Gilles Atlan is the father of Karen. He has four daughters, Karen is the third and is now 13 years old. When she was 6, she was diagnosed with a rare disease: NF2 (neurofibromatosis type 2), it leads to multiple tumors, especially on the cranial and spinal nerves. It is very different from NF1, which is more common. In France, for example, there is no more than 1000 people who suffer from NF2 today. The family realised after a while that there was no existing long term therapy for NF2, and no ongoing novel research to find this long term therapy. There was also no association whose cause was the fight against NF2.
Gilles Atlan found Dr. Nicole Henwood on social media, she was in a similar situation to his own: her son A. J. has NF2 and she couldn’t find any long term therapy for him. Together, they created NF2 BioSolutions in 2018, with the goal to jumpstart novel therapy research on NF2. NF2 BioSolutions’ mission is to find labs and convince them to focus on NF2, to fund them and help them to progress as fast as possible, until they find bigger funding for a trial. The plan worked very well, quickly they convinced several labs to work on NF2.
The organisation started with a consortium where roughly 40 gene and cell therapy experts met in one room with NF2 clinicians and researchers. The goal was to introduce this rare disease to the gene/cell therapy researchers, to understand its particularity and analyze which novel approaches could work for this rare gene mutation. Soon after this, two labs started to work on the disease.
TGE: Could you explain what NF2 exactly is?
Mr. Atlan: The disease creates tumors (schwannomas, meningiomas and ependymomas) in the body on the nervous system (on the nerves in the brain, on the spine, etc. it can basically happen on any nerve). The tumors grow on each nerve, and at one point it starts to press on it and the patient looses the function of the nerve. This leads to deafness, blindness, paralysis, pain, and eventually death. NF2 actually starts when there is a mutation in the NF2 gene (it is a tumor suppressor gene).
TGE: Do you work with other labs since the two that were convinced in the first place?
Mr. Atlan: We have a third lab in the United States, at the Harvard Medical School that has very interesting novel approaches. We gave them a funding to accelerate the research.
So, today, we are supporting the Nationwide Children’s Hospital in Ohio, it is a gene therapy lab. The goal there is to add a healthy gene, so the tumor will not grow. We started to finance them almost two years ago. Tests on mice that have NF2 tumors are starting now.
We also jumpstarted research at the University of Massachusetts. They have a similar approach, but they silenced the muted gene and then added the new one.
Then, we have the lab of Dr. Brenner in Mass General Hospital and Dr Mekalanos at Harvard Medical School. They are using a very novel approach. They use a bacteria and guide it to attack the tumor and destroy it. This is quite advanced now, there are already good results on mice. A biotech is now willing to raise more money to be able to do a trial.
Moreover, we are financing the lab of Dr. Kalamarides in Paris at Pitié-Salpetrière Hospital. He is working on creating mice that may develop a certain type of NF2 tumor (ependymoma). This type of animal model was missing. Indeed, it is hard to make progress and get FDA approval is there are no results on animal model, we cannot jump directly to a patient.
Finally, we jumpstarted a NF2 Biobank. It is always complicated to convince a lab to work with us because why would they focus on this rare disease instead of another? Usually, they agree once you help them to start. How? We bring them a network of experts, clinicians and researchers, and also the tools they need (animal model, cell line of patients and tissue of tumors). We observed that they had a hard time accessing NF2 cell lines and tissues of tumors. So, we launched a NF2 Biobank at the Children’s Brain Tumor Network, at the Children’s Hospital of Philadelphia. They have a non-profit that can manage the whole process of collecting tissue, creating cell lines from the tissue and then sharing it to any lab. The Biobank is crucial to be able for us to give the tools to labs that work, or will work, with NF2 BioSolutions.
TGE: Does the Biobank give the results you were hoping for?
Mr. Atlan: It is sad to say, but we have to compete with thousands of other rare diseases, so we have to facilitate as much as possible the work of the labs that are funded.
We started last summer with the NF2 Biobank. So far we collected more than 50 tissues of patients and we have more on the way. They created also dozens of material transfer agreements between the Biobank and the different institutions, it is a painful process. Every time something has to be transferred from one place to another, they need agreements and that takes time. The goal is to accelerate the sharing of tools and knowledge.
TGE: So you are now working with 5 institutions, how did you decide that?
Mr. Atlan: We don’t know what works and what doesn’t. A treatment might work on a mice but not on a human. So, we don’t want to put all our eggs in the same basket. This is why we try different approaches, and we keep looking for new ones, new technology, that are working for other types of mutations or other types of cancer, and could work on NF2.
TGE: Since you are the only association focusing on NF2, do you feel you created a community?
Mr. Atlan: In terms of NF2 community, we are really focusing on financing research. We are not about supporting patients, it is a totally different space and other organisations are doing that very well. Our goal is really to convince the people that are touched by this disease that we need to be proactive, and if no one is going to push labs to work on it, then it won’t be done. If we don’t do it, no one will do it for us. I think rare diseases have to be advocated by the patients themselves, there is no other choice.
We also work on awareness to make the disease more known. For example, we have webinars every two or three months on different topics. And for the fundraising, the major part happens online (through Facebook and the website), and most donations are small and recurring. Before the Covid-19 pandemic, we had a few fundraising events, but fortunately we were not relying on those physical events.
TGE: Did you have an international vocation since the start?
Mr. Atlan: Because we are a rare disease, we cannot be focused in one country. When we created NF2 BioSolutions we were already spread over the world. Very quickly after the creation, we had this idea of having ambassadors at different locations. An ambassador is someone that can represent us and be the evangelist of our organisation. We really wanted to cover as many countries as possible, from day 1 we were very international. Most of the funds are coming from the US, but we also receive funds from the UK, Germany and France, and we have supporters from many other countries.
It is important to be connected everywhere because this will be useful the day we have a trial. We will have the contacts of patients from a lot of different countries and hopefully it will be possible to do the trial in different locations. We have a lot of legitimacy among the patients, so when we will have a drug, they may listen to us.
TGE: How did you hear about Transnational Giving Europe? How does it fit within your fundraising strategy? Why did you decide to join?
Mr. Atlan: We were raising funds from everywhere, and a lot from Europe, but we knew that the donors weren’t getting tax benefits. Some were asking for it, but they couldn’t get any and were directly giving to the US organisation. So, we wanted to be more attractive for the donor. Since we are all volunteers at NF2 BioSolutions, we wanted an efficient solution, we hadn’t the time to create local non-profits in different countries. In the UK, we had some very motivated people who were with us since day 1, so we created a British non-profit NF2 BioSolutions UK. In Canada, we had no people on the ground, so we went through King Baudouin Foundation. For France and Germany, we needed TGE. Now, donors have tax benefits and we did not lose time and effort at creating a foundation. Even if there is a fee involved with TGE, I think it is still worth it for us. Finally, the different languages on the platform is a huge advantage.
TGE: Was NF2 BioSolutions impacted by the current pandemic? If so, how?
Mr. Atlan: It had some negative and positive effects. For the negative ones, it was on the side of the labs, the research really slowed down. Some of the labs had to shut down almost, send only one person at the time in the lab to maintain what was existing there, but no progress was made.
We first thought that with the pandemic there would be a lot of layoff and people would not donate. But in fact we raised way more this year than the previous year. I think for several reasons. One reason is that people are more at home, they are more on their phone and computer, and since we are mostly present online, they get our message more. Also, the market, the stocks went up a lot this year, so I think people think they made a lot of money and therefore are more generous with their donations as well. We were afraid that Covid-19 would be very hard for the organisation, but for the fundraising side of it, it was good. People had also more time to volunteer for our cause, and to communicate with our members.
Finally, the technology used for the delivery of the Moderna and Pfizer vaccines for Covid-19 is using lipid nanoparticles. The pandemic really boosted that technology, and now it is more approachable because it is been used on millions and millions of people. The manufacturing of this delivery mechanism will be cheaper because it is manufactured everywhere now. We knew about this technology before, but it was not used a lot at all. The pandemic opened our eyes about it. Now we are confident it is really something we should focus on as well, so we don’t have the negative effect of using a virus as delivery mechanism. So from the manufacturing point of view, this new vaccine gives us new possibilities.
TGE: Do you have some projects that will start soon? Can you tell us a word about the future of NF2 BioSolutions?
Mr. Atlan: For the lipid nanoparticles, we are talking with the University of Pennsylvania, the technology for the delivery came from there. We are discussing with researchers on if and how we could use it. We are looking into the possibility of using this delivery mechanism for a “suicide gene” (that destroys the mutated cells that create the tumors). The lab seems very positive on this solution for us. We are discussing about funding them.
Finally, Car T-cell therapy is another approach used a lot for cancer, and we are looking into it. We are in discussion with a few labs for developing that technology.
To learn more about NF2 BioSolutions…
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