The MDS Clinical Research Consortium | Aplastic Anemia and MDS International Foundation

The MDS Clinical Research Consortium

Original Publication Date: 
Wednesday, October 17, 2018
Article Source: 
AAMDSIF Web Content

The MDS Clinical Research Consortium (MDS- CRC) is an unprecedented, six-institution group designed to undertake unique studies and trials to advance treatments and improve outcomes for patients with myelodysplastic syndromes (MDS). Member institutions are six academic medical centers serving a high volume of MDS patients:

  • Cleveland Clinic Taussig Cancer Institute
  • Dana-Farber Cancer Institute
  • H. Lee Moffitt Cancer Center and Research Institute
  •  MD Anderson Cancer Center
  • Weill Medical College of Cornell University
  • Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

The consortium is co-chaired by Amy DeZern, MD, MHS at the Sidney Kimmel Comprehensive Cancer Center/Johns Hopkins School of Medicine, who provides oversight and direction for all scientific matters. Here, Dr. DeZern speaks about the MDS-CRC and effect it has had on specific research that might not have been published as quickly if it not been conducted by the CRC.

  • What is it about the concept and operation of the MDS-CRC that makes it a unique enhancement to traditional MDS research?

This is the first endeavor of its kind in the United States – it’s a wonderful, collaborative effort between six large MDS programs to run trials together in a more efficient way and hopefully bring new drugs to patients sooner.

Are there any studies that would not have been possible without the wider scope that MDS-CRC participation allows?

There are two good examples of this. One is a research study on CMML, a rare MDS/MPD overlap disease. Because it’s an unusual patient population given the disease rarity, having the consortium, in addition to Moffitt as the lead site, to more quickly accrue enough patients for this study was very helpful. A second example is a randomized trial, headed by MD Anderson, of two approved MDS drugs in decreased dose or schedule, azacitidine (Vidaza®) and decitabine (Dacogen®) which are already used in practice at higher doses. Outside the consortium environment this study would not have been possible through more traditional avenues. This trial is going well with good accrual and I think we’ll have some great results for the field in the near term.

One other thing to keep in mind is clinical trials have a lot of regulatory aspects –they are paperwork intensive, and not an altogether efficient process, besides all the aspects of patient care.  So, having several centers working in coordination can be helpful.

  • Are there any CRC studies you have a particular interest in?

Yes, currently there is a study specifically targeting a high-risk form of MDS—those with TP53 mutations which carries a very poor prognosis. There’s a currently Phase I/II trial, led by Moffit Cancer Center, that the CRC sites are participating in that is accruing well. It’s an exciting time in MDS right now and we hope to make progress with a new drug for this high-risk MDS subtype.

  • Do the participating centers have different roles in the CRC or are they all the same kind or level of participation? Does data sharing play a role?

It’s generally the same participation by all centers, though the patient volumes vary some by region – but the experience of planning and running the trials is pretty much the same. Data sharing has been useful to have a larger aggregate group to examine results and statistics from. There have been some successful abstracts that benefitted from having this larger pool of rare disease data.

  • The CRC is an example of a partnership that combines resources to increase the pace and scope of research. Is it working out has you had hoped and is there any ‘next steps’ for the CRC?

Overall it is working very well, and we have been very productive as group since the inception of the CRC. We are currently exploring all ways possible to maintain the funding to continue this endeavor for years to come.