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Inside Clinuvel: effective drug development

Tuesday, July 31st, 2012

Take ten years, half a billion dollars and countless man hours from some of the most highly trained, intelligent individuals on the globe. You still stand a 90% chance of failure, some of which is totally out of your control. This is the apparent reality of modern drug development.

With the odds so stacked against it, it’s little wonder that the drug development sector is one requiring constant evolution in rethinking how to survive. In addition to the ‘regular’ risks of drug development, turmoil in global markets since 2007 has seen risk adverse investors shun drug development and biotechnology stocks for blue chip companies which are perceived as safer. The pressure to perform has increased for those companies who continue to work in the space.

In short, it’s forced even greater creativity to ensure survival and prosperity. (more…)

Living with HV

Wednesday, February 29th, 2012

In the final post from the Roosenboom family, and to help recognise Rare Diseases Day, Simone has penned her own piece on her experiences with Hydroa Vacciniforme. We are grateful to the Roosenboom family for being able to share their story.

Hi, all.

My name is Simone and I’m almost fifteen years old. I got ill when I was nearly six; exactly nine years prior to the day I wrote this. You can read all about that in the previous blogs written by my father.

The first years, my illness troubled me, but it got worse when I turned ten years old. The year 2007 turned out to be a horrific year for me. I got a bit older and more aware of myself and the way my surroundings reacted to my appearance. I looked quite scarred and felt that I was different. Quite a lot of people acted in a way that strengthened that feeling: they looked at me with horror. My parents and I got quite upset with that, even to the point that my mother told these people in anger that I was contagious so they would quickly get away from us. I thought it was funny but in the end it didn’t change anything. (more…)

Hang in there! Take back control! (Part 2)

Monday, February 27th, 2012

We recently invited Richard Roosenboom to share his experiences as a parent of a child with a rare disease. In the coming weeks we will publish part of the Roosenboom’s story in a four post series. In part one of this post Richard described the onset of Simone’s disorder and the road to the diagnosis Hydroa Vacciniforme (HV) some months later.

Our local hospital learned about Simone’s diagnosis with HV and invited us to discuss her condition. They felt that the diagnosis needed confirmation by tests. Yet, as HV itself cannot be confirmed by tests, that meant that Simone would have to undergo a series of examinations to exclude other diseases and disorders. They felt that the diagnosis of HV could be accepted only if all others had been excluded. We learned about some tests being quite painful and harmful to Simone’s skin and took control again: we refused. We decided to accept her having HV and not having her undergo such an ordeal, knowing that even if HV was confirmed, it would change nothing. (more…)

Hang in there! Take back control! (Part 1)

Tuesday, February 21st, 2012

We recently invited Richard Roosenboom to share his experiences as a parent of a child with a rare disease. In the coming weeks we will publish part of the Roosenboom’s story in a four post series. You can read the first post here: Too rare to diagnose: Hydroa Vacciniforme.

In 2003 our daughter Simone (then almost six years old) was diagnosed with Hydroa Vacciniforme (HV) by a leading Dutch dermatologist with over 35 years of experience. He listened carefully to our story, retreated for a moment to think and consult some books, and then came back and pointed out the page in a book where HV was described. He had never seen it in his life! After over three months Simone’s illness finally had a name and a history, and we felt like having a future again. (more…)

Too rare to diagnose: Hydroa Vacciniforme

Friday, February 17th, 2012

We recently invited Richard Roosenboom to share his experiences as a parent of a child with a rare disease. In the coming weeks we will publish part of the Roosenboom’s story in a four post series.

This year, 2012, sees calendars with February 29th: a rare day that is only seen once in every four years. Thus it is logical that this day is chosen as the worldwide Rare Diseases Day: a day where extra attention is sought for many rare diseases and disorders from which adults and children suffer every day.

Often the causes of rare diseases aren’t known. There’s often no treatment (yet). That’s why the world needs Rare Diseases Day: to show that these patients too are entitled to care and treatment, like any other. (more…)

Top five sun and skin myths (part 2)

Wednesday, February 15th, 2012

Myth 4: I need lots of sun exposure to create vitamin D

Vitamin D is a contentious topic in modern medicine and something we’ve blogged about regularly. It’s known to play a role in strengthening bones (with low levels known to contribute to rickets and osteomalacia) and has been linked to the prevention of various diseases, including certain cancers. We know that exposure to sunlight is an efficient way for the body to produce vitamin D, however the UV radiation in sunlight also causes sunburn, skin cancer, premature skin aging and other damage. (more…)

MAA: a moment in Clinuvel’s EPP story

Thursday, February 9th, 2012

Those who have taken an interest in Clinuvel will have learned with joy that, on Monday February 6th, the company announced its first official filing for SCENESSE® (afamelanotide) with the European Medicines Agency. It has taken our teams around six years to arrive at this point. Benchmarked against peer companies, it is a relatively swift development path for a first-in-class drug; we first publicly announced our erythropoietic protoporphyria (EPP) program in September 2006. It is an opportune moment to reflect briefly on how we reached this milestone and then discuss the steps that must be taken from here. (more…)

Low sunscreen use, high melanoma rates: a breakdown of the sunsmart message?

Tuesday, January 24th, 2012

Image from Flickr.com by MuffetIt’s estimated that more than 9,000 Americans will die from melanoma this year and 76,000 new cases of the disease will be diagnosed. Melanoma, an aggressive skin cancer which can spread (metastasize) quickly to other parts of the body, is also the second most common form of cancer in young Americans (those aged 15-29).

Overexposure of skin to ultraviolet (UV) radiation significantly increases an individual’s risk of melanoma, particularly at a young age; just one severe sunburn in childhood can double the lifetime risk of melanoma. Sun protective measures such as clothing and sunscreen are seen as key to reducing melanoma risk. (more…)

EPP results and clinical relevance

Friday, November 4th, 2011

A protoporphyrin IX molecule

Over recent months I have written several times of the need for Clinuvel to prove clinical relevance in our trials with the use of SCENESSE® (afamelanotide) in erythropoietic protoporphyria (EPP). In orphan populations the need to demonstrate how a novel drug assists in their daily activities and improve their lives is at the forefront of the regulators’ minds. And so it should be, after all the objective of the pharmaceutical industry is to develop drugs which address either disease or symptoms adequately and safely. The results the company released yesterday from our Phase II US study of the drug in EPP (CUV030) have given us important data towards demonstrating clinically relevant improvement of patients’ lives. (more…)

Ultraviolet A more cancer-causing than once thought

Tuesday, October 18th, 2011

While the evidence linking sunbeds to the development of skin cancer continues to mount, recent research provides new insight into the underlying mechanisms.

Radiation from both the sun and tanning beds is made up of various wavelengths, including visible light and several types of ultraviolet (UV) radiation. Ultraviolet B (UVB) rays, those which cause sunburn, have long been heralded the culprit which initiates skin cancer. It does this by damaging the genetic information (DNA) within skin cells, forming lesions known as cyclobutane pyrimidine dimers (CPDs) and pyrimidine (6-4) pyrimidone photoproducts (6-4PPs). These lesions create mutations in the DNA which can lead to the development of skin cancer (you can read about this process here). (more…)

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