Rensselaer Polytechnic Institute stem cell meeting report

Last week I attended and spoke at a stem cell meeting at Rensselaer Polytechnic Institute (RPI). It was entitled, “Bioengineering and Stem Cell Research”.

It was a great meeting with many interesting talks. Below I write about some of the talks and themes of the meeting. Still to come later this week I will do a second post entirely focused on the exciting RPE work of Sally Temple, who spoke at the meeting.

Mark Noble

One of the major themes of this meeting was, as Mark Noble put it during his talk, the urgent need for greater precision in defining our cells, whether they are stem cells or derivatives of stem cells. A lack of precise cellular identification interferes, Noble said, with comparing data within labs and between labs, patent evaluation, and therapy development.

Noble also asked how we are going to pay for stem cell therapy development, which is a huge often overlooked issue (see slide above from his talk). One of Noble’s innovative ideas is to focus on already FDA approved drugs, providing an effective shortcut to the bedside. This is a potentially very powerful approach if you can find molecules amongst the already approved ones that do what you want.

Mike West of BioTime also spoke at the meeting. He presented about their neat clonal derivative research from ESCs. The numerous determined stem and progenitor derived in this way have more focused differentiation potential that proves very useful and addresses the concern of heterogeneity of cell populations made directly from human ESCs.

The talk of another speaker, Ron Hart from Rutgers, was somewhat of a cautionary tale. His team was working on IPSC made from patients with Ataxia Telangiectasia (A-T). The IPSC had with compound heterozygous mutations in the ATM gene. At some point in the process of studying these cells they found that a certain mutation was lost. It seemed to become WT and the team’s sense is that a spontaneous gene correction event occurred via the otherwise WT sequences of the mutant ATM alleles. It never occurred to me that a heterozygous line could essentially lose a mutation via gene correction. Another possibility that we should all be on the look out for is contamination of one IPSC line with another.

Overall this was an exciting meeting and I don’t even include in that the tornado watch on the second day. Again, stay tuned for an upcoming blog post on Sally’s work on adult RPEs.

BioTime’s Cell Cure Files IND with FDA for ES cell AMD therapy

Cell Cure LogoStem cell biotech BioTime announced the news today that its subsidiary Cell Cure Neurosciences (Cell Cure) has filed an IND with the FDA for an embryonic stem (ES) cell-based therapy for Dry age-related macular degeneration (AMD). The product to be tested is OpRegen, which the company indicates is “the first IND for an ES cell-based therapy developed in Israel.”

Cell Cure in its PR on this IND also listed three other key bullet points:

  • “IND filed for Phase I/IIa dose escalation trial in patients with dry-AMD
  • No approved therapy exists for dry-AMD, the leading cause of visual impairment in the aging population
  • OpRegen® will be the first preparation of xeno-free RPE cells to be evaluated clinically for dry-AMD”

I’m very curious to see how OpRegen performs in the trial.

This is only the third IND for an ES cell-based therapy with the other two coming from Advanced Cell Technology (ACT) and Geron.

I asked Dr. Mike West, CEO of BioTime, about the trial and he had this to say:

“Age-related degenerative diseases are really the frontier of medicine in our time. The aging of 76 million baby boomers and the consequent tsunami of healthcare costs associated with palliative as opposed to truly therapeutic care is a top national priority. I believe that AMD is only one of numerous examples where the stable engraftment of young healthy cells may provide important new therapeutics for these long-term and expensive problems of aging.”

Macular degeneration of various kinds is a focus of a number of pluripotent stem cell trials. For example, the first ever iPS cell-based clinical study, led by Masayo Takahashi, is also focusing on AMD.

Disclosure. I have no financial interests in BioTime or Cell Cure and have a small long position in ACT. This post is not financial advice.