Past treatments

Children ongoing Acute Lymphoblastic Leukæmia (ALL) best illustrate the need officially mentioned by the authorities (in EMEA’s priority list “For Studies into Off-Patent Pædiatric Medicinal Products”). For all this time, they have used an inadequate formulation of this substance :

• For 6-Mercaptopurine, (i.e. 50mg oral tablets mostly known as Puri-Nethol) children need a dose largely inferior to 50mg every day. One of the consequences is that their mothers or the hospital’s pharmacies have to grind the tablet and dose half, sometimes even a third of it, and then reformulate it.

• For Methotrexate, (i.e. 2,5 mg oral tablets) children need a dose largely superior to 10mg every week, sometimes up to 35 mg. One of the consequences is that their mothers have every week a “Metho day”: the intake is spread throughout the day. In other situations, the hospital’s pharmacies have to grind the tablets and dose the prescribed amount and then reformulate it.

The development of an adapted oral liquid formulation is intended to increase drug efficacy because of its ideal dosage adaptability (Breitkreutz et al. 2007). It will also reduce manipulation risks related to reformulation and dosage adaptation so as to finally increase compliance for heavy treatments with such cytotoxicity.

Neither this extemporaneous preparation done under uncontrolled conditions nor the active compound itself (6MP) have been approved by the authorities for use in ALL children. This compliance issue is essentially a matter of pharmaceutical and industrial development. Only For Children Pharmaceuticals is reformulating 6MP for the maintenance treatment of Acute Lymphoblastic Leukaemia.