Treatment | Clinuvel Pharmaceuticals news and discussion blog

Susceptibility genes for nonsegmental vitiligo

Thursday, October 13th, 2011

Nonsegmental vitiligo is a common pigmentary disorder where the skin gradually loses its colour (melanin) in patches. It is unknown precisely what causes the condition, though it is generally thought to be a result of autoimmune damage to the pigment-producing cells, called melanocytes. Vitiligo is a complex, multifactorial disease, meaning that it occurs as a result of interactions between ones genes and their environment. The fact that vitiligo often presents in close relatives (10-30% of vitiligo patients report a family history of the disorder) indicates that, to some extent, it is a heritable condition. In other words, in some cases a person’s genetic make-up can predispose them to vitiligo. (more…)

Clinical relevance – the value of patient experiences

Friday, September 30th, 2011

Since 2006 Clinuvel has trialed SCENESSE® in a truly unique group of individuals: patients living with erythropoietic protoporphyria (EPP), a rare genetic blood disorder which causes an absolute intolerance to light.

EPP prevents patients from leading ‘normal’ lives, especially outdoors. It is one of the few diseases that manifest clinically with initially invisible symptoms which cause severe dermal pain for several days. This not only presents a challenge for diagnosis and treatment, but also for generating meaningful clinical trial results – those which are measurable numerically and are used by regulatory authorities to evaluate the efficacy of a drug in a patient population. Here, real life patient experiences during a trial can play an important role in providing clinical relevance and analysing hard data. (more…)

The importance of clinical relevance

Tuesday, August 2nd, 2011

Development of novel drugs is truly like no other business: one attempts to address questions that may have never been previously posed – let alone answered – in the pursuit of improving the lives and quality of life of patients. As I eluded to in my recent letter to shareholders, the team is now well into the analysis of results from our erythropoietic protoporphyria (EPP) program; two studies from the US and Europe. This is a complex and time consuming task that requires one to collate and make sense of thousands of data points to answer a seemingly straight forward question: does this trial show that the drug is safe and effective?

Obtaining an answer needs to be understood from the concept of clinical relevance. Put simply, results don’t just need to show that a treatment or intervention has an effect on a disease. Rather, they need to indicate that that effect is relevant to the current clinical understanding, treatment and care for the disease or indication. They need to show that the drug’s effect is having a positive, meaningful impact upon a patient’s prognosis and care. This is a crucial point to consider in the development of protocols and in the careful analysis of results, as it is how regulators will review the results. (more…)

Afamelanotide: an analogue of α-MSH designed to assist in vitiligo repigmentation

Monday, July 25th, 2011

Vitiligo is a common skin depigmentation disorder in which the melanocytes, the skin cells which produce melanin pigment, are progressively lost. Despite this, melanocyte stem cells, or immature melanocytes, are preserved in a special region within hair follicles called the bulge, or niche. Often these cells are able to be stimulated with narrowband ultraviolet B (NB-UVB) phototherapy to mature into fully functional melanocytes which migrate to the epidermis to replace the lost or damaged cells. Upon further exposure to UVB radiation, skin cells (keratinocytes) manufacture a hormone called alpha-melanocyte-stimulating hormone, or α-MSH. Alpha-MSH binds to receptors on the newly formed melanocytes and activates melanin production to repigment areas of skin affected by vitiligo. (more…)

A new treatment regime

Friday, July 15th, 2011

This is the second of a two part series on vitiligo treatment from guest blogger Liz Tyler, to read the first part, click here. Liz runs @VitiligoProject and the Vitiligo Project blog, an initiative she founded to help others, like her, who are living with vitiligo.

I’ve had vitiligo since childhood and, like many others who have the disorder, I find it a challenge to live with. Whether you’re able to accept your vitiligo or not, it’s needless to say it takes a lot of getting used to. For me, I’ve never been completely comfortable with my skin and with a career in marketing and PR, where image is really important, I’ve always been willing to try new treatments and look for therapies that could help cure it. (more…)

Diagnosis and early treatment of vitiligo

Monday, July 11th, 2011

We’re delighted to host guest blogger Liz Tyler. Liz runs @VitiligoProject and the Vitiligo Project blog, an initiative she founded to help others, like her, who are living with vitiligo. This is the first of a two part series on Liz’s personal treatment experience.

I’ve had vitiligo for thirteen years and like many, my journey of living with vitiligo hasn’t been an easy one. In this day and age when we’re all made to think we need to look like Kate Moss – it’s needless to say having patchy skin is a setback. (more…)

Follicular repigmentation in vitiligo – narrowband UVB phototherapy

Wednesday, July 6th, 2011

Figures 1 & 2. A vitiligo patient before and after NB-UVB treatment. Images courtesy of Pearl E Grimes, MD.

Vitiligo is a common skin depigmentation disorder in which the melanocytes, the skin cells which produce melanin pigment, are progressively lost. Clinically, when vitiliginous lesions (the ‘patches’ of skin which have lost their pigment) are repeatedly exposed to an intensive dose of narrowband UVB radiation (308 or 311-313nm), it is common to see small spots, sometimes described as ‘freckles’ or ‘islands’, of repigmentation forming within the lesion. This occurs because the melanocytes producing this melanin have developed from stem cells reserved in the bulge region of the hair follicle, known as the niche. Their development is stimulated by NB-UVB and they migrate to the skin directly surrounding the hair follicle from which they were derived. As the melanocytes continue to travel through the skin and produce melanin, these ‘islands’ begin to spread and merge, eventually creating broader, but seldom perfect, repigmentation in the treated area (see Figures 1 & 2). (more…)

Porphyrias: a disease grouping by cause, not symptoms

Monday, April 18th, 2011

Held biennially, the Porphyrins & Porphyrias conference (P&P) is the world’s largest gathering on the porphyrias – a group of metabolic disorders causing biochemical disruptions in the pathway of the body which synthesizes haem (heme).

As a result of each of these disruptions, the body presents with unique symptoms ranging from skin symptoms and phototoxicity – as those seen in erythropoietic protoporphyria and congenital erythropoietic porphyria – through to acute attacks of abdominal pain, seen most commonly in acute intermittent porphyria. In short, no two porphyrias are clinically identical yet they are discussed as a single group of disorders with a similar cause. As a matter of fact, there are eight variations of porphyrias, each with a specific clinical manifestation of disease. (more…)

‘Measuring’ vitiligo: the challenges of clinical and treatment evaluation

Monday, April 4th, 2011

Since our announcement last year that Clinuvel would commence a new program for SCENESSE® (afamelanotide) in nonsegmental vitiligo, the company has received vast interest in the application of the drug in this disease. Of the enquiries that best captured the essence of this program, one stood out: a US based analyst asked how the company intended to objectively measure the response to treatment, the repigmentation of vitiliginous lesions, in its trial. (more…)

‘The freak out will happen’ – Lee Thomas’ diagnosis of vitiligo

Tuesday, March 22nd, 2011

As part of our ongoing guest blogger series, we’ve invited Lee Thomas to share his experiences on the Clinuvel blog. Lee is an award winning US journalist who has spoken widely about his experiences with vitiligo, including in a documentary entitled ‘Turning White’. You can learn more about Lee at http://www.leethomas.com/

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“What the…?” “Why me…?”

“Where is that trucking school info…?”

“My career is…”

I couldn’t even finish most sentences. (more…)