Beta-thalassemia

Drug: trifluoperazine (The other result, trihydrochloride, is not a drug.)

Trifluoperazine can treat psychotic disorders and anxiety. It can also treat nausea and vomiting caused by chemotherapy.

Beta-thalassemia is a blood disorder that reduces the production of hemoglobin. In people with beta thalassemia, low levels of hemoglobin lead to a lack of oxygen in many parts of the body. Affected individuals also have a shortage of red blood cells, which can cause pale skin, weakness, fatigue, and more serious complications

1.) “..the results reported herein represent clear-cut evidence for the specific binding of chlorpromazine and trifluoperazine to ferrous human adult haemoglobin..”

http://www.ncbi.nlm.nih.gov/pubmed/?term=low+hemoglobin+trifluoperazine

Paolo, A., Alberto, B., Massimo, C. and Antonio, L. (1999), Stabilization of the T-state of ferrous human adult haemoglobin by chlorpromazine and trifluoperazine. Biotechnology and Applied Biochemistry, 30: 185–187. doi: 10.1111/j.1470-8744.1999.tb00911.x

2.) “…Analogous binding behavior has also been obtained with trifluoperazine binding to haemoglobin and myoglobin”

http://www.ncbi.nlm.nih.gov/pubmed/8910864

Bhattacharyya, J., Bhattacharyya, M., Chakraborti, A. S., Chaudhuri, U. and Poddar, R. K. (1996), Trifluoperazine is More Effective than Chlorpromazine in Releasing Oxygen from Haemoglobin and Myoglobin. Journal of Pharmacy and Pharmacology, 48: 965–967. doi: 10.1111/j.2042-7158.1996.tb06013.x

3.) “…, previous research has shown that low hemoglobin level (anemia) is associated with more depressive symptoms. Such an association could be expected since symptoms of low hemoglobin level also often occur when having depressive (or anxiety) symptoms.”

http://www.sciencedirect.com/science/article/pii/S002239991400021X

4.) “…A majority of the Beta-thalassemia patients were also suffered from mild to severe depression, anxiety, and stress.”

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4779154/

Adib-Hajbaghery M, Ahmadi M, S P. Health Related Quality of Life, Depression, Anxiety and Stress in Patients with Beta-Thalassemia Major. Iranian Journal of Pediatric Hematology and Oncology. 2015;5(4):193-205.

Choroideremia

Choroideremia (CHM) is a rare inherited disorder that causes progressive vision loss, ultimately leading to complete blindness.

Drug: Tipifarnib-P1, Enzalutamide (Xtandi)

Tipifarnib is a class of experimental cancer drugs that target protein farnesyltransferase with the downstream effect of preventing the proper functioning of the Ras (protein), which is commonly abnormally active in cancer.

Enzalutamide is a synthetic, non-steroidal pure antiandrogen used for the treatment of metastatic castration-resistant prostate cancer.

1.) There was a case of sudden loss of vision due to breast cancer metastasis to the eyeball.

http://www.ncbi.nlm.nih.gov/pubmed/25038605

2.) There was a case of a pregnant woman with lung cancer presenting with choroidal metastasis.

http://www.ncbi.nlm.nih.gov/pubmed/25962199

Angioimmunoblastic lymphadenopathy with dysproteinemia (AITL)

(AKA Angioimmunoblastic T-cell lymphoma)

Lymphoma is the most common blood cancer. The body has two main types of lymphocytes that can develop into lymphomas: B- lymphocytes (B-cells) and T-lymphocytes (T-cells). Angioimmunoblastic T-cell lymphoma (AITL) is a rare, aggressive (fast-growing) form of peripheral T-cell lymphoma. 

Drug: Digoxin, Penfluridol

Digoxin can treat heart failure and heart rhythm problems.

Penfluridol is a highly potent, first generation antipsychotic.

1.) There was a case of a dog with right-sided congestive heart failure because of a primary intracavitary myocardial lymphoma.

http://www.ncbi.nlm.nih.gov/pubmed/25708789

2.) “Survivors of childhood lymphoma (CL) have markedly increased risk of developing heart failure.”

http://www.ncbi.nlm.nih.gov/pubmed/24948492

Fanconi Anaemia

FA is a rare genetic disease.People with FA have hbone marrow failure, physical abnormalities, organ defects.90 percent of people with Fanconi anemia have impaired bone marrow function that leads to a decrease in the production of all blood cells (aplastic   anemia ).Most develop cancer(AML)FA is the result of a genetic defect in a cluster of proteins responsible for DNA repair

Treatment : Gene Therapy,Blood Transplant

medcines called growth factors (such as erythropoietin, G-CSF, and GM-CSF) can improve blood counts for a short while.

Carbaprostacyclin also called Prostacyclin, PGI2:” When infused in rabbits or dogs, it inhibits ex vivo platelet aggregation”Paper 1Whittle, B.J.R., Moncada, S., Whiting, F., et al. Carbacyclin - a potent stable prostacyclin analogue for the inhibition of platelet aggregation. Prostaglandins 19605-627 (1980). “It inhibits platelet activation”

Barrett's EsophagusBarrett's esophagus is a serious complication of GERD, which stands for gastroesophageal reflux disease.In Barrett's esophagus, normal tissue lining the esophagus changes to tissue that resembles the lining of the intestine.BE has strong associaton with esophageal adenocarcinoma, a very often deadly cancerIn high-grade dysplasia, the risk of developing cancer might be at 10% per patient-year or greater.

Treatment: laser, RadiofrequencyNSAIDS like aspirinStomach acid supressant: Omeprazole (Prilosec), Esomeprazole (Nexium), Lansoprazole (Prevacid) ,Rabeprazole (AcipHex)

Paper1 “Cediranib 20 mg/day in combination with cisplatin and S-1 or capecitabine was tolerable, with no new toxicities identified, and showed preliminary evidence of antitumour activity.”

Phase I study of cediranib in combination with cisplatin plus fluoropyrimidine (S-1 or capecitabine) in Japanese patients with previously untreated advanced gastric cancer.http://www.ncbi.nlm.nih.gov/pubmed/21853311/

it underwent Phase I clinical trials for the treatment of non-small cell lung cancer, kidney cancer, and colorectal cancer in adults. Is a VEGF inhibitor Vascular endothelial growth factor 

Prostatehttp://www.ncbi.nlm.nih.gov/pubmed/24788563

Ovarian cancer“Cediranib, when given orally with chemotherapy and continued as maintenance, yielded a meaningful in progression-free survival in women with recurrent platinum-sensitive ovarian cancer”

http://www.ncbi.nlm.nih.gov/pubmed/27025186

Cediranib with mFOLFOX6 versus bevacizumab with mFOLFOX6 as first-line treatment for patients with advanced colorectal cancer: a double-blind, randomized phase III study (HORIZON III).http://www.ncbi.nlm.nih.gov/pubmed/22965961

Bacterial infection