Tag Archives: advocacy

Episode 27: Going Back to School and Caring for the Caregiver



In Episode 27 of TSC Now, host Dan Klein learns how parents can prepare for the start of a new school year despite the ongoing pandemic. Dan also talks to a licensed professional counselor to get advice on how parents and caregivers can cope with the stress, anxiety and trauma the last year and a half has caused, so that they are looking out for their own well-being in addition to supporting their loved ones with TSC.

First, Dan talked to Lilian Ansari, TSC Alliance Community Programs Resource Advisor (01:13). She gives helpful advice and tips on making decisions about in-person schooling, ensuring that Individualized Education Programs (IEPs) make up for lost learning that may have occurred while students were at home, and how parents can most effectively communicate with their IEP team. Lilian also shares the resources and support available from the TSC Alliance to help parents effectively advocate for their kids in their school system.

You can access all of our School resources here. If you have general school-related questions you can call Shelly Meitzler, Community Program Manager East, at (800) 225-6872 or email her at smeitzler@tscalliance.org.

Then, Dan talks to Latrice Hamilton, a licensed professional counselor at Journey to New Beginnings. With many TSC caregivers feeling the stress and anxiety of the ongoing pandemic in addition to sending kids back to school, Latrice offers some helpful advice on how to manage stress, communicate effectively with loved ones, and ask for help so that parents can make sure they are taking care of their own mental well-being in addition to caring for their loved ones with TSC.

All of the TSC Alliance’s COVID-19 resources can be found at www.tscalliance.org/covid-19. If you need immediate support from the TSC Alliance you can call our emergency hotline 9 am – 9 pm Eastern: (240) 463-7250.

Resources and Links

 


Episode 26: The Road to Newborn Screening in TSC



In Episode 26 of TSC Now, host Dan Klein takes a deep dive into one of the newer and exciting frontiers in TSC research: Newborn Screening. Last Fall, the TSC Alliance hosted an Innovation Workshop that brought together TSC researchers, newborn screening experts and other nonprofits with experience advocating for other diseases to be included in the newborn screening panel to start to set the road map forward. From that meeting the TSC Alliance put a call out to our community for dried blood spots from infants with TSC in certain states to help eventually validate an assay and earlier this month we put out a call for proposals to fund research to start developing that assay. To understand where we need to go and what will be necessary to get tuberous sclerosis complex added to the recommended uniform screening panel (RUSP), Dan spoke to two people who helped organize the Innovation Workshop.

First, Dan spoke to Hope Northrup, MD (01:42), Director of the Division of Medical Genetics, Professor in the Department of Pediatrics, and TSC Clinic Director at the McGovern Medical School at the University of Texas Health Science Center at Houston. Drawing from an impressive career in both TSC and newborn screening, Hope provides a history of newborn screening both in general and in the United States, what they key tenets are to determining whether a disease should be included on the RUSP, and ultimately what the clinical and research benefits are to having TSC included.

Next, Dan spoke to TSC Alliance Chief Scientific Officer Steve Roberds, PhD (23:31), to better understand how newborn screening fits into the larger research efforts of the organization, what steps we have taken to date to move this process forward, and what potential hypotheses we are hoping to test through new research funding. Steve also shares what the timeline and next steps are for both researchers and advocates once an effective and sensitive assay is in place.

The TS Alliance is Seeking Historical Dried Blood Spots and Cord Blood from Individuals with TSC for Newborn Screening Assay Development. Was your child with TSC born in Michigan, New York, Texas, or California? The TSC Alliance is actively seeking access to newborn dried blood spots and stored cord blood from babies born after 1985. If you are interested in donating samples that are potentially stored in your state to the TSC Alliance, please email biosample@tscalliance.org.

The TSC Alliance also recently announced a new funding opportunity for Newborn Screening (NBS) Assay Development. If you are a researcher interested in learning more about this opportunity and submitting a letter of intent you can learn more at www.tscalliance.org/grants. The deadline to submit an LOI is Monday, August 23.

Resources and Links


Episode 25: Lymphangioleiomyomatosis & Worldwide LAM Awareness Month (#WWLAM)



In Episode 25 of TSC Now, host Dan Klein recognizes Worldwide LAM Awareness Month (#WWLAM) by focusing on lymphangioleiomyomatosis (LAM), a rare lung disease affecting women that is characterized by an abnormal growth of smooth muscle cells, especially in the lungs, lymphatic system and kidneys. LAM is caused by mutations in the same gene(s) as TSC and is a common manifestation for women with TSC but can also occur sporadically with no other TSC manifestations. To help raise awareness of LAM on the last day of Worldwide LAM Awareness Day, this episode will focus exclusively on LAM, research being done to develop new treatments and ultimately a cure, and the organization that is leading the charge to improve the lives of women with LAM in the US.

First, Dan talks to Sue Sherman, MHA, Chief Executive Officer of The LAM Foundation (02:18). The LAM Foundation was founded in 1995 by a mother of a woman with LAM with a mission to urgently seeks safe and effective treatments, and ultimately a cure, for lymphangioleiomyomatosis through advocacy and the funding of promising research. They are dedicated to serving the scientific, medical and patient communities by offering information, resources and a worldwide network of hope and support. They discuss how The LAM Foundation has changed over the years, how their investment in research has helped pave the way for breakthroughs, how the organization pivoted during the COVID-19 pandemic, and how they are raising awareness of LAM during Worldwide LAM Awareness month.

Next, Dan talks to Hilaire Lam, PhD (14:22), Research Associate in the Division of Pulmonary and Critical Care Medicine at Brigham and Women’s Hospital and an instructor in medicine at Harvard Medical School. The ultimate goal of Dr. Lam’s research is to leverage mTORC1-driven mitochondrial alterations to uncover therapeutic targets for TSC and LAM. She shares more about the aims of her research, how she first became interested in LAM, what gaps exist in our understanding of LAM and how researchers are currently looking to address those outstanding questions to develop new therapies. She also discusses how she is motivated by women living with LAM and how their relentlessness inspires her to help find a cure.

Earlier this month in recognition of Worldwide LAM Awareness Month, the TSC Alliance hosted an e-webinar titled “LAM in TSC: Surveillance, Treatment and Research” presented by Souheil Y. El-Chemaly, MD, Clinical Director of the Center for LAM Research and Clinical Care at Brigham and Women’s Hospital. The webinar covered surveillance, management and treatment recommendations based on the latest consensus guidelines, clinical trials currently taking place and a look to the future of research in LAM. A recording of the presentation will be available on our e-webinars website soon at tscalliance.org/e-webinars.

Resources and Links

             


Episode 20: The 2021 Virtual March on Capitol Hill



In the first episode of TSC Now, host Dan Klein dives deep on the upcoming Virtual March on Capitol Hill taking place March 1-5, 2021. Due to the ongoing COVID-19 pandemic, the TS Alliance has decided to protect our volunteers by holding the march virtually. The March on Capitol Hill is the TS Alliance’s annual effort to advocate for continued funding for the Tuberous Sclerosis Complex Research Program (TSCRP) a subset of the Congressionally Directed Medical Research Program at the Department of Defense. Through these efforts $97 million has been allocated for the TSCRP since 2001 and last year our advocates successfully advocated for an increase in the annual appropriation to $8 million. If you are interested in volunteering please send an email to grc@tsalliance.org.

First, Dan spoke with Chip Burkhalter (02:13), a parent and TS Alliance Board Member, who came to the March on Capitol Hill for the first time last year. He shares how he first got involved, and why he believes getting connected and volunteering is so important after getting a TSC diagnosis. They also discuss how the TS Alliance advocates have been able to garner bipartisan support for TSCRP funding even in the most partisan environments.

Next, Dan talks to Kaushal Asrani, MBBS, PhD (16:09), a Research Associate in the Department of Pathology, at the Johns Hopkins University School of Medicine. He talks about his research to understand whether lysosomal biogenesis is a potential driver of tumorigenesis and therapeutic target in tuberous sclerosis, from which initial results will be published later this year. They also discuss how TSCRP funding has been pivotal to his work and how connecting with families at last year’s event provides motivation to continue to discover new therapies.

Finally, Dan catches up with Sara Chieffo (25:30), a parent, TS Alliance Board Member and Chair of the Government Relations Committee. She shares what motivated her to get involved in government advocacy, how our volunteers can be effective virtually this year, and why you should get involved too.

Resources and Links
Learn about the TS Alliance’s Government Advocacy Program: https://www.tsalliance.org/engage/government-advocacy/
Learn more about the TSCRP: https://cdmrp.army.mil/tscrp/default
Read an abstract of Dr. Asrani’s TSCRP-funded project:https://cdmrp.army.mil/search.aspx?LOG_NO=TS180078
Meet Dr. Asrani’s Team at Johns Hopkins: http://labs.pathology.jhu.edu/lotan/our-team/
Learn about what goes into our advocacy efforts by enrolling in TSC Academy, the TS Alliance’s e-learning platform: www.tscacademy.org


Episode 11: A #March4TSCresearch Recap & Update on COVID-19



In episode 11 of TSC Now, host Dan Klein recaps our annual March on Capitol Hill, which took place on March 4, 2020 in Washington, DC and brought together more than 100 volunteers from across the country to advocate for continued funding for the Tuberous Sclerosis Complex Research Program (TSCRP) at the Department of Defense. He then shares resources the TS Alliance has created to address the ongoing Coronavirus (COVID-19) pandemic.

First, Dan talks to Mark Vieth (2:04), Senior Vice President at Cavarocchi Ruscio Dennis Associates, who serves as a consultant for the TS Alliance’s advocacy efforts and helps prepare our volunteers for their meetings with their representatives. They discuss what goes on at the Training Dinner before March on Capitol Hill, what key messages volunteers should bring to their meetings, and why this effort has been so successful over the years.

Next, Dan interviews Bridget Britt (10:54), an advocate and mom from California. She shares why she advocates and how coming to March on Capitol Hill gives her strength and purpose.

Dan then talks to Sam Barth (15:42), a graduate student working in Kim Raab-Graham’s lab at Wake Forest University Medical School. Sam shares how the March on Capitol Hill provides meaningful context for the work he does in the lab and also discusses how the funding his mentor Kim received through the TSCRP helped generate new insights on TSC.

Dan then shares resources the TS Alliance has developed in response to the ongoing COVID-19 pandemic (25:44), including a FAQ guide for parents and caregivers, resources for TSC medical professionals, and a schedule of upcoming webinars, virtual Town Halls and Open Forums. All of these resources can be found at tsalliance.org/COVID-19.

Finally, Dan continues his Get to Know the TS Alliance segment by talking to Justin Martucci (28:40), Development Services Manager at the TS Alliance. They talk about how Justin has spearheaded the TS Alliance database cleanup and transition to new platforms, what he does when he’s not in the office, and how he is managing working from home during the pandemic.

In the April episode of TSC Now, Dan will be diving deeper into how the COVID-19 pandemic is affecting the TSC community and he wants to hear from you! Leave a voicemail at (240) 638-4644 with your first name, where you are calling from and how the pandemic has affected you, and your call may be played on the April episode!

March on Capitol Hill Resources:
For more information on the Tuberous Sclerosis Complex Research Program (TSCRP), visit: https://cdmrp.army.mil/tscrp/default
To read about Dr. Kim Raab-Graham’s research funded by the TSCRP, visit: https://cdmrp.army.mil/tscrp/research_highlights/17graham_highlight
For more information on the TS Alliance’s Government Advocacy Program, visit: https://www.tsalliance.org/engage/government-advocacy/
If you are interested in participating in our government advocacy program, send us an email to grc@tsalliance.org.

COVID-19 Resources:
For all of the TS Alliance’s resources on COVID-19, visit: tsalliance.org/COVID-19
To download a FAQ guide for parents and caregivers, visit: http://www.tsalliance.org/wp-content/uploads/2020/03/COVID-19-FAQ.pdf
For resources for TSC medical professionals, go to: https://www.tsalliance.org/covid-19-resources-for-tsc-medical-professionals/
To see a schedule and register for our upcoming webinars, visit: https://www.tsalliance.org/upcoming-covid-19-and-tsc-webinars-and-virtual-town-halls/
To find recordings of previous webinars, visit: https://www.tsalliance.org/covid-19-webinar-and-virtual-town-hall-recordings/