Nitisinone has been found in early clinical trials to reduce homogentisic acid (HGA) levels by 95%, resulting in patients reporting significantly reduced joint and back pain. (2) Researchers believe that if nitisinone is administered early enough it could prevent the toxic acid from building up, (3) allowing sufferers to lead a normal, pain-free life.The University of Liverpool and the Royal Liverpool University Hospital have conducted research, assessing how alkaptonuric mice responded to the potential treatment nitisinone. Mice with the same gene abnormality as AKU patients were studied. Just like AKU patients, their urine goes black, and they have high levels of HGA in their blood and urine.
Researchers were able to show that the drug nitisinone, which is about to be studied internationally as part of a clinical trial, works really well in these mice. Nitisinone reduced the amount of HGA in the blood and the number of pigmented cells in their knee joints. Research showed that nitisinone is effective in preventing the onset of the disease if it is used at the earliest signs of the condition, which could appear in the younger years of a patient’s life. They found that if the drug is administered throughout a patient’s lifespan it has the potential to completely prevent joint disease. You can read the official research paper online.
Clinical trials, lasting three years, were also conducted by the National Institute of Health (NIH) into the use of nitisinone as a treatment for AKU. Evidence showed that nitisinone reduced homogentisic acid (HGA) levels by up to 95%. Read the official research paper for free on the NIH website.
For more information about nitisinone and the DevelopAKUre trial, please contact the DevelopAKUre team. Your doctor may also be able to give you more information and background about nitisinone.
DevelopAKUre is a series of major international clinical trials, run by a consortium of 12 European partners. It aims to study a potential new drug, called nitisinone, and assess its potential effectiveness in treating the rare disease, alkaptonuria (AKU).
DevelopAKUre is co-funded by a grant from the European Commission. This website is run by a UK patient group, the AKU Society. Learn more about AKU on the AKU Society's What is AKU page.
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