Approval processes for new drugs and novel medical technologies for rare diseases

Luminesce Alliance recently prepared a parliamentary response to The House of Representatives Standing Committee on Health, Aged Care and Sport inquiry into the approval processes for new drugs and novel medical technologies in Australia, with a particular focus on those for the treatment of rare diseases and conditions where there is high and unmet clinical need.


The publicly available paper presents six case studies of children and their families who have a rare disease and the challenges and barriers that currently exist in accessing new therapeutic and technological responses that are at hand.

What the case studies also validate, is there is no more exciting time for Australia to capitalise on the full value of our country’s health system and research in collaboration with the entire sector. The gains to be made in enhancing the research, development, trialling and implementation of novel therapies and medical technologies for the treatment of rare disease in the paediatric setting in particular, are enormous.


Advanced therapeutics – Transforming the way we care for children webinar Friday 27th Nov 2020 2:00pm AEDT

Luminesce Alliance partner, the Sydney Children’s Hospital Network, will be hosting a webinar on Advanced therapeutics – Transforming the way we care for children.
The speakers will be Professor Ian Alexander and Associate Professor Michelle Farrar.

They will be discussing gene therapy and Spinal Muscular Atrophy (SMA) and how an Australian-first SMA trial is leading to better outcomes for children with this devastating disease.

Further details and how you can register for the webinar can be found on the link below.



Sydney Children’s Hospitals Network and CAR T Cell immunotherapy for paediatric bone tumours

Luminesce Alliance partner, the Sydney Children’s Hospitals Network has published an online paper on the use of Chimeric Antigen Receptor (CAR)-T cell therapy for patients with B cell malignancies, (such as oestosacroma and Ewing Sarcoma) in Cancer Gene Therapy.

Vector and CAR T-cell production are currently being developed at Westmead prior to early phase clinical trials.


Opening the black box of childhood cancer

Opening the black box of childhood cancer – results from a national personalised medicine program with significant outcomes for kids with aggressive cancer.

Luminesce Alliance partners the Children’s Cancer Institute and the Sydney Children’s Hospital Network has published an online paper in Nature Medicine outlining the first major results from  the Zero Childhood Cancer Personalised Medicine Program.

 Key results from the paper are:

  • The paper shares the results from a study of the first 250 children to be enrolled on the ZERO national clinical trial.
  • The lead authors for the paper were ZERO researchers, Associate Professors Paul Ekert, Mark Cowley, and David Ziegler, with the support of a vast team of researchers and clinicians.
  • In the study, the molecular basis of a child’s cancer was able to be found in more than 90% of cases. And in 70% of cases, at least one new potential treatment option was able to be identified within a clinically relevant timeframe of 9 weeks.
  •  32% of the children went on to receive the recommended personalised treatment and then had their response to treatment tracked over time, of these, 70% had a positive response: their tumour either stopped growing (40% of cases) or shrank (another 30% of cases). In some cases, the tumour completely regressed.
  • These promising results prove that understanding cancer at the molecular level can change outcomes for children with the most aggressive cancers, who in many cases have no other treatment options available.