Samumed, a biotech founded over 10 years ago in southern California is a company working on exploring the field of regenerative medicine. Their goal is to build drug development platform for restoring the health of any tissue in the human body. So far little has been known about their approach. Let’s take a deeper look at how they plan to achieve their goal.
Broadly speaking their platform is aimed at three distinct classes of disease:
- Degenerative diseases - develop drugs that renew / regrow a given tissue that has degenerated
- Oncology (cancer) - develop drugs that suppress prolifactory effect of cancer on tissue
- Diseases where inflammation is the driving factor - develop drugs that suppress immune system attacking tissues in our bodies
As an example, one degenerative disease today that has no cure is the wearing out of cartilage in the knee joint. Samumed is working on a small molecule drug that regrows the cartilage.
What is driving their discoveries? It all starts with stem cells that in the embryonic stage of human life are all the same (right after sperm meets the egg). Those cells have the capacity to turn into any other cell in the body. As the fetus starts growing those stem cells divide into more of the same cells that then start to communicate with each other and start to partially differentiate into progenitor stem cells.
Progenitor stem cells start to form around week 8 and are the different types of stem cells that then form the base for related kinds of cells in the adult body. For example, mesenchymal progenitor stem cells are the basis for bone and cartilage. Dermal progenitor stem cells are the basis for hair and skin cells. Ependymal progenitor stem cells are basis for neurons, spinal cord hematopoietic progenitor cells are found in bone marrow, blood, such as T cells etc. Epithelial stem cells responsible for cells present in internal organs.
Each type of progenitor stem cells can only further differentiate into “their” type of cells, ependymal stem cells cannot for example create bone cells (that is the job of mesenchymal stem cells). We have about 15 different types of progenitive stem cells in our bodies. All these 15 types of stem cells generate all subsequent cells for tissues in our bodies (while in the womb). By the time the baby is born all original stem cells (non-differentiated embryonic stem cells) are gone. However the progenitor stem cells stay with us embedded in the local tissue of the given part of our bodies until the day we die. Some estimate there are on the order of ~100,000 progenitor stem cells in our bodies. That number is low (when compared to an estimated 37 billion of total cells in our bodies). Their job is to regrow into the given cell type when damage, aging occur to maintain the health of the specific tissues that they are responsible for. For example, when we break a bone, mesenchymal progenitor stem cells start working by first multiplying and then turning into bone building cells to repair the damage. The key biological pathway that regulates this process is called Wnt.
As we age various signaling pathways of the Wnt start to become deregulated; they are no longer functioning optimally. Every time the given pathway gets out of a healthy band there is a disease associated with it: if there’s not enough bone replenishment we end up with osteoporosis, not enough hair being regrown: we go bold, too many colon cells: colon cancer etc. Imbalance of Wnt pathway leads to disease (however that imbalance gets triggered, lifestyle choices, or simply aging).
The goal of Samumed is to develop small molecule drugs that regulate Wnt pathways back into nominal functional bands.
Wnt pathway has been discovered many decades ago. Its existence has been discovered while researching cancer and there’s been numerous attempts at controlling it with drugs before.
How is Samumed then able to leverage regulation of Wnt pathways where others have failed before? Recent interview with Osman Kibar, Samumed CEO, sheds some light [source]
“Targeting Wnt can be tricky,” Kibar said, “because Wnt receptors have multiple purposes and the Wnt pathway is diverse enough, rich enough, that it always finds a way to bypass your drug intervention, which makes the efficacy go down.
What’s more, hitting Wnt receptors hard can backfire. A drug may have its intended effect, but it may disrupt other processes, leading to side effects and safety concerns. So, instead of aiming at Wnt receptors on the cell membrane, Samumed’s drugs target CLK and DYRK, kinase enzymes that are upstream of the Wnt pathway.”
“Our approach to drugging the Wnt pathway is as natural as it gets because we are not fighting the body’s own machinery or telling it to do something it wasn’t doing anyway,” Kibar said. “Instead, once we bring a certain signaling level back into balance, the progenitor stem cells and all the associated biological processes, they kick into autopilot and then the body knows exactly how to restore health.”
“Because we’re not going against the current but rather working with the body’s own machinery, both the hardware and the software, we are able to achieve such safety,” he said.
A recent compassionate use of Samumed’s oncology drug for one terminal pancreatic patient yielded reversal of the disease.
A couple of drugs in Samumed’s clinical trials pipeline:
- Phase 3: Single injection into worn out joints that regrows cartilage.
- Phase 2: Topical cream that regrows hair in balding men.
- Phase 1: Alzheimer’s Disease (pill), Oncology – Solid Tumors, Liquid Tumors (Hematological Malignancies) (pill), Degenerative Disc Disease, Pulmonary Fibrosis, skin lotion that eliminated wrinkles, psoriasis, Tendinopathy (lotion over damaged tendon)
Samumed’s overall approach can be summed up as a “spare parts” business for the human body that regenerates tissue by tissue. With each drug optimized for targeting the particular disease they are “de-aging” the human body part by part.
