5 Groundbreaking Regenerative Medicine Clinical Trials
October 2018

There are currently over 5,000 registered clinical trials testing regenerative medicine  – specifically involving stem cell research – on In a field that’s developing so rapidly with such a high level of innovation, we think it’s important that we recognise the progress (and challenges) of researchers around the world.

While it’s difficult – if not impossible – to choose just 5 from thousands of truly groundbreaking studies, we’ve done our best to narrow it down to showcase a wide range of clinical trials that are testing regenerative medicines on a wide range of conditions.


1. Stem Cells From Baby Teeth Shown to Regrow Damaged Dental Tissue

A Phase 1 clinical trial in China that’s being co-led by Songtao Shi, professor and Chair in the Department of Anatomy and Cell Biology in Penn’s School of Dental Medicine in Pennsylvania, USA, has given patients sensation back in their teeth. 30 children received the experimental treatment which uses human deciduous pulp stem cells (hDPSC) extracted from baby teeth to regrow dental pulp.  So far, it’s proven to be a safe and effective treatment and the team is waiting on FDA approval to start clinical trials in the United States.


2. A Novel Immunotherapy Approach Causes Patient’s Breast Cancer To Disappear

In an ongoing phase II trial led by Steven A. Rosenberg, the chief of the Surgery Branch at NCI’s Center for Cancer Research (CCR), a patient – who was unresponsive to other treatments – received a modified form of adoptive cell transfer (ACT) that uses tumour-infiltrating lymphocytes (TILs) to target tumour cell mutations. The novel approach to immunotherapy was developed by the National Cancer Institute and can identify mutations present in a cancer that aren’t recognised by the immune system. After treatment, the cancer disappeared and hasn’t returned for nearly two years.

Those involved in the trial are hoping for a larger study that allows them to test the therapy on a wider range of patients with different kinds of cancer.


3. Phase I/II Study Testing Therapy That Would Eliminate The Need for Chronic Treatment in Severe Hemophilia A

BioMarin Pharmaceutical Inc. is evaluating an investigational gene therapy called valoctocogene roxaparvovec on patients with and without pre-existing AAv5 antibodies. They are currently testing for safety and efficacy before expanding the study to treat patients across all sub-groups. Hemophilia A is a genetic disorder that prevents people living with the disease from forming blood clots. While there is a standard treatment, many patients are still at risk for and experience excessive bleeding that causes debilitating joint damage. Press releases from BioMarin suggest researchers are optimistic but they are still waiting on decisions regarding future trials from the FDA and other regulatory bodies.

4. For the First time, a Clinical Trial Will Investigate Stem Cell Therapy For Severe Traumatic Injury

After receiving funding from the Medical Technology Consortium (MTEC) and Memorial Hermann Foundation, The University of Texas Health Science Center at Houston (UHealth) will begin the first-ever Phase II trial that will investigate stem cell therapy for early treatment and prevention of complication associated with severe traumatic injuries. The study will test an investigational clinical product called MultiStem, which will be provided by Athersys, Inc.

According to prior research and pre-clinical trials over the last 10 years, MultiStem has been studied with high hopes that it can help improve recovery and reduce the severity of complications when administered after an acute neurological injury.  With traumatic injury being the third-leading cause of death in the US, the Phase II trial represents exciting potential in the field


5. Allogeneic Mesenchymal Stem Cells Treating Infants With A Rare Heart Condition

The University of Maryland, Johns Hopkins University and Longeveron have joined forces for a clinical trial that will treat 30 infants with Hypoplastic Left Heart Syndrome (HLHS) with the aim of improving ventricular function.

The study is intended to evaluate the safety and feasibility of intramyocardial injections of allogeneic mesenchymal cells during surgeries on HLHS pediatric patients. With 20 patients being treated with the injection and 10 acting as controls, the trial should be completed by October 2020 with results published in a study by October 2022.

Of course, creating a list isn’t the only way to acknowledge and engage with key players in the field.

Our Chief  Executive Officer, Ajan Reginald, attended the Cell & Gene Therapy Strategy Meeting in Zurich on September 26th to learn more about emerging biotech and pharma companies around the world. He engaged with primary decision-makers to discuss past, current and future projects related to Cell Therapy. Among the topics discussed were: Immune Deficiencies, Cell Engineering, CAR-T, Biotherapeutics, Cellular Immunotherapy, Regenerative Medicine, and Supply Chain and Manufacturing.

To keep up to date on other events like the Cell & Gene Therapy Strategy Meeting, visit our Events page.

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