Stem cell research and treatments represent exciting advancements in medicine. This innovation and our patients’ needs drive everything that we do. All of our efforts are focused on bringing life-saving medicines to the market.
With more than 20 tissue-specific regenerative medicines in our pipeline, we want the public to understand what stem cells are, their potential in treating a range of formerly untreatable conditions, and why their use in medicine has been considered controversial in the past.
- Different Stem Cells Serve Different Purposes
There are four major types of stem cells:
Embryonic Stem Cells: ES Cells are cells derived from early stage pre-implantation embryos. In order to harvest these stem cells, an embryo has to be fertilized in a laboratory as opposed to inside the female body. ES cells are pluripotent, meaning they can divide into more stem cells and can become other types of cells in the body.
Adult Stem Cells (Tissue-Specific Stem Cells): Adult stem cells (found in both children and in adults) come from developed organs and tissue. They can self-renew indefinitely to replenish dying cells and to regenerate damaged tissue but they have limited differentiations..
Induced Pluripotent Stem Cells (iPSCs): Scientists have recently discovered how to reprogram adult stem cells to act more like pluripotent, embryonic stem cells. They have the potential to produce new cells for any organ or tissue in the body and can be made from someone’s own skin, potentially preventing rejection from the immune system.
Cord Blood Stem Cells: After childbirth, stem cells can be harvested from the umbilical cord and frozen for future use. These stem cells can produce all other cells found in blood, including cells of the immune system. This makes them especially useful in treating blood diseases like Leukemia.
- Regenerative Medicine and Tissue Engineering Are Different
Tissue engineering is an interdisciplinary field which applies the principles of engineering and science to develop biological substitutes that restore, maintain, or improve tissue function. The key word here is develop. Tissue engineering has the capacity to build biologic materials.
Regenerative medicine doesn’t require the production or growth of biological substitutes. Rather, regenerative medicine is any therapy aimed at restoring function.
- Stem Cells Could be Used to Treat Everything from Heart Disease to Menopause
Because stem cells can differentiate themselves into a range of adult cells, they can potentially treat any disease or condition that causes and/or is perpetuated by the destruction of cells and tissues.
At Celixir, we’ve focused our efforts on how iMP cells (Integral Membrane Protein) can treat patients with heart disease. Around the world, scientists have found stem cells to be useful in treating Parkinson’s Disease, Diabetes, Leukemia and even menopause. Scientists are also closer than ever to finding a cure for Human Immunodeficiency Virus (HIV) because of extensive stem cell research.
- Stem Cells Could Regenerate Tissues, Bones and Cartilage
Cell-based bone and cartilage replacement is an evolving therapy that could help amputees, those have lost limbs in an accident, and those with autoimmune diseases that attack and destroy cartilage tissues in the body. While many animals have the ability to regenerate or replace lost or damaged appendages, humans, unfortunately, do not.
But, back in 2017, scientists from the University of New South Wales (UNSW) transplanted multipotent stem cells into areas of damaged tissue. Controlled repair of tissue seemed to be observed. Human trials are expected to begin this year.
- There is Controversy Surrounding Stem Cell Research and Treatment
The controversy surrounding stem cell research and treatments is of a moral nature rather than scientific. Namely, opponents believe that harvesting embryonic stem cells and using fertilized embryos is unethical as they believe first stage embryos should have the same rights as fully developed humans.
On the other side of the argument, supporters believe that embryos aren’t yet humans. Donor couples whose eggs and sperm were used to create the embryo give their consent in putting forth their embryo to be used in valuable, potentially life-changing scientific research.
Now, with iPSCs, there’s less of a need for human embryos in research which has alleviated some concern for opponents.
- Embryonic Stem Cells Were First Identified Less Than 40 Years Ago
Scientists only just discovered how to harvest embryonic stem cells from mice in 1981. Just 17 years later, scientists created a method to do the same with human embryos, effectively growing embryonic cells in laboratories. In 2006, a team of scientists in Japan successfully reprogrammed adult cells to create iPSCs, putting to rest some of the ethical and moral debates surrounding stem cell research. Today, clinical trials using stem cells are being approved around the world, including Celixir’s own Heartcel in Europe and the US.
- Stem Cells Have Been Proven to Work
As more and more trials are being approved, it’s becoming increasingly evident that stem cell therapies are effective in treating a range of diseases and conditions. While, as mentioned, there has been controversy surrounding stem cell therapies on a moral basis, the success of trials in both animals and humans suggests that stem cells do work. New, exciting research from Universities and biotech companies around the world is being published weekly. For a summary of 11 stem cell studies set to revolutionize healthcare, click here.
- You Can Preserve Your Child’s Stem Cells
Stem cell banks can store stem cells derived from amniotic fluid or umbilical cords for future use. It’s easy to collect, with no risk for the mother or the child, and can be used to treat over 80 diseases. Cord blood can treat both children and adults, although adults need two cord blood samples compared to just the one that children need.
Cord Blood banks now exist in every developing country and within most developing nations, with approximately 500 operating worldwide. Today, the question isn’t so much should you store the cord blood of your offspring for future use, but whether to support public vs. private cord blood banks.
- It Takes Time for Treatments to be Approved
Recently, regulatory bodies like the FDA have released new guidelines to ensure the delivery of safe and effective regenerative medicine advanced therapies (RMATs). As it stands, stem cell-derived products that are minimally altered and that are used for the same purpose in both donor and host do not need premarket approval. Products that do not fall under this umbrella, though, are regulated as drugs, biologics or devices. In this case, the drug must be tested on animals before the company can submit an application to the FDA. The FDA then reviews the application to assure that the proposed studies/clinical trials do not place human subjects at unreasonable risk of harm.
From there, the drug is approved for Phase 1 testing with 20-80 healthy volunteers. Phase 1 emphasizes safety. Phase 2 then involves hundreds of patients with a focus on effectiveness. Afterward, the FDA and sponsors discuss how large-scale studies in Phase 3 will begin. Phase 3 involves thousands of patients and studies different populations, dosages, and the use of the drug combined with other drugs. After another review meeting with the FDA and sponsors, the company submits an NDA, formally asking for approval for marketing in the US. The FDA has 60 days to review the application. In June of this year, the FDA approved Celixir’s Investigational New Drug application (IND) for Heartcel, an exciting and significant regulatory milestone that will allow Celixir to conduct potentially pivotal trials with Hearcel.