GDF11 | Adult MSCs > MS | NYSCF Mito/NT

While I attended many sessions during the week at ISSCR, a few caught my attention. Those were topical for me as they touched on some previous reporting I had done and featured updated information which I want to share.

As a non-scientist the finer details of the technical presentations I leave to the experts. However, as I appreciated from Rusty Gage's talk pushing those neurons to bridge helps... I try to do my homework and as an objective observer I process that in relation to how it might impact the field and patients in particular.

So in that spirit onto the highlights - GDF11 first.

The fountain of youth, as it was often referred to when young blood was first presented as the ultimate elixir. The property identified within the crimson tide was GDF11, a protein that evidentially is in abundance in young blood but over time depletes to the point where its reduced percentage in aged blood has been identified as the potential treatment target for restorative therapeutics. Enter the debate...

Who wouldn't want to believe in a solution for age related degenerative conditions via a shot, transplant or cell stimulant. It’s the ultimate product - one, if produced, would mint the next superpower in the guise of an all powerful corporate entity. Hopefully with a humanitarian bent for universal application and charity - otherwise we're in for the equivalent of eugenics, not via genetic engineering but through class access.

Amy Wagers of Harvard, who leads the team that presented the controversial data on GDF11, again presented data during the closing Plenary session at ISSCR 2015. She reiterated that GDF11 was indeed responsible for stimulating the rejuvenation of aged tissue. What was interesting was her resounding affirmation of her previous team’s work with additional clarification of the decrease in aged sampling while postulated the reasons for metabolic interaction. With this new evidence it seems there is a case for continued debate between Novartis and Wager’s group - with the ball now firmly in the big pharma’s court.

A good background piece by Sara Reardon on the controversy can be found here.

Another topic which has been a source of scientific debate, is the efficacy potential of adult MSCs to treat Multiple Sclerosis (“MS”). Much has been written on the potential immunological benefits of MSCs, as well as the therapeutic value in a whole host of degenerative conditions. This potential has to some extent broadened the expectation factor when the reality has yet to be fully materialized in clinical results and approved products with meaningful cost to efficacy ratios. Neuronal diseases are by far the most challenging and a source of patient desperation given they are largely unmet medical conditions - MS being one such disease.

There have been various reports of autologous stem cells being used systemically and directly into the CNS for MS yet none were administered under a FDA approved clinical trial setting and therefore the results, however promising in certain reported cases were not recognized by the community. This therefore makes the situation all the more pressing for validating studies on severe unmet medical conditions - not just in the US but in all territories internationally where stem cell therapies are being applied. 

Most recently in Canada they have begun the 1st Canadian MSC trial for MS and the community eagerly awaits the data.

In the US it has largely gone unreported that there is an open FDA approved adult MSC-NP clinical trial at the Tisch School of Medicine in NY with Dr. Violaine Harris & team driving the science and Dr. Saud Sadiq, as the investigator. During the session interim data was presented from 10 patients that had been dosed with 8 to 10m MSCs due to have 3 separate administration sessions. This report was post the 2nd dosing. Safety was shown with mild transient headaches reported (24 to 48 hrs duration). On the effect signal, although it’s still early and only 10 patients, there were no worsening of the disease in any subjects, with 4 showing mild improvement with standing and movement and 3 patients with good improvement. No doubt the full data set will be of interest and it’s a positive sign that under clinical conditions there is a biological effect which may be attributable to the cells. One can postulate that with more potent technology this effect may be accentuated and patients without much recourse will be recruited for larger studies to speed the path forward. Positive presentation.

The last of the three sessions I will relay here is the talk by Mitsutoshi Yamada, a postdoc fellow who works with Dieter Elgi’s at the New York Stem Cell Foundation (“NYSCF”). What I took away from the talk was the viability issue of using screening technology to assist in the process of ensuring safe and effective translation of mitochondrial replacement therapy. It was shown in this study that there was no adverse developmental effects using the NT process, which seemed positive and that there was also solid reporting of integrity analysis. The issue of reported “drift” I believe was tweeted once or twice on the presentation at the time, which was a noted issue requiring follow-up. Paper to be published shortly.

As mitochondrial replacement therapy is being considered in the US and the legal authorization to proceed in the UK allowed it’s fitting that the science investigation continues apace to answer the open questions and look to increase the accumulating knowledge surrounding the technology. Infertility is a curse and a growing problem in today’s stress filled and environmentally polluted world.

As a related aside, I coincidentally stopped at a Poster number looking for a specific study, which I thought would still be up, but which had been taken down and replaced. In its stead was an iPS Egg poster from Taiwan. 

I spoke with the investigators and was happy to learn that they are actively pursuing the iPS Egg creation concept for infertility treatment. They relayed that Taiwan has the highest rate of infertility in the world and are near their goal of producing iPS Eggs. I wished them well.

This sculpture by my Mother was created some ten years ago for all those women that suffer from infertility - she’s screaming to the heavens…it weighs 500 kilos and was made in Norway of granite - it rests now in the Pyrenees, closer to the sky...

Cheers