Rapamycin, also known as Sirolimus is a drug normally used to prevent organ transplant rejection. It has use as a coating for coronary stents and treating a rare lung disease as well. It is also known in the pharmacy market by its trade name, Rapamune. It is also famous as a cancer drug.
Isolated for the first time in 1972 from the samples of a bacterium found on Easter Island, Sirolimus was first used as an anti-fungal agent. Subsequently, it was discovered that this wonder drug had tremendous immunosuppressive and antiproliferative properties. This made the physicians abandon the use of the drug as an anti-fungal agent.
The role of Mikhail Blagosklonny:
Mikhail Blagosklonny, a professor of Oncology at the Rosewell Park Cancer Institute in Buffalo, New York, has conducted tremendous research on cancer and its therapies. Additionally, in his role as Editor-in-Chief of Oncotarget, he has conducted and published extensive research on the subject of aging. He is renowned for advocating the use of this cancer drug, Rapamycin for the purpose of increasing the life span of the human being. His contribution to this field of science is immense and unparalleled.
Reasons why Rapamycin is one of the best medicines we’ve never heard of:
• Immunosuppressant properties:
In the case of organ transplants, the principal reaction of the human body is to reject the new transplanted organ. In such an eventuality, the transplantation becomes a failure. Hence, doctors prescribe anti-rejection drugs also known as calcineurin inhibitors or immunosuppressive drugs. These drugs can have an adverse effect on the kidneys over a long period. It is here that Rapamycin scores over the normal immunosuppressive drugs. Rapamycin displays a lower level of toxicity towards kidneys.
• Best for treating patients with hemolytic-uremic syndrome:
This is a rare disease caused due to a combination of anemia, kidney failure, and a low platelet count. This leads to a major renal failure. You can undergo dialysis on a permanent basis. The only corrective measure is to have kidney transplantation. When you do so, you have to take calcineurin inhibitors. Under such circumstances, there is every chance that the transplanted kidney can contract the same disease. The use of Rapamycin reduces this probability a great deal because of its low level of toxicity towards kidneys.
• Treatment of Lymphangioleiomyomatosis (LAM):
The name of the disease can sound frightening but the disease is usually not. This is because it is an extremely rare lung disease usually affecting women of childbearing age. Rapamycin has the distinction of being the primary drug approved to cure this ailment. LAM infiltrates the lung tissue with the mutations of the Tuberous Sclerosis Complex gene (TSC2) thereby activating the mTOR signaling gateway. This factor results in the release of the Lymphangiogenic growth factors. The importance of Rapamycin is that it blocks this gateway.
• Coronary Stent Coating:
One of the major applications of Rapamycin is in the coating of the coronary stents. They do it to prevent the occurrence of re-stenosis in the arteries following the procedure of balloon angioplasty. Studies have shown that patients who have used this coating of Sirolimus show a lower rate of the occurrence of re-stenosis. The main advantage of using these Sirolimus coated stents is that you need not repeat the procedure often.
• Treatment of Cancer:
The antiproliferative properties of Sirolimus come to the forefront while treating cancer patients. When taken in right doses, Rapamycin has the capacity of enhancing the immune response of the body towards the tumors. Alternatively, it promotes tumor regression as well. Therefore, this drug has great utility in reducing the risk of cancer in patients with organ transplants.
• Treatment of Tuberous Sclerosis Complex (TSC):
TSC is a congenital disorder that can cause benign tumor growth in areas such as brain, heart, kidneys, and other organs as well. Research showed that mTOR inhibitors play a great role in suppressing TSC in children as well as in adults. Studies also showed that the tumors started to grow again after discontinuation of Rapamycin treatment.
• Treatment of Facial angiofibromas:
More than 80% of people with TSC suffer from Facial angiofibromas. This is a kind of a rash or spots usually found on the nose and cheeks of the affected person. You can observe the formation of the rash in the shape of a butterfly. In fact, they consist of fibrous tissues and blood vessels. Dermabrasion or laser treatment is a cosmetic cure. Rapamycin is a better cure. You can use this drug in the form of creams, gels, or ointments to cure this problem.
• Increase longevity of the person: (Research in progress)
Dr. Mikhail Blagosklonny has conducted tremendous research in this subject. Carrying out tests on mice, he has proved that the use of Rapamycin on aging mice has resulting in enhancing the ability of these mice to deal with tuberculosis. Initial tests on humans have shown an immunological rejuvenation in elderly patients when administered a dose of Rapamycin prior to an influenza vaccination. However, there is no conclusive evidence that this drug can increase the longevity of the person. Dr. Blagosklonny is of the firm belief that this drug can in fact increase human longevity. The scientists are in the process of conducting tests on the marmoset monkey at present.
• Treatment of Alzheimer’s disease: (Research in progress)
Research shows that Sirolimus has a record of reducing the brain lesions and preventing cognitive defects in mice. This can be a possible cure for the Alzheimer’s disease.
• Treatment of Muscular Dystrophy: (Research in progress)
Researchers have conducted tests on mice by administering nanoparticles coated in Sirolimus. The resultant effect was an increase in the grip strength of the mice by about 30%. The tests also showed a positive effect by increasing the cardiac functions in mice. Hence, this can be a potential cure for Muscular Dystrophy.
• Treatment of Systemic Lupus Erythematosus (SLE): (Research in progress)
SLE patients can develop resistance to immunosuppressant drugs. Sirolimus acts by blocking the activation of the mTORC1 resulting in the suppression of mTORC2. This can result in a cure for the SLE. Research has shown that the use of Rapamycin has resulted in reversing the activation of mTORC1 in mice. This is a positive step towards finding the right cure.
We have seen 11 ways reasons why Rapamycin can prove to be one of the best medicines one has never heard of. Of course, research is still going on in certain treatment procedures but the initial results are encouraging enough to have high hopes. Dr. Mikhail Blagosklonny has played a significant part in pioneering the use of Rapamycin to treat a variety of ailments including advocating the increase in the human life span.