Thursday, August 22, 2013

ONE21: A Community United Will Achieve Great Things

The latest from the ONE21 initiative - please share on blogs, on FB and by word of mouth - the biobank is close, within our grasp...

ONE21. A community united will achieve great things.

ONE21 is a new community-wide initiative of parents, researchers, and advocates for Down syndrome coming together for a common goal:  to give DS research the support -- and voice -- that it so urgently needs.

To help advance DS research to the next level, ONE21 seeks to fund the development of a dedicated and centralized DS biobank that will be open to all DS researchers, regardless of institutional affiliation.

What is a biobank? It's a large medical-grade refrigeration unit used to store high-quality samples of blood, tissue, DNA, and other specimens researchers need to build a more thorough understanding of DS.

Biobanks have been in use since the late 1990's, and are already commonplace in other research areas, including autism, cystic fibrosis, Duchenne muscular dystrophy, and psoriasis.

A biobank will go a long way toward advancing the science of DS faster, making therapies and best practices available sooner, so our loved ones with DS can live longer, healthier, and more vibrant lives.

What You Can Do Right Now
The ONE21 campaign to fund a dedicated DS biobank will roll out in the coming weeks. Until then, here are two easy steps you can take today to help:

  1. Spread Awareness: the biggest barrier to achieving more for our children with DS through research is the lack of knowledge. Talk with other parents and advocates about the need for  a dedicated DS biobank - a critical tool that will advance research in speech and communications, congenital heart defects, childhood leukemia, early cognitive decline, thyroid issues, and others. Without a biobank, progress for our loved ones with DS will remain slow.
  2. Invite others to join ONE21: The ONE21 campaign is about what can be achieved today a community working together for the greater good. Ask your friends, families and others to learn more -- and lend their voices to the cause -- by signing up at
Dr. Alberto Costa is largely credited for publishing the first study to show a drug could normalize the growth and survival of new brain cells essential for memory and spatial navigation in a mouse model for Down syndrome (DS). Since his daughter was born with DS 18 years ago, Dr. Costa has dedicated his research to the study of DS. He is also credited for completing one of the first randomized human clinical trials of a DS cognition drug called memantine, which is already FDA-approved for the treatment of Alzheimer’s disease.

Dr. Costa was recently recruited by Case Western Reserve University to continue his groundbreaking work and, among many important projects he is involved with, he is collaborating with a leading pharmaceutical company to see if a drug in development, named RG1662, can reverse the effects of the GABA neurotransmitter, which inhibits brain activity. This seems to be one of the mechanisms responsible for the memory and learning problems in people with DS.

We asked Dr. Costa his views on the state of DS research today and what is needed to advance further, faster.


Vicki said...

Wow, Rebecca! You scored a video interview with Dr. Costa, that's great. So this does make it clearer in terms of what ONE21 is trying to do. I am curious about the current state of the data bank: how many samples do they have, from what part of the country, ages of people in there and who is using this biobank currently.

I think families will need lots of reassurance that their samples would be safe and kept private and used for good purposes (for instance, how can we be sure that they won't be used by companies seeking to create even more prenatal tests for Ds?). I am curious what there plans are for how to accomplish this and how to recruit the actual samples. What channels are they going through -- pediatricians, Ds groups, geneticists, what? -- to encourage people to give the samples??

I don't expect you to answer these questions! I am just saying these are the thoughts that are sparked when I contemplate this. Thank you for the work you are doing here!!

Becca said...

Just to clarify, this is just a copy/paste from the ONE21 e-mail I received. Not my interview, not my words. However, stay tuned, there will be far more information coming that will answer the other questions Victoria has!