Cell therapy is the use of cells and tissues to regenerate, repair, and in some instances, even enhance human health. Traditionally, the modern medical system has relied on a pharmaceutical based approach. This lead to the rise of the U.S. Food and Drug Administration (FDA) which was founded in 1906, making it 111 years old this year. Unfortunately, our approach to human health has not experienced a dramatic shift over the past 100+ years, and as a society, we still tend to rely on a drug-based approach.
Although variations exist, drugs generally work by binding to receptors on the cell surface or by exerting an enzymatic effect to regulate the rate of an internal chemical reaction. Pharmaceuticals can frequently modulate the human system with great effect, but rarely do they have the capacity to regenerate or restore entire tissues or systems within the human body.
In contrast, cell therapy works because cells are powerful factories that can exert therapeutic effects through a wide range of strategies, including honing to sites of injury, exerting paracrine effects, and in some cases, differentiating into new types of cells and tissues. This versatility makes cell therapy extremely powerful and gives it the potential to reverse previously untreatable diseases. Needless to say, it is an exciting time in history.
For this reason, cell therapy – or more likely, cell therapy in combination with pharmaceuticals – will be the future of human health.
Eight Benefits of Cell Therapy
Below, we profile eight strengths of cell therapy that will propel it into the medical mainstream.
1. Treating Conditions with No Known Cure
Cell therapies have the potential to treat disease conditions with no known cure. There are many conditions that are now incurable, such as vision loss. While there has never been a treatment capable of reversing permanent vision loss, clinical trials are investigating the potential to restore it.
Specifically, there is a cell therapy clinical trial underway at the RIKEN Center for Developmental Biology (Japan), in which autologous iPSC-derived retinal pigment epithelial (RPE) cells are being transplanted into human patients with age-related macular degeneration. Although the trial was initially suspended due to safety concerns, it is now being resumed using allogeneic iPSC-derived RPE cells.
2. Reversal and Repair of Disease
Cell therapies also have the potential to reverse and repair diseases have traditionally been “managed.” For example, current drugs can slow the decline in functionality associated with multiple sclerosis (MS), but cannot stop or reverse the symptoms. However, in April 2016, a Phase I Clinical Trial conducted by the Tisch MS Research of New York (TMSRCNY) announced positive results from a FDA-approved Phase I stem cell trial for MS.
In this trial, MS patients received multiple spinal injections of neural progenitors derived from bone marrow mesenchymal stem cells (called MSC-NPs), and it was the first time in human history that a treatment method demonstrated reversal of established disability in MS patients. Tisch MSRSN is now pursing a Phase II clinical trial to explore the potential of stem cells to reverse MS.
3. Natural and Effective
Cells are powerful factories that are naturally present within the human system. Moreover, some cell types have intrinsic mechanisms that can assist with promoting repair. As we like to say at BioInformant, “We are not made of drugs, we are made of cells.”
For example, mesenchymal stem cells (MSCs) can hone to sites of injury, synthesize and secrete a variety of macromolecules to exert paracrine effects, and influence their local microenvironment. MSCs can also take up exogenous DNA and keep introduced genes, an attribute that may allow use of the cells in therapeutic delivery of molecules to target regions of the body.
Additionally, MSCs can form a variety of cell types in the laboratory, including those of both intra- and extra-mesenchymal lineage. These cell types include: fat (adipocytes), bone (osteoblasts), skin (dermal cells), nerve (neural cells), cartilage (chondrocytes), muscle (skeletal myocytes), tendons (tenocytes), marrow stroma, ligaments, and more.
With potentially hundreds of therapeutic cell types to explore, being a natural component of the human body is clearly a therapeutic advantage.
4. Lowered Costs by Eliminating Continuous Drug Use
For many disease conditions, such as diabetes, patients are required to take a prescription on a daily basis. A cell therapy approach would substantially reduce the healthcare costs of this disease by providing a one-time (or limited-time) treatment. Similarly, cell therapy approaches to pain management would substantially reduce the costs of opioid-based pain medications. There are many examples of how cell therapy treatments could make pharmaceutical treatments obsolete.
5. Overcoming Tissue and Organ Donor Shortages
Another strength of cell therapies is that it could overcome donor shortages. For many disease conditions, the only known cure is a tissue or organ transplant. Examples of transplanted tissues and organs include the heart, kidneys, lungs, pancreas, pancreatic islet cells, and more. However, if a cell therapy approach could convert a diabetic patient’s own tissue into insulin-producing cells, it would overcome the problem of donor shortage and remove the risk of transplant rejection.
For this reason, the following companies are exploring cell-based solutions to diabetes:
- Janssen Pharmaceuticals
- Sernova Corporation
The same principle is true for every type of tissue and organ transplantation.
6. Replacing Invasive Surgeries
Cell therapies have the potential to replace many risky, costly, and invasive surgeries. For example, the Regenexx treatment pioneered by Dr. Chris Centeno is an autologous same-day, minimally manipulated MSC procedure that is legal in the U.S., because it meets the FDA’s requirements for not requiring a clinical trial process.
In this cell therapy procedure, a proprietary two fraction isolation, which is a “buffy coat” plus another source of stem cells within the bone marrow, is administered to a patient in a 3-step process. The first step is a pro-inflammatory injection for which Regenexx received a recent patent. The second step is implanting the stem cells via precise guidance. The third step is a “fertilizer” type step, adding in platelet-based growth factors.
Interestingly, this cell-based procedure is capable of repairing most ACL tears that would have traditionally been treated surgery. With an estimated 100,000 ACL surgeries performed in the U.S. each year, this cell therapy alternative represents a paradigm shift. The Regenexx procedure may also prevent other surgeries, such as shoulder rotator cuff tear surgeries, hip surgeries, and more.
7. Augmenting Traditional Surgery
Most surgeries create an enormous amount of inflammation (swelling) and fibrotic activity (scarring). Therefore, the future of surgery will likely involve combining the structural elements provided by a surgeon’s skills with the anti-inflammatory and anti-fibrotic elements of cell therapies. The most likely solution is that autologous or allogeneic MSCs will be administered to patient immediately post-surgery to support healthy regulation of the patient’s response to surgery. The reason for use of MSCs is that they are safe, easily sourced from adipose tissue and bone marrow, and thoroughly documented to have strong anti-inflammatory and anti-fibrotic properties.
Currently, the Mayo Clinic is testing the use of autologous cord blood cells for the treatment of Hypoplastic Left Heart Syndrome (HLHS). It is executing is a Phase I study to determine the safety and feasibility of injections of autologous umbilical cord blood (UCB) cells into the right ventricle of HLHS children undergoing a scheduled Glenn surgical procedure.
The investigators are doing this study to find out if autologous stem cells from the individual’s own umbilical cord blood can be used to strengthen the muscle of the right side of their heart. This will help determine the safety and feasibility of using cell-based regenerative therapy as an additional treatment to support surgical management of HLHS.
8. Cell Therapies Used in Conjunction with Pharmaceuticals
Cell therapies also have the potential to work synergistically with pharmaceutical drugs. For example, evidence indicates that intravenous administration of MSCs may improve several metrics of cardiac function, potentially making them a synergistic to medications traditionally prescribed to cardiac patients, such as amlodipine, diltiazem, and felodipine, and others.
One company leading the way is the area of cell therapeutics used in conjunction with pharmaceutical drugs is Stem Cell Medicine Ltd. in Israel. As Frida Grynspan, VP of R&D and Site Manager for Stem Cell Medicine Ltd., told BioInformant in an interview, “Stem cells used in conjunction with pharmaceuticals are the future of medicine.” Not surprisingly, Stem Cell Medicine’s mission is to “develop and commercialize cellular therapies in combination with pharmaceutical products to be used for tissue repair and for the treatment of inflammatory, immunological and neurological disorders.”
Other Reasons Cell Therapy is the Future of Medicine?
Do you have other reasons that cell therapy is the future of medicine? Add them in the comments below and we’ll add them when we update this post.