability of these cells to regenerate themselves. Against this background, it is evident that there is an urgent need for new drugs to treat CLL.

CD-160130 is such a drug candidate. After thorough ex vivo trials on patient blood and some initial toxicological and pharmacological tests, it is now planned to go through the formal preclinical phase. Both in the laboratories of the Medicines Company in Leipzig and at the Duke University (Medical Center) in Durham, North Carolina, CD-160130 has had a better effect on killing (apoptosis) leukaemia cells than other tested drugs.
Despite the fact that this substance is still in a very early phase of development, some definite advantages over competitive drugs have already become apparent:

• CD-160130 induces the apoptosis in cells obtained from CLL patients with high selectivity. This is a clear advantage over cytostatic agents, which have a nonspecific effect against other rapidly growing cells. This is particularly relevant since CLL is characterized by a disturbed apoptosis rather than excessive cell proliferation.
• According to the literature to date, it is unlikely that treatment with CD-160130 will affect the stem cells of the bone marrow.
• CD-160130 has a synergistic effect with an agent commonly used in CLL, thus opening up the possibility of a combined therapy having a significantly improved effectiveness and/or reduced side effects.
• First results have shown that CD-160130 may be particularly suited for patients having an unfavorable prognosis due to a chromosome mutation on the leukaemia cell (e.g. 11q23.3).
• CD-160130 can be administered orally, giving it a clear advantage over substances which have to be infused - increasing the patient's quality of life.

Even at this early stage of development, CD-160130 has been given Orphan Drug Designation for the treatment of chronic lymphocytic leukaemia by the European Commission being registered in the Community Register for Orphan Medical Products for Human Use under number EU/3/07/487.