UCLA: Utilizing Patient's Own Cells for Individualized Stem Cell Therapies

Researchers at UCLA believe using the patient’s own cells to create stem cells for therapeutic purposes is the future of medicine. A recently published study by scientists at UCLA demonstrates how specialized proteins change the cellular characteristics of skin cells to create induced pluripotent stem cells. These stem cells have the ability to turn into any cell type within the body. Also at UCLA, a clinical trial which uses the baby’s own blood-forming stem cells to treat the immune deficiency condition ADA-SCID, better known as “bubble baby disease,” was recently awarded a $20M grant from the California Institute for Regenerative Medicine.

The research into the creation of induced pluripotent stem cells from skin cells is led by Kathrin Plath Ph.D. and Jason Ernst Ph.D. Their study could improve the method for creating healthy tissue that can be used to treat a number of diseases. Though induced pluripotent stem cells were first created over a decade ago, scientist have not had detailed information on how the process actually worked until now.

One way stem cell therapy can be done is by removing skin cells from a patient, reprogramming them, and then reintroducing them into that person’s body. The reprogramming process is done by adding proteins called “transcription factors.” Since the process uses the individual’s own cells, they are a perfect match. That means they can treat or even reverse a disease without the need for immune suppression.

In this study, scientists added the transcription factors to mouse skin cells and mapped their interaction with the cells’ DNA. Researchers made two key discoveries:

1. Transcription factors shut down the skin cell’s identity and activate pluripotency at the same time.
2. Three of the four factors must work together to locate and regulate the DNA locations important to the process.

Researchers used this data to predict which additional transcription factors might improve the process and added a fifth transcription factor. The new combination suppressed the tissue-specific cell’s identity more effectively, which in turn increased the efficiency of the cell reprogramming process by a hundredfold. Understanding exactly how transcription factors change a cell’s identity, and increasing the efficiency of the process, are considered two critical steps in the effort to combat many deadly diseases using stem cell-derived tissues or cells.

In a separate research project, UCLA’s clinician-scientist Dr. Donald Kohn will lead a clinical trial meant to improve the treatment options for children with Severe combined immunodeficiency, SCID, or bubble baby disease.

SCID is a rare genetic disorder characterized by an immune system so compromised that it is considered almost absent. Because of this, people with SCID must live their lives in a sterile environment.

However, a stem cell gene therapy currently only available at UCLA and in London has cured 30 out of 30 babies to date. This treatment removes the baby’s own blood-forming stem cells and fixes the broken genes to eliminate the disease-causing mutations and the associated debilitating symptoms. The corrected cells are then transplanted back into the baby.

According to the article for the UCLA’s Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research News Site, the CIRM funding will allow researchers to test a method that freezes the blood stem cells after they have been genetically corrected. This would allow for:

• Better testing of the cells prior to transplant
• More time to customize treatment plans based on patient needs
• The transportation of cells from the gene correction facility back to a transplant center closer to a patient’s home.

Biotechnology Trade Show Event at UCLA: 

To meet face to face with the inovative researchers at UCLA and discuss their equipement needs, laboratory equipment suppliers should plan on displaying their products at the the 39th Semiannual Biotechnology Vendor Showcase™ Event at UCLA. This event will be held on Thursday April 20, 2017, where more than 200 life scientists are expected to attend. Last year, researchers came from 46 different research buildings and 80 on-campus departments.

To learn more about participating in this popular event, call (530) 272-6675 or visit the link below:

Science professionals attend for free. Click the link below to for more information and to preregister.