Projects

Stepchild drugs and therapies that were selected by Cinderella Therapeutics for further investigation:

 

Progress reports:

– Tapering.   Tapering medication for paroxetine shortly available. Gradual dose reduction after long-term use of drugs such as antidepressants, sleeping pills and painkillers is something that both patients and doctors would like but that is not possible because the drug is currently not available in gradually decreasing amounts. Cinderella is developing dose-reduction blister packs. Read the full report.

 

Final reports:

– Ataluren: an experimental drug for Duchenne muscular dystrophy. After a clinical study with this drug was halted, the manufacturer refused to make it available to patients who had benefited from it.

– Baclofen, a registered drug for muscle cramps. It is also a promising drug for the treatment of severe alcohol addiction, but the mainstream medical community in this country has shown no interest. Read the full report.

– Galactosemia: a hereditary disease with ‘stepchild’ status in which the body is unable to break down galactose. It is treated with a modified diet that restricts the intake of galactose. A more effective mode of treatment is needed – enzyme restitution appears to be a promising option.

– Mexiletine: an experimental drug for the treatment of the very rare hereditary muscle disease non-dystrophic myotonia. This drug was no longer available in the Netherlands and the health insurance companies were refusing to reimburse its costs.

– Anti-GD2 for the treatment of neuroblastoma. This antibody significantly improves survival in children with stage 4 neuroblastoma (very poor prognosis). It is not available in this country. Treatment of Dutch pediatric patients involves spending 6 months in the US at great cost.

– Radio-immunotherapy for cancer with anti-Tenascin C. This radioactively labeled antibody had promising results both in animal experiments and in patients with brain tumors. Its development stagnated because the indispensable participation of nuclear physicians could not be realized.

 

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– Foscan: drug used for photodynamic therapy of head and neck cancers that are untreatable with surgery or radiotherapy. This treatment is mainly needed in countries where radiotherapy is not widely available and where these types of cancer are commonly seen, such as in Indonesia. Its development is being hampered by Foscan’s exorbitant costs.
– Ibogaine: a hallucinogenic substance from plants with proven potential for treating severe addiction. Ibogaine is widely used in alternative and complementary medicine but has dangerous side effects. Researchers in the Netherlands who want to further investigate this drug cannot get hold of it.
– Medical devices. Each year students at Delft University of Technology develop dozens of promising new medical therapeutic applications, together with doctors, of which only a small percentage ever reaches patients.
– Tetrahydrocannabinolic acid (THCA): a natural substance derived from the cannabis plant that has anti-inflammatory properties but without the undesirable hallucinatory properties of other substances in cannabis. Cinderella has been asked to help develop therapeutic applications of THCA.
– Low-dose naltrexone: patient groups propagate its use as complementary therapy for multiple sclerosis, Crohn’s disease and other auto-immune diseases, as well as for HIV and cancer. Naltrexone can only be obtained with a prescription and low-dose tablets are not available. In mainstream medicine the drug is used in high doses as an addiction treatment medication and to treat intolerable itching.

 

Projects

Cinderella is interested in compounds showing promise in preclinical research and phase-1 studies.
The efficacy and therapeutic ratio of these drugs will be demonstrated or disproved in a short period of time and in a small number of patients.

 

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