Transendocardial Autologous Serum Autologous Mesenchymal Stem Cell delivery using Transendocardial Delivery For Heart Failure Trial

Funding Type: 
Disease Team Planning
Grant Number: 
ICOC Funds Committed: 
Public Abstract: 

There is a need for new heart failure therapies. Today, those over 40 years of age have a 1 in 5 chance of developing heart failure. 5% of total US health care costs are attributable to these patients. Heart failure with ischemic etiology is a significant economic burden. Therapies are today limited to heart transplant, left ventricular assist devices, and palliative medical therapy. The only therapies in development not aimed at merely slowing deterioration of the heart or replacing heart function completely are cell based therapies. To date, results using adult autologous cells by our group, as well as others, in this population are promising.

By advancing therapeutic strategies based on autologous cells with noted plasticity, or the ability to differentiate into other cells, many of the preclinical and clinical hurdles to advance other potentially more promising cell types into the clinic will be solved. Through our efforts with autologous cells, our group believes we have made great strides on solving the delivery, retention, imaging, and clinical trial design issues for all cell based therapies in heart failure.

We propose to clinically develop a special autologous stem cell therapeutic for heart failure that has enormous promise based on our efforts to date. We intend to perform this work working with leading public and private California institutions, to publish this work to broadly advance the field, and to develop a road map such that this work paves the way for future embryonic strategies that may be not just therapeutic, but restorative.

The work in question will require tissue culture and processing in a quality controlled fashion appropriate for clinical use, the establishment of laboratory test controls to validate each therapeutic dose which will be a separate manufacturing lot per the Food and Drug Administration guidelines, animal preclinical studies to demonstrate safety, clinical protocol development and IND preparation, hospital institutional review board approval, hospital contracting, and ultimately clinical trials in patients with heart failure.

Statement of Benefit to California: 

Applicant institution, {REDACTED}, has been a California company since 1998 and employs 18 Californians with good jobs having excellent benefits. {REDACTED} is primarily focused on cell based therapy and its potential to treat unmet diseases in cardiology, such as ischemic heart failure.

The applicant seeks support to develop a solution for heart failure, a significant chronic disease in desperate need of new therapies from which California’s health care system is under an enormous economic burden.

Success in the proposed development efforts in collaboration with private and public California institutions, will benefit the State of California and its citizens on many levels. Success will reduce the cost of heart failure health care within California and beyond, and will create high quality biotechnology and medical device jobs within the state of California. In turn these jobs will improve the tax base in the state and provide for broad benefits to the State. Success will also herald the leadership in clinical medicine and basic research in the State of California, which will help draw other capable researchers and biotechnology entrepreneurs to the State. If patents are filed because of discoveries made they will directly benefit California through arrangements with the California Institute of Regenerative Medicine.

This is likely true for all potential embryonic stem cell (ESC) therapies. The difference here, is that autologous stem cell (ASC) strategies are far more proven in preclinical and clinical studies and there is a clear pathway to commercialization from a clinical and regulatory perspective. The numbers are also compelling – the potential average sales price for a product based around the approach outlined here would likely exceed $10,000 and be in the market after two stages of clinical investigation in five years. First year sales could easily be 10,000 procedures in the USA making this a $100,000,000 revenue generating business very quickly. As the cell processing will be labor intensive even with robotic automation, it will create a great many jobs. Further, due to the autologous nature of the product and the process of shipping source tissue for processing and final therapeutic material for treatment – these valuable jobs are highly unlikely to migrate overseas. A rough estimate is that success would create in excess of 1000 jobs in the state of California with an average annual salary of $100,000.00.

This project is state of the art and will benefit California regardless if successful by the experience and its import for other cell based therapies. This project will have significant implication for other ESC and ASC therapies in cardiology and beyond.