Homoeopathy in Hemophilia

 

http://homoeopathyinhaemophilia.com/

Prof. (Dr.) Tapas Kumar Kundu

B.H.M.S (Cal), P.G.D.P.C., M.S. (Ibms)

Professor of Medicine

Motiwala Homoeopathic.Medical College and Hospital, Nashik, Maharashtra, India.

• Recipient of Rajiv Gandhi Vidya Gold Award-2012
• Recipient of Best Teacher Award of MUHS-2013.

Consulting Homoeopath and Psychological Counsellor

HIH Nashik (Chapter):- 1-A, Sairaj Apartment, Behind Ichchhamani Lawns, Upnagar, Nashik (Maha)- 422006, India.
Camp: - First Sunday of Every Month
Time: - 10 a.m. – 2 p.m.
Cell: +91-9373908112
E-mail: dr_kundu2002@yahoo.co.in

A Letter From A Hemophiliac: Dear Lady Gaga, I've Never Drank Blood In My Life

Matthew Gates
Broadcast Journalism Student at Hampton University

 


"I'll have a cheeseburger with a cup of blood on the side," said no hemophiliac, ever. Let me start this off by saying I am not writing out of anger or spite. During your recent interview on The Tonight Show Starring Jimmy Fallon when asked about your character on American Horror Story: Hotel, you said, "Mine in particular is a hemophiliac or "vampire," so I need to drink blood in order to survive." I understand that you meant no harm by this comment, but hear me out. I was born with this bleeding disorder and have never seen fangs in my mouth or have drank blood. I've come to understand that the only people that will truly comprehend the extent of hemophilia, are people living with it, so please don't feel singled out.
Hemophilia is a genetic disorder caused by missing or defective clotting protein within the blood. According to the US Centers for Disease Control and Prevention, Hemophilia occurs in approximately 1 in 5,000 live births. Currently, the exact number of people living with hemophilia in the US is not known. However, the CDC estimates there to be about 20,000 males with Hemophilia within the United States based on expected births and deaths since 1994. I have been an active member of the Hemophilia community for 20 years, attending conferences and seminars, and have seen zero pairs of fangs.
When we have any form of physical trauma, we have a risk of profuse internal and or external bleeding. The main medication used to treat the various forms of Hemophilia is a medication we call Factor. This medicine is administered intravenously, through a vein. My fellow hemophiliacs and I practice a routine regimen called prophylaxis, pushing us to take this medicine three times a week. Let me reiterate, we don't have to drink blood, we have to take shots of our clotting Factor, to survive.

Although our medicine is advanced today, the road to clean and reliable medicine was not easy. There was a time in which the demand for our hemophiliac product was so high it overpowered the quality of it. According to the CDC, until 1992, all factor replacement products were made from human plasma. Within B.L. Evatt's, World Federation Of Hemophilia's publication of, " The Tragic History Of Aids In The Hemophilia Population, 1982-1984," it is highlighted that our medicine was once infected with blood-borne diseases, due to unsuccessful screenings of the collected blood. This crisis unconsciously affected many innocent hemophiliacs, who wanted nothing more than to be healthy. I personally have never had a chance to meet my hemophiliac grandfather, who I was named after, because he died as a result of being effected by this crisis.

As I've said before, I'm not writing this out of anger or spite. The way Hemophilia is perpetrated within the media, school textbooks, medical world, etc. is very skewed. Gaga, I will always support you as an artist and overall person. It is understood that you spoke on Hemophilia the best way you could. Hopefully this will give you some insight to Hemophilia, our lack of fangs, and treatment for the disorder. The next time I take my clotting Factor, I'll be sure to throw my paws up.

2015 Reno and Las Vegas Hemophilia Walks Raise Record Funds! October 5, 2015 By Mitch Truswell

Walks and 5Ks held in Reno and Las Vegas brought in a record $61,000 in donations to the Nevada Chapter of the National Hemophilia Foundation.


LAS VEGAS - Walkers and runners in Reno and Las Vegas in September raised a record amount of donations for the Nevada Chapter of the National Hemophilia Foundation, surpassing last year’s record amount.
More than $61,000 was raised through the efforts of walk participants and supporters for Nevada’s only nonprofit organization advocating on behalf of the inherited bleeding disorders community.
The majority of the funds will allow the chapter to continue efforts to send 150 kids with inherited bleeding disorders, along with their siblings, to a special medically supervised summer camp at no cost to them.
There is no cure for hemophilia or von Willebrand Disease, meaning those affected rely on life saving medicine, called factor, to prevent bleeds. While extremely effective, factor medication is expensive. The Chapter will also use walk funds to help clients without medical insurance, or without the financial ability, to get their needed medication.
“The support we continue to receive from across Nevada is absolutely humbling,” said Kelli Walters, Executive Director of the Nevada Chapter of the National Hemophilia Foundation. “We cannot continue our efforts to improve the lives of those with inherited bleeding disorders without the community’s involvement and help!”
The Reno Hemophilia Walk & 5K event was held September 12, 2015 at Virginia Lake Park. The Las Vegas Hemophilia Walk & 5K was held September 26, 2015 at Floyd Lamb Park at Tule Springs. Both walks were sponsored by the Hemophilia Treatment Center of Nevada which provides diagnosis, treatment, medical referrals and comprehensive clinics to all people with inherited bleeding and clotting disorders in Las Vegas, Reno and throughout the state. It is the only federally recognized hemophilia treatment center (HTC) in the state.

About Nevada Chapter of the National Hemophilia Foundation
NHF Nevada was founded in 1990 and helps meet the vast education and support needs of the bleeding disorders community in Nevada. There are approximately 20,000 people living with Hemophilia in the United States. It is an expensive disease where life-long management of Hemophilia places a large financial burden on individuals and families. Additionally, NHF Nevada provides services to those affected by von Willebrand disease, and clotting disorders such as Thrombophilia, which causes the blood to clot excessively. Every dollar raised at this event will help the bleeding disorder community in Nevada.
Filed Under: Press Release Wire

FDA Approves Octapharma’s NUWIQ To Treat Hemophilia A

by PPN Staff 

The FDA has approved Octapharma’s NUWIQ, antihemophilic factor (recombinant), an IV therapy for adults and children living with hemophilia A. The approval includes on-demand treatment and control of bleeding episodes, routine prophylaxis to reduce the frequency of bleeding episodes and perioperative management of bleeding.

NUWIQ is the first B-domain–deleted recombinant factor VIII (FVIII) derived from a human cell line, not chemically modified or fused with another protein, designed for the treatment of patients with hemophilia A, congenital FVIII deficiency.

Up to 16,000 individuals in the United States have hemophilia A. Although there are already therapies for hemophilia A, significant challenges remain, including development of inhibitors and the need for multiple infusions on a prophylactic basis, the company said.

The initial global clinical study program for NUWIQ commenced with a pharmacokinetic (PK) evaluation in an open-label, multicenter clinical trial of 22 previously treated patients (PTPs). These patients consisted of 20 adults and two adolescents. In this study, NUWIQ demonstrated a mean half-life of 17.1 hours using a one-stage clotting assay in adults. NUWIQ was also evaluated in children using a one-stage clotting assay with a mean half-life of 11.9 hours for ages 2 to 5 years and a mean half-life of 13.1 hours for ages 6 to 12 years. These PK results for mean half-life were longer than earlier generations of recombinant FVIII products currently available in the United States.

The second set of global clinical studies for NUWIQ also evaluated overall efficacy and tolerability in three prospective, open-label clinical studies in PTPs with severe hemophilia A. From all clinical studies, 135 patients were treated with NUWIQ, including 74 adults, three adolescents between ages 12 and 17 years and 58 pediatric patients between ages 2 and 11 years. All these patients were treated with a total of 16,134 infusions over 15,950 exposure days using NUWIQ.

In a study of 32 adults, overall prophylactic efficacy of NUWIQ for spontaneous bleeds was rated as excellent or good in 92% of patients. In a study of 59 children, prophylactic efficacy for spontaneous bleeds was rated as excellent or good in 97% of patients. The mean annualized bleeding rates (ABR) for spontaneous bleeds during prophylaxis were approximately 1.5 in children and 1.2 in adults. For hemophilia A patients receiving NUWIQ prophylaxis compared with on-demand treatment, the ABR was reduced 96% for adults and 93% for children.

Treatment of breakthrough bleeds during NUWIQ prophylaxis was rated as excellent or good in all of 30 (100%) bleeds in adults and 89 of 108 bleeds (82%) in children. For on-demand treatment with NUWIQ in 20 adults and two adolescents, efficacy for the treatment of bleeds was excellent or good in 931 of 986 bleeds (94%). Overall efficacy in surgical prophylaxis was rated excellent or good in 32 of 33 procedures (97%) using NUWIQ.

In all clinical studies, NUWIQ had a total of seven reported adverse events. Each of these adverse events occurred one time with a rate of 0.7% across all 135 patients. These events were parathesia, headache, injection site inflammation, injection site pain, back pain, vertigo and dry mouth.

Octapharma USA will offer hemophilia A patients educational and support services in connection with the introduction of NUWIQ, which should be available by early 2016.

NUWIQ is not indicated for the treatment of vonWillebrand disease.

“We are pleased that the treatment options for adults and children with hemophilia A continue to advance with ever more innovative therapies being approved for the U.S.,”  said Val Bias, CEO of the National Hemophilia Foundation. “The continued commitment to develop life-enhancing products for the bleeding-disorders community is absolutely vital. Empowering patients and providers with treatment options, as well as education and support programs, is extremely important to people living with hemophilia A.”

—From company press materials

A Hope For Hemophilia Patients

 For the first time, chromosomal defects responsible for hemophilia have been corrected in patient-specific iPSCs using CRISPR-Cas9 nucleases. Asian Scientist Newsroom | August 4, 2015 | In the Lab

                    

AsianScientist (Aug. 4, 2015) - Sufferers of hemophilia live in a perpetual state of stress and anxiety: their joints wear down prematurely and they have bleeding episodes that feel like they will never end. Their bodies lack the ability to make the clotting factor responsible for the coagulation of blood so any cut or bruise can turn into an emergency without immediate treatment.

Hemophilia A occurs in about 1 in 5,000 male births and almost half of severe cases are caused by identified chromosomal inversions. In a chromosomal inversion, the order of the base pairs on the chromosome are reversed so the gene doesn’t express properly and the sufferer lacks the blood coagulation factor VIII (F8) gene, which causes blood to clot in healthy people.

A Korean team led by director of the Center for Genome Engineering Kim Jin-Soo, Institute for Basic Science (IBS) and Professor Kim Dong-Wook at Yonsei University has experimented with hemophilia A-derived induced pluripotent stem cells (iPSCs) and hemophiliac mice and found a way to correct this inversion and reverse the clotting factor deficiency that causes hemophilia A.

This was the first time that iPSCs—which possess the ability to change into any cell type in the body—have been used in such a procedure. The researchers first collected urinary cells from patients with chromosomal inversions causing haemophilia, transforming them into iPSC cells. Then, they used CRISPR-Cas9 nucleases (Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR associated protein 9) to flip the F8 gene around, allowing it to be read correctly.

The corrected-iPSCs were then induced to differentiate into mature endothelial cells which expressed the F8 gene in the correct orientation. To verify that the process worked, the endothelial cells with the inversion-corrected genes were transplanted into F8 deficient mice (mice with hemophilia A) and the mice started producing the F8 clotting factor on their own, which essentially cured them of hemophilia A.

        “We used CRISPR RGENs [RNA-guided engineered nucleases] to repair two recurrent, large chromosomal inversions responsible for almost half of all severe hemophilia A cases,” Kim Jin-Soo said.

        “To the best of our knowledge, this report is the first demonstration that chromosomal inversions or other large rearrangements can be corrected using RGENs or any other programmable nuclease in patient iPSCs,” Kim Dong-Wook added.

Importantly, the studied showed no evidence of off-target mutations resulting from the procedure, suggesting that it was only the desired parts of genome were changed.

These findings open the door for further testing and if the results are anything like the mice trials, the future of this treatment looks promising.

The article can be found at: Park et al. (2015) Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9

Source: Institute for Basic Science; Photo: Shutterstock. Disclaimer: This article does not necessarily reflect the views of AsianScientist or its staff.

https://www.facebook.com/Hemophilia-Awareness-446712415406740/timeline/?ref=bookmarks 

Roche hemophilia drug wins fast-track FDA designation

The logo of Swiss pharmaceutical company Roche is seen at a plant in the central Swiss village of Rotkreuz in this November 6, 2013 file photo.

Reuters/Arnd Wiegmann

Roche said on Friday it had won breakthrough therapy designation from the U.S. Food and Drug Administration for an experimental hemophilia medicine, aiming for a piece of the $11 billion hemophilia drug market.

The Swiss drugmaker said its U.S.-based Genentech unit's ACE910 secured the fast-track designation as the company prepares separate Phase III trials in 2015 and 2016, the first in patients with hemophilia A with factor VIII inhibitors and the second for patients without inhibitors.

It represents a threat to more traditional treatments from Novo Nordisk and Baxalta, the target of a $30 billion takeover attempt by Shire.

Hemophilia A is a rare genetic disorder that prevents blood clotting. Patients receive lifesaving infusions of clotting factors, but development of inhibitors in many of those being treated interferes with efforts to control their bleeding.

With the market for hemophilia medications expected to grow to $11 billion next year, Roche's ACE910 drug is closely watched because it could change the way the disease is treated.

"FDA has granted breakthrough therapy designation for ACE910, recognizing an unmet need for patients with inhibitors and the promise of these early data," Sandra Hornung, Roche's chief medical officer, said in a statement.

Last year, Roche said early data indicated encouraging reduction in bleeding rates in all patients.

In 2012, U.S. regulatory changes created the breakthrough therapy designation, allowing the FDA to expedite development and review of drugs whose preliminary clinical evidence indicates substantial improvement over existing therapies.

(Reporting by John Miller. Editing by Jane Merriman)

CDC Findings Suggest Hemophilia Carriers Vulnerable to Joint Damage

In a newly highlighted study, the US Centers for Disease Control and Prevention (CDC) concluded that hemophilia carriers showed evidence of joint abnormalities as early as the pre-teen years regardless of the severity of bleeding symptoms. Results of the study were first published in “Females with FVIII and FIX Deficiency have Reduced Joint Range of Motion,” in August 2014 in the American Journal of Hematology. The lead author was Robert Sidonio, MD, MS, Department of Pediatrics, Division of Hematology/Oncology, Vanderbilt University Medical Center in Nashville, TN.

To learn whether hemophilia carriers reported joint bleeding and showed physical signs of joint damage or destruction, CDC looked at joint abnormalities among 451 women presumed to be hemophilia carriers aged 2-69 years. The women were enrolled in a national public health tracking project called the Universal Data Collection (UDC) system. The UDC was created in 1998 by the CDC, in cooperation with the federally funded hemophilia treatment center (HTC) network, to collect vital health information on individuals with bleeding disorders in the US.

Data for the study were gathered by either an HTC physical therapist or other trained healthcare provider, who collected information on specific participant characteristics, such as race/ethnicity, income and educational level (demographic information), as well as information on bleeding and infectious disease history, and range of movement measurements in five joints (right and left shoulders, elbows, hips, knees and ankles).

CDC’s most prominent findings were:

• The proportion of female hemophilia carriers reporting at least one joint bleed in the last six months increased as the severity of hemophilia worsened
• Approximately one in seven females with mild hemophilia reported at least one joint bleed in the last six months. Mild hemophilia means they have 6% to 40% of normal clotting ability.
• Approximately one in three females with moderate hemophilia reported at least one joint bleed in the last six months. Moderate hemophilia means they have 1% to 5% of normal clotting ability.
• Approximately half of females with severe hemophilia reported at least one joint bleed in the last six months. Severe hemophilia means they have less than 1% of normal clotting ability.

Hemophilia carriers showed signs of joint abnormalities as reflected by reduced joint range of movement, which worsened with increasing levels of severity of hemophilia.

These findings suggest that joint bleeding might be occurring even before a carrier’s adolescent years. Sidonio and his co-investigators also acknowledge that this research is preliminary and that the next step is to document joint disease with X-rays and other tools.

 Source: CDC release dated March 26, 2015