Whilst the extent of our medical knowledge is impressive, if you look closely, we have so much yet to learn.
In reality, we are only putting on bandaids everywhere in the hope that the body can do the rest. The advent of stem cells, though, changed that. Suddenly, we could grow organs and program cells for a specific function. With so many complex and debilitating diseases out there that can shut down your normal way of being, it’s little wonder that cell therapy is on the front page of most medical news these days.
Stem cells are the body’s raw materials that can originate, or give rise to, other specialized cells, such as muscle, brain, nerve, or organ cells. This, very simply stated, boosts the body’s own healing abilities by triggering certain cells to grow and regenerate, depending on the disease.
- An evolution in technologies
- Range of applications
- Ongoing research
Cell therapies use different types of stem cells. These are embryonic stem cells, adult and induced pluripotent stem cells. To give you an idea, adult cells can only give rise to one specific set of cell types whereas the other 2 types can become many different types of cells.
An example of adult stem cells are blood cells or the mesenchymal stem cells found in the bone marrow and fat tissue. These are often used in regenerative medicine such that cell therapy is used for neurological diseases because the cells specifically target neurons and the immune system.
Earlier cell therapy in around 2017, involved CAR-T cells taken from a patient’s blood to attack cancer cells. However, they didn’t work on all types of cancer cells. This technology still exists today but you’ll also hear about gene editing, donor cell therapies, and personalized therapies. The idea is to tailor the stem cell gene therapy solution to the patient rather than hope and pray for the best.
The list of diseases and neurological injuries being researched for cellular therapies is vast. Everything from cancer to Parkinson’s, autism and spinal diseases as well as sports injuries are being investigated for cell therapy. Essentially, these are debilitating disorders that often involve intrusive treatment. That’s why cell therapy is bringing potential solutions and hope to those affected, including their families.
Whilst the FDA has approved cell therapy trials, there are few treatments that have fully received the complete approval. Therefore, today, anyone exploring cell therapy should check with the clinic what FDA file number the treatment is registered against. This gives people reassurance that the process is underway.
The medical industry has many challenges but these are the main ones for cell therapy:
- The regulatory environment
- Limited large scale clinical trials
- Specialist administration
The pharmaceutical and biotechnology world has just faced its biggest challenge yet. The recent pandemic shows how increased collaboration can reduce timelines for approvals when it comes to vaccines. Now, the industry is under pressure to implement those lessons learnt and to keep evolving to become nimbler and more effective.
Stem cells were traditionally sourced for cell therapy from embryos. This opened up a complex ethical debate which is why there were very limited clinical trials, and for good reason.
Since then though, research has uncovered a more practical source of stem cells, mainly from the patients themselves. For many cell therapy programs, stem cells can come from muscle or fat. Then again, there are also willing donors on the market. That means that today we can actually conduct a clinical trial much more easily which is good news for the cell therapy progress we hope for.
Another limitation of cell therapy is that it needs specialist training before it can be administered. It isn’t intrusive and usually only involves only a few needles but the specialists still need to know what they’re doing. On the other hand, researchers hope to get to the point where cell therapy could even be administered at home or at least as an outpatient.
Researchers and doctors alike are all excited about the recent acceleration in research in cell therapy. This could further enhance the following trends:
- Scalable manufacturing
- Gene editing therapies
- T-cell therapy
Currently, the global manufacturing footprint is insufficient to meet the expected demand for cell therapy. This is partly due to funding issues and partly because research was slower until now. However, there is an exciting trend going on with pharmaceuticals starting to partner more competitively with manufacturers to enable large-scale manufacturing capabilities.
Whilst any cell therapy clinical trial usually has very promising results, this isn’t the case for every disease in practice. So, for example, it didn’t always work for cancer. Nowadays though, gene editing is improving accuracy and making the cells more specific to target the disease in question. This is making them much more effective at destroying, for example, specific tumors.
The industry expects greater collaboration amongst its key players, with the regulators, alongside accelerated research programs. Overall, gene and cell therapy are exploding to treat a wide range of diseases. Never before has biotechnology been so exciting nor has it ever leveraged our own healing abilities to this level. It’s a new revolution that will take us beyond our wildest dreams.