The Possible Future Treatment of Sickle cell anemia"
Source: "The Possible Future Treatment of Sickle cell anemia" Tag: sickle cell anemia pathophysiology at
geneticdisorders.info
geneticdisorders.info
Authors;
* Professor Pranab Kumar Bhattacharya MD(cal) FIC path(Ind)- Professor and Head,* Miss Upasana Bhattacharya- daughter of Prof.PK Bhattacharya ** Mr. Rupak Bhattacharya Bsc(cal) MSc(JU),** Mr Ritwik Bhattacharya B.com(cal); **Miss Rupsa Bhattacharya** Mr .Soumak Bhattacharya BHM, MSc-student(PUSHA) New Delhi;All of Residence- 7/51 Purbapalli, Sodepur, Dist 24 parganas(north) kol-110, ***Oindrila Mukherjee*** Mrs. Dalia Mukherjee BA(Hons) cal;*** Mr Debasis Mukherjee Bsc(cal). Dr. Ranu Roy Biswas MD(cal) Asst Professor. *Dr. Anuradha De DCP(cal) MD(AIIMS) Associate Professor; *Dr. Soma Das MBBS(cal) DCH(cal) Demonstrator; **
· Affiliation-* Department of Pathology , Calcutta School of Tropical Medicine, CR Avenue ,Kol-73; W.B; India ** 7/51 Purbapalli, Po-Sodepur; Dist 24 pargnas( West Bengal) Kol-20*** Swamijinagar, South Habra, 24 Parganas(north) West Bengal.
Scientists have learned a great deal about sickle cell anaemia during the past 30 years - what causes it, how it affects the patient, and how to treat some of its complications. They also have begun to have success in developing drugs that will prevent the symptoms of sickle cell anaemia and procedures that should ultimately provide a possible cure.
Some researchers are focusing on identifying drugs that will increase the level of fetal hemoglobin in the blood. studies have shown that these people have less severe cases of the disease. Fetal hemoglobin seems to prevent
sickling of red cells, and cells containing fetal hemoglobin tend to survive longer in the bloodstream.
Hydroxyurea appears to work primarily by stimulating production of fetal hemoglobin. There is some evidence that administering hydroxyurea with erythropoietin, a genetically engineered hormone that stimulates red cell
production, may make hydroxyurea work more better. This combination approach offers the possibility that lower doses of hydroxyurea can be used to achieve the needed level of fetal haemoglobin to reduce side effects of both toxic drugs to search for safer agents that are just as effective.'Butyrate,- a simple fatty acid tha is widely used as a food additive, is
also being investigated as an agent that may increase fetal hemoglobin production. Clotrimazole, an over-the-counter medication commonly used to treat fungal infections, is under investigation as a treatment to prevent the loss of
water from RBC that contributes to sickling. Bone marrow transplantation has been shown to provide a cure for severely affected children with sickle cell disease. Researchers are working on techniques to f urther reduce some of the risks of bone marrow transplantation for patients with sickle cell disease. The ultimate cure for sickle cell anemia may be gene therapy. Gene therapy offers enormous promise as a potential curative therapy for SCD, but concerns over the safety of random genomic insertion must first be resolved . Pre-clinical studies in mice have provided the proof of principle that transduction of bone marrow stem cells with lentiviral vectors that express a beta-globin gene can prevent Hb S polymerization in vivo. The wide range of abnormalities engendered by the sickle cell mutation offers several other opportunities for therapeutic interventions. For example, the NIH Road Map is supporting ongoing investigations in which high-through put screening approaches are used to discover novel low -molecular-weight compounds that can alter key aspects of the disease, I ncluding hemoglobin polymerization, expression of Hb F, and leukocyte adhesion. Current clinical trials are evaluating the efficacy of Ca2+- sensitive Gardos channel inhibitors , with or without hydroxyurea, in preventing dehydration of erythrocytes . Vasoactive drugs (e.g., NO, sildenafil, endothelin antagonists) are being evaluated for the treatment i f pulmonary hypertension. Statins are of potentially great interest since they can increase NO production and reduce leukocyte adhesion . Selective antagonists & Intravenous gammaglobulins are currently under clinical evaluation following a study demonstrating a dose-dependent reduction in leukocyte adhesion and in the number of interactions between rbc and wbc, accompanied by improvements inmicrocirculatory blood flow and survival of sickle transgenic mice . Almost a century after SCD was first Scientists have learned a great deal about sickle cell anaemia during the past 30 years - what causes it, how it affects the patient, and how to treat some of its complications. They also have begun to have success in developing drugs that will prevent the symptoms of sickle cell anaemia and procedures that should ultimately provide a possible cure.
Some researchers are focusing on identifying drugs that will increase the level of fetal hemoglobin in the blood. studies have shown that these people have less severe cases of the disease. Fetal hemoglobin seems to prevent
sickling of red cells, and cells containing fetal hemoglobin tend to survive longer in the bloodstream.
Hydroxyurea appears to work primarily by stimulating production of fetal hemoglobin. There is some evidence that administering hydroxyurea with erythropoietin, a genetically engineered hormone that stimulates red cell
production, may make hydroxyurea work more better. This combination approach offers the possibility that lower doses of hydroxyurea can be used to achieve the needed level of fetal haemoglobin to reduce side effects of both toxic drugs to search for safer agents that are just as effective.'Butyrate,- a simple fatty acid tha is widely used as a food additive, is
also being investigated as an agent that may increase fetal hemoglobin production. Clotrimazole, an over-the-counter medication commonly used to treat fungal infections, is under investigation as a treatment to prevent the loss of
described, we may be towards the dawn of a new era in which a physician one day might be able to use genetic information to select one or more drugs that target specific aspects of disease pathophysiology that are
relevant to a particular patient with SCD
Copy Right- Copy Right of this article belongs to Professor Pranab kumar Bhattacharya and his first degree relatives only as per copy right act & rules of Intellectual Property Right Rules 3D/107/1201 a,b/ RDF Copy Right rules/ SPARC copy Right rules-2006/ and Protect intellectual Property Right(PIP) copy right rules of USA-2012.Please do not Infringe and be enough careful for your own safety if you are not direct Blood relation to prof Pranab Kumar Bhattacharya . No person, No NGOS [ except the authors& first degree relatives] in the state of West Bengal or in any states of India or in any abroad countries are authorized to use this article, with any meaning full, scientific sentences or with scientific and meaning full words laid out in this article either in the class room/ or in mass teaching programme including CME or in any form what so ever it is with any content of this article or while in writing any book or for his/her personal/ home use, or collective works or for any future Research or implementation as a policy matter or,[ except the authors ]or by Xeroxing and distributing the article/ or by printing/saving/broadcasting the article from any website of internet services,displayed without proper copy right clearance from the authors or from his family members or future copy right owner by written forms.
relevant to a particular patient with SCD
Copy Right- Copy Right of this article belongs to Professor Pranab kumar Bhattacharya and his first degree relatives only as per copy right act & rules of Intellectual Property Right Rules 3D/107/1201 a,b/ RDF Copy Right rules/ SPARC copy Right rules-2006/ and Protect intellectual Property Right(PIP) copy right rules of USA-2012.Please do not Infringe and be enough careful for your own safety if you are not direct Blood relation to prof Pranab Kumar Bhattacharya . No person, No NGOS [ except the authors& first degree relatives] in the state of West Bengal or in any states of India or in any abroad countries are authorized to use this article, with any meaning full, scientific sentences or with scientific and meaning full words laid out in this article either in the class room/ or in mass teaching programme including CME or in any form what so ever it is with any content of this article or while in writing any book or for his/her personal/ home use, or collective works or for any future Research or implementation as a policy matter or,[ except the authors ]or by Xeroxing and distributing the article/ or by printing/saving/broadcasting the article from any website of internet services,displayed without proper copy right clearance from the authors or from his family members or future copy right owner by written forms.
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