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Distinguished member
Jun 28, 2015
Long Beach
Is anyone else registered and participating in the ALS Therapy Development Institute Precision Medicine Program?

I ask because there are open participant spots (280 filled, 20 open).

I don't know if participants will be able to benefit from the neuron cell lines grown from the selected participants DNA and stem cells, and their use in research, but having more potential chances of getting treatments tested on your specific Cell-DNA is better than waiting on neuron specific clinical trials.

The Precision Medicine Program launched in August 2014 with the goal of enrolling 20-25 patients. As a result of the Ice Bucket Challenge that summer, the Institute was able to expand enrollment to include 300 patients. The 100th participant enrolled in April 2015 and 200th in September 2015. We expect to complete enrollment of the 300th by June 2016. That cohort will be known as PMP 1.0. In April 2016 we announced PMP 2.0, an extension o the PMP to enrollment up to 450 more people and achieve specific goals for specific types of data. This pie chart depicts the total number of Patients Enrolled (purple), the number of those Scheduled for Enrolled (orange) and the number of Funded Slots Open (blue) for PMP 1.0. As we launch into PMP 2.0 we will update this graph to encapsulate the crucial participation of PMP 2.0 enrollees as well.

*Graphed data last updated Monday, May 01, 2017
Patients Enrolled (280), Scheduled for Enrollment (0), Open Slots (20)
I am.

I had my visit 2 years ago. While I think it worth doing I agree not to expect anthing from the cell lines. Mine have not yet had reprogramming to IPS cells yet. Have yours? I think they do not have resources to move too quickly.

Are they doing the skin biopsies on pmp 2 participants? I thought they were not?
No skin biopsy (not yet), only blood sampling.

One motive for the question is if anyone has been given notice of programming of IPS cells. I have not been given notice of any cell development action.

I would think the cell development would be best if targeted to use samples from living participants, to speed successful lab results to actual human patient trials (to overcome the mice model to human transition delay and problems). Wishful thinking, but better to have hope than defeat.

Resources and funding...

Of course now they can get cell lines from blood but don't think tdi is doing that.

I think Answer ALS is doing similar though not sharing. May be wrong I am not in that study.

If tdi found something already available that helped a group ( they are planning this in groups not 1 by 1 I was told- groups by phenotype/ genotype) it might help any survivors but the fda has taken a dim view of therapies only studied in ips cells. C9 antisense was first studied in ips as we did not have a good mouse. Fda said no dice without animal studies. To be fair, mouse studies showed some issues that the cells did not including a n increased risk for lymphoma
I just completed all their questionaires hoping to participate.
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