Newsletter - April 2014
Cord Blood Stem Cell Infusion for Children with Acquired Hearing Loss
During the last 25 years, stem cells derived from umbilical cord blood have been used in more than 30,000 transplants performed worldwide for the treatment of over 80 diseases and disorders in both adults and children. The evolution of stem cell therapies has paved the way for further research being conducted through FDA-regulated clinical trials to uncover their potential in regenerative medicine applications.
With this potential in mind, researchers at Florida Hospital for Children are conducting an FDA-regulated clinical trial to determine the safety and feasibility of using a child's stem cells derived from their own umbilical cord blood as a treatment for sensorineural hearing loss. The study is supported by Cord Blood Registry®.
In the United States, approximately 15% of children suffer from low or high-frequency hearing loss (1). The most common type of hearing loss, especially at high frequencies, is sensorineural. Acquired sensorineural hearing loss results from damage to hair cells in the inner ear (cochlea) and can be caused by illness, medication, noise exposure, birth injury, or head trauma. A child's ability to hear affects the development of language skills, and hearing impairments can lead to poor academic and social development (2).
Hearing aids and cochlear implants have become welcome standards in assisting children with acquired hearing loss, but these devices don't actually repair associated damage to the cochlea, cochlear nerve or brain stem. Aiming to restore normal hearing without the use of a prosthesis, the study will investigate whether using cord blood stem cells to help trigger the body's own repair mechanisms could provide a non-invasive therapeutic option for acquired hearing loss that does not exist today. The trial follows promising evidence from preclinical studies suggesting that the infusion of human umbilical cord stem cells may help repair damaged cells in the inner ear in ways that could lead to hearing improvement.
The study has a primary objective of determining the safety of using cord blood stem cells in a select pediatric patient population while also assessing whether this approach to treatment improves inner ear function, speech, and language development. Researchers will follow 10 children, ranging in age from 6 weeks to 6 years, who have been diagnosed with acquired hearing loss for less than 18 months and who have their own cord blood unit processed and stored under Cord Blood Registry's strict quality controls. Children with a known genetic cause of deafness are ineligible for study participation. Patients will receive one intravenous infusion of their own umbilical cord blood stem cells. All patients will return for follow-up at 1 month, 6 months, and 1 year post-treatment. To date, three children have been treated and six more are in the enrollment process.
A growing body of research provides evidence that stem cells derived from cord blood can be used to repair damaged or diseased tissue or organs. Cord Blood Registry is the only family cord blood bank to partner with leading research institutions to establish FDA-regulated clinical trials exploring the potential regenerative ability of newborn stem cells to help treat conditions that have no cure today. The results of the acquired hearing loss study and others may spur a new era in treating common neurological conditions impacting children and adults.
- Niskar, AS, et al. Prevalence of hearing loss among children 6 to 19 years of age: the Third National Health and Nutrition Examination Survey. JAMA.1998; 279: 1071-1075.
- Tierney, CD, et al. Development of children who have hearing impairment. Pediatr Rev 29, e72-73; discussion e73 (2008)
Hearing Loss Association of America
The Hearing Loss Association of America® (HLAA) is the nation's foremost membership and advocacy organization for people with hearing loss. The mission of HLAA is to open the world of communication for people with hearing loss through information, education, support and advocacy.
The HLAA website, Hearingloss.org, provides accurate and timely information about: hearing aids and cochlear implants; hearing assistive technology; hearing loss management; advocacy efforts on federal and state levels; communication strategies for family and friends; and resources to help you and your family live successfully with hearing loss.
HLAA's Walk4Hearing® is the largest walk of its kind taking place in multiple cities across the country. Every spring and fall thousands of walkers - children and their families, young adults, young at heart and everyone in between - form teams and walk in their communities to increase public awareness about hearing loss, help eradicate the stigma associated with it, and raise funds for programs and services.
Eight-year old Katherine Pawlowski is this year's Walk4Hearing ambassador; her goal is to raise awareness about people "just like her", meaning people with hearing loss. Visit www.walk4hearing.org to find out how you can "step up for people with hearing loss".
Convention 2014, June 26 - 29 in Austin, Texas, is the nation's most extensive educational program and trade show for people with hearing loss. This year highlights include:
- - Classical music composer Richard Einhorn will deliver keynote address, A Life in Music After Hearing Loss; Richard suffered sudden sensorineural hearing loss in 2010.
- - Emerging Technologies Symposium moderated by the Consumer Electronics Foundation (CEA Foundation) with presentations by member companies.
- - Educational workshops on interesting and relevant topics such as hearing technology, relationships and communication, and employment issues for people with hearing loss.
- - Exhibit Hall full of the latest technology and services.
- - Get Acquainted Party on opening night.
- - Off-site event to the historic Scholz Bier Garten for BBQ and swing dancing.
- - Awards Breakfast and Ceremony honoring volunteers across the country.
- - Fiesta Saturday evening banquet with entertainment.
- - First-timer and Early-bird discounts plus free exhibit hall passes.
- - Complimentary registration for veterans with hearing loss who served in OIF and OEF.
Published bi-monthly since 1980, the award-winning Hearing Loss Magazine tells real-life stories of people with hearing loss and their families, informs readers of cutting-edge technology, and provides updates about HLAA's advocacy efforts to create a better future for people with hearing loss. Hearing Loss Magazine is included with your membership. The HLAA e-News of current events complements the magazine.
Public policy and advocacy staff advocate for consumer issues such as the hearing aid tax credit initiative, captioning, affordable hearing health care, insurance coverage, cochlear implant reimbursement, newborn infant hearing screening, and telecommunications compatibility. HLAA has representation on corporate advisory boards, government agencies, coalitions, educational institutions and nonprofit organizations.
HLAA's national support network includes the Washington, D.C. area office, state organizations, as well as local chapters that offer regular meetings, emotional support, camaraderie, and opportunities for advocacy on state and local levels.
In addition to these programs, HLAA offers a parent-to-parent resource site, educational webinars, and will soon launch the Hearing Loss Support Specialist Training, an online course designed for people who work with people with hearing loss. To learn more about HLAA, visit Hearingloss.org.
Why Hasn't Private Cord Blood Banking Grown Faster?
This simple question has been lingering unanswered over private cord blood banks around the world for years. Despite compelling data on the growing therapeutic use of cord blood, both in terms of treatable diseases and numbers of therapies, often medical doctors are still reluctant to embrace the value of family cord blood banking. In many countries, such as Brazil, this sector is still plagued with mostly unfounded controversy, such as the "public versus private" banking debate.
Hematology reviews show that there have been over 30 thousand cord blood transplants around the world to date. Confidential data archived by the Parentís Guide to Cord Blood Foundation show that by the end of 2013 there were over 3 million private cord blood units in storage and over one thousand therapies (both autologous and allogeneic) using units released from private banks.
Data from the USA's National Cancer Institute (Figure 1) indicate that the odds of having cancer increase with age, so that cord blood in private banks will be increasingly useful to families only after 30 to 40 years. Currently the average age of privately stored cord blood units is between 5 and 10 years, so the likelihood of these units being used to treat cancer is still low. Indeed, according to the Parent's Guide to Cord Blood, in some parts of the world most sibling transplants released from private banks are not for cancer, but for non malignant acquired and hereditary blood disorders, especially Sickle Cell and Thalassemia.
Looking deeper into the characteristics which contribute to the slow growth of the cord blood banking sector, it is never a trivial task to sell a medical service directly to mass consumer markets, especially when your service is perceived as complicated and new. In reality, this service should not be perceived differently than other major medical innovations which required substantial time to prove themselves, after reliable data could be collected along many years.
Thus, it is natural that physicians are the key stepping stone for outreach to parents. Selling a service that is perceived as hematologic/oncologic to someone who delivers babies is also a challenge. The doctor needs to be convinced that a harmless decision such as the collection of cord blood, with apparently no immediate consequence to the baby, may become a life saving measure for that child later on. Cord blood collection needs to become one of the many precautionary measures so many Ob/Gynís are used to prescribe, like ultra-sound examination and pre-natal care, among many others. Afterall, the likelihood of a child to need therapy with cord blood, especially for indications associated with prematurity, is not that different from many conditions potentially detected during pregnancy.
Physicians will certainly be more open to support cord blood banking if they are supplied with updated, quality, scientific evidence. Unfortunately, storing cord blood is still not a consensus from societies of medical professionals. Coupling all these challenges, those private cord blood banks that make false promises and sensationalist statements, certainly do not contribute to the credibility building process of the sector.
Hiring the right person as a sales representative ("rep") visiting physicians for a private cord blood bank is crucial. The mix of determination, selling skills, scientific curiosity and sense of purpose is not an easy one to find. Keeping the reps continuously motivated requires somewhat of a preachers' approach. These professionals need to buy in to the cord blood banking concept at a very deep philosophical level, realizing they do sell a service that may cure cancer, and this is a big deal! This will be the key pillar which will keep them focused, day in and day out, when they wake up early, often to hear derogatory comments from poorly informed doctors who may ridicule or discredit what they do.
The private bank must make a long term commitment to the sales rep model, as it takes at least 24 months for reps to payback their cost (Figure 2). There are four distinct stages a rep goes through until they reach their breakeven point, where the rep is generating enough revenue to cover their cost. First, due to the training complexity of private cord blood banking, it normally takes around three months for a rep to generate their first sales. Fairly quickly after that, a small group of doctors, namely early adopters, normally start referring new clients. Evolving to the next stage, when actual long term relationships are established, can take up to one more year or sometimes may not happen at all. This is the most important step for a rep to overcome, which will come to prove them as an effective relationship builder. Expanding this initial portfolio of loyal doctors is slow; literally a process of converting one doctor at a time.
In addition to personal visits to physicians by sales reps, private cord blood banks may apply internet marketing strategies that reach customers directly with little cost, and thus lower the aquisition cost per customer. Internet marketing strategies include social media outreach and targeted advertising.
Maintaining a loyal portfolio of doctors, however, does not depend solely on the continuous efficiency of the rep's work. It is imperative that the private cord blood bank as an organization (sales, operations, laboratory, customer service, etc...) consistently delivers on it's promises. A fair analogy is that of a good restaurant, whose greatness starts inside the kitchen, first and foremost, and not primarily because of marketing buzz. One final note, based on our experience, is that clients are loyal to a brand and are not a rep's property to be transfered from company to company, wherever the rep chooses to work.
In summary, the combination of all factors mentioned above explain why private cord blood banking may be an enticing sector for investment, but a challenging one when it comes to growth. The reason is fairly simple: it is difficult and expensive to sell this service.