At a glance: Sickle-cell trial drug MX-1520

What are sickle-cell disorders? They are a group of rare genetic diseases that affect red blood cells, causing them to become sickle-shaped and obstruct blood vessels, creating extreme pain, permanent organ damage or even death.

It is most common in people of African and Caribbean descent and around 200 to 300 babies a year are born with sickle-cell disorders in the UK. PR efforts have recently centred on screening.

Tell us about the new drug

US-based biotech firm Medinox has carried out pre-clinical studies on rats of a drug, MX-1520, that uses a type of vanilla, called vanillin. Once it enters the bloodstream, trials have so far found a reduction in ‘sickling’. Taking vanillin orally would not be effective because it is broken down in the intestine.

Isn’t Media Strategy trying to boost awareness of sickle-cell disorders?

Yes, that’s right. The agency won the account last year (PRWeek, 5 March 2004) to promote NHS screening programmes for sickle cell and a related condition, thalassaemia. It has targeted midwives, GPs and nurses, in addition to media relations work with Afro-Caribbean ethnic media in an attempt to help community leaders spread the message among social groups at the highest risk.

Are there other treatments available?

Relatively few. Bristol-Myers Squibb’s Droxia (hydroxyurea) is not licensed for use in this country. Bone marrow transplant – a high-risk and difficult operation – is seen as the only effective treatment at present. So a drug such as MX-1520, if it proves effective in humans, could be a step forward. But as yet, the Sickle Cell Society and the NHS say they have no information on the drug, which reinforces the fact that its use is a long way off. Control of sickle-cell pain is currently achieved through analgesics ranging from paracetamol to codeine phosphate and anti-inflammatories. For more serious cases, diamorphine – heroin – is used.

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