stem cells

Satellite Bio is named descriptively for the way its platform works. Out of stealth in the past few months with what you might call a middle ground approach to generative medicine between stem cell therapy and organ transplant, the company takes its name from the tissue therapy constructs they surgically implant in patients.

“We build these constructs which are implantable tissue therapeutics. They look like thin squishy jellyfish. They are loaded up with cells, and we then introduce these to patients with minimally invasive surgery. They can then perform their functions and graph into the patient and vascularize and develop a new blood supply of their own. We can place these virtually any place in the body. They function from a satellite location to the host organ.”

That’s Dave Lennon, CEO of Satellite Bio joining us on the program today to tell us about this fascinating new technology. Is this stem cell therapy 2.0? How much space do these constructs take up in the body in their satellite location? How far is the company to date? Have they begun trials? What are the challenges they face, and who will be the first patients to benefit?

There are drug trial failures. And there are drug trial failures.

The recent move by drug giant Biogen to halt their Alzheimer’s drug has not only been a setback for the company—their stock fell precipitously on the news—it’s also been a major setback for the whole research community focused on this disease. The Biogen trial was one of the last of the major drug companies with high hopes for a significant therapeutic step forward for the neurodegenerative disease. The last five years has seen one big pharma players after another throw in the towel.

Larry Goldstein joins us today to look at the field and give his assessment. He’s been studying Alzheimer’s at his lab at UC San Diego for a number of years. His number one takeaway? Let’s move on from all eggs in the amyloid hypothesis basket—something he did years ago.

Have we made any progress in ten years? Are there any cool new tools on the bench? And have we improved diagnoses at least?

Guest: Liran Shlush, Researcher, Princess Margaret Cancer Center

Listen (4:44) Preleukemic stem cells

Listen (7:42) Implications for AML therapy and diagnosis

Listen (2:21) Implications for other cancers

Listen (6:14) Importance of population genetics tools

One of the common topics on our show these days is how researchers can ask better questions. It takes being master at science, but also being connected with patients and their health problems.

Today's guest, Liran Shlush, a cancer researcher at Princess Margaret Cancer Center in Toronto, solved this by being one of those rare physician-scientists. And it's paying off big time.

In February, Liran and a group from the lab of John Dick at the University of Toronto published a major finding into the biology of acute myeloid leukemia (AML). In today's interview, Liran tells us about the discovery of what he terms "pre-leukemic" stem cells. These mutant stem cells go on to form cancerous cells. When AML patients are treated with chemo, the cancerous cells are killed, but the mutant stem cells are still there and can cause recurrence. The finding holds a lot of promise for both treatment, earlier diagnosis, and new screening for AML and perhaps other cancers as well.

Liran was a post doc at the time of the discovery. He tells of his journey from Israel to John Dick's lab in Canada and the lessons along the way. What led him to ask the right questions?

"The scientific lesson I learned--and I was lucky to learn it early in my career--" he says, "is that nothing in biology makes sense except in the light of evolution."

Podcast Sponsor: Integrated DNA Technologies - Introducing the evolution of NGS capture panels

Guests:

Larry Goldstein, PhD, Director, UCSD Stem Cell Program
Bio and Contact Info

Listen (3:16) Where are we at today with stem cell research?

Listen (5:14) One step beyond animal models

Listen (3:58) Why are stem cells so useful?

Listen (4:46) Promising applications for rare diseases

Listen (6:00) Funding has been a special challenge

Listen (3:22) Is there still a PR problem?

There are an estimated 7,000 rare or orphan diseases. But there are currently only treatments for about 5% of them. In this series, we've been exploring the promise of various new technologies to come up with more treatments and more cures. Today we are going to discuss the promising research with stem cells.

Scientists have made dramatic advances with stem cells over the past ten years. Dr. Larry Goldstein, Director of the Stem Cell Program at UCSD is one of them. He’s been using stem cells as a new technology for understanding disease, such as the rare Niemann Pick Type C disease. Stem cell therapy is now FDA approved for certain cancers and is at the heart of numerous clinical trials for various diseases.

Larry discusses the unique challenge that funding has proved for stem cell research and discusses whether there is still a PR problem holding back future research.

For more info, get the free eBook from Rare Genomics Institute

"Rare Disease Horizons: The Hope of New Research and Technology" is underwritten by the Rare Genomics Institute.

Podcast brought to you by: Ingenuity Variant Analysis - Identify causal variants from human sequencing data in just hours.

Guest: Gajus Worthington, CEO, Fluidigm Bio and Contact Info

Chapters: (Advance the marker)

0:39 Looking at heterogeneity

6:18 How is your technology driving the study of single cells

13:35 Overcoming skepticism

18:29 The promise of IPS dependent on looking at cells separately

21:11 Single cell genomics will expand to every area of cell biology

25:31 Are you the next big Illumina?

30:01 BONUS: Chip holds as much plumbing as a one-thousand-room hotel

Gajus Worthington has a message. Molecular biologists have been studying the genetics of individual cells (single cell) for a long time. But the amount of manual labor required has been prohibitive to achieving sufficiently large data sets. Fluidigm, a tools company in South San Francisco, co-founded by Worthington, is changing that. Now a single micro fluidics chip that contains as much plumbing as a one-thousand-room hotel is driving the adoption of single cell genomics research. In today's interview, Worthington, also CEO of Fluidigm, explains the company's technology and some of its applications.

We've heard a lot lately about the heterogeneous nature of cancer tumors. But there is heterogeneity everywhere, including stem cells. "Some iPS (induced pluripotent stem) cells are more "gifted" than others," Worthington explains. "To make iPS achieve its promise, and the promise is amazing, you have to understand the mechanism by which these "gifted" cells go from one type to another." One assumes the company's name comes from the two words "fluidics" and "paradigm." Fluidigm's technology is enabling researchers to make a shift in paradigm necessary to go ever deeper into the complexity of biology. Will they be the next Illumina?