Gene Therapy a Huge Advancement for Rare Blood Disorders like Sickle Cell Disease

An Orphan Disease is considered a 'rare disease' that affects less than 200,000 people in the US. Health News from NPR by Richard Knox on December 07,2013 found that genetic rare conditions that affect children without a immune system like the "Boy in the Bubble" also known as SCID-XI can live a quality of life without germ threats from Gene Therapy. Sickle Cell Disease Anemia is a genetic inherited disorder that affects the blood cells ability to carry oxygen and nutrients to the body and tissues under certain conditions. This is debilitating and recurrence of crisis causes damage and failure of the major organs and joints. Delaware State University, a local researcher, Dr. Kmiech is leading the way in Gene Therapy Research to cure Sickle Cell Disease Anemia. "

"Scientists now report that 8 out of 9 young children given gene therapy for a type of severe combined immunodeficiency disease, called SCID-X1, are alive and living amid the everyday microbial threats that would otherwise have killed them. The oldest is just over 3 years old. Equally important, these children are so far showing no signs of the cancers that developed a decade ago in 5 out of 20 children given an earlier version of the same gene therapy. Those treatment-related cancers temporarily brought such gene therapy experiments to a screeching halt. The latest gene therapy experiment used a "self-inactivating" virus to deliver the corrective gene into the children's blood stem cells. This new viral vector apparently doesn't flip switches that give rise to leukemia, as the old vector did. We're not saying these children are completely free from any risk," says investigator Williams, who was to report on the trial Saturday morning at the American Society of Hematology meeting in New Orleans. "But we're quite happy with the results we have so far and feel confident that the vector is safer in every way we can look." The cost of gene therapy can be costly ($125,000-250,000) but the individual and family must balance the risk versus the benefits and make an informed decision to go forward with Gene Therapy. New technology uses a safer re-engineered virus, 'vector' to correct the gene would to cause leukemia in some cases. "Williams says two of the eight surviving children — one in Paris, the other treated in Boston and living in his native Argentina — had the new gene therapy more than three years ago, when they were infants. They have reached the age when some of the children in the earlier experiment developed leukemia." Children's Hospital of Philadelphia reported in the Associated Press today 'unprecedented success' in treating children with Acute Lymphocytic Leukemia (ALL). A 8-year old with an aggressive form of leukemia was the first to be given Gene Therapy at CHOP and shows no signs of cancer 2 years later. "Within the next year, investigator Williams says they're planning to test gene therapy for sickle cell disease and thalassemia, two much more common blood disorders that together affect hundreds of thousands of people in this country."

Dart Paratransit proposes a Hike in Transit Fees

    According to the News Journal, Dart Para Transit Service Plans to “re-design” the Paratransit route system model that could triple the bus fares for thousands of Delawareans living with disabilities from $2.00-$7.00 one-way.   The Department of Transportation is proposing fare increases and re-structuring to improve the efficiency of services for the disabled and to reduce costs.  Changes to para-transit proposed: 

§  ¾ mile fixed bus route pick-ups

§  1 hour window pick-up times

§  Seniors >65 years of age will no longer automatically qualify

    Transportation Secretary Shailen Bhatt says “the current paratransit model is unsustainable and inefficient.  A program that cost $15 million to run in 2000, now costs $50 million - a 233-percent increase in costs.  When something increases 233 percent in price, it restricts what you can do elsewhere.  For a number of years, we have been wanting to increase fixed-route service. It's been something we've been unable to achieve. We know what the problems are."
Delaware Transit Corp, Rich Paprcka said, “"Right now, we have no flexibility to negotiate. If someone calls up the day before, and we're already overbooked -we end up having to put more people on the buses than we want to.  That's part of what's driving service down. That's why people are complaining about service being late. We're just over capacity."

    Why?  Because, this a serious public-health issue!   According to the News Journal Editorial, “people with disabilities are one of the most undermployed populations in the country ….. being unable to participate in everyday life and meet their personal needs to an even greater extent.  There are hundreds of Delawareans who utilize Paratransit just to live a quality of life independently and within the community.  Having to wait 1 up to an hour safely alone at a fixed bus route could cost people their jobs and/or the inability to perform activities of daily living.    Debra McCann, who sits on the state Developmental Disabilities Council came up with a few solutions.  She hopes to see the agency introduce cheaper, smaller vehicles for riders who don't require wheelchair lifts,  and improve communication between dispatchers, drivers and riders.  Yes, not all people with disabilities require use of a wheel chair but depend on these essential services to live independently, like Autism, Cerebal Palsey, Sickle Cell Anemia, Rheumatoid Arthritis and ect.

The Delaware Transit Corporation will conduct public hearings through Nov. 6 to gather comments on a proposed Transit Redesign Plan. Each will take place from 4 to 7 p.m.:

* Oct. 29 at the Wilmington Doubletree Hotel, 700 King St., Wilmington, 19801
* Oct. 30 at Delaware Technical Community College, 100 Campus Drive, Dover, 19904
* Nov. 5 at the Cheer Community Center, 20520 Sand Hill Road, Georgetown, 19947
* Nov. 6 at the Embassy Suites, 654 S. College Ave., Newark, 19713.
Comments may be submitted through Nov. 15 to RightFitTransit@state.de.us or to P.O. Box 1670,
Wilm, DE 19899-1670, or online at www.DartFirstSate.com/publichearing

     KYLE GRANTHAM/The News Journal

http://pqasb.pqarchiver.com/delawareonline/doc/1443679146.html?FMT=FT&FMTS=ABS:FT&fmac=abeb787d7c93083aa651742ba00a15c3&date=Oct+22%2C+2013&author=Melissa+Nann+Burke&desc=Fare+hike+part+of+%27redesign%27+proposal

Do you think the state should increase the bus fare rates for people with disabilities?  What kind of solutions do you propose to fill the budget gap for an essential service for Delawareans living with disabilities?

Buzzy helps people who suffer from Needle Phobia

      Are you afraid of needles or cringe when you have to get an injection or needle?   Do you suffer from Needle Phobia?  Well, Help is on the Way.  A ER Pediatrician named Dr. Amy Baxter  has won a $1 million grant to study a product  that disrupts pain signals called, "Buzzy."   The Buzzy prototype operates on the "Gate Control" theory which interrupts pain signals traveling up the spinal cord to the brain.  It also distracts and numbs the pain from the cool speed vibrations.
   
    "The vibrating bee attaches to thin ice packs that look like wings and then is placed above the location of the pain. The vibration also dilates nearby veins, Baxter says, making it easier to stick a needle in successfully."

YouTube

    Over the past year, the 4-year-old company's sales nearly tripled, Baxter says
"We haven't advertised," she says. "It's really been patients telling other patients, and moms telling other moms."   Buzzy is available on:   buzzy4shots.com and Amazon.com.

                                                           Why not, Check It Out!

Re-Blogged from NPR News:  "An Innovation for Pain Relief That's Worthy of Some Buzz"
   by Emily Stiner

http://www.npr.org/blogs/alltechconsidered/2013/10/08/230541468/an-innovation-for-pain-relief-thats-worthy-of-some-buzz?utm_medium=Email&utm_source=share&utm_campaign=

Blue Tie Program-Blood Saves Lives

       Journalist, Tom Coyne from the Associated Press reports Blood Banks are declaring fewer critical shortages because there is a dwindling demand for blood.  This is a result of fewer elective surgeries and medical advances in the OR curve blood loss.  Nevertheless, people who suffer from Sickle Cell Disease may require life sustaining blood transfusions to treat sickle cell pain crisis and prevent stroke and other complications.   (News Journal, 10/13/2013, Blood banks change approach as the demand dwindles away).
     Sickle Cell disease affects mostly African-Americans in the U.S. but Hispanics are the fastest growing population in the states.  According to the American Red Cross, African-Americans can provide blood which more closely match the blood of these patients.  But, only 1% of African-Americans donate blood while making up 12-14% of the US population.    

    "Under the program, blood donors who identify themselves as being African-American or Black, can have a “blue tag” attached to their blood collection bag.  This indicates that the donated unit of blood may be matched to a patient with sickle cell disease. If a patient with sickle cell disease does not need the blood within 21 days, or if there is not a match, the donated blood will be available for any patient in need.  Blood types O and B, the blood types of about 70 percent of African-Americans, are the blood types most in demand. Your blood donation can help African-Americans suffering from diseases such as sickle cell anemia, cancer and heart disease."

    Blood Centers of the Pacific reports that African-Americans face other serious medical conditions that may require blood transfusions such as hypertension, kidney disease and premature births.  When blood is closely matched, there is also less chance of developing antibodies from the transufusion which is minimizes the risks for a blood transfusion reaction.  

http://apps.pathology.jhu.edu/blogs/pathology/donors-needed

      Enough said,  Why not Consider being a "Blue Tie Tag Program Donor?  Tell us what are the barriers that prevent the African-American community from donating blood? 

To find out more information, click on:   http://www.redcrossblood.org/info/northernca/importance-african-american-blood-donors

  

Obama Care-Where Do We Go From Here

Get Covered: A one-page guide to the

Health Insurance Marketplace

      Even though the Government is Shut Down, about 100,000 Delawareans still remain uninsured.  The opportunity to purchase medical insurance in the marketplace is a step in the right direction.  To find out what you and/or family are eligible for,

Go to:  https://www.healthcare.gov/.  Remember, the insurance monthly rate you qualify for is based on a number of factors:  household size, income and age.  If you have a pre-existing condition, you will not be denied coverage under the Affordable Healthcare Act. 

     You’re considered covered if you have Medicare, Medicaid, CHIP, any job-based plan, anyplan you bought yourself, COBRA, retiree coverage, TRICARE, VA health coverage, or some other kinds of health coverage.

On April 1, 2014, you’ll have to pay a penalty of $95 per adult, $47.50 per child, or 1% of your income (whichever is higher). The fee increases every year. Some people may qualify for an exemption to this fee. 

    Let us know about your experience applying for health insurance through the Delaware Market Exchange.

All the best,
Dr. Anderson

“....the work goes on, the cause endures, the hope still lives, and the dream shall never die.”  Former Senator, Ted Kennedy

 

2nd Annual Sickle Cell Awareness 5K Walk or Run

Living a Healthy Lifestyle,

    Successfully raising over $15,000 for the 2nd Annual Sickle Cell Awareness 5K Walk or Run held on

September 14, 2013 at Brandywine Park, was truly awe inspiring.  Seeing people in my community of all colors, shapes-sizes and ages walking, running will help increase their overall health and well-being.  African-Americans are at higher risk of developing Diabetes, Hypertension and Heart Disease than Caucasians as well as Obesity.  Continuing to make exercise and healthy eating habits apart of your lifestyle takes discipline and commitment.  But there rewards are great!  Continue to take your family to the park on weekends.   Even if it's just for 30 minutes,  Is Definitely a step in the right direction. 

All the best,
Dr. Nina

                                              http://www.delawareonline.com/apps/pbcs.dll/article?AID=2013309240019&nclick_check=1

Obama Care Starts October 1st

        For the 100,000 Delawareans who are uninsured, October 1st is the first day that one can purchase insurance on the Market through the Exchanges.  Many have seen news articles and commercials vaguely explain how the exchanges work.  The price for Health Insurance policy has not been so easy to understand.  I can honestly say, that I am still perplexed about my options but Health Navigators will be able to assist.  There will be a Gold, Silver and Bronze option and the deductibles will be based your Age and Income level.
 
                            The News Journal article explains the range of the premiums. 
 

Sickle Cell is on the Rise

     In some parts of Africa, the number of babies born with Sickle Cell Anemia is steadily rising. 

    In developed countries, genetic testing are non-existent or may not provided before and/or during pregnancy. Newborn screening to test for sickle cell disease and trait is also non-existent.

   According to NPR news, the vast majority of kids in developing countries don't get diagnosed or treated for sickle cell anemia. Up to 90 percent of these children will die in the first five years of their life.

 The World Health Organization has long recognized the importance of sickle cell anemia as a global health issue. 

 Fast Facts:  1 in 10 African-Americans and 1 in 100 Hispanics in the U.S. carry the Sickle     Cell Trait

http://www.npr.org/blogs/health/2013/07/17/202628070/sickle-cell-anemia-is-on-the-rise-worldwide?utm_medium=Email&utm_source=share&utm_campaign=

Do you think there needs to be increased awareness about sickle cell trait in the U.S. and more genetic testing for all pregnant women?

 

Delaware Compassion Center’s for Marijuana

   Compassion Center for Medical Marijuana in Delaware was approved by Governor Markell despite federal laws from the U.S. Department of Justice that state employees could be subject to prosecution for dispensing Medical Marijuana.

   Sickle Cell Anemia was not a disease was not one of the medical conditions authorized to treat
chronic pain. 

    "Physicians may authorize a medical marijuana use to treat symptoms of cancer, HIV-related illnesses, Hepatitis C, Lou Gehrig’s disease, Alzheimer’s disease or posttraumatic stress disorder. Conditions that cause severe, debilitating pain, wasting syndrome, intractable nausea and seizures also fall under the law."

                                            Governor Markell moving ahead with Medical Marijuana

             Do you think Sickle Cell Disease Anemia should be authorized for Medical Marijuana? Are there other medical conditions that should be considered?




        Picture: News Journal

 

Decriminalizing Drug Abuse-Portugal Style Law

     The use of substance abuse is on the rise and incarceration rates in the U.S. are the   highest in the world.  African-American and Latino men are incarcerated at significantly higher rates for non-violent offenses such as drug possession than whites.  The country of Portugal  in 2001 Decriminalized (reduce or abolish criminal penalties), all drugs use which was a Public Health Crisis.   Under Portugal law, people found guilty of possessing drugs in small amounts are referred for mental health services, consisting of a psychologist, social worker and legal advisor for appropriate medical and rehab treatment.  This approach is offered (but can be refused) without criminal punishment and/or jail time.  The substances listed in the Law 30/2000  (cocaine,  heroin, methamphetamines, marijuana ect.) are illegal in Portugal-but using these drugs are nothing more than a misdemeanor, much the same as a parking  violation.  Did this new policy work in Portugal?   Yes, in the five years after all personal possession of drugs were decriminalized, illegal drug use among teens in Portugal declined and rates of new HIV infections caused by sharing of dirty needles dropped.  The number of people seeking treatment for drug addiction more than doubled.  

          On the contrary, people who suffer with chronic pain from sickle cell disease, arthritis, cancer and others may need pain medications and other treatments just to live a decent  quality of life.  But, there are a very small percentage of people who battle substance abuse from the use of pain killers and other ‘illegal’ drugs.         Oftentimes, they will end up buying medications and/or drugs on the street, doing prison time or at worst die from an overdose.

        It’s time for our legislatures take a look at how we treat people who suffer from Drug Addictions and perhaps provide more mental health and rehabilitative  services instead of a sentence for jail time (Global Commission on Drug Policy, 2012).