Stories that caught our eye: FDA grants orphan drug status to CIRM-funded therapy; stunning discovery upends ideas of cell formation; and how tadpoles grow new tails

Gut busting discovery

Intestinal stem cells: Photo courtesy Klaus Kaestner, Penn Institute for Regenerative Medicine

It’s not often you read the word “sensational” in a news release about stem cells. But this week researchers at the University of Copenhagen released findings that are overturning long-held ideas about the development of cells in our stomachs. So perhaps calling it “sensational” is not too big a stretch.

In the past it was believed that the development of immature cells in our stomachs, before a baby is born, was predetermined, that the cells had some kind of innate sense of what they were going to become and when. Turns out that’s not the case. The researchers say it’s the cells’ environment that determines what they will become and that all cells in the fetus’ gut have the potential to turn into stem cells.

In the “sensational” news release lead author, Kim Jensen, says this finding could help in the development of new therapies.

“We used to believe that a cell’s potential for becoming a stem cell was predetermined, but our new results show that all immature cells have the same probability for becoming stem cells in the fully developed organ. In principle, it is simply a matter of being in the right place at the right time. Here signals from the cells’ surroundings determine their fate. If we are able to identify the signals that are necessary for the immature cell to develop into a stem cell, it will be easier for us to manipulate cells in the wanted direction’.

The study is published in the journal Nature.                             

A tale of a tail

African clawed frog tadpole: Photo courtesy Gary Nafis

It’s long been known that some lizards and other mammals can regrow severed limbs, but it hasn’t been clear how. Now scientists at the University of Cambridge in the UK have figured out what’s going on.

Using single-cell genomics the scientists were able to track which genes are turned on and off at particular times, allowing them to watch what happens inside the tail of the African clawed frog tadpole as it regenerates the damaged limb.

They found that the response was orchestrated by a group of skin cells they called Regeneration-Organizing Cells, or ROCs. Can Aztekin, one of the lead authors of the study in the journal Science, says seeing how ROCs work could lead to new ideas on how to stimulate similar regeneration in other mammals.

“It’s an astonishing process to watch unfold. After tail amputation, ROCs migrate from the body to the wound and secrete a cocktail of growth factors that coordinate the response of tissue precursor cells. These cells then work together to regenerate a tail of the right size, pattern and cell composition.”

Orphan Drug Designation for CIRM-funded therapy

Poseida Therapeutics got some good news recently about their CIRM-funded therapy for multiple myeloma. The US Food and Drug Administration (FDA) granted them orphan drug designation.

Orphan drug designation is given to therapies targeting rare diseases or disorders that affect fewer than 200,000 people in the U.S. It means the company may be eligible for grant funding toward clinical trial costs, tax advantages, FDA user-fee benefits and seven years of market exclusivity in the United States following marketing approval by the FDA.

CIRM’s President and CEO, Dr. Maria Millan, says the company is using a gene-modified cell therapy approach to help people who are not responding to traditional approaches.

“Poseida’s technology is seeking to destroy these cancerous myeloma cells with an immunotherapy approach that uses the patient’s own engineered immune system T cells to seek and destroy the myeloma cells.”

Poseida’s CEO, Eric Ostertag, said the designation is an important milestone for the company therapy which “has demonstrated outstanding potency, with strikingly low rates of toxicity in our phase 1 clinical trial. In fact, the FDA has approved fully outpatient dosing in our Phase 2 trial starting in the second quarter of 2019.”

One thought on “Stories that caught our eye: FDA grants orphan drug status to CIRM-funded therapy; stunning discovery upends ideas of cell formation; and how tadpoles grow new tails

  1. Gut Busting Discovery

    The development stem cells from immature to mature requires many stages of cell growth and differentiation. Each stage of progenitor cells has different genotype and phenotype. Therefore each stage of cells responses differently to the growth factors. Therefore, receptor of growth factors and genetic markers are important parameters to indicate the stage of maturation of cells in organ.

    The development of human and animals are regulated by the cycles of growth factors and hormones. In the early stage of life, although the structure of organs looks similar to aldult but they are not yet fully developed and functioning. All the cells in fetus can be considered immature which are progenitor cells from different stages of stem cell development. The existence of specific growth factor may trigger the cell to growth and differentiate to become more mature. Therefore, different organs have different time-line to become fully developed and functioning organs depending on existence of growth hormones in cycle.

    Tale of A Tail Single -genomic facilitated the determination of the existence of a group of skin cells so-called Regeneration -Organizing Cells playing roles to regenerate the damaged limb.

    Frog tadpoles are primative animals. The structure of genomic DNA is simplicity. Single genomic DNA can easily be used to analyze structure and expression of genomic DNA. Human is considered more developed and complicated. The structure of DNA is big and complex. Single genomic DNA is not be able to compare the diversity of specific cell type from one individual to the other. In addition, human is completely lack of potential to regenerate the limbs and therefore human may not has this gene for regeneration functioning.

    Clinical Study of CAR-T cells for Multiple Myeloma.

    The effectiveness of immunotherapy for cancer is very depended on the ability of immune cells to adhere and extravasate the tumor . Tumor is heterogeneous and instability of genetic in tumor cell cause genotype and phenotype changing from time to time. This may modulate the identity of antigen tumor and affect affinity of binding between tumor antigen and antigen receptor from CAR-T cells. The short-term of CAT-T cells treatment may more effective than long-term. The mutation in genetic antigen may cause antigen tumor does not recognize the binding site on CAR -T cells. Therefore, long-term treatment of CAR-T cells required the consistent observation of the stability of tumor antigen.

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