CART T Cells via mRNA-LNPs Update 12/2023

Date: December 2023
Author: Dr. Steven M. Albelda

ROTZ Foundation Donation Summary

Upon completing the 9th Annual fundraising event, we are incredibly proud and grateful to announce the 9th Annual Event raised $70,975 bringing the total amount raised to $551,410 since inception.

In this article, Dr. Steven Albeda explains how the fund's dontations have provided the research team the ability to test cutting edge novel ideas critical to developing treatments. Below, read about advancements made in ongoing treatment research as well as tests towards developing a newer approach in LNPs.

Key Updates

Dr. Steven Albeda and his team at Penn Medicine are making progress in clinical trials and LNP projects:

1. We are making progress in getting the clinical trial for the FAP-CARTs started. Dr. Carl June, using institutional funds, has agreed to help fund the first trial here at Penn. We are including lung cancer patients. Over the next year, we will be working on getting the final data we need to submit to the FDA and get started.
2. We are making progress on the mRNA lipid nanoparticle (LNP) project and are in discussions with Johnson and Johnson and a new startup company to fund future studies.
3. Penn recently hired one of our former trainees, Dr. Jarrod Predina, as our new thoracic surgeon. Jarod is working in our lab and is going to be taking over the mRNA project.

Potential for LNP Treatments and Fund Importance

Our lab spent many years developing treatments where we injected a modified cold virus (an adenovirus) expressing an immunologic activator (interferon) directly into tumors with some success. We now think we can replicate and improve on this approach in an easier and safer fashion by using the LNPs that encode a message for interferon. Studies are underway to test this idea and to optimize its efficacy.

The Thoracic Oncology Laboratory has funding from a number of sources, including government grants and research agreements with pharmaceutical and biotech companies. Traditionally, these funding sources are quite conservative and only want to fund "sure things" with lots of preliminary data. However, that is not how the discovery of cutting edge science works. There is a need to try new, creative, risky, and unproven approaches. Some succeed, but most don’t.

This "novel idea space" is where the Rotz Foundation has been of special importance to the lab by helping to support these more novel “high risk - high gain” projects needed to produce the preliminary data that can then be used to obtain government or industry support. For example, ROTZ Foundation funding was used to help support the FAP CAR T projects, ultimately leading to the NIH grants and industry support that will lead to human clinical trials.

More recently, the funds have supported the NK cell projects, and over the next year will be used to support the new project to generate CAR T Cells inside the body with a simple injection of mRNA. We are hopeful that these projects will also lead to government and industry support and ultimately to clinical trials.