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The Children’s Tumor Foundation issued the following press release on November 5, 2020
The Children’s Tumor Foundation (CTF), the foremost funder of neurofibromatosis (NF) research, and PLOS ONE, a leading peer-reviewed scientific journal, are pleased to release the first output of the Drug Discovery Initiative Registered Reports (DDIRR) Award, which CTF and PLOS ONE first announced in 2017. The Registered Reports model, first piloted by CTF and PLOS ONE, ensures that all valid research – both successes and failures in the lab and clinics – are made public.
The publication, titled Evaluating modified diets and dietary supplement therapies for reducing muscle lipid accumulation and improving muscle function in neurofibromatosis type 1 (NF1), by Dr. Aaron Schindeler, PhD, associate professor at the University of Sydney, reports findings on L-Carnitine as an effective dietary supplement for reducing muscle lipid accumulation and potentially addresses muscle weakness and fatigue often observed in NF1 patients. The result of the study led to the opening of a clinical trial for NF1 patients.
The Funder-Publisher Partnership around the DDIRR program allows researchers to preregister their study, obtain an in-principle acceptance (IPA) to publication in the journal PLOS ONE, and obtain funding from the Children’s Tumor Foundation to conduct their proposed experiments. Both CTF and PLOS ONE maintain a separate and independent review process to align proposed research with both the funder mission as well as to safeguard soundness, accuracy, and scientific rigor. Through this model, before any experiments start, researchers have the opportunity to address reviewer remarks and submit revisions of their protocol until it is ready for publication.
The innovation of this new funding model creates multiple benefits for all participants. Researchers are able to make adjustments to their protocols before they start any lab experiment, funders are ensured that the quality of the proposed research is high quality, and the publisher secures a manuscript that has robust reproducibility in line with the mission and ethos of the journal. In addition, the published manuscript links to the original registered protocol deposited on the Open Science Framework portal for full transparency and traceability.
“We are very proud to see the first manuscript of this new award program out, the transparency and the quantity of the protocol adds to the soundness of the research, independent from the nature of the outcome,” said Salvatore La Rosa, PhD, Chief Scientific Officer, Children’s Tumor Foundation. “Positive or negative results are equally valuable when a robust protocol is applied, and this model allows for either to emerge.”
“I am delighted that our partnership with the Children’s Tumor Foundation has resulted in the first publication of a preregistered study,” adds Joerg Heber, PLOS Editorial Director and Editor-in-Chief of PLOS ONE. “This scheme ensures the robustness of the research conducted and is a win for openness and transparency in research.”
Click here to read the published study.
Click here for information about the clinical trial.
To learn more about the work of the Children’s Tumor Foundation, visit ctf.org.