NZORD – the New Zealand Organisation for Rare Disorders
NZORD Newsletter 2012 #6 — 28 November 2012
In this issue:
1 – Putting patient and family interests into newborn screening criteria.
2 – Common themes as groups respond to Ministry consultation on payments to family carers.
3 – Two significant clinical trials with New Zealand connections.
4 – Plain packaging submission to Ministry of Health tobacco control team.
5 – Recommended reading: The Immortal Life of Henrietta Lacks, by Rebecca Skloot.
***************************************************************************************************
1 – Putting patient and family interests into newborn screening criteria.
Over a decade of NZORD’s advocacy for patient and family interests in health policy has taught us that talking the language of officials and professionals is an important step in having our messages taken seriously. So NZORD took the lead in publishing how family interests and ethics must influence screening policy. See Screening criteria: the need to deal with new developments and ethical issues in newborn metabolic screening. We are proud to have the valued support of the Save Babies Through Screening Foundations in the USA and UK, and the Genetic Alliance in the USA, in the preparation of this article, along with valued help from two New Zealand academics with editing and technical aspects.
The article is published online in the Journal of Community Genetics, October 2012. We propose that decision criteria for metabolic screening in the newborn period should be adapted to specifically include patient and family interests, community values, patients’ rights, duties of government and healthcare providers, and ethical arguments for action in the face of uncertainty. Here is an open access PDF version of the article.
2 – Common themes as groups respond to Ministry consultation on payments to family carers.
The Ministry of Health’s consultation on payments to family carers has now closed and decisions will be made over the next few months. Wide ranging discussions took place among support groups during the consultation period and there was widespread concern at themes contained in the Ministry’s document.
NZORD is concerned that the Ministry’s defeat on this issue at the Human Rights Review Tribunal, and in two major court cases, has negatively influenced the policy direction they are signalling, and even suggests an attempt to relitigate the case through the policy setting process. This is very disappointing. Family carers deserve a more respectful process that is based on sound principles. Read more about a better approach to this policy issue in the submissions made by the Carers Alliance and also by NZORD.
3 – Two significant clinical trials with New Zealand connections.
Possibly missed by many in the constant stream of news about research activities under way, is a planned new clinical trial by NZ-based Neuren Pharmaceuticals who specialise in drugs for brain injury and neurodegeneration. They have successfully completed a phase 1 safety trial and submitted for approval of their candidate drug NNZ-2566 for a phase 2 trial for Rett syndrome, a very rare neurodegenerative condition. It is exciting to see such projects emerging from New Zealand universities, and also great to see attention being paid to rare conditions. Read more in the Neuren press release.
Also this month, Living Cell Technologies received approval for a clinical trial of its porcine cell encapsulation technology for the treatment of Parkinson’s disease. This follows successful earlier trails of the same technology for the treatment of unstable diabetes. Read more about the ongoing diabetes trial. This is another project where the innovation and basic research originated in New Zealand.
4 – Plain packaging submission to Ministry of Health tobacco control team.
NZORD supports the introduction of plain tobacco packaging and the other recommendations set out in the proposal under consultation by the Ministry of Health. We support this proposal because the health outcomes of tobacco use, exert an indirect but significant impact on the rare disease population. Smoking is a significant risk factor for a range of diseases that are high on health priority lists and therefore take a significant slice of the health budget. As a result, patients with rare diseases are pushed further down the priority list. Here is our submission in Word and PDF.
5 – Recommended reading: The Immortal Life of Henrietta Lacks, by Rebecca Skloot.
Her name was Henrietta Lacks, but scientists know her as HeLa. She was a poor Southern tobacco farmer who worked the same land as her slave ancestors, yet her cells—taken without her knowledge—became one of the most important tools in medicine. HeLa cells were vital for developing the polio vaccine; uncovered secrets of cancer, viruses, and the atom bomb’s effects; helped lead to important advances like in vitro fertilization, cloning, and gene mapping; and have been bought and sold by the billions.
Yet Henrietta Lacks remains virtually unknown, buried in an unmarked grave. Henrietta’s family did not learn of her “immortality” until more than twenty years after her death, when scientists investigating HeLa began using her husband and children in research without informed consent. And though the cells had launched a multimillion-dollar industry that sells human biological materials, her family never saw any of the profits. As Rebecca Skloot so brilliantly shows, the story of the Lacks family—past and present—is inextricably connected to the dark history of experimentation on African Americans, the birth of bioethics, and the legal battles over whether we control the stuff we are made of.
Intimate in feeling, astonishing in scope, and impossible to put down, The Immortal Life of Henrietta Lacks captures the beauty and drama of scientific discovery, as well as its human consequences. Read more about Henrietta in Wikipedia. The book is available at Amazon.
Contact:
John Forman
Executive Director, NZORD
New Zealand Organisation for Rare Disorders
PO Box 38-538, Wellington Mail Centre, 5045
228 Tinakori Rd, Thorndon, Wellington, 6011
New Zealand
Ph +64 4 471 2226
Mob +64 27 240 3377
Email: exec.director@nzord.org.nz
Website: www.nzord.org.nz
